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A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (PROPEL)

Primary Purpose

Pompe Disease (Late-onset)

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Cipaglucosidase Alfa
Miglustat
Alglucosidase Alfa
Placebo
Sponsored by
Amicus Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pompe Disease (Late-onset) focused on measuring Pompe, rhGAA

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Subject must provide signed informed consent prior to any study-related procedures being performed.
  2. Male and female subjects are ≥ 18 years old and weigh ≥ 40 kg at screening.
  3. Female subjects of childbearing potential and male subjects must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug.

  4. Subject must have a diagnosis of LOPD based on documentation of one of the following:

    1. deficiency of GAA enzyme
    2. GAA genotyping

  5. Subject is classified as one of the following with respect to ERT status:

    1. ERT-experienced, defined as currently receiving standard of care ERT (alglucosidase alfa) at the recommended dose and regimen (ie, 20 mg/kg dose every 2 weeks) for ≥ 24 months
    2. ERT-naïve, defined as never having received investigational or commercially available ERT
  6. Subject has a sitting FVC ≥ 30% of the predicted value for healthy adults (National Health and Nutrition Examination Survey III) at screening.

  7. Subject performs two 6MWTs at screening that are valid, as determined by the clinical evaluator, and that meet all of the following criteria:

    1. both screening values of 6MWD are ≥ 75 meters
    2. both screening values of 6MWD are ≤ 90% of the predicted value for healthy adults
    3. the lower value of 6MWD is within 20% of the higher value of 6MWD

Exclusion Criteria

  1. Subject has received any investigational therapy or pharmacological treatment for Pompe disease, other than alglucosidase alfa, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before Day 1 or is anticipated to do so during the study.
  2. Subject has received gene therapy for Pompe disease

  3. Subject is taking any of the following prohibited medications within 30 days before Day 1:

    • miglitol (eg, Glyset)
    • miglustat (eg, Zavesca)
    • acarbose (eg, Precose or Glucobay)
    • voglibose (eg, Volix, Vocarb, or Volibo)

    Note: None of these medications have a half-life that, when multiplied by 5, is longer than 30 days.

  4. Subject requires the use of invasive or noninvasive ventilation support for > 6 hours per day while awake.
  5. Subject has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221.
  6. Subject has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the subject or may compromise his/her ability to comply with or adversely impact protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms.
  7. Subject, if female, is pregnant or breastfeeding at screening.
  8. Subject, whether male or female, is planning to conceive a child during the study.
  9. Subject does not have documentation of diagnosis of Pompe disease and refuses to undergo genetic testing.

Sites / Locations

  • Neuromuscular Research Center
  • University of Arkansas for Medical Sciences
  • University of California, Irvine
  • UF Helath: University of Florida Clinical Research Center
  • University of South Florida Research Center
  • Emory Clinic
  • Indiana University Health Neuroscience Center
  • University of Kansas Medical Center
  • University of Minnesota Clinical Research Unit
  • Washington University School of Medicine
  • Billings Clinic
  • Hackensack University Medical Center
  • The Feinstein Institute for Medical Research
  • NYU School of Medicine
  • Duke University Medical Center
  • University of Cincinnati Neurology
  • Cincinnati Children's Hospital Medical Center
  • The Ohio State University Wexner Medical Center
  • Oregon Health & Science University
  • Penn State Health Milton S. Hershey Medical Center
  • University of Pennsylvania
  • University of Pittsburgh
  • University of Texas Health Science Center San Antonio
  • University of Utah, Center for Clinical and Translational Sciences
  • Lysosomal and Rare Disorders Research
  • Hospital Universitario Austral
  • Royal Brisbane & Women's Hospital
  • Royal Adelaide Hospital
  • Monash Medical Centre
  • Westmead Hospital
  • Medizinische Universität Innsbruck
  • UZ Leuven
  • University Clinical Centre of the Republic of Srpska
  • UMHAT Alexandrovska
  • Heritage Medical Research Clinic, University of Calgary
  • McMaster University Medical Centre
  • Aarhus Universitets Hospital
  • Rigshospitalet Copenhagen Neuromuscular Center
  • Hôpital Neurologique Pierre Wertheimer
  • Hôpital Raymond Poincaré
  • Hôpital Salengro
  • Hôpital de la Timone
  • Hôpital Pasteur
  • Friedrich-Baur Institut
  • Universitätsklinikum Bonn
  • Universitätsklinikum Münster
  • Universitätsklinikum Halle (Saale)
  • Eginition Hospital
  • Semmelweis University, Institute of Genomic Medicine and Rare Disease
  • University of Pécs
  • University of Szeged
  • UOC di Neurologia e Malattie Neuromuscolari
  • UOC Genetica Medica
  • Hokkaido University Hospital
  • Fukuoka University Hospital
  • Kagoshima University Hospital
  • Izumi City General Hospital
  • The Jikei University Hospital
  • National Center of Neurology and Psychiatry
  • Pusan National University
  • Seoul National University Hospital
  • Erasmus MC
  • University of Auckland
  • Szpital Uniwersytecki w Krakowie
  • Centrum Medyczne
  • University Medical Centre Ljubljana
  • Hospital de la Santa Creu I Sant Pau
  • Sahlgrenska University Hospital
  • National Taiwan University Hospital
  • Queen Elizabeth Hospital Birmingham
  • Cambridge University Hospitals
  • Royal Free Hospital NHS
  • Salford Royal NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Cipaglucosidase Alfa/Miglustat

Alglucosidase Alfa/Placebo

Arm Description

Participants received cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W).

Participants received alglucosidase alfa co-administered with placebo Q2W.

Outcomes

Primary Outcome Measures

Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD)
The efficacy of cipaglucosidase alfa/miglustat co-administration on ambulatory function was measured by the 6MWT. The 6MWD, measured in meters, is the distance walked on the 6MWT. A greater distance indicated greater endurance. An increase from baseline indicated improvement.

Secondary Outcome Measures

Change From Baseline to Week 52 in Sitting Forced Vital Capacity (FVC; % Predicted)
The efficacy of cipaglucosidase alfa/miglustat co-administration on pulmonary function was measured by sitting FVC (% predicted). FVC is a standard pulmonary function test used to quantify respiratory muscle weakness.
Change From Baseline to Week 52 in the Manual Muscle Test (MMT) Score for the Lower Extremities
The total score for the MMT lower extremity strength included the following 8 body parts: right/left hip flexion, right/left hip abduction, right/left knee flexion and right/left knee extension. The MMT lower extremity score ranged from 0 to 40, with lower scores indicating weaker muscle strength. An increase from baseline indicated increased muscle strength.
Change From Baseline to Week 26 in 6MWD
The 6MWD, measured in meters, is the distance walked on the 6MWT.
Change From Baseline to Week 52 in the Total Score for the Patient- Reported Outcomes Measurement Information System (PROMIS®) - Physical Function
Physical Function Short Form 20a (v2.0) consisted of 20 questions. The first 14 questions were each scored on a scale from 1 to 5 as follows: 1 = unable to do; 2 = with much difficulty; 3 = with some difficulty; 4 = with a little difficulty; 5 = without any difficulty; the next 6 questions were each scored on a scale from 1 to 5 as follows: 1 = cannot do; 2 = quite a lot; 3 = somewhat; 4 = very little; 5 = not at all. The total score was calculated by summing up scores (1 to 5) across all items. Total scores range from 20 to 100. A higher score represented a better outcome.
Change From Baseline to Week 52 in the Total Score for the PROMIS® - Fatigue
Fatigue Short Form 8a consisted of 6 questions, each scored on a scale from 1 to 5 as follows: 1 = not at all; 2 = a little bit; 3 = somewhat; 4 = quite a bit; 5 = very much; and 2 questions, each scored on a scale from 1 to 5 as follows: 1 = never; 2 = rarely; 3 = sometimes; 4 = often; 5 = always. The total score was calculated by summing up scores (1 to 5) across all items. A lower score represented lower fatigue symptoms.
Change From Baseline to Week 52 in the Total Score for the Gait, Stairs, Gowers' Maneuver, and Chair (GSGC)
The GSGC consisted of a 10-meter walk for evaluation of gait, a 4-stair climb, Gowers' maneuver, and arising from a chair. Results of the GSGC included the time required to complete the individual tests, individual scores for each of the tests (1 to 7 points for each of gait, 4-stair climb, and Gowers' maneuver, and 1 to 6 points for arising from a chair), and a total score. GSGC total score was the sum of the component scores from the 4 functional tests. The total score ranged from a minimum of 4 points (normal performance) to a maximum of 27 points (worst performance).
Change From Baseline to Week 52 in Rasch-Built Pompe-Specific Activity (R-PAct) Total Score
The R-PAct scale was an 18-item questionnaire to measure limitations in activities and restriction in social participation. Possible responses to questions were as follows: unable to perform, able to perform, but with difficulty, and able to perform without difficulty. The total score was calculated by summing up the observed scores across the 18 items and it ranged from 0 to 36, with higher values representing lower level of disease impact on the muscles.
Change From Baseline to Week 52 in European Quality of Life-5 Dimensions 5 Response Levels (EQ-5D-5L) Based on the EuroQol Visual Analogue Scale (EQ VAS) Quantitative Score
The EuroQol Visual Analogue Scale (EQ VAS) is a vertical visual analogue scale that records the respondent's own assessment of his or her overall health status at the time of completion. Scores range from 0 (the worst health you can imagine) to 100 (the best health you can imagine). Higher EQ VAS scores represent an improved sense of overall health while lower scores represent a worsening of overall health.
Change From Baseline to Week 52 in Sitting Slow Vital Capacity (SVC) % Predicted
SVC is a standard pulmonary function test used to quantify respiratory muscle weakness.
Change From Baseline to Week 52 in Maximal Inspiratory Pressure (MIP) % Predicted
The percent predicted values of MIP were calculated as: % predicted = (actual result / predicted result)* 100, where the predicted results were obtained using the reference equations from Uldry and Fitting (1995).
Change From Baseline to Week 52 in Maximal Expiratory Pressure (MEP) % Predicted
The percent predicted values of MEP were calculated as: % predicted = (actual result / predicted result) * 100, where the predicted results were obtained using the reference equations from Uldry and Fitting (1995).
Change From Baseline to Week 52 in Sniff Nasal Inspiratory Pressure (SNIP) % Predicted
The percent predicted values of SNIP were calculated as: % predicted = (actual result / predicted result) * 100, where the predicted results were obtained using the reference equations from Evans and Whitelaw (2009).
Change From Baseline to Week 52 in % Predicted 6MWD
The % predicted 6MWD = (actual 6MWD / predicted 6MWD) * 100. The predicted values were calculated using Enright And Sherrill 1998 Reference Equations.
Change From Baseline to Week 52 in the Quantitative Muscle Test (QMT) Values
QMT was measured using the hand-held dynamometer. Larger values (in kg) indicated greater muscle strength.
Change From Baseline to Week 52 in Other MMT Scores
Each manual muscle test was evaluated on a scoring scale from 0 to 5, as follows: 0 = no muscle movement; 1 = visible muscle movement, but no movement at the joint; 2 = movement at the joint, but not against gravity; 3 = movement against gravity, but not against added resistance; 4 = movement against resistance, but less than normal; 5 = normal strength. Upper extremity score was the sum of scores for right/left shoulder abduction, right/left shoulder adduction, right/left elbow extension, and right/left elbow flexion, with the total score ranging from 0 to 40. Proximal muscle group score, the sum of scores for right/left hip flexion, right/left hip abduction, right/left shoulder abduction, and right/left shoulder adduction, with the total score ranging from 0 to 40. MMT total score was the sum of the lower and upper extremity scores and ranged from 0 to 80. Lower scores indicated lower overall muscle strength. An increase from baseline indicated improvement in muscle strength.
Change From Baseline to Week 52 in Maximum Vital Capacity (Maximum VC) % Predicted
Maximum VC is the greater of the two VC values (FVC or SVC).
Change From Baseline to Week 52 in PROMIS-Dyspnea and Upper Extremities Total Scores
The Upper Extremities Short Form 7a consisted of 7 items each scored on a decreasing scale from 1 to 5 as follows: 1 = unable to do; 2 = with much difficulty; 3 = with some difficulty; 4 = with a little difficulty; 5 = without any difficulty. Dyspnea Severity Short Form 10a consisted of 10 items each scored on a scale from 0 to 3 as follows: 0 = no shortness of breath; 1 = mildly short of breath; 2 = moderately short of breath; 3 = severely short of breath. A total score was generated for each instrument by adding up each item. Total scores for upper extremities range from 7 to 35. Total scores for dyspnea range from 0 to 30. A higher score for upper extremities represented improvement in symptoms. A lower score for dyspnea severity represented improvement in symptoms.
Change From Baseline in the Time to Complete Individual GSGC Component Tests and Timed Up and Go (TUG) Test at Week 52
Motor function test assessed the time to complete individual GSGC component tests (10-meter walk, 4- stair climb, Gowers' maneuver, and arise from a chair) and the TUG test. The TUG test assessed the time a subject needed to rise from a chair, walk 3 meters, turn around, walk back to the chair, and sit down.
Physician's Global Impression of Change (PGIC) Overall Status at Week 52
Physician's Global Impression of Change is based on a single item that is scored on a 7-point rating scale ranging from 1 "very much worse" to 7 "very much improved". A tertiary response variable (improving, declining, stable) was defined as follows: "Improving", which consisted of improved, moderately improved, and very much improved; "Declining", which consisted of worse, moderately worse, and very much worse; and "Stable", which equaled to no change.
Subject's Global Impression of Change (SGIC) at Week 52
The SGIC is designed to record the participants' impression of their functional status since starting study drug using a 7-point scale ranging from 1 "very much worse" to 7 "very much improved". A tertiary response variable (improving, declining, stable) was defined as follows: "Improving", which consisted of improved, moderately improved, and very much improved; "Declining", which consisted of worse, moderately worse, and very much worse; and "Stable", which equaled to no change.
Number of Participants Improving on Both 6MWD and % Predicted FVC at Week 52
A composite subject-level response of the 2 relevant clinical outcomes, 6MWD and FVC (% predicted), was assessed. Prespecified thresholds were used for assessment of improvement consistent with published minimal clinically important difference values for comparable instruments in similar disease.
Number of Participants With Treatment-Emergent Anti-Drug Antibodies (ADAs)
Treatment-emergent ADAs were defined as participants who had seroconverted or boosted their preexisting ADA during the study period.
Change From Baseline to Week 52 in Urinary Hexose Tetrasaccharide (Hex4) Level
Levels of urinary Hex4, a biomarker of disease substrate, were measured. The assay specifically targets Hex4, the glucose tetrasaccharide (Glc4), which is a biomarker of glycogen storage.
Change From Baseline to Week 52 in Serum Creatine Kinase (CK) Level
Change from baseline to Week 52 in serum CK level. CK levels were measured as part of the serum chemistry panel.
Population Pharmacokinetics (PK): Maximum Observed Concentration (Cmax) of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Experienced Participants Using Plasma Total GAA Protein Level by Signature Peptide Assay and Plasma Miglustat Concentration
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-experienced participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Population PK: Area Under the Concentration-Time Curve (AUC) of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Experienced Participants Using Plasma Total GAA Protein Level by Signature Peptide Assay and Plasma Miglustat Concentration
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-experienced participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Population PK: Cmax of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Participants
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-naïve participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Population PK: AUC of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Subjects
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-naïve participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Noncompartmental Analysis: Cmax of Plasma Total GAA Protein by Signature Peptide T09 in ERT-Naïve Subjects
A noncompartmental analysis was performed on ERT-naïve subjects, who underwent serial PK sampling during the study. On Day 1, serial blood samples were collected for ERT-naïve participants just prior to initiation of cipaglucosidase alfa/alglucosidase alfa infusion (time 0) and at 1, 2, 3, 3.5, 4, 4.5, 6, 8, 10, and 24 hours after the start of cipaglucosidase alfa/alglucosidase alfa infusion for plasma total human acid α-glucosidase (GAA) protein signature peptide T09 and plasma miglustat determinations.
Noncompartmental Analysis: AUC From Time 0 (Predose) to the Time of Last Quantifiable Concentration of Plasma Total GAA Protein by Signature Peptide T09 in ERT-Naïve Subjects
A noncompartmental analysis was performed on ERT-naïve subjects, who underwent serial PK sampling during the study. On Day 1, serial blood samples were collected for ERT-naïve participants just prior to initiation of cipaglucosidase alfa/alglucosidase alfa infusion (time 0) and at 1, 2, 3, 3.5, 4, 4.5, 6, 8, 10, and 24 hours after the start of cipaglucosidase alfa/alglucosidase alfa infusion for plasma total GAA protein signature peptide T09 and plasma miglustat determinations.
Comparison of Cmax of Cipaglucosidase Alfa in ERT-Experienced and ERT-Naïve Populations
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Comparison of AUC of Cipaglucosidase Alfa in ERT- Experienced and ERT-Naïve Populations
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.

Full Information

First Posted
October 10, 2018
Last Updated
August 25, 2023
Sponsor
Amicus Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT03729362
Brief Title
A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
Acronym
PROPEL
Official Title
A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
December 4, 2018 (Actual)
Primary Completion Date
December 15, 2020 (Actual)
Study Completion Date
January 15, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amicus Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a phase 3 double-blind randomized study to study the efficacy and safety of intravenous ATB200 Co-administered with oral AT2221 in adult subjects with Late Onset Pompe Disease compared with Alglucosidase Alfa/placebo.
Detailed Description
This is a double-blind, randomized, multicenter, international study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who have received enzyme replacement therapy with alglucosidase alfa (ie, ERT-experienced) or who have never received ERT (ie, ERT naïve) compared with alglucosidase alfa/placebo. The study will consist of a screening period up to 30 days, a 12-month treatment period, and a 30 day safety follow-up period. Subjects who complete this study will have the option to participate in an open label extension study to receive ATB200/AT2221 under a separate protocol. Enzyme replacement therapy-experienced subjects will continue to take alglucosidase alfa during the screening period; treatment with alglucosidase alfa will then be replaced by study drug (ATB200/AT2221 or alglucosidase alfa/placebo) on the same schedule without interruption (ie, every 2 weeks). Infusion visits will be scheduled every 2 weeks throughout the study; assessments (eg, clinical laboratory tests) for initial safety monitoring will be performed at these visits for the first 6 weeks of the study. Study visits that include efficacy, safety, and other assessments will be scheduled approximately every 3 months and may occur over 2 days, provided all study assessments and procedures (with the exception of pharmacokinetic [PK] sample collection) are performed before administration of study drug. Efficacy assessments (ie, functional assessments) include evaluation of ambulatory function (6MWT), motor function tests (Gait, Stair, Gowers' maneuver, and Chair [GSGC] test and Timed Up and Go [TUG] test), muscle strength (manual muscle testing and quantitative muscle testing), and pulmonary function tests (forced vital capacity [FVC], slow vital capacity [SVC], maximal inspiratory pressure [MIP], maximal expiratory pressure [MEP], and sniff nasal inspiratory pressure [SNIP]). Patient reported outcomes (Rasch-built Pompe-specific Activity [R PAct] Scale, EuroQol 5 Dimensions 5 Levels Instrument [EQ-5D-5L], Patient-Reported Outcomes Measurement Information System [PROMIS®] instruments for physical function, fatigue, dyspnea, and upper extremity, and Subject's Global Impression of Change). The Physician's Global Impression of Change will also be performed. Pharmacodynamic assessments include measurement of biomarkers of muscle injury (creatine kinase [CK]) and disease substrate (urinary hexose tetrasaccharide [Hex4]). Blood samples will be collected for determination of total GAA protein levels and AT2221 concentrations in plasma for a population PK analysis. Safety assessments include monitoring of adverse events (AEs), including infusion associated reactions (IARs), clinical laboratory tests (chemistry, hematology, and urinalysis), vital signs, physical examinations including weight, electrocardiograms (ECGs), and immunogenicity. Concomitant medications and nondrug therapies will also be recorded.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease (Late-onset)
Keywords
Pompe, rhGAA

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
125 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cipaglucosidase Alfa/Miglustat
Arm Type
Experimental
Arm Description
Participants received cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W).
Arm Title
Alglucosidase Alfa/Placebo
Arm Type
Active Comparator
Arm Description
Participants received alglucosidase alfa co-administered with placebo Q2W.
Intervention Type
Biological
Intervention Name(s)
Cipaglucosidase Alfa
Other Intervention Name(s)
ATB200
Intervention Description
Participants received an intravenous (IV) infusion dose over a 4-hour duration every 2 weeks (Q2W).
Intervention Type
Drug
Intervention Name(s)
Miglustat
Other Intervention Name(s)
AT2221
Intervention Description
Participants received weight-based doses 1 hour prior to cipaglucosidase alfa infusion Q2W.
Intervention Type
Biological
Intervention Name(s)
Alglucosidase Alfa
Other Intervention Name(s)
Myozyme
Intervention Description
Participants received an IV infusion dose over a 4-hour duration Q2W.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Miglustat matching placebo was administered orally 1 hour prior to alglucosidase alfa infusion Q2W.
Primary Outcome Measure Information:
Title
Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD)
Description
The efficacy of cipaglucosidase alfa/miglustat co-administration on ambulatory function was measured by the 6MWT. The 6MWD, measured in meters, is the distance walked on the 6MWT. A greater distance indicated greater endurance. An increase from baseline indicated improvement.
Time Frame
Baseline, Week 52
Secondary Outcome Measure Information:
Title
Change From Baseline to Week 52 in Sitting Forced Vital Capacity (FVC; % Predicted)
Description
The efficacy of cipaglucosidase alfa/miglustat co-administration on pulmonary function was measured by sitting FVC (% predicted). FVC is a standard pulmonary function test used to quantify respiratory muscle weakness.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in the Manual Muscle Test (MMT) Score for the Lower Extremities
Description
The total score for the MMT lower extremity strength included the following 8 body parts: right/left hip flexion, right/left hip abduction, right/left knee flexion and right/left knee extension. The MMT lower extremity score ranged from 0 to 40, with lower scores indicating weaker muscle strength. An increase from baseline indicated increased muscle strength.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 26 in 6MWD
Description
The 6MWD, measured in meters, is the distance walked on the 6MWT.
Time Frame
Baseline, Week 26
Title
Change From Baseline to Week 52 in the Total Score for the Patient- Reported Outcomes Measurement Information System (PROMIS®) - Physical Function
Description
Physical Function Short Form 20a (v2.0) consisted of 20 questions. The first 14 questions were each scored on a scale from 1 to 5 as follows: 1 = unable to do; 2 = with much difficulty; 3 = with some difficulty; 4 = with a little difficulty; 5 = without any difficulty; the next 6 questions were each scored on a scale from 1 to 5 as follows: 1 = cannot do; 2 = quite a lot; 3 = somewhat; 4 = very little; 5 = not at all. The total score was calculated by summing up scores (1 to 5) across all items. Total scores range from 20 to 100. A higher score represented a better outcome.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in the Total Score for the PROMIS® - Fatigue
Description
Fatigue Short Form 8a consisted of 6 questions, each scored on a scale from 1 to 5 as follows: 1 = not at all; 2 = a little bit; 3 = somewhat; 4 = quite a bit; 5 = very much; and 2 questions, each scored on a scale from 1 to 5 as follows: 1 = never; 2 = rarely; 3 = sometimes; 4 = often; 5 = always. The total score was calculated by summing up scores (1 to 5) across all items. A lower score represented lower fatigue symptoms.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in the Total Score for the Gait, Stairs, Gowers' Maneuver, and Chair (GSGC)
Description
The GSGC consisted of a 10-meter walk for evaluation of gait, a 4-stair climb, Gowers' maneuver, and arising from a chair. Results of the GSGC included the time required to complete the individual tests, individual scores for each of the tests (1 to 7 points for each of gait, 4-stair climb, and Gowers' maneuver, and 1 to 6 points for arising from a chair), and a total score. GSGC total score was the sum of the component scores from the 4 functional tests. The total score ranged from a minimum of 4 points (normal performance) to a maximum of 27 points (worst performance).
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in Rasch-Built Pompe-Specific Activity (R-PAct) Total Score
Description
The R-PAct scale was an 18-item questionnaire to measure limitations in activities and restriction in social participation. Possible responses to questions were as follows: unable to perform, able to perform, but with difficulty, and able to perform without difficulty. The total score was calculated by summing up the observed scores across the 18 items and it ranged from 0 to 36, with higher values representing lower level of disease impact on the muscles.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in European Quality of Life-5 Dimensions 5 Response Levels (EQ-5D-5L) Based on the EuroQol Visual Analogue Scale (EQ VAS) Quantitative Score
Description
The EuroQol Visual Analogue Scale (EQ VAS) is a vertical visual analogue scale that records the respondent's own assessment of his or her overall health status at the time of completion. Scores range from 0 (the worst health you can imagine) to 100 (the best health you can imagine). Higher EQ VAS scores represent an improved sense of overall health while lower scores represent a worsening of overall health.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in Sitting Slow Vital Capacity (SVC) % Predicted
Description
SVC is a standard pulmonary function test used to quantify respiratory muscle weakness.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in Maximal Inspiratory Pressure (MIP) % Predicted
Description
The percent predicted values of MIP were calculated as: % predicted = (actual result / predicted result)* 100, where the predicted results were obtained using the reference equations from Uldry and Fitting (1995).
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in Maximal Expiratory Pressure (MEP) % Predicted
Description
The percent predicted values of MEP were calculated as: % predicted = (actual result / predicted result) * 100, where the predicted results were obtained using the reference equations from Uldry and Fitting (1995).
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in Sniff Nasal Inspiratory Pressure (SNIP) % Predicted
Description
The percent predicted values of SNIP were calculated as: % predicted = (actual result / predicted result) * 100, where the predicted results were obtained using the reference equations from Evans and Whitelaw (2009).
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in % Predicted 6MWD
Description
The % predicted 6MWD = (actual 6MWD / predicted 6MWD) * 100. The predicted values were calculated using Enright And Sherrill 1998 Reference Equations.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in the Quantitative Muscle Test (QMT) Values
Description
QMT was measured using the hand-held dynamometer. Larger values (in kg) indicated greater muscle strength.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in Other MMT Scores
Description
Each manual muscle test was evaluated on a scoring scale from 0 to 5, as follows: 0 = no muscle movement; 1 = visible muscle movement, but no movement at the joint; 2 = movement at the joint, but not against gravity; 3 = movement against gravity, but not against added resistance; 4 = movement against resistance, but less than normal; 5 = normal strength. Upper extremity score was the sum of scores for right/left shoulder abduction, right/left shoulder adduction, right/left elbow extension, and right/left elbow flexion, with the total score ranging from 0 to 40. Proximal muscle group score, the sum of scores for right/left hip flexion, right/left hip abduction, right/left shoulder abduction, and right/left shoulder adduction, with the total score ranging from 0 to 40. MMT total score was the sum of the lower and upper extremity scores and ranged from 0 to 80. Lower scores indicated lower overall muscle strength. An increase from baseline indicated improvement in muscle strength.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in Maximum Vital Capacity (Maximum VC) % Predicted
Description
Maximum VC is the greater of the two VC values (FVC or SVC).
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in PROMIS-Dyspnea and Upper Extremities Total Scores
Description
The Upper Extremities Short Form 7a consisted of 7 items each scored on a decreasing scale from 1 to 5 as follows: 1 = unable to do; 2 = with much difficulty; 3 = with some difficulty; 4 = with a little difficulty; 5 = without any difficulty. Dyspnea Severity Short Form 10a consisted of 10 items each scored on a scale from 0 to 3 as follows: 0 = no shortness of breath; 1 = mildly short of breath; 2 = moderately short of breath; 3 = severely short of breath. A total score was generated for each instrument by adding up each item. Total scores for upper extremities range from 7 to 35. Total scores for dyspnea range from 0 to 30. A higher score for upper extremities represented improvement in symptoms. A lower score for dyspnea severity represented improvement in symptoms.
Time Frame
Baseline, Week 52
Title
Change From Baseline in the Time to Complete Individual GSGC Component Tests and Timed Up and Go (TUG) Test at Week 52
Description
Motor function test assessed the time to complete individual GSGC component tests (10-meter walk, 4- stair climb, Gowers' maneuver, and arise from a chair) and the TUG test. The TUG test assessed the time a subject needed to rise from a chair, walk 3 meters, turn around, walk back to the chair, and sit down.
Time Frame
Baseline, Week 52
Title
Physician's Global Impression of Change (PGIC) Overall Status at Week 52
Description
Physician's Global Impression of Change is based on a single item that is scored on a 7-point rating scale ranging from 1 "very much worse" to 7 "very much improved". A tertiary response variable (improving, declining, stable) was defined as follows: "Improving", which consisted of improved, moderately improved, and very much improved; "Declining", which consisted of worse, moderately worse, and very much worse; and "Stable", which equaled to no change.
Time Frame
Week 52
Title
Subject's Global Impression of Change (SGIC) at Week 52
Description
The SGIC is designed to record the participants' impression of their functional status since starting study drug using a 7-point scale ranging from 1 "very much worse" to 7 "very much improved". A tertiary response variable (improving, declining, stable) was defined as follows: "Improving", which consisted of improved, moderately improved, and very much improved; "Declining", which consisted of worse, moderately worse, and very much worse; and "Stable", which equaled to no change.
Time Frame
Week 52
Title
Number of Participants Improving on Both 6MWD and % Predicted FVC at Week 52
Description
A composite subject-level response of the 2 relevant clinical outcomes, 6MWD and FVC (% predicted), was assessed. Prespecified thresholds were used for assessment of improvement consistent with published minimal clinically important difference values for comparable instruments in similar disease.
Time Frame
Week 52
Title
Number of Participants With Treatment-Emergent Anti-Drug Antibodies (ADAs)
Description
Treatment-emergent ADAs were defined as participants who had seroconverted or boosted their preexisting ADA during the study period.
Time Frame
Baseline up to Week 52
Title
Change From Baseline to Week 52 in Urinary Hexose Tetrasaccharide (Hex4) Level
Description
Levels of urinary Hex4, a biomarker of disease substrate, were measured. The assay specifically targets Hex4, the glucose tetrasaccharide (Glc4), which is a biomarker of glycogen storage.
Time Frame
Baseline, Week 52
Title
Change From Baseline to Week 52 in Serum Creatine Kinase (CK) Level
Description
Change from baseline to Week 52 in serum CK level. CK levels were measured as part of the serum chemistry panel.
Time Frame
Baseline, Week 52
Title
Population Pharmacokinetics (PK): Maximum Observed Concentration (Cmax) of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Experienced Participants Using Plasma Total GAA Protein Level by Signature Peptide Assay and Plasma Miglustat Concentration
Description
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-experienced participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Time Frame
Days 1 and 364 (Week 52)
Title
Population PK: Area Under the Concentration-Time Curve (AUC) of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Experienced Participants Using Plasma Total GAA Protein Level by Signature Peptide Assay and Plasma Miglustat Concentration
Description
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-experienced participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Time Frame
Days 1 and 364 (Week 52)
Title
Population PK: Cmax of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Participants
Description
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-naïve participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Time Frame
Days 1 and 364 (Week 52)
Title
Population PK: AUC of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Subjects
Description
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-naïve participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Time Frame
Days 1 and 364 (Week 52)
Title
Noncompartmental Analysis: Cmax of Plasma Total GAA Protein by Signature Peptide T09 in ERT-Naïve Subjects
Description
A noncompartmental analysis was performed on ERT-naïve subjects, who underwent serial PK sampling during the study. On Day 1, serial blood samples were collected for ERT-naïve participants just prior to initiation of cipaglucosidase alfa/alglucosidase alfa infusion (time 0) and at 1, 2, 3, 3.5, 4, 4.5, 6, 8, 10, and 24 hours after the start of cipaglucosidase alfa/alglucosidase alfa infusion for plasma total human acid α-glucosidase (GAA) protein signature peptide T09 and plasma miglustat determinations.
Time Frame
Day 1
Title
Noncompartmental Analysis: AUC From Time 0 (Predose) to the Time of Last Quantifiable Concentration of Plasma Total GAA Protein by Signature Peptide T09 in ERT-Naïve Subjects
Description
A noncompartmental analysis was performed on ERT-naïve subjects, who underwent serial PK sampling during the study. On Day 1, serial blood samples were collected for ERT-naïve participants just prior to initiation of cipaglucosidase alfa/alglucosidase alfa infusion (time 0) and at 1, 2, 3, 3.5, 4, 4.5, 6, 8, 10, and 24 hours after the start of cipaglucosidase alfa/alglucosidase alfa infusion for plasma total GAA protein signature peptide T09 and plasma miglustat determinations.
Time Frame
Day 1
Title
Comparison of Cmax of Cipaglucosidase Alfa in ERT-Experienced and ERT-Naïve Populations
Description
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Time Frame
Days 1 and 364 (Week 52)
Title
Comparison of AUC of Cipaglucosidase Alfa in ERT- Experienced and ERT-Naïve Populations
Description
On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.
Time Frame
Days 1 and 364 (Week 52)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subject must provide signed informed consent prior to any study-related procedures being performed. Male and female subjects are ≥ 18 years old and weigh ≥ 40 kg at screening. Female subjects of childbearing potential and male subjects must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug. Subject must have a diagnosis of LOPD based on documentation of one of the following: deficiency of GAA enzyme GAA genotyping Subject is classified as one of the following with respect to ERT status: ERT-experienced, defined as currently receiving standard of care ERT (alglucosidase alfa) at the recommended dose and regimen (ie, 20 mg/kg dose every 2 weeks) for ≥ 24 months ERT-naïve, defined as never having received investigational or commercially available ERT Subject has a sitting FVC ≥ 30% of the predicted value for healthy adults (National Health and Nutrition Examination Survey III) at screening. Subject performs two 6MWTs at screening that are valid, as determined by the clinical evaluator, and that meet all of the following criteria: both screening values of 6MWD are ≥ 75 meters both screening values of 6MWD are ≤ 90% of the predicted value for healthy adults the lower value of 6MWD is within 20% of the higher value of 6MWD Exclusion Criteria Subject has received any investigational therapy or pharmacological treatment for Pompe disease, other than alglucosidase alfa, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before Day 1 or is anticipated to do so during the study. Subject has received gene therapy for Pompe disease Subject is taking any of the following prohibited medications within 30 days before Day 1: miglitol (eg, Glyset) miglustat (eg, Zavesca) acarbose (eg, Precose or Glucobay) voglibose (eg, Volix, Vocarb, or Volibo) Note: None of these medications have a half-life that, when multiplied by 5, is longer than 30 days. Subject requires the use of invasive or noninvasive ventilation support for > 6 hours per day while awake. Subject has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221. Subject has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the subject or may compromise his/her ability to comply with or adversely impact protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms. Subject, if female, is pregnant or breastfeeding at screening. Subject, whether male or female, is planning to conceive a child during the study. Subject does not have documentation of diagnosis of Pompe disease and refuses to undergo genetic testing.
Facility Information:
Facility Name
Neuromuscular Research Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85028
Country
United States
Facility Name
University of Arkansas for Medical Sciences
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Facility Name
University of California, Irvine
City
Irvine
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
UF Helath: University of Florida Clinical Research Center
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
University of South Florida Research Center
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Emory Clinic
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Indiana University Health Neuroscience Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66205
Country
United States
Facility Name
University of Minnesota Clinical Research Unit
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Billings Clinic
City
Billings
State/Province
Montana
ZIP/Postal Code
59101
Country
United States
Facility Name
Hackensack University Medical Center
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
Facility Name
The Feinstein Institute for Medical Research
City
Manhasset
State/Province
New York
ZIP/Postal Code
11030
Country
United States
Facility Name
NYU School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10017
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
University of Cincinnati Neurology
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45219
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
The Ohio State University Wexner Medical Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Penn State Health Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
University of Texas Health Science Center San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
University of Utah, Center for Clinical and Translational Sciences
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
Lysosomal and Rare Disorders Research
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
Hospital Universitario Austral
City
Buenos Aires
ZIP/Postal Code
B1629ODT
Country
Argentina
Facility Name
Royal Brisbane & Women's Hospital
City
Herston
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Facility Name
Royal Adelaide Hospital
City
Adelaide
State/Province
South Australia
ZIP/Postal Code
5000
Country
Australia
Facility Name
Monash Medical Centre
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia
Facility Name
Westmead Hospital
City
Westmead
ZIP/Postal Code
2145
Country
Australia
Facility Name
Medizinische Universität Innsbruck
City
Innsbruck
Country
Austria
Facility Name
UZ Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
University Clinical Centre of the Republic of Srpska
City
Banja Luka
ZIP/Postal Code
78000
Country
Bosnia and Herzegovina
Facility Name
UMHAT Alexandrovska
City
Sofia
Country
Bulgaria
Facility Name
Heritage Medical Research Clinic, University of Calgary
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 4Z6
Country
Canada
Facility Name
McMaster University Medical Centre
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Aarhus Universitets Hospital
City
Aarhus N
ZIP/Postal Code
8200
Country
Denmark
Facility Name
Rigshospitalet Copenhagen Neuromuscular Center
City
Copenhagen
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Hôpital Neurologique Pierre Wertheimer
City
Bron
ZIP/Postal Code
69677
Country
France
Facility Name
Hôpital Raymond Poincaré
City
Garches
ZIP/Postal Code
92380
Country
France
Facility Name
Hôpital Salengro
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
Hôpital de la Timone
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
Hôpital Pasteur
City
Nice
ZIP/Postal Code
06001
Country
France
Facility Name
Friedrich-Baur Institut
City
Munich
State/Province
Bavaria
ZIP/Postal Code
80336
Country
Germany
Facility Name
Universitätsklinikum Bonn
City
Bonn
State/Province
NRW
ZIP/Postal Code
53105
Country
Germany
Facility Name
Universitätsklinikum Münster
City
Münster
State/Province
NRW
ZIP/Postal Code
48149
Country
Germany
Facility Name
Universitätsklinikum Halle (Saale)
City
Halle (Saale)
State/Province
Sachsen-Anhalt
ZIP/Postal Code
06120
Country
Germany
Facility Name
Eginition Hospital
City
Athens
State/Province
Attica
ZIP/Postal Code
11528
Country
Greece
Facility Name
Semmelweis University, Institute of Genomic Medicine and Rare Disease
City
Budapest
ZIP/Postal Code
1083
Country
Hungary
Facility Name
University of Pécs
City
Pécs
ZIP/Postal Code
7623
Country
Hungary
Facility Name
University of Szeged
City
Szeged
ZIP/Postal Code
6725
Country
Hungary
Facility Name
UOC di Neurologia e Malattie Neuromuscolari
City
Messina
State/Province
NAP
ZIP/Postal Code
98125
Country
Italy
Facility Name
UOC Genetica Medica
City
Napoli
State/Province
NAP
ZIP/Postal Code
80131
Country
Italy
Facility Name
Hokkaido University Hospital
City
Sapporo
State/Province
Hokkaido
ZIP/Postal Code
060 8648
Country
Japan
Facility Name
Fukuoka University Hospital
City
Fukuoka
ZIP/Postal Code
814-0180
Country
Japan
Facility Name
Kagoshima University Hospital
City
Kagoshima
Country
Japan
Facility Name
Izumi City General Hospital
City
Osaka
Country
Japan
Facility Name
The Jikei University Hospital
City
Tokyo
ZIP/Postal Code
105-8471
Country
Japan
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
Country
Japan
Facility Name
Pusan National University
City
Yangsan
State/Province
Gyeongsangnam-do
ZIP/Postal Code
50612
Country
Korea, Republic of
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Erasmus MC
City
Rotterdam
ZIP/Postal Code
3015GD
Country
Netherlands
Facility Name
University of Auckland
City
Auckland
Country
New Zealand
Facility Name
Szpital Uniwersytecki w Krakowie
City
Małogoskie
ZIP/Postal Code
31-066
Country
Poland
Facility Name
Centrum Medyczne
City
Rzeszów
ZIP/Postal Code
35-326
Country
Poland
Facility Name
University Medical Centre Ljubljana
City
Ljubljana
ZIP/Postal Code
1000
Country
Slovenia
Facility Name
Hospital de la Santa Creu I Sant Pau
City
Barcelona
ZIP/Postal Code
08026
Country
Spain
Facility Name
Sahlgrenska University Hospital
City
Gothenburg
ZIP/Postal Code
41345
Country
Sweden
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
Queen Elizabeth Hospital Birmingham
City
Birmingham
Country
United Kingdom
Facility Name
Cambridge University Hospitals
City
Cambridge
Country
United Kingdom
Facility Name
Royal Free Hospital NHS
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Salford Royal NHS Foundation Trust
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
34800400
Citation
Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforet P, Toscano A, Castelli J, Diaz-Manera J, Goldman M, van der Ploeg AT, Bratkovic D, Kuchipudi S, Mozaffar T, Kishnani PS; PROPEL Study Group. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial. Lancet Neurol. 2021 Dec;20(12):1027-1037. doi: 10.1016/S1474-4422(21)00331-8. Erratum In: Lancet Neurol. 2023 Oct;22(10):e11.
Results Reference
derived

Learn more about this trial

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

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