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Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen (APOLLO-LOPD)

Primary Purpose

Pompe's Disease

Status
Recruiting
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
ALGLUCOSIDASE ALFA
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pompe's Disease

Eligibility Criteria

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

Inclusion criteria :

  • Patients (or and patient's legal guardian) must provide written informed consent prior to any study-related procedures

  • The patient must be ≥ 3 years of age at the time of enrollment.

    1. For patient ≥ 3-year and < 5-year old: must be able to walk 10 meters or climb 4-step stairs independently.
    2. For patients ≥5-year old

    i. Must be able to ambulate 40 meters in 6 minutes without assistance ii. Must be able to successfully perform repeated forced vital capacity (VC) measurements in upright position of ≥ 30% predicted and ≤85% predicted.

  • The patient has confirmed Pompe's Disease with at least 2 of the following condition,

    1. GAA enzyme deficiency from any tissue source.
    2. 2 confirmed GAA gene mutations.
    3. muscle pathology meet the diagnosis of Pompe disease.
  • The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol.
  • The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline.

Exclusion criteria:

  • Use of invasive ventilatory support (Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.)
  • Use of non-invasive ventilatory support while awake and in an upright position. (Non-invasive ventilation is defined as any form of ventilatory support applied without the use of an endotracheal tube.)
  • Previously treated with Enzyme Replacement Treatment.
  • A Female patient of childbearing potential with a positive pregnancy test.
  • Wheelchair dependent.
  • The patient has a major congenital anomaly.
  • The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
  • The patients with ≥5-year old are unable to ambulate 40 meters without assistance or unable to successfully perform repeated FVC of >30% and <85% predicted (upright).

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sites / Locations

  • Investigational SiteRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Alglucosidase Alfa

Arm Description

A dose of 20 mg/kg body weight once every 2 weeks for a minimum of 52 weeks

Outcomes

Primary Outcome Measures

Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old
This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.
Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Number of participants with adverse events Number of AEs
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.

Secondary Outcome Measures

Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients ≥5-year old
MMT has been reported most often as a summary score of a total number of proximal, distal, and axial muscle groups tested bilaterally or as a proximal score that sums a number of proximal muscle groups from the upper and lower extremities.
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
The Quick Motor Function Test is a reliable and valid test for assessing motor function in patients with Pompe's disease.
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
The 12-Item Short Form Health Survey (SF-12) was developed for the Medical Outcomes evaluation of patients with chronic conditions.

Full Information

First Posted
December 15, 2020
Last Updated
September 26, 2023
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT04676373
Brief Title
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
Acronym
APOLLO-LOPD
Official Title
A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 10, 2021 (Actual)
Primary Completion Date
July 25, 2024 (Anticipated)
Study Completion Date
July 25, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

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

5. Study Description

Brief Summary
Primary Objective: To evaluate the effect of one-year Alglucosidase alfa treatment on motor function [Six-minute walk test (6MWT) and lung function predicted Forced vital capacity (FVC)] among Chinese Late Onset Pompe Disease patients above 5 years old. To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years old. Secondary Objective: To evaluate the effect of one-year treatment with Alglucosidase alfa on improvement of manual muscle test (MMT), Maximal inspiratory and expiratory pressure (MIP and MEP)], Quick Motor Function Test scores, and health-related quality of life (SF-12) among LOPD patients over 5 years old.
Detailed Description
Study duration per participants is approximatively 56 weeks including a 52-week treatment period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe's Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Alglucosidase Alfa
Arm Type
Experimental
Arm Description
A dose of 20 mg/kg body weight once every 2 weeks for a minimum of 52 weeks
Intervention Type
Drug
Intervention Name(s)
ALGLUCOSIDASE ALFA
Other Intervention Name(s)
MYOZYME
Intervention Description
Pharmaceutical form:solution for infusion Route of administration: intravenous
Primary Outcome Measure Information:
Title
Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old
Description
This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.
Time Frame
Baseline to 12 months
Title
Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old
Description
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Time Frame
Baseline to 12 months
Title
Number of participants with adverse events Number of AEs
Description
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Time Frame
From signing the ICF to the 30th day after the last dosage of the study medications
Secondary Outcome Measure Information:
Title
Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old
Description
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Time Frame
Week 52
Title
Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old
Description
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Time Frame
Week 52
Title
Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients ≥5-year old
Description
MMT has been reported most often as a summary score of a total number of proximal, distal, and axial muscle groups tested bilaterally or as a proximal score that sums a number of proximal muscle groups from the upper and lower extremities.
Time Frame
Week 52
Title
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
Description
The Quick Motor Function Test is a reliable and valid test for assessing motor function in patients with Pompe's disease.
Time Frame
Week 52
Title
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
Description
The 12-Item Short Form Health Survey (SF-12) was developed for the Medical Outcomes evaluation of patients with chronic conditions.
Time Frame
Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria : Patients (or and patient's legal guardian) must provide written informed consent prior to any study-related procedures The patient must be ≥ 3 years of age at the time of enrollment. For patient ≥ 3-year and < 5-year old: must be able to walk 10 meters or climb 4-step stairs independently. For patients ≥5-year old i. Must be able to ambulate 40 meters in 6 minutes without assistance ii. Must be able to successfully perform repeated forced vital capacity (VC) measurements in upright position of ≥ 30% predicted and ≤85% predicted. The patient has confirmed Pompe's Disease with at least 2 of the following condition, GAA enzyme deficiency from any tissue source. 2 confirmed GAA gene mutations. muscle pathology meet the diagnosis of Pompe disease. The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol. The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline. Exclusion criteria: Use of invasive ventilatory support (Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.) Use of non-invasive ventilatory support while awake and in an upright position. (Non-invasive ventilation is defined as any form of ventilatory support applied without the use of an endotracheal tube.) Previously treated with Enzyme Replacement Treatment. A Female patient of childbearing potential with a positive pregnancy test. Wheelchair dependent. The patient has a major congenital anomaly. The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities. The patients with ≥5-year old are unable to ambulate 40 meters without assistance or unable to successfully perform repeated FVC of >30% and <85% predicted (upright). The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Trial Transparency email recommended (Toll free number for US & Canada)
Phone
800-633-1610
Ext
option 6
Email
Contact-US@sanofi.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
Investigational Site
City
China
Country
China
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

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Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen

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