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Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa (Baby-COMET)

Primary Purpose

Glycogen Storage Disease Type II

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
avalglucosidase alfa
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Glycogen Storage Disease Type II

Eligibility Criteria

0 Days - 12 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Participants must have confirmed diagnosis of infantile-onset Pompe disease defined as: the presence of 2 lysosomal acid α-glucosidase (GAA) pathogenic variants and a documented GAA deficiency from blood, skin, or muscle tissue; or the presence of 1 GAA pathogenic variant and a documented GAA deficiency from blood, skin and muscle tissue in 2 separate samples (from either 2 different tissues or from the same tissue but at 2 different sampling dates).
  • Participants must have established cross-reactive immunological material (CRIM) status available prior to enrollment.

  • Participants must have cardiomyopathy at the time of diagnosis: ie, left ventricular mass index (LVMI) equivalent to mean age specific LVMI

    • +1 standard deviation for participants diagnosed by newborn screening or sibling screening;
    • +2 standard deviation for participants diagnosed by clinical evaluation.
  • Parents or legally authorized representative(s) must be capable of giving signed informed consent.

Exclusion Criteria:

  • Participants with symptoms of respiratory insufficiency, including any ventilation use (invasive or noninvasive) at the time of enrollment.
  • Participants with major congenital abnormality.
  • Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
  • Participant received enzyme-replacement therapy (ERT) with recombinant human acid α glucosidase (rhGAA) from any source.
  • Participant who has previously been treated in any clinical trial of avalglucosidase alfa.
  • Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.

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

Sites / Locations

  • Boston Children's Health Physicians-Site Number:8400002Recruiting
  • Duke University Medical Center-Site Number:8400004Recruiting
  • Seattle Childrens Hospital and Regional Medical Center-Site Number:8400003Recruiting
  • Investigational Site Number :0560001Recruiting
  • Investigational Site Number :1560002Recruiting
  • Investigational Site Number :1560001Recruiting
  • Investigational Site Number :2760001Recruiting
  • Investigational Site Number :3800002Recruiting
  • Investigational Site Number :3800001Recruiting
  • Investigational Site Number :5280001Recruiting
  • Investigational Site Number :1580001Recruiting
  • Investigational Site Number :8260001Recruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Avalglucosidase alfa

Arm Description

Administered intravenously every 2 weeks

Outcomes

Primary Outcome Measures

Proportion of participants who are alive and free of invasive ventilation at Week 52

Secondary Outcome Measures

Proportion of participants who are alive and free of invasive ventilation at 12 and 18 months of age
Proportion of participants who are alive at Week 52
Proportion of participants who are alive at 12 and 18 months of age
Proportion of participants who are free of ventilator use (invasive and non-invasive separate and combined) at Week 52
Proportion of participants who are free of supplemental oxygen use at Week 52
Change from baseline to Week 52 in left ventricular mass (LVM)-Z score
Change from baseline to Week 52 in Alberta Infant Motor Scale (AIMS) score
Change from baseline to Week 52 in body length Z-scores
Change from baseline to Week 52 in body weight Z-scores
Change from baseline to Week 52 in head circumference Z-scores
Change from baseline to Week 52 in body length percentiles
Change from baseline to Week 52 in body weight percentiles
Change from baseline to Week 52 in head circumference percentiles
Change from baseline to Week 52 in urinary Hex4
Number of participants experiencing at least 1 treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR)
Number of participants with abnormalities in physical examinations
Number of participants with potentially clinically significant abnormality (PCSA) in clinical laboratory results
Number of participants with PCSA in vital signs measurements
Number of participants with PCSA in 12-lead electrocardiogram (ECG)
Incidence of treatment-emergent anti-drug antibodies (ADA)
Plasma concentration of avalglucosidase alfa

Full Information

First Posted
May 25, 2021
Last Updated
September 26, 2023
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT04910776
Brief Title
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
Acronym
Baby-COMET
Official Title
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 1, 2021 (Actual)
Primary Completion Date
December 27, 2024 (Anticipated)
Study Completion Date
March 27, 2026 (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
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: Study duration: Screening - up to 4 weeks; Primary Analysis Period (PAP) - 52 weeks; Extended Treatment Period (ETP) - 52 weeks; Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years. Treatment duration: Up to 4 years Visit frequency: every other week and potentially every week
Detailed Description
Study duration may be variable by country, but until avalglucosidase alfa is approved in the patient's country or up to 4.08 years, whichever comes first.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glycogen Storage Disease Type II

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Avalglucosidase alfa
Arm Type
Experimental
Arm Description
Administered intravenously every 2 weeks
Intervention Type
Drug
Intervention Name(s)
avalglucosidase alfa
Other Intervention Name(s)
GZ402666
Intervention Description
Sterile lyophilized powder intravenous (IV) infusion
Primary Outcome Measure Information:
Title
Proportion of participants who are alive and free of invasive ventilation at Week 52
Time Frame
Week 52
Secondary Outcome Measure Information:
Title
Proportion of participants who are alive and free of invasive ventilation at 12 and 18 months of age
Time Frame
at 12 and 18 months of age
Title
Proportion of participants who are alive at Week 52
Time Frame
Week 52
Title
Proportion of participants who are alive at 12 and 18 months of age
Time Frame
at 12 and 18 months of age
Title
Proportion of participants who are free of ventilator use (invasive and non-invasive separate and combined) at Week 52
Time Frame
Week 52
Title
Proportion of participants who are free of supplemental oxygen use at Week 52
Time Frame
Week 52
Title
Change from baseline to Week 52 in left ventricular mass (LVM)-Z score
Time Frame
Week 52
Title
Change from baseline to Week 52 in Alberta Infant Motor Scale (AIMS) score
Time Frame
Week 52
Title
Change from baseline to Week 52 in body length Z-scores
Time Frame
Week 52
Title
Change from baseline to Week 52 in body weight Z-scores
Time Frame
Week 52
Title
Change from baseline to Week 52 in head circumference Z-scores
Time Frame
Week 52
Title
Change from baseline to Week 52 in body length percentiles
Time Frame
Week 52
Title
Change from baseline to Week 52 in body weight percentiles
Time Frame
Week 52
Title
Change from baseline to Week 52 in head circumference percentiles
Time Frame
Week 52
Title
Change from baseline to Week 52 in urinary Hex4
Time Frame
Week 52
Title
Number of participants experiencing at least 1 treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR)
Time Frame
Week 52, Week 212
Title
Number of participants with abnormalities in physical examinations
Time Frame
Week 52, Week 208
Title
Number of participants with potentially clinically significant abnormality (PCSA) in clinical laboratory results
Time Frame
Week 52, Week 208
Title
Number of participants with PCSA in vital signs measurements
Time Frame
Week 52, Week 208
Title
Number of participants with PCSA in 12-lead electrocardiogram (ECG)
Time Frame
Week 52, Week 208
Title
Incidence of treatment-emergent anti-drug antibodies (ADA)
Time Frame
Week 52, Week 208
Title
Plasma concentration of avalglucosidase alfa
Time Frame
at Day 1, Week 12, and Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
0 Days
Maximum Age & Unit of Time
12 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Participants must have confirmed diagnosis of infantile-onset Pompe disease defined as: the presence of 2 lysosomal acid α-glucosidase (GAA) pathogenic variants and a documented GAA deficiency from blood, skin, or muscle tissue; or the presence of 1 GAA pathogenic variant and a documented GAA deficiency from blood, skin and muscle tissue in 2 separate samples (from either 2 different tissues or from the same tissue but at 2 different sampling dates). Participants must have established cross-reactive immunological material (CRIM) status available prior to enrollment. Participants must have cardiomyopathy at the time of diagnosis: ie, left ventricular mass index (LVMI) equivalent to mean age specific LVMI +1 standard deviation for participants diagnosed by newborn screening or sibling screening; +2 standard deviation for participants diagnosed by clinical evaluation. Parents or legally authorized representative(s) must be capable of giving signed informed consent. Exclusion Criteria: Participants with symptoms of respiratory insufficiency, including any ventilation use (invasive or noninvasive) at the time of enrollment. Participants with major congenital abnormality. Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease). Participant received any Pompe disease specific treatment, eg enzyme-replacement gene therapy (ERT). Participant who has previously been treated in any clinical trial of avalglucosidase alfa. Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures. 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
Boston Children's Health Physicians-Site Number:8400002
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
Individual Site Status
Recruiting
Facility Name
Duke University Medical Center-Site Number:8400004
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Individual Site Status
Recruiting
Facility Name
Seattle Childrens Hospital and Regional Medical Center-Site Number:8400003
City
Seattle
State/Province
Washington
ZIP/Postal Code
98040
Country
United States
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :0560001
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :1560002
City
Qingdao
ZIP/Postal Code
266000
Country
China
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :1560001
City
Shanghai
ZIP/Postal Code
200127
Country
China
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :2760001
City
Gießen
ZIP/Postal Code
35390
Country
Germany
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :3800002
City
Monza
State/Province
Lombardia
ZIP/Postal Code
20900
Country
Italy
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :3800001
City
Firenze
ZIP/Postal Code
50139
Country
Italy
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :5280001
City
Rotterdam
ZIP/Postal Code
3015 CN
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :1580001
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :8260001
City
London
State/Province
London, City Of
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
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

Learn more about this trial

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

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