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Clinical Study for IOPD Participants Less Than or Equal to 6 Months of Age to Evaluate Efficacy and Safety of Enzyme Replacement Therapy (ERT) With Avalglucosidase Alfa (Baby-COMET)

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ClinicalTrials.gov Identifier: NCT04910776
Recruitment Status : Recruiting
First Posted : June 2, 2021
Last Update Posted : September 5, 2021
Sponsor:
Information provided by (Responsible Party):
Sanofi

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 male and female participants less than or equal to 6 months of age 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

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type II Drug: avalglucosidase alfa Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Less Than or Equal to 6 Months of Age With Infantile-Onset Pompe Disease (IOPD)
Actual Study Start Date : September 1, 2021
Estimated Primary Completion Date : May 2024
Estimated Study Completion Date : December 2026


Arm Intervention/treatment
Experimental: Avalglucosidase alfa
Administered intravenously every 2 weeks
Drug: avalglucosidase alfa
Sterile lyophilized powder intravenous (IV) infusion
Other Name: GZ402666




Primary Outcome Measures :
  1. Proportion of participants who are alive and free of invasive ventilation at Week 52 [ Time Frame: Week 52 ]

Secondary Outcome Measures :
  1. 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 ]
  2. Proportion of participants who are alive at Week 52 [ Time Frame: Week 52 ]
  3. Proportion of participants who are alive at 12 and 18 months of age [ Time Frame: at 12 and 18 months of age ]
  4. Proportion of participants who are free of ventilator use and free of supplemental oxygen use at Week 52 [ Time Frame: Week 52 ]
  5. Change from baseline to Week 52 in LVM-Z score [ Time Frame: Week 52 ]
  6. Change from baseline to Week 52 in AIMS score [ Time Frame: Week 52 ]
  7. Change from baseline to Week 52 in body length Z-scores [ Time Frame: Week 52 ]
  8. Change from baseline to Week 52 in body weight Z-scores [ Time Frame: Week 52 ]
  9. Change from baseline to Week 52 in head circumference Z-scores [ Time Frame: Week 52 ]
  10. Change from baseline to Week 52 in body length percentiles [ Time Frame: Week 52 ]
  11. Change from baseline to Week 52 in body weight percentiles [ Time Frame: Week 52 ]
  12. Change from baseline to Week 52 in head circumference percentiles [ Time Frame: Week 52 ]
  13. Change from baseline to Week 52 in urinary Hex4 [ Time Frame: Week 52 ]
  14. Number of participants experiencing at least 1 treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR) [ Time Frame: Week 52, Week 212 ]
  15. Number of participants with abnormalities in physical examinations [ Time Frame: Week 52, Week 208 ]
  16. Number of participants with potentially clinically significant abnormality (PCSA) in clinical laboratory results [ Time Frame: Week 52, Week 208 ]
  17. Number of participants with PCSA in vital signs measurements [ Time Frame: Week 52, Week 208 ]
  18. Number of participants with PCSA in 12-lead electrocardiogram (ECG) [ Time Frame: Week 52, Week 208 ]
  19. Incidence of treatment-emergent anti-drug antibodies (ADA) [ Time Frame: Week 52, Week 208 ]
  20. Plasma concentration of avalglucosidase alfa [ Time Frame: at Day 1, Week 12, and Week 52 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 6 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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, 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04910776


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext option 6 Contact-US@sanofi.com

Locations
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Belgium
Investigational Site Number 0560001 Recruiting
Leuven, Belgium, 3000
Germany
Investigational Site Number 2760001 Recruiting
Gießen, Germany, 35390
Italy
Investigational Site Number 3800001 Recruiting
Firenze, Italy, 50139
Taiwan
Investigational Site Number 1580001 Recruiting
Taipei, Taiwan, 100
Sponsors and Collaborators
Sanofi
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Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT04910776    
Other Study ID Numbers: EFC14462
2020-004686-39 ( EudraCT Number )
U1111-1246-6645 ( Registry Identifier: ICTRP )
First Posted: June 2, 2021    Key Record Dates
Last Update Posted: September 5, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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://www.clinicalstudydatarequest.com/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Lysosomal Storage Diseases