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

  • STATUS
    Recruiting
  • End date
    Aug 28, 2026
  • participants needed
    18
  • sponsor
    Sanofi
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Updated on 19 July 2024
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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

Description

Study duration may be variable by country, including at least completion of the PAP and ETP, and up to 4.08 years.

Details
Condition Glycogen Storage Disease Type II
Age 12months or below
Treatment avalglucosidase alfa
Clinical Study IdentifierNCT04910776
SponsorSanofi
Last Modified on19 July 2024

Eligibility

 Yes No Not Sure

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
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