Clinical trial NCT06666413: China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Official title: A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Glycogen Storage Disease Type II
Pompe's Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2024-10-30
Enrollment: 13
Sex: ALL
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT.

Study details include:

* The study duration: total study duration is approximately 64 weeks.

  * Screening period of up to 8 weeks
  * Treatment period of 52 weeks
  * Follow-up period of 4 weeks.
* The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.


Eligibility criteria: Inclusion Criteria:

* Participant must be \<18 years of age, at the time of signing the informed consent.
* Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations.
* Participants must have documented cardiomyopathy at the time of diagnosis.
* Contraceptive use should be consistent with local regulations Participant's parent/legally authorized representative (LAR) must be capable of giving signed informed consent.

Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:

* Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
* Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
* Participants who have received an ERT other than alglucosidase alfa or avalglucosidase alfa, or any other treatment for Pompe disease, including gene therapy prior to the enrollment.
* Participants who have received alglucosidase alfa or avalglucosidase alfa less than 1 week prior to the first dose of avalglucosidase alfa given as IMP Participants who are anticipated to take prohibited therapy (ie, any other treatment for Pompe disease) during this study.
* Participants who have taken other investigational drugs (not Pompe disease specific) within 30 days or 5 elimination half-lives in blood of that drug before enrollment, whichever is longer, or are anticipated to take any other concurrent investigational treatments.
* Participants 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.

Primary outcomes:
measure: Incidence of AEs, SAEs, AESIs including IARs during the TE period
timeFrame: From baseline to Week 56
measure: Abnormality in clinical laboratory, vital signs, and ECG parameters during the TE period
timeFrame: From baseline to Week 56
Secondary outcomes:
measure: Proportion of participants who are alive and free of invasive ventilation at Week 52
timeFrame: At Week 52
measure: Change from baseline to Week 52 in LVM Z score evaluated by echocardiography
timeFrame: From baseline to Week 52
measure: The absolute change in body growth Z scores from baseline to Week 52
timeFrame: From baseline to Week 52
measure: The percentiles of the body growth Z scores changes from baseline to Week 52
timeFrame: From baseline to Week 52
measure: Change from baseline to Week 52 in distance walked during six-minute walk test (6MWT) (in applicable participants)
timeFrame: From baseline to Week 52
measure: Change from baseline to Week 52 in quick motor function test (QMFT) score (in applicable participants)
timeFrame: From baseline to Week 52
measure: Number of motor development milestones achieved as evaluated by Motor Milestone Checklist at Week 52
timeFrame: At Week 52
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Investigational Site Number: 1560001
Study officials:
References: