China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
AgeUp to 17
SponsorGenzyme, a Sanofi Company
About this trial
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. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks) * The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.
Eligibility criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Avalglucosidase alfa
Treatment groups
13 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov