China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Recruiting
Phase 4
Interventional Study
Glycogen Storage Disease Type II
Pompe's Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- Up to 17
- Sex
- Any
- Trial phase
- Phase 4
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 13 (estimated)
- Sponsor
- Genzyme, a Sanofi Company · Industry
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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
* …
Trial Locations
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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 defici…
Contacts
Trial Transparency email recommended (Toll free for US & Canada)
800-633-1610
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