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.
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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

contact-us@sanofi.com

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