Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
PEARL
NCT04532047Phase 1Recruiting
- Sponsor
- University of California, San Francisco
- Start
- Jul 2021
- Primary completion
- Jul 2031
- Modality
- Replacement Enzymes
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of laronidase, idursulfase, elosulfase alfa, galsulfase, vestronidase alfa and 2 more in mucopolysaccharidosis type I, mucopolysaccharidosis type II, Mucopolysaccharidosis type IV A and 6 more, sponsored by University of California, San Francisco. Primary endpoints: Number of participants with treatment-related adverse events at 6 years, Number of participants to receive the full initial and subsequent doses of ERT at 6 years and 2 more. Started Jul 2021; status: recruiting.
02
Drugs
laronidaseidursulfaseelosulfase alfagalsulfasevestronidase alfa
03
Conditions
mucopolysaccharidosis type Imucopolysaccharidosis type IIMucopolysaccharidosis type IV Amucopolysaccharidosis type VImucopolysaccharidosis type VII
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