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Clinical trial

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) Homozygous or Compound Heterozygous for the G6PC1 c.247C>T (p.R83C) Variant

NCT06735755 is registered in ClinicalTrials.gov with Beam Therapeutics Inc. as lead sponsor.

NCT NCT06735755
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) Homozygous or Compound Heterozygous for the G6PC1 c.247C>T (p.R83C) Variant
Lead sponsor
Beam Therapeutics Inc.
Phase
Phase 1/2
Overall status
Recruiting
Start date
December 6, 2024
Completion date
December 30, 2027
Enrollment
36 (estimated)
Sites
3

Conditions

As the registry lists them; a condition with its own page is linked.

glycogen storage disease type ia