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)

Recruiting Phase 1/2
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Summary
This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant and to determine the optimal biological dose.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2024-12-11; most recent amendment 2025-12-09.
Trial Details
NCT Number NCT06735755
Lead Sponsor Beam Therapeutics Inc.
Conditions Glycogen Storage Disease Type Ia
Enrollment 36 participants
Start Date 2024-12-06
Primary Completion 2027-12-30 (estimated)
Study Completion 2027-12-30 (estimated)
Updated on ClinicalTrials.gov 2025-12-15