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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)

StatusRecruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study sites: 2 to 3v3
  2. Study sites: 1 to 2v2
  3. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT06735755
Lead Sponsor Beam Therapeutics Inc.
Conditions
(from sponsor)		 Glycogen Storage Disease Type Ia
Related terms Hepatorenal form of glycogen storage disease
Enrollment 36 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov