clinical trial · NCT06735755
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)
Glycogen Storage Disease Type IaBEAM-301: Single dose of BEAM-301 administered by IV
brief 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.
started
Dec 6, 2024
primary completion
Dec 30, 2027
completion
Dec 30, 2027
last updated
Dec 15, 2025
official 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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol