RecruitingPhase 1Phase 2NCT06735755

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)

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

Sponsor

Beam Therapeutics Inc.

Enrollment

36 participants

Start Date

Dec 6, 2024

Study Type

INTERVENTIONAL

Conditions

Glycogen Storage Disease Type Ia

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.

Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called BEAM-301: Single dose of BEAM-301 administered by IV for people with glycogen storage disease type ia. The study is currently recruiting participants at 3 locations. People eligible for this study include aged 18 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGBEAM-301: Single dose of BEAM-301 administered by IV

BEAM-301 is designed to correct the G6PC1 c.247C\>T allele via an A:T-to-G:C base-pair substitution, resulting in restoration of G6Pase-α catalytic activity.