● Recruiting

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)

Condition: Glycogen Storage Disease Type Ia · Sponsor: Beam Therapeutics Inc.

PhasePhase 1/Phase 2

Planned participants36

Who can joinAll sexes, 18 Years to no upper limit

Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Medical Information · 857-327-8641 · clinicalinfo@beamtx.com

Always discuss trial participation with your own doctor first.

Locations (3)

Clinical Study Site	Orange, California, United States	Recruiting
Clinical Study Site	Farmington, Connecticut, United States	Recruiting
Clinical Study Site	Houston, Texas, United States	Recruiting

Follow this study

Get one email when the public record changes — results posted, or the study's status changes. Nothing else, ever.

We email about this public record only. Unsubscribe anytime with one click. Never medical advice.

Is this your study? This page was generated automatically from the public registry record. Sponsors can claim it — free — to add branding and verified contact routing. Claim this page →

This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.

Source record: clinicaltrials.gov/study/NCT06735755