Apply to trial NCT06735755

A few quick questions so the study team can decide if you might be a fit.

RecruitingDrug 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)

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.

How this works

  1. Answer a few questions

    About 5 to 10 minutes. Skip-friendly where possible.

  2. We connect you with the study team

    When a site picks up your application, we introduce you to their team directly — with you on the thread.

  3. The team reaches out to schedule screening

    Usually within a few business days, via the contact you give.

We save your progress under this email and copy you when we connect you with the study team.

By continuing, you agree to our terms of service.