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NCT06735755 · ClinicalTrials.gov registry record · Phase 1
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 study of Glycogen Storage Disease Type Ia, sponsored by Beam Therapeutics.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 36
- Enrollment target
- 3
- Study locations
NCT06735755: Recruiting Phase 1 study of Glycogen Storage Disease Type Ia, sponsored by Beam Therapeutics.
NCT06735755 is a Phase 1 study of Glycogen Storage Disease Type Ia that is actively recruiting participants, run by Beam Therapeutics. The registered enrollment target is 36 participants, below the 44-participant average among 5 other Glycogen Storage Disease Type Ia trials with a reported enrollment target (18% lower). The trial reports 3 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06735755, a Phase 1 study of Glycogen Storage Disease Type Ia, is actively recruiting participants, sponsored by Beam Therapeutics.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 36 participants
- Enrollment target
- 3
- Study locations
Study 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.
Conditions Studied
Interventions
- DRUG BEAM-301: Single dose of BEAM-301 administered by IV
Study Locations (3)
California
- Clinical Study Site - Orange
Connecticut
- Clinical Study Site - Farmington
Texas
- Clinical Study Site - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2024-12-06 |
| Est. Completion | 2027-12-30 |
| Phase | Phase 1 |
What NCT06735755 shows while recruiting
NCT06735755 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 36 participants, a relatively small participant target, below the 44-participant average among 5 other Glycogen Storage Disease Type Ia trials with a reported enrollment target (18% lower).
The record links to 1 condition, with Glycogen Storage Disease Type Ia appearing as the primary indexed condition, and to 1 intervention - of which BEAM-301: Single dose of BEAM-301 administered by IV is the first listed.
NCT06735755 reports a single indexed study location in California, Connecticut, Texas.
Frequently Asked Questions
What is clinical trial NCT06735755 about?
NCT06735755 is a clinical study titled "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.
What is the current status of trial NCT06735755?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 36 participants. The study started on 2024-12-06. Estimated completion is 2027-12-30.
What conditions does trial NCT06735755 study?
This clinical trial studies the following conditions: Glycogen Storage Disease Type Ia.
What interventions are being tested in trial NCT06735755?
The interventions under investigation include: BEAM-301: Single dose of BEAM-301 administered by IV (DRUG).
Who is sponsoring clinical trial NCT06735755?
This trial is sponsored by Beam Therapeutics, which has 5 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06735755 being conducted?
This trial has 3 study locations across California, Connecticut, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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