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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 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.

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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

Glycogen Storage Disease Type Ia

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

FieldValue
Enrollment Target 36 participants
Start Date 2024-12-06
Est. Completion 2027-12-30
Phase Phase 1

Sponsor

Beam Therapeutics

5 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06735755

The ClinicalTrials.gov registry entry for NCT06735755 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 36 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 44-participant average among 5 other Glycogen Storage Disease Type Ia trials with a reported enrollment target (18% lower). The listed sponsor is Beam Therapeutics, which has 5 total studies on file at ClinicalTrials.gov.

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 3 study locations spanning 3 distinct geographic areas - top geographies include 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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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.