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)

Part of paid clinical trials in Orange, California.

Sponsor
Beam Therapeutics Inc.
Study ID
NCT06735755
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Glycogen Storage Disease Type Ia

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • BEAM-301: Single dose of BEAM-301 administered by IV — DRUG
    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.

Study Details

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.

Key Dates

Start date
Dec 6, 2024
Status verified
Dec 2025
Primary completion
Dec 30, 2027
Completion
Dec 30, 2027

Study Design

Enrollment
36 participants (estimated)
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: BEAM-301
    BEAM-301 consists of adenine base editor messenger ribonucleic acid (mRNA) and single guide ribonucleic acid (gRNA) formulated in lipid nanoparticles (LNPs) for intravenous (IV) administration.

Primary Outcome Measure

To evaluate the safety and tolerability of BEAM-301 in patients with GSDIa with a G6PC1 c.247C>T (p.R83C) variant by measuring incidence of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and dose-limiting toxicities (DL [ Time Frame: BEAM-301 administration through month 24 ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Clinical Study SiteOrangeCalifornia92868-
Clinical Study SiteFarmingtonConnecticut06030-
Clinical Study SiteHoustonTexas77030-

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By research site
Clinical Study Site· Orange, CAClinical Study Site· Farmington, CTClinical Study Site· Houston, TX

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