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)

NCT06735755 | Recruiting Phase 1 FDA Drug

• 1 other identifier: BTX-301-001
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 3

Brief 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

Glycogen Storage Disease Type Ia

Keywords

GSDIa; GSD1a

Outcome Measures

Primary Outcomes (1)

• 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

  BEAM-301 administration through month 24

Secondary Outcomes (6)

• Absolute and change from baseline in time to hypoglycemia or metabolic decompensation during a controlled fasting challenge

  Baseline to month 24

• Changes from baseline in starch supplementation dose

  Baseline to month 24

• Level of serum glucose and metabolic parameters (lipid profile and uric acid) over time

  Baseline to month 24

• BEAM-301 PK parameters (Cmax)

  Baseline to month 24

• BEAM-301 PK parameters (AUC)

  Baseline to month 24

Study Arms (1)

BEAM-301

EXPERIMENTAL

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.

Drug: BEAM-301: Single dose of BEAM-301 administered by IV

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.

BEAM-301

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females age ≥18 years of age at the time of consent.
• Diagnosis of GSDIa and homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant (confirmed by genetic testing).
• History of at least 1 episode of hypoglycemia \<60 mg/dL within the 2 years prior to signing the ICF.

You may not qualify if:

• Liver transplant recipient/on waiting list for liver transplant or known history of liver cirrhosis.
• Presence of liver adenoma \>5 cm in size based on liver MRI or liver ultrasound performed at screening or within 6 months prior to screening.
• Presence of liver adenoma \>3 cm and ≤5 cm with a documented annual growth rate of ≥0.5 cm per year.
• Have aspartate transaminase or alanine transaminase \>upper limit of normal (ULN).
• Total bilirubin levels \>ULN; if documented Gilbert's Syndrome, total bilirubin \>2 × ULN.
• Previous treatment with any cell-, gene-, or viral vector-derived therapy Hospitalization for management of hypoglycemia within 4 weeks prior to signing the ICF.
• Have triglycerides \>1000 mg/dL or a history of pancreatitis or a history of acute pancreatitis within 5 years that does not have a known and corrected etiology (eg, cholecystectomy for gallstone pancreatitis).

Sponsors & Collaborators

• Beam Therapeutics Inc.: lead

Study Sites (3)

Clinical Study Site

Orange, California, 92868, United States

RECRUITING

Clinical Study Site

Farmington, Connecticut, 06030, United States

RECRUITING

Clinical Study Site

Houston, Texas, 77030, United States

RECRUITING

MeSH Terms

Conditions

Hepatorenal form of glycogen storage disease

Central Study Contacts

Medical Information

CONTACT

857-327-8641; clinicalinfo@beamtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: up to 4 planned, single-ascending-dose, dose-exploration cohorts

Study Record Dates

• First Submitted: December 6, 2024
• First Posted: December 16, 2024
• Study Start: December 6, 2024
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): December 30, 2027
• Last Updated: December 15, 2025

Record last verified: 2025-12

Locations

US (3)