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)

Phase 1

Recruiting

• Conditions: Glycogen Storage Disease Type Ia

• Registration Number: NCT06735755
• Lead Sponsor: Beam Therapeutics Inc.

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-12
• Study Start: 2024-12-06
• Study First Submitted: 2024-12-06
• Study First Submitted QC: 2024-12-11
• Study First Posted: 2024-12-16
• Last Update Submitted: 2025-04-22
• Last Update Posted: 2025-04-24
• Primary Completion: 2027-12-30
• Study Completion: 2027-12-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

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

Exclusion Criteria

1. Liver transplant recipient/on waiting list for liver transplant or known history of liver cirrhosis.
2. 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.
3. Presence of liver adenoma >3 cm and ≤5 cm with a documented annual growth rate of ≥0.5 cm per year.
4. Have aspartate transaminase or alanine transaminase >upper limit of normal (ULN).
5. Total bilirubin levels >ULN; if documented Gilbert's Syndrome, total bilirubin >2 × ULN.
6. Previous treatment with any cell-, gene-, or viral vector-derived therapy Hospitalization for management of hypoglycemia within 4 weeks prior to signing the ICF.
7. 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).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Absolute and change from baseline in time to hypoglycemia or metabolic decompensation during a controlled fasting challenge	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
BEAM-301 PK parameters (half-life)	Baseline to month 24
Changes from baseline in starch supplementation dose	Baseline to month 24

Trial Locations

Locations (1)

Clinical Study Site; 🇺🇸 Farmington, Connecticut, United States