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
• Last Update Submitted: 2024-12-11
• Study First Posted: 2024-12-16
• Last Update Posted: 2024-12-16
• 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.

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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).

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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
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
BEAM-301 PK parameters (half-life)	Baseline to month 24

Trial Locations

Locations (1)

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