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) Homozygous or Compound Heterozygous for the G6PC1 c.247C>T (p.R83C) Variant

Beam Therapeutics Inc.1 site in 1 country36 target enrollmentDecember 6, 2024

ConditionsGlycogen Storage Disease Type Ia

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

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Glycogen Storage Disease Type Ia
Sponsor: Beam Therapeutics Inc.
Enrollment: 36
Locations: 1
Primary Endpoint: 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
Status: Recruiting
Last Updated: 4 months ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

December 6, 2024

End Date

December 30, 2027

Last Updated

4 months ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Beam Therapeutics Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•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.

Exclusion Criteria

•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).

Outcomes

Primary Outcomes

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

Secondary Outcomes

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)