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)

Recruiting now · Phase 1/Phase 2

Conditions studied: Glycogen Storage Disease Type Ia

In brief

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 facts

Study ID

NCT06735755

Run by

Beam Therapeutics Inc.

People needed

36

Starts

2024-12-06

Expected to finish

2027-12-30

Last updated by the study team

2025-12-15

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

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).

Where it is running

• Clinical Study Site — Orange, California, United States (enrolling)
• Clinical Study Site — Farmington, Connecticut, United States (enrolling)
• Clinical Study Site — Houston, Texas, United States (enrolling)

Full record on ClinicalTrials.gov

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