Actively Recruiting
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)
Led by Beam Therapeutics Inc. · Updated on 2025-12-15
36
Participants Needed
3
Research Sites
159 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males or females age 6518 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 will not qualify if you...
- Liver transplant recipient, on waiting list for liver transplant, or known history of liver cirrhosis.
- Presence of liver adenoma larger than 5 cm based on liver MRI or ultrasound at screening or within 6 months prior.
- Presence of liver adenoma between 3 cm and 5 cm with documented annual growth rate of 0.5 cm or more per year.
- Aspartate transaminase or alanine transaminase above the upper limit of normal.
- Total bilirubin levels above the upper limit of normal; if Gilbert's Syndrome, total bilirubin greater than 2 times upper limit.
- Previous treatment with any cell-, gene-, or viral vector-derived therapy.
- Hospitalization for management of hypoglycemia within 4 weeks prior to signing the consent form.
- Triglycerides above 1000 mg/dL or history of pancreatitis or acute pancreatitis within 5 years without known and corrected cause (e.g., gallstone pancreatitis with cholecystectomy).
AI-Screening
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Trial Site Locations
Total: 3 locations
1
Clinical Study Site
Orange, California, United States, 92868
Actively Recruiting
2
Clinical Study Site
Farmington, Connecticut, United States, 06030
Actively Recruiting
3
Clinical Study Site
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
M
Medical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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