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) Homozygous or Compound Heterozygous for the G6PC1 c.247C>T (p.R83C) Variant
Led by Beam Therapeutics Inc. · Updated on 2025-12-15
36
Participants Needed
3
Research Sites
N/A
Total Duration
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AI-Summary
What this Trial Is About
Researchers are studying BEAM-301, an investigational treatment, in adults with Glycogen Storage Disease Type Ia (GSDIa) who have a specific genetic variant (G6PC1 c.247C>T). This Phase 1/2 trial aims to evaluate the safety, tolerability, and effectiveness of BEAM-301 and to find the best dose for treatment. The study is open-label and involves multiple centers. Participants will receive a single intravenous dose of BEAM-301, which contains messenger RNA and guide RNA formulated in lipid nanoparticles designed to correct the genetic mutation causing GSDIa. This dose-exploration study will assess how the treatment behaves in the body and its effects over a 24-month period. During the study, participants will be monitored for treatment-emergent adverse events and dose-limiting toxicities. Researchers will also evaluate changes in episodes of low blood sugar, metabolic stability during fasting, starch supplementation needs, and blood levels of glucose and other metabolic markers. Pharmacokinetic parameters such as peak concentration, area under the curve, and half-life of BEAM-301 will be measured. The total study duration for each participant spans up to 24 months after treatment.
CONDITIONS
Brief 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 18 years or older at the time of consent
- Diagnosis of Glycogen Storage Disease Type Ia confirmed by genetic testing showing homozygous or compound heterozygous G6PC1 c.247C>T (p.R83C) variant
- History of at least one episode of hypoglycemia below 60 mg/dL within the 2 years before signing the informed consent form
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 by liver MRI or ultrasound within 6 months prior to screening
- Presence of liver adenoma between 3 cm and 5 cm with documented growth rate of 0.5 cm or more per year
- Elevated aspartate transaminase or alanine transaminase above the upper limit of normal
- Total bilirubin level above the upper limit of normal; if Gilbert's Syndrome is present, total bilirubin above twice the upper limit of normal
- Previous treatment with any cell-, gene-, or viral vector-derived therapy
- Hospitalization for hypoglycemia management within 4 weeks prior to signing the informed consent form
- Triglycerides above 1000 mg/dL or history of pancreatitis without corrected cause in the last 5 years
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 24 months
Participants receive a single dose of BEAM-301 administered intravenously to evaluate safety and efficacy in Glycogen Storage Disease Type Ia.
Regular visits for safety and metabolic assessments over 24 months
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
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3
Clinical Study Site
Houston, Texas, United States, 77030
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Research Team
M
Medical Information
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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