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Phase 12 Study to Test Safety and Effectiveness of BEAM-301 for Adults with Glycogen Storage Disease Type Ia Having G6PC1 c.247C>T Variant
Led by Beam Therapeutics Inc. · Updated on 2025-12-15
36
Participants Needed
3
Research Sites
N/A
Total Duration
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 12 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)
Research Team
M
Medical Information
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