Evaluating BEAM-301 for treating Glycogen Storage Disease Type Ia

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

This study is testing a new treatment called BEAM-301 to see if it is safe and effective for adults with Glycogen Storage Disease Type Ia who have a specific genetic variant.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Beam Therapeutics Inc. (Industry-sponsored)
• Locations: 3 sites (Orange, California and 2 other locations)
• Trial ID: NCT06735755 on ClinicalTrials.gov

What this trial studies

This Phase 1/2 study is designed to assess the safety, tolerability, and efficacy of a single intravenous dose of BEAM-301 in adult patients diagnosed with Glycogen Storage Disease Type Ia who carry a specific genetic variant. The study will involve multiple centers and will focus on patients who are homozygous or compound heterozygous for the G6PC1 c.247C>T (p.R83C) variant. The primary goal is to determine the optimal biological dose while monitoring for any adverse effects and therapeutic benefits.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a confirmed diagnosis of GSDIa and the specific genetic variant.

Not a fit: Patients who have undergone a liver transplant or have significant liver complications may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of Glycogen Storage Disease Type Ia, potentially reducing the frequency of hypoglycemic episodes.

How similar studies have performed: While this approach is novel, similar studies targeting genetic variants in metabolic disorders have shown promise in the past.

Eligibility criteria

Inclusion Criteria:

1. Males or females age ≥18 years of age at the time of consent.
2. Diagnosis of GSDIa and homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant (confirmed by genetic testing).
3. History of at least 1 episode of hypoglycemia \<60 mg/dL within the 2 years prior to signing the ICF.

Exclusion Criteria:

1. Liver transplant recipient/on waiting list for liver transplant or known history of liver cirrhosis.
2. 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.
3. Presence of liver adenoma \>3 cm and ≤5 cm with a documented annual growth rate of ≥0.5 cm per year.
4. Have aspartate transaminase or alanine transaminase \>upper limit of normal (ULN).
5. Total bilirubin levels \>ULN; if documented Gilbert's Syndrome, total bilirubin \>2 × ULN.
6. Previous treatment with any cell-, gene-, or viral vector-derived therapy Hospitalization for management of hypoglycemia within 4 weeks prior to signing the ICF.
7. 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 this trial is running

Orange, California and 2 other locations

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

Study contacts

• Study coordinator: Medical Information

Conditions Glycogen Storage Disease Type Ia; GSDIa; GSD1a