Evaluating BEAM-301 for treating Glycogen Storage Disease Type Ia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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
• Email: clinicalinfo@beamtx.com
• Phone: 857-327-8641

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Home lactate and Accu‑Chek glucose meter accuracy for people with glycogen storage disease

  This project will see if a handheld Lactate Plus meter and an Accu‑Chek glucometer give results that match lab blood tests in people with glycogen storage disease types Ia, Ib, and XI.

  Recruiting Connecticut Children's Medical Center · Hartford, Connecticut · NCT07459582

• Evaluating the accuracy of home lactate meters in patients with Glycogen Storage Disease Type Ia

  This study tests if home lactate meters give accurate readings for people with Glycogen Storage Disease Type Ia by comparing them to lab results.

  Recruiting Connecticut Children's Medical Center · Hartford, Connecticut · NCT06843330

• Monitoring program for patients with Glycogen Storage Disease Type Ia

  This study is tracking the long-term health and safety of a treatment called DTX401 in people with Glycogen Storage Disease Type Ia to see how it helps them over at least 10 years.

  Recruiting at 17 sites Ultragenyx Pharmaceutical Inc · Orange, California and 18 other locations · NCT06636383

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.