RecruitingPhase 1/2

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)

United States36 participantsStarted 2024-12-06

Plain-language summary

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.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Males or females age ≥18 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.

Exclusion criteria

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

What they're measuring

To evaluate the safety and tolerability of BEAM-301 in patients with GSDIa with a G6PC1 c.247C>T (p.R83C) variant by measuring incidence of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and dose-limiting toxicities (DL

Timeframe: BEAM-301 administration through month 24

Trial details

NCT IDNCT06735755

SponsorBeam Therapeutics Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-12-30

Contact for this trial

Medical Information

857-327-8641 clinicalinfo@beamtx.com

View on ClinicalTrials.gov More Glycogen Storage Disease Type Ia trials