BEAM-301 for Glycogen Storage Disease

Recruiting at 1 trial location

Overseen By Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Beam Therapeutics Inc.

Disqualifiers: Liver transplant, Liver adenoma, Pancreatitis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 4-6 weeks

36 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for adults with Glycogen Storage Disease Type Ia (GSDIa) who have specific genetic variants. It's not clear what excludes someone from participating, but typically, trials require stable health and no conflicting medications or conditions.

Inclusion Criteria

History of at least 1 episode of hypoglycemia <60 mg/dL within the 2 years prior to signing the ICF

I have GSDIa and carry two copies of the specific G6PC1 gene mutation.

Exclusion Criteria

Have aspartate transaminase or alanine transaminase >upper limit of normal (ULN)

Total bilirubin levels >ULN; if documented Gilbert's Syndrome, total bilirubin >2 × ULN

I have had a liver transplant, am waiting for one, or have liver cirrhosis.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single ascending dose of BEAM-301 to evaluate safety, tolerability, and efficacy

4-6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Treatment Details

Interventions

• BEAM-301

Trial Overview The study tests BEAM-301, a new treatment given as a single IV dose to see if it's safe and effective for GSDIa. The trial will gradually increase the dose to find the best amount that works without causing harm.

Participant Groups

Group I: BEAM-301 Experimental Treatment 1 Intervention

BEAM-301 consists of adenine base editor messenger ribonucleic acid (mRNA) and single guide ribonucleic acid (gRNA) formulated in lipid nanoparticles (LNPs) for intravenous (IV) administration.

BEAM-301 is already approved in United States for the following indications:

Approved in United States as BEAM-301 for:

• Glycogen Storage Disease Type Ia (GSDIa)

Who Is Running the Clinical Trial?

Beam Therapeutics Inc.

Lead Sponsor

Trials

5

Recruited

1,200+