← Back to Search

Enzyme Replacement Therapy

BEAM-301 for Glycogen Storage Disease

Phase 1 & 2

Recruiting

Research Sponsored by Beam Therapeutics Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of GSDIa and homozygous or compound heterozygous for the G6PC1 c.247C>T (p.R83C) variant (confirmed by genetic testing)

Be older than 18 years old

Must not have

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial will test a new drug, BEAM-301, on adult patients with a specific genetic condition called GSDIa. The study will focus on evaluating the safety and effectiveness of the drug

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Since this is an early-phase trial of BEAM-301, exact side effects aren't listed yet. Generally, IV treatments can cause reactions at the injection site, allergic responses, or flu-like symptoms.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

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

Select...

I have a liver tumor larger than 5 cm, confirmed by MRI or ultrasound.

Select...

My liver tumor is between 3 and 5 cm and grows more than 0.5 cm a year.

Select...

I haven't had cell, gene, or viral therapies and wasn't hospitalized for low blood sugar in the last month.

Select...

My triglycerides are over 1000 mg/dL or I've had pancreatitis in the last 5 years without a known cause.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: BEAM-301Experimental Treatment1 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.

0 / 6Eligibility criteria met