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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests BEAM-301, a new treatment for individuals with Glycogen Storage Disease Type Ia (GSDIa), a condition affecting sugar processing in the body. Researchers aim to determine the safety and effectiveness of this treatment and identify the optimal dose. BEAM-301 is administered through an IV and uses special molecules to edit genes. Adults with GSDIa who have experienced low blood sugar episodes in the past two years may be suitable candidates for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative treatment.

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.

Is there any evidence suggesting that BEAM-301 is likely to be safe for humans?

Research has shown that BEAM-301 yields promising results in early tests. In studies with mice that have a similar genetic condition, the treatment corrected the disease's effects, suggesting potential effectiveness in humans. However, as this is a Phase 1/2 trial, the treatment remains in the early stages of human testing. While some initial safety information exists, further research is necessary to fully understand human tolerance. In these early phases, the trial aims to carefully assess safety and determine the appropriate dose. Participants will be closely monitored for any side effects, a standard procedure in such trials. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

BEAM-301 is unique because it uses a cutting-edge approach called adenine base editing to treat Glycogen Storage Disease. Unlike standard treatments that manage symptoms, BEAM-301 aims to correct the underlying genetic mutation, offering a potential one-time solution. It combines mRNA and gRNA, packaged in lipid nanoparticles, for targeted intravenous delivery, which is a novel method not seen in current therapies. Researchers are excited about this treatment because it has the potential to provide a more effective and long-lasting solution compared to existing options.

What evidence suggests that BEAM-301 might be an effective treatment for Glycogen Storage Disease?

Research has shown that BEAM-301, the treatment under study in this trial, may help treat Glycogen Storage Disease Type Ia (GSDIa). In earlier studies, BEAM-301 corrected up to 60% of the G6PC1-R83C mutation in liver cells. This correction improved blood sugar levels and resolved metabolic issues related to the disease. The treatment employs a method called base editing, which accurately targets and changes the faulty part of the gene. These early results suggest that BEAM-301 could be a promising new approach to managing GSDIa. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. investors.beamtx.com investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-presents-preclinical-data-highlighting-0

Press Release Today's data continue to show that treatment with BEAM-301 yielded potent and durable liver editing that translated into normalization of blood ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06735755

NCT06735755 | A Phase 1/2, Dose-Exploration Study to ... 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 ...

3. nature.com nature.com/articles/s41467-024-54108-1

Base-editing corrects metabolic abnormalities in a ... BEAM-301 can correct up to ~60% of the G6PC1-R83C variant in liver cells, restores blood glucose control, improves metabolic abnormalities of the disease, and ...

4. crisprmedicinenews.com crisprmedicinenews.com/clinical-trial/glycogen-storage-disease-type-ia-gsdia-nct06735755/

Glycogen Storage Disease Type Ia, GSDIa, (NCT06735755) 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 ...

5. sec.gov sec.gov/Archives/edgar/data/1745999/000095017025063955/beam-ex99_1.htm

Beam Therapeutics Reports First Quarter 2025 Financial ... BEAM-301 is an investigational in vivo base editing treatment designed to correct the R83C mutation, the most common disease-causing mutation ...

6. nichd.nih.gov nichd.nih.gov/newsroom/news/021925-gene-editing-technique-GSD-Ia

Gene editing technique corrects glycogen storage disease ... They tested the treatment, called BEAM-301, in a mouse model they developed that has the human version of the variant G6PC1 gene. The DNA ...

7. trialx.com trialx.com/clinical-trials/listings/297059/a-phase-12-dose-exploration-study-to-evaluate-the-safety-and-efficacy-of-beam-301-in-patients-with-glycogen-storage-disease-type-ia-gsdia/

A Phase 1/2, Dose-Exploration Study to Evaluate the ... This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in ...