LY3884961 for Gaucher Disease

Recruiting in Palo Alto (17 mi)

+7 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: Prevail Therapeutics

Must be taking: ERT, SRT

Must not be taking: Chaperone therapy, Immunosuppressants

Disqualifiers: Neurological signs, Bone disease, Splenectomy, others

No Placebo Group

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial tests a new drug called LY3884961 in adults with specific symptoms of Gaucher Disease. The study aims to find the safest and most effective dose by observing how patients' bodies react to different amounts of the drug. Researchers will monitor safety, side effects, and changes in disease symptoms over several years.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must have been on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years and on a stable dose for at least 3 months before joining. You cannot use GD-related chaperone therapy within 4 weeks before starting the trial.

How does the drug LY3884961 differ from other treatments for Gaucher Disease?

LY3884961 is a novel gene therapy approach for Gaucher Disease, which aims to address the root cause by delivering a functional copy of the GBA1 gene to cells, potentially offering a more long-term solution compared to existing enzyme replacement therapies that require regular infusions.¹ ² ³ ⁴ ⁵

Research Team

Sarah Neuhaus, DO

Principal Investigator

Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company

Eligibility Criteria

Adults aged 18-65 with Gaucher Disease (GD) and specific GBA1 mutations can join this trial. They must have been on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years, but not responding well enough. Participants need to use effective birth control and cannot donate blood for a year.

Inclusion Criteria

Patients must agree to abstain from blood donations for at least the first year of the study

I am between 18 and 65 years old.

My GBA1 mutations have been officially confirmed.

See 3 more

Exclusion Criteria

My heart condition is stable and not considered serious by my doctor.

I have never received gene or cell therapy.

I am not on any immunosuppressants or steroids not allowed by the study.

See 15 more

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

6 weeks

Treatment

Participants receive a single intravenous dose of LY3884961 and are evaluated for safety, tolerability, immunogenicity, biomarkers, and efficacy

18 months

Follow-up

Participants are monitored for safety, immunogenicity, and selected biomarker and efficacy parameters

42 months

Treatment Details

Interventions

LY3884961 (Monoclonal Antibodies)
Methylprednisolone (Corticosteroid)
Prednisone (Corticosteroid)
Sirolimus (Immunosuppressant)

Trial OverviewThe study tests different doses of LY3884961 in adults with GD's peripheral symptoms. It's an open-label trial where everyone knows they're getting the drug, lasting about 5 years with close monitoring for safety, immune response, biomarkers, and effectiveness.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: LY3884961Experimental Treatment1 Intervention

LY3884961 is an advanced therapy investigational medicinal product administered as a single intravenous infusion.