PR001 for Gaucher Disease

Recruiting at 4 trial locations

Overseen By Prevail Therapeutics

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Prevail Therapeutics

Must not be taking: Substrate reduction therapy, Immunosuppressants

Disqualifiers: Significant CNS disease, Independent gait, Severe peripheral symptoms, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

7 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires that participants do not use certain medications, including substrate reduction therapy for Gaucher disease and some other prohibited medications, herbals, or over-the-counter agents. The protocol does not specify all medications that must be stopped, so it's best to discuss your current medications with the study team.

What is the purpose of this trial?

J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2). For each patient, the study will be approximately 5 years in duration. During the first 12 months after dosing, patients will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed up for an additional 4 years to monitor safety and changes on selected biomarkers and clinical outcomes.

Research Team

Hamzeh Migdadi, M.D.

Principal Investigator

Prevail Therapeutics

Eligibility Criteria

This trial is for infants with Type 2 Gaucher Disease (GD2) who have a clinical diagnosis confirmed by the central lab. They must not have had prior gene or cell therapy, and should not be on treatments that could affect the study results. Parents or guardians need to consent and provide information about the child's health.

Inclusion Criteria

My guardian understands the study's risks and can consent for me.

I have been diagnosed with GD2.

Patient has a reliable informant (i.e., parent/legal guardian) willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities (including providing input into the rating scales)

Exclusion Criteria

I can stop my blood thinner medication for 7 days before and 48 hours after certain procedures.

I am not on any immunosuppressants or steroids, except for skin conditions or asthma.

Your lab test results at screening show significant abnormalities.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

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

12 months

Follow-up

Participants are monitored for safety and changes in biomarkers and clinical outcomes

4 years

Treatment Details

Interventions

• PR001

Trial Overview The safety and effectiveness of LY3884961 (formerly PR001) are being tested in this Phase 1/2 trial over approximately five years. The first year focuses on how well patients tolerate the drug, its side effects, immune response, biomarkers, and initial efficacy.

Participant Groups

Group I: Low Dose Experimental Treatment 4 Interventions

Group II: High Dose Experimental Treatment 4 Interventions

Who Is Running the Clinical Trial?

Prevail Therapeutics

Lead Sponsor

Trials

5

Recruited

190+

Eli Lilly and Company

Industry Sponsor

Trials

2,708

Recruited

3,720,000+

Dr. Daniel Skovronsky

Eli Lilly and Company

Chief Medical Officer since 2018

MD from Harvard Medical School

David A. Ricks

Eli Lilly and Company

Chief Executive Officer since 2017

BSc from Purdue University, MBA from Indiana University