PR001 for Gaucher Disease

Recruiting in Palo Alto (17 mi)

+4 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Prevail Therapeutics

No Placebo Group

Breakthrough Therapy

Trial Summary

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.

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)

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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.

See 12 more

Treatment Details

Interventions

PR001 (Enzyme Replacement Therapy)

Trial OverviewThe 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

2Treatment groups

Experimental Treatment

Group I: Low DoseExperimental Treatment4 Interventions

Group II: High DoseExperimental Treatment4 Interventions