Who is eligible for clinical trial NCT04411654?

Participants must meet the protocol's inclusion criteria for Gaucher Disease, Type 2. The stated age range is 0 Months to 24 Months. Confirm all requirements with the study coordinator.

Does clinical trial NCT04411654 provide travel or cost reimbursement?

Check with the site coordinator at the participating facility for travel and lodging stipend availability, and for which routine-care costs are covered.

🏛️ Verified NIH Record • Updated 2026-08-18

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) (NCT04411654)

An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose LY3884961 in Infants With Type 2 Gaucher Disease

🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic Sponsor: Prevail Therapeutics

🩺 Plain-English Summary (8th-Grade Level)

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.

Who can join: To join this study, participants generally need to meet the listed inclusion criteria (4 items) and avoid the listed exclusions (14 items).

Location: UCSF Benioff Children's Hospital, 747 52nd St — Oakland, California

Age range: 0 Months to 24 Months

🧪 Interventions in This Study

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

Bi-allelic GBA1 mutations consistent with a diagnosis of GD2 confirmed by the central laboratory.
Clinical diagnosis of GD2
Parent/legal guardian is capable of providing signed informed consent; including compliance with the requirements and restrictions listed in the informed consent form (ICF) in this protocol.
Patient has a parent/legal guardian 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).

Usually NOT eligible if any of the following apply:

Exclusion Criteria:
Significant CNS disease other than GD2 that may be a cause for the patient's symptoms or interfere with study objectives.
Achieved independent gait.
Severe peripheral symptoms of GD which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
Concomitant disease, condition, or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
Use of any substrate reduction therapy (SRT) for GD treatment.
Use of prohibited medications, herbals, or over-the-counter agents as listed in the protocol.
Any type of prior gene or cell therapy.
Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified immunosuppression.
Participation in another investigational drug or device study within the past 3 months.
Brain MRI (magnetic resonance imaging) and MRA (magnetic resonance angiography) showing clinically significant abnormality deemed a contraindication to intracisternal injection.
Clinically significant laboratory test result abnormalities assessed at screening.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (6 Sites)

UCSF Benioff Children's Hospital, 747 52nd St — Oakland, California
University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue — Minneapolis, Minnesota
NYU Medical Center, 305 Second Ave, Suite 16 — New York, New York
Children's Hospital of Pittsburgh, 4401 Penn Avenue — Pittsburgh, Pennsylvania
Lysosomal & Rare Disorders Research and Treatment Center — Fairfax, Virginia
Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road — Manchester,

📞 Contact the Study Coordinator

Phone: (877) 827-3222

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

💰 Cost, Insurance & Patient Rights

100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

Explore by condition: All Gaucher Disease, Type 2 clinical trials

💡 How to Participate

Check the checklist above to see if you may qualify.
Contact the study coordinator using the phone/email above.
Ask about the visit schedule, what is covered at no cost, and any travel support.
Confirm with your own doctor before making a decision.

🏛️ View Official NIH Record ↗