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

March 26, 2026 updated by: Prevail Therapeutics

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
7

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United Kingdom
      • Manchester, United Kingdom, M13 9WL
        • Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road
  • United States
    • California
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital, 747 52nd St
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh, 4401 Penn Avenue
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • Lysosomal & Rare Disorders Research and Treatment Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 second to 2 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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).

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.
  • Contraindications or intolerance to radiographic visualization methods (e.g. MRI, MRA, CT), and intolerance to contrast agents used for MRI or CT scans.
  • Contraindications to general anesthesia or sedation.

Other protocol-defined inclusion/exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: High Dose
Drug: Prednisone
Administered orally as concomitant medication, followed by dose tapering.
Drug: Methylprednisolone
Single IV pulse administered as concomitant medication.
Drug: Sirolimus
Loading dose, followed by maintenance doses, followed by dose tapering; administered as concomitant medication.
Genetic: LY3884961
Participants will receive a single dose of LY3884961 administered intracisternally.
Experimental: Low Dose
Drug: Prednisone
Administered orally as concomitant medication, followed by dose tapering.
Drug: Methylprednisolone
Single IV pulse administered as concomitant medication.
Drug: Sirolimus
Loading dose, followed by maintenance doses, followed by dose tapering; administered as concomitant medication.
Genetic: LY3884961
Participants will receive a single dose of LY3884961 administered intracisternally.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Number of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Events leading to discontinuation
Time Frame: Year 5
Year 5
Immunogenicity of AAV9 and GCase in blood
Time Frame: Up to Year 2
Up to Year 2
Immunogenicity of AAV9 and GCase in CSF
Time Frame: Up to Year 1
Up to Year 1

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Time to death
Time Frame: Baseline until event or study completion, up to Year 5
Baseline until event or study completion, up to Year 5
Time to clinical event
Time Frame: Baseline until event or study completion, up to Year 5
Clinical event defined as tracheostomy/invasive ventilation, and/or percutaneous endoscopic gastrostomy (PEG) tube placement, and/or nasogastric (NG) tube placement
Baseline until event or study completion, up to Year 5
Change in GCase (glucocerebrosidase) enzyme activity levels in blood
Time Frame: Up to Year 5
Up to Year 5
Change in GCase enzyme activity levels in CSF (cerebrospinal fluid)
Time Frame: Up to Year 3
Up to Year 3
Change in glycolipid levels in blood
Time Frame: Up to Year 5
Up to Year 5
Change in glycolipid levels in CSF
Time Frame: Up to Year 3
Up to Year 3
Individual Vector Shedding data
Time Frame: Up to Year 5
Up to Year 5
Change in cognitive function
Time Frame: Months 6,12 and up to Year 2
Measured using Bayley Scales of Infant and Toddler Development (BSID-III)
Months 6,12 and up to Year 2
Change in cognitive function
Time Frame: Study Month 12 and up to Study Year 2
Measured using Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV) as appropriate. (Not all patients begin the study at birth. Only patients who are age 36 months at the designated study visits will be assessed using this measure)
Study Month 12 and up to Study Year 2
Change in motor skills
Time Frame: Months 6, 12 and up to Year 2
Change from baseline in motor function using Gross Motor Function Measure (GMFM-88).
Months 6, 12 and up to Year 2
Change in motor skills
Time Frame: Months 6, 12 and up to Year 2
Change from baseline in motor function using the BSID-III.
Months 6, 12 and up to Year 2
Change in Clinical Global Impressions (Severity)
Time Frame: Months 6, 12 and up to Year 2
Change from baseline in the clinical severity of illness (CGI-Severity {CGI-S}).
Months 6, 12 and up to Year 2
Clinical Global Impressions (Improvement)
Time Frame: Months 6, 12 and up to Year 2
Clinical improvement from baseline (CGI-Improvement [CGI-I]).
Months 6, 12 and up to Year 2
Change in adaptive behavior and functioning
Time Frame: Months 6 and 12 and up to Year 2
Change from baseline in adaptive functioning using the Vineland Adaptive Behavior Scale (VABS-2) (2nd edition)
Months 6 and 12 and up to Year 2
Change in most troubling symptoms
Time Frame: Months 6, 12 and up to Year 2
Change from baseline in the Visual Analog Scale for the Most Troubling Symptoms (VAS-MTS)
Months 6, 12 and up to Year 2
Change in behavioral symptoms
Time Frame: Months 6, 12 and up to Year 2
Change from baseline in the Child Behavior Checklist (CBCL)
Months 6, 12 and up to Year 2

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Prevail Therapeutics

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Eli Lilly and Company

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Hamzeh Migdadi, M.D., Prevail Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 29, 2021

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 1, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 1, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 11, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 29, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 2, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 1, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 26, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • J3Z-MC-OJAB (PRV-GD2-101)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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