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Gaucher Disease, Type 2Phase 1/2NCT04411654

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

A Phase 1/2 study testing LY3884961, Methylprednisolone and Sirolimus in people with Gaucher Disease, Type 2, run by Prevail Therapeutics at 6 sites in 2 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 0 to 24 months
Conditions the study is for
Gaucher Disease, Type 2
What is being tested?
LY3884961, Methylprednisolone and Sirolimus
Could I receive a placebo?
No, everyone receives an active treatment
How many people
7 participants
Where
6 sitesin 2 countries
Study dates
29 Jun 2021to Feb 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 0 to 24 months
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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).

Who cannot join

  • 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.
Show all 14Show fewer
  • 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.

Trial locations and contacts

6 sites in 2 countries.

6 sites in 2 countries

United States5 sites

  • Lysosomal & Rare Disorders Research and Treatment Center

    Fairfax, Virginia, 22030

  • University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue

    Minneapolis, Minnesota, 55454

  • NYU Medical Center, 305 Second Ave, Suite 16

    New York, New York, 10003

  • UCSF Benioff Children's Hospital, 747 52nd St

    Oakland, California, 94609

  • Children's Hospital of Pittsburgh, 4401 Penn Avenue

    Pittsburgh, Pennsylvania, 15224

Other trials in United States

United Kingdom1 site

  • Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road

    Manchester, M13 9WL

Other trials in United Kingdom

About this study

Official title: 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.

Being tested
LY3884961, Methylprednisolone, Sirolimus, Prednisone
Keywords
Gaucher Disease, GD, Gaucher, Type 2 Gaucher, Neuronopathic Gaucher, nGD, AAV9, GBA, Gene Therapy, Glucocerebrosidase, GBA1 mutation, Infants
Medical subject headings
Gaucher Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LY3884961Genetic

    Participants will receive a single dose of LY3884961 administered intracisternally.

  • MethylprednisoloneDrug

    Single IV pulse administered as concomitant medication.

  • SirolimusDrug

    Loading dose, followed by maintenance doses, followed by dose tapering; administered as concomitant medication.

  • PrednisoneDrug

    Administered orally as concomitant medication, followed by dose tapering.

Study groups in NCT04411654
Study groupRoleWhat they receive
Low DoseExperimentalNot described
Receives: Genetic: LY3884961; Drug: Methylprednisolone; Drug: Sirolimus; Drug: Prednisone
High DoseExperimentalNot described
Receives: Genetic: LY3884961; Drug: Methylprednisolone; Drug: Sirolimus; Drug: Prednisone

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Events leading to discontinuation

    Measured: Year 5

  2. Immunogenicity of AAV9 and GCase in blood

    Measured: Up to Year 2

  3. Immunogenicity of AAV9 and GCase in CSF

    Measured: Up to Year 1

Secondary outcomes (16)

  1. Time to death

    Measured: Baseline until event or study completion, up to Year 5

  2. Time to clinical event

    Clinical event defined as tracheostomy/invasive ventilation, and/or percutaneous endoscopic gastrostomy (PEG) tube placement, and/or nasogastric (NG) tube placement

    Measured: Baseline until event or study completion, up to Year 5

  3. Change in cognitive function

    Measured using Bayley Scales of Infant and Toddler Development (BSID-III)

    Measured: Months 6,12 and up to Year 2

  4. Change in cognitive function

    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)

    Measured: Study Month 12 and up to Study Year 2

  5. Change in motor skills

    Change from baseline in motor function using Gross Motor Function Measure (GMFM-88).

    Measured: Months 6, 12 and up to Year 2

  6. Change in motor skills

    Change from baseline in motor function using the BSID-III.

    Measured: Months 6, 12 and up to Year 2

  7. Change in Clinical Global Impressions (Severity)

    Change from baseline in the clinical severity of illness (CGI-Severity {CGI-S}).

    Measured: Months 6, 12 and up to Year 2

  8. Clinical Global Impressions (Improvement)

    Clinical improvement from baseline (CGI-Improvement [CGI-I]).

    Measured: Months 6, 12 and up to Year 2

  9. Change in adaptive behavior and functioning

    Change from baseline in adaptive functioning using the Vineland Adaptive Behavior Scale (VABS-2) (2nd edition)

    Measured: Months 6 and 12 and up to Year 2

  10. Change in most troubling symptoms

    Change from baseline in the Visual Analog Scale for the Most Troubling Symptoms (VAS-MTS)

    Measured: Months 6, 12 and up to Year 2

  11. Change in behavioral symptoms

    Change from baseline in the Child Behavior Checklist (CBCL)

    Measured: Months 6, 12 and up to Year 2

  12. Change in GCase (glucocerebrosidase) enzyme activity levels in blood

    Measured: Up to Year 5

  13. Change in GCase enzyme activity levels in CSF (cerebrospinal fluid)

    Measured: Up to Year 3

  14. Change in glycolipid levels in blood

    Measured: Up to Year 5

  15. Change in glycolipid levels in CSF

    Measured: Up to Year 3

  16. Individual Vector Shedding data

    Measured: Up to Year 5

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Jun 2020
  2. Study start29 Jun 2021
  3. Primary completionFeb 2027 (estimated)
  4. Study completionFeb 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
7 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04411654
Sponsor's protocol ID
J3Z-MC-OJAB (PRV-GD2-101)
Status last verified
Oct 2026
First submitted
11 May 2020
Last updated
22 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing LY3884961, Methylprednisolone and Sirolimus in people with Gaucher Disease, Type 2, run by Prevail Therapeutics at 6 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 22 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Events leading to discontinuation (time frame: Year 5). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04411654.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2026.

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