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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

7 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-06-29

Study Completion Date

2028-05-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Gaucher Disease, Type 2

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Blinded assessor used in secondary outcome measures

Study Groups

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Low Dose

Group Type EXPERIMENTAL

LY3884961

Intervention Type GENETIC

Participants will receive a single dose of LY3884961 administered intracisternally.

Methylprednisolone

Intervention Type DRUG

Single IV pulse administered as concomitant medication.

Sirolimus

Intervention Type DRUG

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

Prednisone

Intervention Type DRUG

Administered orally as concomitant medication, followed by dose tapering.

High Dose

Group Type EXPERIMENTAL

LY3884961

Intervention Type GENETIC

Participants will receive a single dose of LY3884961 administered intracisternally.

Methylprednisolone

Intervention Type DRUG

Single IV pulse administered as concomitant medication.

Sirolimus

Intervention Type DRUG

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

Prednisone

Intervention Type DRUG

Administered orally as concomitant medication, followed by dose tapering.

Interventions

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LY3884961

Participants will receive a single dose of LY3884961 administered intracisternally.

Intervention Type GENETIC

Methylprednisolone

Single IV pulse administered as concomitant medication.

Intervention Type DRUG

Sirolimus

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

Intervention Type DRUG

Prednisone

Administered orally as concomitant medication, followed by dose tapering.

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

0 Months

Maximum Eligible Age

24 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No