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

  • STATUS
    Recruiting
  • End date
    Sep 19, 2028
  • participants needed
    15
  • sponsor
    Prevail Therapeutics
Updated on 26 September 2021
type 1 gaucher disease

Summary

PRV-GD2-101 is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose 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 PR001 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.

Details
Condition Gaucher Disease
Treatment prednisone, Methylprednisolone, Sirolimus, PR001
Clinical Study IdentifierNCT04411654
SponsorPrevail Therapeutics
Last Modified on26 September 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Bi-allelic GBA1 mutations consistent with a diagnosis of GD2 confirmed by the central laboratory
Neurological signs and/or symptoms consistent with diagnosis of GD2
Parent/legal guardian has the ability to understand the purpose and risks of the study and provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations
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)

Exclusion Criteria

Diagnosis of a significant CNS disease other than GD2 that may be a cause for the patient's GD symptoms or may confound 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 GD treatment-related substrate reduction therapy
Use of strong inhibitors or inducers of cytochrome P450 3A4 (CYP3A4) or P-glycoprotein (P-gp) medications, herbals, or over-the-counter agents
Any type of prior gene or cell therapy
Immunizations (live vaccines) in the prior 4 weeks
Use of blood thinners. Antiplatelet therapies are acceptable if the patient is medically able to temporarily stop them from 7 days prior to dosing and through at least 48 hours after the intracisternal injection and lumbar puncture
Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified (topical preparations for dermatological conditions are allowed)
Participation in another investigational drug or device study within the past 6 months
Brain MRI (magnetic resonance imaging) and MRA (magnetic resonance angiography) showing clinically significant abnormality considered to prevent 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
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