Summary

Eligibility
for people ages 0 months to 24 months (full criteria)
Location
at Oakland, California and other locations
Dates
study started
estimated completion

Description

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. 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.

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

Keywords

Gaucher Disease, Type 2, Gaucher Disease, GD, Gaucher, Type 2 Gaucher, Neuronopathic Gaucher, nGD, AAV9, GBA, Gene Therapy, Glucocerebrosidase, GBA1 mutation, Infants, Sirolimus, Prednisone, Methylprednisolone, Methylprednisolone Acetate, Methylprednisolone Hemisuccinate, Prednisolone, Prednisolone acetate, Prednisolone hemisuccinate, Prednisolone phosphate

Eligibility

You can join if…

Open to people ages 0 months to 24 months

  • Bi-allelic GBA1 mutations consistent with a diagnosis of GD2 confirmed by the central laboratory.
  • Clinical 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).

You CAN'T join if...

  • 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.
  • Live vaccine Immunizations within 4 weeks, or non-live vaccines within 2 weeks prior to the start of required immunosuppressive regimen.
  • 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 or inhaled preparations for dermatological conditions or asthma are allowed).
  • 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.

Locations

  • UCSF Benioff Children's Hospital, 5700 Martin Luther King Jr Way accepting new patients
    Oakland California 94609 United States
  • University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue accepting new patients
    Minneapolis Minnesota 55454 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Prevail Therapeutics
ID
NCT04411654
Phase
Phase 1/2 Gaucher Disease Research Study
Study Type
Interventional
Participants
Expecting 15 study participants
Last Updated