This study is in progress, not accepting new patients

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

a study on Gaucher Disease

Summary

Eligibility
for people ages 0 months to 24 months (full criteria)
Location
at UCSF
Dates
study started
study ends around
Principal Investigator
by Paul Harmatz (ucsf)
Headshot of Paul Harmatz
Paul Harmatz

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, Methylprednisolone, Sirolimus, Prednisone

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 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).

You CAN'T join if...

  • 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.

Locations

  • UCSF Benioff Children's Hospital, 747 52nd St
    Oakland California 94609 United States
  • University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue
    Minneapolis Minnesota 55454 United States

Lead Scientist at University of California Health

  • Paul Harmatz (ucsf)
    Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.

Details

Status
in progress, not 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
About 7 people participating
Last Updated