Phase 1/2a Clinical Trial of PR001A in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

  • End date
    Jun 15, 2027
  • participants needed
  • sponsor
    Prevail Therapeutics
Updated on 13 May 2022


The study PRV-PD101 is evaluating a potentially disease-modifying, single-dose gene therapy for patients with Parkinson’s disease with GBA1 mutations (PD-GBA).
PD-GBA is driven by mutations in the gene GBA1. This gene contains the instructions for making the lysosomal enzyme beta-glucocerebrosidase, or GCase, which is needed for the disposal and recycling of glycolipids — a type of cellular component that is known to accumulate with aging. PD-GBA patients have mutations in at least one chromosomal copy of GBA1, while Gaucher disease patients have mutations in both chromosomal copies. Without enough GCase, glycolipids accumulate, causing lysosomal dysfunction and aggregation of the protein α-Synuclein in cells, which is believed to lead to the inflammation and neurodegeneration behind PD-GBA.
PRV-PD101 delivers PR001, the well-studied viral vector AAV9 carrying a healthy copy of the GBA1 gene to the brain. This is administered by a one-time injection into an area above the spinal canal called the cisterna magna — a direct, non-surgical technique that has been used safely in humans for a century.
The U.S. FDA has granted Fast Track Designation for PR001 for the treatment of PD-GBA. It has also granted Orphan Drug Designation for PR001 for the treatment of patients with Gaucher disease.
PROPEL, the Phase 1/2 clinical trial of PR001 for the treatment of PD-GBA patients, is now recruiting as a multicenter, open-label, ascending dose, first in-human study. The purpose is to evaluate the safety of intracisternal PR001 administration in patients with moderate to severe Parkinson's disease with at least 1 pathogenic GBA1 mutation. Two escalating dose cohorts are planned (low dose and high dose). 


Condition Parkinson's disease, Parkinson's disease
Treatment prednisone, Methylprednisolone, Sirolimus, PR001A
Clinical Study IdentifierNCT04127578
SponsorPrevail Therapeutics
Last Modified on13 May 2022


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Inclusion Criteria

Body weight range of 40 kg (88 lbs) to 110 kg (242 lbs) and a BMI of 18 to 34 kg/m2
Diagnosis of Parkinson's Disease (PD) per UK Parkinson's Disease Society Brain Bank Clinical Diagnostic Criteria
Hoehn and Yahr Stage III-IV (as determined in OFF state)
Stable use of background medications at least 8 weeks prior to investigational product (IP) administration, including but not limited to those used for treatment of PD. Gaucher Disease-PD patients receiving GD treatments should be on a stable regimen of their ERT or substrate replacement therapy (SRT) medication for at least 3 months prior to screening
At least 1 pathogenic GBA1 mutation confirmed by the central laboratory
Negative screening test for Mycobacterium tuberculosis (MTB) or documented negative MTB test within 1 year prior to Screening
Patient and/or patient's legally authorized representative (LAR) 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 study partner/informant (e.g., family member, friend) 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). The study partner should have regular contact with the patient (in person or via phone/video communication). The study partner must sign a separate partner informed consent form (ICF) indicating that she/he understands the study requirements and is willing to participate and attend study visits requiring study partner input
Women of nonchildbearing potential must be either surgically sterile or postmenopausal. Men and women of childbearing potential must use a highly effective method of contraception consistently and correctly for the duration of the study including the long-term follow-up
Men must agree to abstain from sperm donation for the duration of the study, including long-term follow-up
Women must agree to abstain from egg donation for the duration of the study, including long-term follow-up
Women of childbearing potential cannot be pregnant or lactating/breastfeeding and must have a negative result for serum pregnancy test at Screening
Patient is generally ambulatory, not dependent on walker or wheelchair
Patient is living in the community (i.e., not in nursing home); some levels of assisted living may be permitted at the discretion of the Investigator
Pneumococcal and shingles vaccines are required within 10 years of screening (allowed to be performed during screening but must be given at least 4 weeks prior to start of the immunosuppressant treatment)
Patient is up to date with age and gender-appropriate cancer screening as per local standard of care
Other protocol-defined inclusion

Exclusion Criteria

Other protocol-defined criteria may apply
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