Adeno-associated viral vector serotype 9 expressing codon-optimized human GBA gene for Parkinson's disease with GBA1 mutation

Inclusion criteria

• {"criterion_text":"- 1. Men or women aged 35 to 80 years (inclusive)"}
• {"criterion_text":"- 10. Women of childbearing potential cannot be pregnant or lactating/breastfeeding"}
• {"criterion_text":"- 2. Body weight range of ≥40 kg (88 lbs) to ≤110 kg (242 lbs)"}
• {"criterion_text":"- 3. Diagnosis of PD per UK Parkinson’s Disease Society Brain Bank Clinical Diagnostic Criteria"}
• {"criterion_text":"- 4. Hoehn and Yahr Stage III-IV (when off of PD medication)"}
• {"criterion_text":"- 5. 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"}
• {"criterion_text":"- 6. At least 1 pathogenic GBA1 mutation confirmed by the central laboratory. Patients with bi-allelic GBA1 mutations and/or monogenic forms of PD will not be eligible. Only patients with low (< lower limit of normal) GCase enzyme activity measured in the blood will be eligible for participation."}
• {"criterion_text":"- 7. Negative screening test for Mycobacterium tuberculosis (MTB)"}
• {"criterion_text":"- 8. Patient and/or patient’s legally authorized representative has the ability to understand the purpose and risks of the study and provide written informed consent"}
• {"criterion_text":"- 9. Patient has a reliable study partner (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"}

Exclusion criteria

• {"criterion_text":"- 1. The diagnosis of a significant central nervous system (CNS) disease other than PD"}
• {"criterion_text":"- 10. Participants who are legally incompetent due to severe PD symptoms and/or cognitive impairment are not eligible for participation."}
• {"criterion_text":"- 2. Montreal Cognitive Assessment (MoCA) score of <14"}
• {"criterion_text":"- 3. Spinal, cervical, or brain MRI/magnetic resonance angiography (MRA) indicating clinically significant abnormality"}
• {"criterion_text":"- 4. Hypersensitivity or contraindications to corticosteroid and/or sirolimus use"}
• {"criterion_text":"- 5. Concomitant disease or condition within 6 months of Screening that could interfere with, or treatment of which might interfere with, the conduct of the study or that would, in the opinion of the Investigator, pose an unacceptable safety risk to the patient or interfere with the patient’s ability to comply with study procedures"}
• {"criterion_text":"- 6. Clinically significant abnormalities in laboratory test results at Screenin"}
• {"criterion_text":"- 7. Participation within 3 months prior to Screening in another therapeutic investigational drug or device study with purported disease-modifying effects on PD, unless it can be documented that the patient received placebo."}
• {"criterion_text":"- 8. History of deep brain stimulator placement, focused ultrasound, or surgery for PD"}
• {"criterion_text":"- 9. Any type of prior gene or cell therapy"}

Primary endpoints

• {"endpoint_text":"- Incidence and severity of treatment-emergent AEs and SAE","definition_or_measurement_approach":""}
• {"endpoint_text":"- Incidence of procedure or treatment-emergent safety findings","definition_or_measurement_approach":""}
• {"endpoint_text":"- Treatment emergent and change from baseline in immunogenicity of adeno-associated viruses serotype 9 and glucocerebrosidase","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Change from baseline in glycolipid and glucocerebrosidase levels, as well as glucocerebrosidase enzyme activity in blood","definition_or_measurement_approach":""}
• {"endpoint_text":"- Change from baseline in glycolipid and glucocerebrosidase levels, as well as glucocerebrosidase enzyme activity in cerebrospinal fluid","definition_or_measurement_approach":""}

Netherlands

Earliest CTIS Part Ii Submission Date

26-05-2025

Latest Decision Or Authorization Date

16-06-2025

Processing Time Days

21

Number Of Sites

1

Number Of Participants

5

Primary sponsor

Full Name

Prevail Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Pharmaceutical Product Development LLC

Responsibilities

Specialty lab samples logistics; other sponsorDuties codes include 15 and 4

Name

PPD International Holdings LLC

Responsibilities

Sponsor duties code: 4

Name

PPD Development LP

Responsibilities

Responsibilities include codes 1,10,11,12,2,5,6 (roles as listed in sponsorDuties)

Name

Bioclinica Inc.

Responsibilities

Medical Image Analyses (sponsorDuties code: 15)

Name

WCG Clinical Inc.

Responsibilities

Scale Management, Rater Training, eCOA, Clinical Data Analytics; sponsorDuties codes: 15 and 7

Third parties

• {"country":"United States","full_name":"Arup Laboratories Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties codes: 7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Sbh Sciences Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"sponsorDuties codes: 15; value: Travel, reimbursement, accommodations, Patient facing material","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Quanterix Corp.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"National Jewish Health","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Nextcea Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"F. Hoffmann-La Roche AG","duties_or_roles":"sponsorDuties codes: 15 (Digital Biomarkers, ePRO); 7","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"sponsorDuties codes: 15 (specialty lab samples logistics); 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: 1, 10, 11, 12, 2, 5, 6","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Veeva Systems Inc.","duties_or_roles":"sponsorDuties codes: 15 (eTMF)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"sponsorDuties codes: 15 (Medical Image Analyses)","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Cleveland Clinic Foundation","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"sponsorDuties codes: 15 (Scale Management, Rater Training, eCOA, Clinical Data Analytics); 7","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Centogene GmbH","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC (Boston address)","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Unisphere Travel Ltd. Inc.","duties_or_roles":"sponsorDuties codes: 15 (Travel Reimbursement, accomodations)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC (Richmond address)","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}