A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease
Withdrawn before enrolling · Phase 1/Phase 2
Conditions studied: GBA1 Parkinson Disease
In brief
The goal of this clinical trial is to learn about the safety of CAP-003 gene therapy in adults with GBA1 associated Parkinson's Disease. It will also provide information about whether CAP-003 demonstrates efficacy in these adults. Participants will have a single intravenous infusion of CAP-003 and visit the clinic regularly for 2 years for checkups and tests.
Key facts
- Study ID
- NCT07011771
- Run by
- Capsida Biotherapeutics, Inc.
- People needed
- 0
- Starts
- 2025-08-18
- Expected to finish
- 2026-06-17
- Last updated by the study team
- 2026-06-22
Who can join
Age: 21 and older, up to 75. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Male or female, 21 to 75 years
- Has diagnosis of Parkinson's disease (PD) per UK Parkinson's Disease Society Brain Bank Clinical Diagnostic Criteria;
- Has modified Hoehn and Yahr Stage I to III in the 'OFF' state;
- Presence of a pathogenic or likely pathogenic GBA1 mutation confirmed;
- Must be generally ambulatory, not dependent on wheelchair;
- Has a body weight of ≥40 kg (88 lb) to ≤110 kg (242 lb) and a body mass index (BMI) of 18 to 34 kg/m2;
- Participant has a reliable study partner/informant (eg, family member, friend) willing and able to participate in the trial as a source of information on the participant's health status and cognitive and functional abilities;
- Is living in the community (ie not in a nursing home)
You may not qualify if…
- Presence of a bi-allelic GBA1 mutation, or presence of LRRK2 2019S or other LRRK2 mutation;
- Diagnosis of significant central nervous system (CNS) disease other than PD that may be a cause for the participant's PD symptoms or may confound study objectives;
- Montreal Cognitive Assessment (MoCA) score of ≤22;
- History of deep brain stimulator placement, focused ultrasound therapy, or other intercranial surgery for PD;
- Hypersensitivity or contraindications to corticosteroid;
- Prior gene or cell therapy;
- Positive test result for anti-capsid total antibodies (tAb);
- Unable to undergo lumbar puncture;
- Diagnosis of Gaucher disease;
- Clinically significant abnormalities in safety lab tests, vital signs;
- Other illnesses or medications that may affect the interpretation of the study results.
Where it is running
- Rush University Medical Center — Chicago, Illinois, United States
- New York Presbyterian Hospital-Columbia University Medical Center — New York, New York, United States
- University of Pennsylvania — Philadelphia, Pennsylvania, United States
- Baylor College of Medicine — Houston, Texas, United States
- Inland Northwest Research — Spokane, Washington, United States
Full record on ClinicalTrials.gov
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