A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease (NCT07011771) | Clinical Trial Compass
WithdrawnPhase 1/2
A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease
Stopped: Per protocol stopping rule was met for a separate trial for a different disease state and different experimental therapy that utilized a similar technology.
United States0Started 2025-08-18
Plain-language summary
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.
Who can participate
Age range
21 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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)
Exclusion Criteria:
* 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 sign…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of treatment emergent adverse events (safety and tolerability)