NCT07011771WithdrawnPhase 1/2
A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease
A Phase 1/2 Dose-Escalation Trial to Evaluate Safety, Tolerability, and Efficacy of a Single Dose of CAP-003 Gene Therapy Administered to Patients With Parkinson's Disease With GBA1 Gene Mutation (PD-GBA)
Source: ClinicalTrials.gov · Last updated 22 Jun 2026
Reason given: Per protocol stopping rule was met for a separate trial for a different disease state and different experimental therapy that utilized a similar technology.
Brief summary (registry, in English)
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.
- Status (registry)
- Withdrawn
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 0 (actual)
- First submitted
- 2 Jun 2025
- Start date
- 18 Aug 2025
- Primary completion date
- 17 Jun 2026
- Lead sponsor
- Capsida Biotherapeutics, Inc.
- Diseases
- Parkinson disease
- NCT number
- NCT07011771
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior gene or cell therapy;
- Positive test result for anti-capsid total antibodies (tAb);
Other illness
- 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;
- History of deep brain stimulator placement, focused ultrasound therapy, or other intercranial surgery for PD;
- Hypersensitivity or contraindications to corticosteroid;
- Diagnosis of Gaucher disease;
- Other illnesses or medications that may affect the interpretation of the study results.
Other
- Presence of a bi-allelic GBA1 mutation, or presence of LRRK2 2019S or other LRRK2 mutation;
- Montreal Cognitive Assessment (MoCA) score of ≤22;
- Unable to undergo lumbar puncture;
- Clinically significant abnormalities in safety lab tests, vital signs;
Who the study is looking for
- 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)
Age in the registry: 21 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (5)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Rush University Medical Center | Chicago | United States | Unknown status |
| Baylor College of Medicine | Houston | United States | Unknown status |
| New York Presbyterian Hospital-Columbia University Medical Center | New York | United States | Unknown status |
| University of Pennsylvania | Philadelphia | United States | Unknown status |
| Inland Northwest Research | Spokane | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.