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A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease

WithdrawnPhase 1, Phase 2

Sponsor: Capsida Biotherapeutics, Inc.

Conditions: GBA1 Parkinson Disease

Interventions: gene therapy, gene therapy

Countries: United States

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.

Eligibility overview

Sex: ALL

Age: 21 Years to 75 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

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 signs;
* Other illnesses or medications that may affect the interpretation of the study results.

Locations (5)

Chicago, Illinois, United States
New York, New York, United States
Philadelphia, Pennsylvania, United States
Houston, Texas, United States
Spokane, Washington, United States