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

Launched by CAPSIDA BIOTHERAPEUTICS, INC. · Jun 2, 2025

Keywords

ClinConnect Summary

This clinical trial is investigating the safety of a new gene therapy called CAP-003 for adults with a specific type of Parkinson's disease linked to a genetic mutation known as GBA1. The trial aims to find out if this treatment is effective in helping patients. Eligible participants are adults aged 21 to 75 who have been diagnosed with Parkinson's disease and have a confirmed GBA1 mutation. They should be able to walk independently and have a reliable support person, like a family member or friend, to help monitor their health during the study.

Participants will receive a single infusion of CAP-003 through an intravenous (IV) line and will need to visit the clinic regularly for two years to undergo check-ups and tests. It's important to know that the trial is not yet recruiting participants, so if you or someone you know is interested, there will be more information available when it starts.

Gender

ALL

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.