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

Korábbi kezelés

• Prior gene or cell therapy;
• Positive test result for anti-capsid total antibodies (tAb);

Más betegség

• 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.

Egyéb

• 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;

• 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)