RECRUITING

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

Description

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.

Conditions

GBA1 Parkinson Disease
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Related Conditions:

GBA1 Parkinson Disease

Study Overview

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Study Details

Study overview

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.

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)

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

Condition
GBA1 Parkinson Disease
Intervention / Treatment

-

Contacts and Locations

Chicago

Rush University Medical Center, Chicago, Illinois, United States, 60612

Kansas City

University of Kansas Medical Center, Kansas City, Kansas, United States, 66160

New York

Columbia University Medical Center, New York, New York, United States, 10032

Houston

Baylor College of Medicine, Houston, Texas, United States, 77030

Kirkland

Booth Gardner Parkinson's Care Center - Evergreen Neuroscience Institute, Kirkland, Washington, United States, 98034

Spokane

Inland Northwest Research, Spokane, Washington, United States, 99202

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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)
  • * 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.

Ages Eligible for Study

21 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Capsida Biotherapeutics, Inc.,

Melanie Brandabur, MD, STUDY_DIRECTOR, Capsida Biotherapeutics

Study Record Dates

2029-11