SUSPENDED

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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)

Quick Facts

Study Start:2025-08-18
Study Completion:2029-11
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:SUSPENDED

Study ID

NCT07011771

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.

Ages Eligible for Study:21 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Male or female, 21 to 75 years
  2. * Has diagnosis of Parkinson's disease (PD) per UK Parkinson's Disease Society Brain Bank Clinical Diagnostic Criteria;
  3. * Has modified Hoehn and Yahr Stage I to III in the 'OFF' state;
  4. * Presence of a pathogenic or likely pathogenic GBA1 mutation confirmed;
  5. * Must be generally ambulatory, not dependent on wheelchair;
  6. * 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;
  7. * 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;
  8. * Is living in the community (ie not in a nursing home)
  1. * Presence of a bi-allelic GBA1 mutation, or presence of LRRK2 2019S or other LRRK2 mutation;
  2. * 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;
  3. * Montreal Cognitive Assessment (MoCA) score of ≤22;
  4. * History of deep brain stimulator placement, focused ultrasound therapy, or other intercranial surgery for PD;
  5. * Hypersensitivity or contraindications to corticosteroid;
  6. * Prior gene or cell therapy;
  7. * Positive test result for anti-capsid total antibodies (tAb);
  8. * Unable to undergo lumbar puncture;
  9. * Diagnosis of Gaucher disease;
  10. * Clinically significant abnormalities in safety lab tests, vital signs;
  11. * Other illnesses or medications that may affect the interpretation of the study results.

Contacts and Locations

Principal Investigator

Melanie Brandabur, MD
STUDY_DIRECTOR
Capsida Biotherapeutics

Study Locations (Sites)

Rush University Medical Center
Chicago, Illinois, 60612
United States
University of Kansas Medical Center
Kansas City, Kansas, 66160
United States
New York Presbyterian Hospital-Columbia University Medical Center
New York, New York, 10032
United States
Baylor College of Medicine
Houston, Texas, 77030
United States
Booth Gardner Parkinson's Care Center - Evergreen Neuroscience Institute
Kirkland, Washington, 98034
United States
Inland Northwest Research
Spokane, Washington, 99202
United States

Collaborators and Investigators

Sponsor: Capsida Biotherapeutics, Inc.

  • Melanie Brandabur, MD, STUDY_DIRECTOR, Capsida Biotherapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-08-18
Study Completion Date2029-11

Study Record Updates

Study Start Date2025-08-18
Study Completion Date2029-11

Terms related to this study

Keywords Provided by Researchers

  • GBA1
  • Gene Therapy
  • Parkinson Disease

Additional Relevant MeSH Terms

  • GBA1 Parkinson Disease