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

NCT07011771 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: CAP-003-101
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 5

Brief Summary

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

Keywords

GBA1; Gene Therapy; Parkinson Disease

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment emergent adverse events (safety and tolerability)

  Incidence of treatment emergent adverse events assessed through clinical safety laboratory tests (hematology, chemistry, liver function and urinalysis), ECG, vital sign measurements and physical examinations

  2 years

Secondary Outcomes (5)

• Efficacy: Glucosylsphingosine (GluSph) biomarker change

  Baseline, week 4, week 8, week 12, month 6, month 12, month 18, month 24 or end of trial

• Efficacy: Glucosylsphingosine (GluSph) biomarker change

  Baseline, week 12, month 6, month 18

• Efficacy: β-Glucocerebrosidase (GCase) biomarker change

  Baseline, week 4, week 8, week 12, month 6, month 12, month 18, month 24 or end of trial

• Efficacy: β-Glucocerebrosidase (GCase) biomarker change

  Baseline, week 12, month 6, month 18

• Efficacy: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) disease rating change

  Baseline, month 6, month 12, month 18, month 24 or end of trial

Study Arms (2)

Dose Level 1

EXPERIMENTAL

Participants will receive a single dose of CAP-003, administered IV

Drug: gene therapy

Dose Level 2

EXPERIMENTAL

Participants will receive a single dose of CAP-003, administered IV

Drug: gene therapy

Interventions

gene therapy DRUG

IV gene therapy

Dose Level 1

Eligibility Criteria

• Age: 21 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Capsida Biotherapeutics, Inc.: lead

Study Sites (5)

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

New York Presbyterian Hospital-Columbia University Medical Center

New York, New York, 10032, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Inland Northwest Research

Spokane, Washington, 99202, United States

Location

MeSH Terms

Conditions

Parkinson Disease

Interventions

Genetic Therapy

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics; Genetic Engineering; Genetic Techniques; Investigative Techniques

Study Officials

• Melanie Brandabur, MD

  Capsida Biotherapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2025
• First Posted: June 10, 2025
• Study Start: August 18, 2025
• Primary Completion: June 17, 2026
• Study Completion: June 17, 2026
• Last Updated: June 22, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)