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Gene therapy trial targets Parkinson's at its genetic root

NCT ID NCT07011771

Disease control Sponsor: Capsida Biotherapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled This study
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a one-time gene therapy called CAP-003 in adults with Parkinson's disease caused by a GBA1 gene mutation. The goal is to see if it is safe and if it can help control the disease. Participants receive a single IV infusion and are monitored for two years.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Started

Aug 2025

Finished

Jun 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

21 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

GBA1-related Parkinson disease, susceptibility Parkinson disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GBA1 Parkinson disease

Contacts and locations

Locations

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Inland Northwest Research

    Spokane, Washington, 99202, United States

  • New York Presbyterian Hospital-Columbia University Medical Center

    New York, New York, 10032, United States

  • Rush University Medical Center

    Chicago, Illinois, 60612, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.