GBA1 Parkinson DiseasePhase 1/2NCT07011771
A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease
A Phase 1/2 study testing gene therapy in people with GBA1 Parkinson Disease, run by Capsida Biotherapeutics, Inc. at 5 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 21 to 75 years
- Conditions the study is for
- GBA1 Parkinson Disease
- What is being tested?
- gene therapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 5 sitesUnited States
- Study dates
- 18 Aug 2025to 17 Jun 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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;
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- 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Rush University Medical Center
Chicago, Illinois, 60612
Baylor College of Medicine
Houston, Texas, 77030
New York Presbyterian Hospital-Columbia University Medical Center
New York, New York, 10032
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Inland Northwest Research
Spokane, Washington, 99202
About this study
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)
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.
Read the detailed description
This is a Phase 1/2, first-in-human, open-label, multi-centre, dose escalation trial to assess the safety, tolerability, and efficacy of a single intravenous (IV) dose of CAP-003 administered to adult patients aged 21 to 75 with Parkinson's Disease associated with GBA1 mutation.
Approximately 31 participants will be dosed in this trial. Phase 1 is a dose escalation phase that will dose approximately 6 participants divided into 2 cohorts (Cohort 1 and Cohort 2) while Phase 2 will have 1 dose cohort and dose approximately 25 participants. Participants in Phase 1 will be dosed sequentially in each cohort. Phase 2 will allow participants to be dosed concurrently if safety and tolerability data from Phase 1 are deemed acceptable.
Participants will receive a single IV infusion of CAP-003 and will then be followed for 2 years with safety measures, assessments to measure changes from Baseline in motor and non-motor scales, cognitive function, patient and clinical global impression of severity and improvement, disease progression, sleep scale and suicide severity rating.
Upon completion of the study or at the participant's final visit they will be invited to participate in a 3 year safety follow up study.
- Conditions
- GBA1 Parkinson Disease
- Being tested
- gene therapy
- Keywords
- GBA1, Gene Therapy, Parkinson Disease
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
gene therapyDrug
IV gene therapy
gene therapyDrug
IV gene therapy
| Study group | Role | What they receive |
|---|---|---|
| Dose Level 1 | Experimental | Participants will receive a single dose of CAP-003, administered IV Receives: Drug: gene therapy |
| Dose Level 2 | Experimental | Participants will receive a single dose of CAP-003, administered IV Receives: Drug: gene therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 2 years
Secondary outcomes (5)
Efficacy: Glucosylsphingosine (GluSph) biomarker change
Percent change from baseline in GluSph levels in plasma. Higher percent decrease is a better outcome.
Measured: Baseline, week 4, week 8, week 12, month 6, month 12, month 18, month 24 or end of trial
Efficacy: Glucosylsphingosine (GluSph) biomarker change
Percent change from baseline in GluSph levels in CSF. Higher percent decrease is a better outcome.
Measured: Baseline, week 12, month 6, month 18
Efficacy: β-Glucocerebrosidase (GCase) biomarker change
Percent change from baseline in GCase protein levels in plasma. Higher percent increase is a better outcome.
Measured: 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
Percent change from baseline in GCase protein levels in CSF and plasma. Higher percent increase is a better outcome.
Measured: Baseline, week 12, month 6, month 18
Efficacy: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) disease rating change
Change from baseline in MDS-UPDRS Parts II and III. A decrease in score indicates improvement of symptoms.
Measured: Baseline, month 6, month 12, month 18, month 24 or end of trial
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2025
- Study start18 Aug 2025
- Primary completion17 Jun 2026
- Study completion17 Jun 2026
Why it stopped: Per protocol stopping rule was met for a separate trial for a different disease state and different experimental therapy that utilized a similar technology.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GBA1 Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Capsida Biotherapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Melanie Brandabur, MD, Study director, Capsida Biotherapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07011771
- Sponsor's protocol ID
- CAP-003-101
- Status last verified
- Jun 2026
- First submitted
- 2 Jun 2025
- Last updated
- 22 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT07011771
- Acronym / sponsor ID
- CAP-003-101
- Conditions
- GBA1 Parkinson Disease
- Keywords
- GBA1, Gene Therapy, Parkinson Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Per protocol stopping rule was met for a separate trial for a different disease state and different experimental therapy that utilized a similar technology.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Capsida Biotherapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 18 Aug 2025 (actual)
- Primary completion
- 17 Jun 2026 (actual)
- Study completion
- 17 Jun 2026 (actual)
- First posted
- 10 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a Phase 1/2, first-in-human, open-label, multi-centre, dose escalation trial to assess the safety, tolerability, and efficacy of a single intravenous (IV) dose of CAP-003 administered to adult patients aged 21 to 75 with Parkinson's Disease associated with GBA1 mutation.
Approximately 31 participants will be dosed in this trial. Phase 1 is a dose escalation phase that will dose approximately 6 participants divided into 2 cohorts (Cohort 1 and Cohort 2) while Phase 2 will have 1 dose cohort and dose approximately 25 participants. Participants in Phase 1 will be dosed sequentially in each cohort. Phase 2 will allow participants to be dosed concurrently if safety and tolerability data from Phase 1 are deemed acceptable.
Participants will receive a single IV infusion of CAP-003 and will then be followed for 2 years with safety measures, assessments to measure changes from Baseline in motor and non-motor scales, cognitive function, patient and clinical global impression of severity and improvement, disease progression, sleep scale and suicide severity rating.
Upon completion of the study or at the participant's final visit they will be invited to participate in a 3 year safety follow up study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dose Level 1 | Experimental | Drug: gene therapyParticipants will receive a single dose of CAP-003, administered IV |
| Dose Level 2 | Experimental | Drug: gene therapyParticipants will receive a single dose of CAP-003, administered IV |
Who may qualify
- Ages
- 21 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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;
Key exclusion criteria (11 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| gene therapyIV gene therapy | Drug | Dose Level 1 |
| gene therapyIV gene therapy | Drug | Dose Level 2 |
Primary outcome
Incidence of treatment emergent adverse events (safety and tolerability)
Time frame: 2 years
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
5 secondary outcomes
Efficacy: Glucosylsphingosine (GluSph) biomarker change
Time frame: Baseline, week 4, week 8, week 12, month 6, month 12, month 18, month 24 or end of trial
Efficacy: Glucosylsphingosine (GluSph) biomarker change
Time frame: Baseline, week 12, month 6, month 18
Efficacy: β-Glucocerebrosidase (GCase) biomarker change
Time frame: 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
Time frame: Baseline, week 12, month 6, month 18
Efficacy: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) disease rating change
Time frame: Baseline, month 6, month 12, month 18, month 24 or end of trial
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2026.
- Individual participant dataSharing planned: No
Related trials for GBA1 Parkinson Disease
Not yet recruiting
Understanding Alpha-Synuclein Spread in Parkinson's Disease Through Blood Biomarkers and Neuroimaging
Parkinson's Disease (PD), GBA1 Parkinson Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Melanie Brandabur, MD study director
Capsida Biotherapeutics
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing gene therapy in people with GBA1 Parkinson Disease, run by Capsida Biotherapeutics, Inc. at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment emergent adverse events (safety and tolerability) (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2026.