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PD - Parkinson's DiseasePhase 1CT1-DAP001NCT06482268

Transplantation of Human iPS Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II)

A Phase 1 study testing Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001) in people with PD - Parkinson's Disease, run by University of California, San Diego at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

To evaluate the safety and efficacy of transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors, CT1-DAP001, into the corpus striatum in patients with Parkinson's disease Read more about this study

Who is it for?
Ages 40 to 75 years
Conditions the study is for
PD - Parkinson's Disease
What is being tested?
Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
7 participants
Where
1 siteUnited States
Study dates
1 Jun 2024to May 2028
Phase
Phase 1
Are there results?
Not yet: the study is under way

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 40 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • The subject has a diagnosis of PD (clinically established or clinically probable) in accordance with the MDS Clinical Diagnostic Criteria for Parkinson's Disease (2015).
  • The subject has an inadequate response to drug treatments.
  • The subject is ≥ 40 years and ≤ 75 years of age at the time of informed consent.
  • The subject has had PD for at least 5 years.
  • The subject has both ON and OFF (as demonstrated by the MDS-UPDRS Part III and a symptom diary).
  • The subject does not have a debilitating dyskinesia score greater than or equal to 3 on the MDS-UPDRS.
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  • The subject is in stage 2 or higher on the Hoehn and Yahr scale at OFF time.
  • The subject is in stage 3 or lower on the Hoehn and Yahr scale at ON time.
  • The subject has an L-dopa response of 30% or more without influence of antiparkinsonian drugs.
  • The subject has the following organ functions as determined by laboratory tests at Screening visit:
  • Neutrophil count ≥ 2,000/μL
  • Platelet count ≥ 5.0 × 104/μL
  • AST, ALT ≤ 3.0 × upper limit of normal
  • Total bilirubin ≤ 1.5 × upper limit of normal
  • eGFR ≥ 60 mL/min/1.73 m2 (As part of Creatinine testing, an estimated glomerular filtration rate (mL/min/1.73 m2)will be calculated based on the CKD-EPI 2021 equation)
  • The subject is willing to avoid pregnancy using abstinence, highly effective means of birth control, surgical sterility, or menopause.
  • The subject is willing to comply with the protocol-required assessments.
  • The subject provides written informed consent to participate in the study. If the subject cannot sign due to physical constraints, verbal consent may be provided with signature of a Legally Authorized Representative.

Who cannot join

  • The subject has an abnormal brain MRI suggestive of brain pathology other than Parkinson's disease.
  • Atypical parkinsonism (Parkinsonism-Plus syndrome, secondary parkinsonism, hereditary parkinsonism).
  • The subject has clinical indication or diagnosis of abnormal immune function.
  • The subject has been diagnosed with a major neurocognitive disorder such as dementia, or is high risk for this.
  • The subject has bleeding tendency or abnormal coagulation function as evidenced by platelets <50 or PT/PTT > 1.5x normal.
  • The subject is HBs antigen-positive, or HBs antibody- or HBc antibody-positive with evidence of HBV-DNA.
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  • The subject is anti-HIV antibody positive.
  • The subject is anti-HTLV-1 antibody-positive.
  • The subject has active infection such as hepatitis C or syphilis (STS/TPHA).
  • The subject has hypersensitivity or contraindication to tacrolimus, concomitant drugs (e.g., levodopa, carbidopa, MRI contrast), and/or their components.
  • Contraindications to general anesthesia as evaluated by subject matter experts.
  • The subject has a serious allergy to a component (e.g., gentamicin, component of bovine origin, or component of porcine origin) used in the preparation of the study product.
  • The subject has any of the following conditions/diseases concurrently:
  • Active malignancy
  • Epilepsy
  • Psychiatric disease (e.g., uncontrolled anxiety or depression, bipolar disorder, schizophrenia)
  • Diabetes mellitus with poorly controlled blood glucose (glycosylated hemoglobin > 9.0%, or fasting plasma glucose (FPG) ≥ 200 mg/dL (11.1 mmol/L).
  • Other serious concurrent diseases (e.g., cerebrovascular disorder, heart disease, chronic respiratory disease, inadequately controlled hypertension) as determined by the investigator.
  • The subject has a history of any of the following:
  • Prior malignancy < 5 years prior to Screening. Patients who had prior malignancies within 5 years and in complete remission with expected survival of more than 5 years are not excluded
  • Epilepsy
  • Cerebral hemorrhage or stroke
  • Psychiatric disease (e.g., uncontrolled anxiety or depression, bipolar disorder, schizophrenia)
  • Congenital long QT syndrome
  • Pallidotomy, thalamotomy, or Deep Brain Stimulation
  • The subject is pregnant or lactating or does not agree to avoid pregnancy throughout the study.
  • The subject has undergone transplantation of human iPSC-derived dopaminergic progenitors.
  • The subject, in the opinion of the investigator or sub investigator, is not appropriate to conduct the study safely.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Alpha Stem Cell Clinic contact

(858) 249-4020

AlphaStemCellClinic@health.ucsd.edu

United States1 site, 1 recruiting

  • University of California, San Diego

    La Jolla, California, 92037

    Alpha Stem Cell Clinic, 858-249-4020, AlphaStemCellClinic@health.ucsd.edu

    Miguel Alvendia, 858-249-4020, AlphaStemCellClinic@health.ucsd.edu

    Recruiting

Other trials in United States

About this study

Official title: An Investigator-initiated Clinical Trial of Safety and Efficacy of Transplantation of Human Induced Pluripotent Stem Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II)

To evaluate the safety and efficacy of transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors, CT1-DAP001, into the corpus striatum in patients with Parkinson's disease

Read the detailed description

Single-center, open-label, uncontrolled. The primary objective of this study is to evaluate the safety of CT1-DAP001 in subjects with Parkinson's disease by determining the incidence and severity of adverse events, especially graft expansion, after transplantation into the corpus striatum. Other objectives are to evaluate the efficacy of CT1-DAP001 through the assessment of Parkinson's disease symptoms and clinical severity or progression.

Being tested
Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)
Keywords
Parkinson's Disease, Ataxia, Dopaminergic, Dyskinesia, PD, Neurodegenerative Disease, Brain Disease, CNS, Movement Disorder, Central Nervous System Disease, FDOPA, MRI, Corpus striatum
Medical subject headings
Parkinson Disease, Ataxia, Dyskinesias, Neurodegenerative Diseases, Brain Diseases, Movement Disorders, Central Nervous System Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)Combination product

    Also called Investigational imaging - FDOPA & FEPPA, Investigational device - Sunovion needle

    Investigational PET agents will be synthesized at UCSD and administered to subjects according to the local institutional guidelines. IND applications for these agents are linked with the IND application for the study product, CT1-DAP001. The IND applications for the PET radiopharmaceuticals contains the manufacturing method, specifications, quality testing, clinical usage, and safety and efficacy information for individual PET agents.

    Investigational Cell Injector Suniviion needle: The investigational instrument will be used to administer dopaminergic progenitors into the putamen. After aspirating cells, the instrument will be attached to a Leksell stereotactic frame to administer the cells into the brain.

Study groups in NCT06482268
Study groupRoleWhat they receive
Single-center, open-label, uncontrolledOtherTo evaluate the safety and efficacy of transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors, CT1-DAP001, into the corpus striatum in patients with Parkinson's disease
Receives: Combination Product: Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence And Severity Of Adverse Events

    For adverse events reported, the severity will be assessed, and the incidence in the treatment period will be determined.

    Measured: 24 months

  2. ACCEPTABILITY

    Assessed by presence or absence of graft expansion (> 3 cm3) in the brain at 24 months after transplantation

    Measured: 24 months

Secondary outcomes (6)

  1. Improvement or worsening of involuntary movements

    Assessed by dyskinesia score, measured as the change in dyskinesia score from Baseline to each post-transplantation time point.

    Measured: 24 months

  2. ACCURACY

    Assessed by FDOPA (MRI) of tissue expansion The expansion on MRI will be assessed at each time point of measurement

    • day before transplantation,
    • immediately after transplantation,
    • day after transplantation,
    • 4 weeks, 12 weeks, 6 months, 18 months, (if clinically indicated)
    • 12 months, 24 months after transplantation

    Measured: 24 months

  3. MDS-UPDRS Part III totalscore (at OFF time)

    This endpoint is defined as the change in UPDRS Part III score at OFF time from Baseline to each post-transplantation time point. The efficacy will be assessed based on MDS-UPDRS Part III total score at OFF time at 24 months after cell transplantation.

    1. Excellent response defined as a decrease of ≥ 5
    2. Good response defined as a decrease of 0 to 4

    Measured: 24 months

  4. MDS-UPDRS Part III totalscore (at ON time)

    This endpoint is defined as the change in UPDRS Part III score at ON time from Baseline to each post-transplantation time point.

    Measured: 24 months

  5. Average daily ON duration (with or without dyskinesia) and OFF duration

    This endpoint is defined as the change in daily ON duration (with or without dyskinesia) or OFF duration from Baseline to each post-transplantation time point.

    Measured: 24 months

  6. L-dopa equivalent dose

    This endpoint is defined as the change in L-dopa equivalent dose from Baseline to 4 weeks, 12 weeks, 6 months, 12 months, 18 months, or 24 months after transplantation.

    Formula to convert to the L-dopa dose:

    L-dopa 100 mg = pramipexole salt 1 mg = ropinirole 5 mg = rotigotine 7.5 mg = bromocriptine 10 mg = pergolide 1 mg = cabergoline 1.5 mg = selegiline 10 mg = amantadine 100 mg = apomorphine 10 mg

    Measured: 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Jul 2024
  2. Study start1 Jun 2024
  3. Primary completion28 Dec 2027 (estimated)
  4. Study completionMay 2028 (estimated)

Resources

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
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Model description
This study will be conducted as a single-center, open-label, uncontrolled study to evaluate the safety of transplantation of CT1-DAP001 as the primary objective, and as a secondary objective, to evaluate efficacy. Seven (7) subjects with PD will be followed up for 24 months after transplantation of approximately 4.2-5.4 × 106 dopaminergic progenitors per side into the bilateral putamen.
Masking
None (open label)
Enrolment
7 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06482268
Sponsor's protocol ID
808955
Acronym
CT1-DAP001
Status last verified
Aug 2026
First submitted
25 Jun 2024
Last updated
17 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001) in people with PD - Parkinson's Disease, run by University of California, San Diego at 1 site 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 18 inclusion and 28 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: looking for participants now, as of the sponsor's last update on 17 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Alpha Stem Cell Clinic ((858) 249-4020, AlphaStemCellClinic@health.ucsd.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence And Severity Of Adverse Events (time frame: 24 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06482268.

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 17 Aug 2026.

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