RecruitingPhase 1

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

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)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
40 Years to 75 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the safety of CT1-DAP001 and how well it works for Parkinson’s disease. It places cells made from human stem cells into a brain area called the striatum; these cells are meant to develop into dopamine-making cells. It is for adults ages 40–75 who have had Parkinson’s for at least 5 years and whose medicines do not work well enough.

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

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have confirmed or likely Parkinson’s disease based on the study’s diagnosis rules.
  • Be ages 40–75 when giving consent and have had Parkinson’s for at least 5 years.
  • Have symptoms that are not controlled well enough by medicines, but improve at least 30% with L-dopa under study testing rules.
  • Have both ON times, when medicine helps, and OFF times, when symptoms return, shown by movement tests and a symptom diary.
  • Meet the study’s symptom rating limits: stage 2 or higher during OFF times and stage 3 or lower during ON times. Have no disabling unwanted movements rated 3 or higher.
  • Meet the study’s blood cell count, liver test, and kidney test limits.
  • Agree to avoid pregnancy using one of the study’s accepted methods.
  • Agree to study tests and give written consent. If physical limits prevent signing, give spoken consent with a legally authorized person’s signature.

Who can't join

  • Have a brain MRI showing another brain problem, or have a different type of condition that causes Parkinson-like symptoms.
  • Have abnormal immune function, dementia or a high risk of it, or bleeding or blood-clotting problems.
  • Have hepatitis B test results listed by the study, positive HIV or HTLV-1 antibody tests, or an active infection such as hepatitis C or syphilis.
  • Cannot safely receive general anesthesia, which puts you to sleep for surgery, or required medicines such as tacrolimus, levodopa, carbidopa, or MRI dye. Also excluded: serious allergy to materials used to make the study cells.
  • Have active cancer, poorly controlled diabetes, or another serious illness. Cancer within the past 5 years may also exclude you, unless it is in complete remission and expected survival is more than 5 years.
  • Have current or past epilepsy or certain mental health conditions, such as uncontrolled anxiety or depression, bipolar disorder, or schizophrenia. Also excluded: a past stroke or brain bleed, or an inherited heart rhythm condition called long QT syndrome.
  • Have had deep brain stimulation, brain surgery called pallidotomy or thalamotomy, or a prior transplant of the same type of stem-cell-derived dopamine cells.
  • Are pregnant or breastfeeding, will not agree to avoid pregnancy during the study, or the study doctor believes participation would not be safe.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: 1. 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). 2. The subject has an inadequate response to drug treatments. 3. The subject is ≥ 40 years and ≤ 75 years of age at the time of informed consent. 4. The subject has had PD for at least 5 years. 5. The subject has both ON and OFF (as demonstrated by the MDS-UPDRS Part III and a symptom diary). 6. The subject does not have a debilitating dyskinesia score greater than or equal to 3 on the MDS-UPDRS. 7. The subject is in stage 2 or higher on the Hoehn and Yahr scale at OFF time. 8. The subject is in stage 3 or lower on the Hoehn and Yahr scale at ON time. 9. The subject has an L-dopa response of 30% or more without influence of antiparkinsonian drugs. 10. The subject has the following organ functions as determined by laboratory tests at Screening visit: 1. Neutrophil count ≥ 2,000/μL 2. Platelet count ≥ 5.0 × 104/μL 3. AST, ALT ≤ 3.0 × upper limit of normal 4. Total bilirubin ≤ 1.5 × upper limit of normal 5. 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) 11. The subject is willing to avoid pregnancy using abstinence, highly effective means of birth control, surgical sterility, or menopause. 12. The subject is willing to comply with the protocol-required assessments. 13. 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. Exclusion Criteria: 1. The subject has an abnormal brain MRI suggestive of brain pathology other than Parkinson's disease. 2. Atypical parkinsonism (Parkinsonism-Plus syndrome, secondary parkinsonism, hereditary parkinsonism). 3. The subject has clinical indication or diagnosis of abnormal immune function. 4. The subject has been diagnosed with a major neurocognitive disorder such as dementia, or is high risk for this. 5. The subject has bleeding tendency or abnormal coagulation function as evidenced by platelets \<50 or PT/PTT \> 1.5x normal. 6. The subject is HBs antigen-positive, or HBs antibody- or HBc antibody-positive with evidence of HBV-DNA. 7. The subject is anti-HIV antibody positive. 8. The subject is anti-HTLV-1 antibody-positive. 9. The subject has active infection such as hepatitis C or syphilis (STS/TPHA). 10. The subject has hypersensitivity or contraindication to tacrolimus, concomitant drugs (e.g., levodopa, carbidopa, MRI contrast), and/or their components. 11. Contraindications to general anesthesia as evaluated by subject matter experts. 12. 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. 13. The subject has any of the following conditions/diseases concurrently: 1. Active malignancy 2. Epilepsy 3. Psychiatric disease (e.g., uncontrolled anxiety or depression, bipolar disorder, schizophrenia) 4. Diabetes mellitus with poorly controlled blood glucose (glycosylated hemoglobin \> 9.0%, or fasting plasma glucose (FPG) ≥ 200 mg/dL (11.1 mmol/L). 5. Other serious concurrent diseases (e.g., cerebrovascular disorder, heart disease, chronic respiratory disease, inadequately controlled hypertension) as determined by the investigator. 14. The subject has a history of any of the following: 1. 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 2. Epilepsy 3. Cerebral hemorrhage or stroke 4. Psychiatric disease (e.g., uncontrolled anxiety or depression, bipolar disorder, schizophrenia) 5. Congenital long QT syndrome 6. Pallidotomy, thalamotomy, or Deep Brain Stimulation 15. The subject is pregnant or lactating or does not agree to avoid pregnancy throughout the study. 16. The subject has undergone transplantation of human iPSC-derived dopaminergic progenitors. 17. The subject, in the opinion of the investigator or sub investigator, is not appropriate to conduct the study safely.

Study sites (1)

CA