Actively Recruiting
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
Led by University of California, San Diego · Updated on 2025-01-29
7
Participants Needed
1
Research Sites
165 weeks
Total Duration
On this page
Sponsors
U
University of California, San Diego
Lead Sponsor
S
Sumitomo Pharma America, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of transplanting human induced pluripotent stem cell-derived dopaminergic progenitors, called CT1-DAP001, into the brain region called the corpus striatum in people with Parkinson's disease. This open-label, uncontrolled phase I/II study aims primarily to assess possible side effects, especially graft expansion, and also to observe changes in Parkinson's symptoms and disease progression. Participants will receive the investigational CT1-DAP001 cells transplanted into the putamen using a specialized cell injector attached to a stereotactic frame. PET agents are also used according to institutional guidelines to assist in the procedure. The study is conducted at a single center and does not involve comparison groups or placebo. The transplantation is carefully managed with close monitoring. During the 24-month study period, participants will undergo evaluations for safety and acceptability, including monitoring adverse events. Researchers will assess quality of life, Parkinson's symptom scores (MDS-UPDRS Part III at ON and OFF times), daily ON and OFF durations, and medication doses. Participants will follow protocol-required assessments and laboratory tests, with attention to organ function and disease status throughout the study.
CONDITIONS
Brief Title
Transplantation of Human iPS Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Parkinson's disease according to the MDS Clinical Diagnostic Criteria (2015)
- Inadequate response to drug treatments
- Age between 40 and 75 years at informed consent
- Parkinson's disease duration of at least 5 years
- Presence of both ON and OFF symptom phases
- Debilitating dyskinesia score less than 3 on MDS-UPDRS
- Hoehn and Yahr stage 2 or higher at OFF time
- Hoehn and Yahr stage 3 or lower at ON time
- L-dopa response of 30% or more without other antiparkinsonian drugs
- Adequate organ function based on lab tests (neutrophils, platelets, liver enzymes, bilirubin, kidney function)
- Willingness to avoid pregnancy and comply with study assessments
- Provided informed consent
You will not qualify if you...
- Abnormal brain MRI indicating pathology other than Parkinson's disease
- Atypical parkinsonism or secondary/hereditary parkinsonism
- Abnormal immune function or active infections (e.g., hepatitis, HIV, HTLV-1, syphilis)
- Major neurocognitive disorders such as dementia or high risk for dementia
- Bleeding disorders or abnormal coagulation
- Positive for hepatitis B markers with active viral DNA
- Hypersensitivity or contraindication to tacrolimus or related drugs
- Contraindications to general anesthesia
- Serious allergies to study product components
- Concurrent serious diseases (malignant neoplasm, epilepsy, psychiatric disorders, uncontrolled diabetes, cerebrovascular or heart disease, chronic respiratory disease, uncontrolled hypertension)
- History of malignancy within 5 years unless in remission with good prognosis
- History of cerebral hemorrhage, stroke, or epilepsy
- Previous pallidotomy, thalamotomy, or deep brain stimulation
- Pregnancy or lactation
- Prior transplantation of human iPSC-derived dopaminergic progenitors
- Investigator judgment of unsafe participation
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single transplantation with follow-up over 24 months
Participants receive a transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors into the corpus striatum using a stereotactic frame and investigational cell injector.
Multiple visits for transplantation and monitoring over 24 months
Duration - Up to 24 months
Participants are monitored for safety, efficacy, and clinical progression of Parkinson's disease after the transplantation.
Regular visits during the 24 months following transplantation
Trial Site Locations
Total: 1 location
1
University of California, San Diego
La Jolla, California, United States, 92037
Actively Recruiting
Research Team
C
Christian Fulinara
D
Donna Brusch
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
OTHER
Number of Arms
1
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