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)
University of California, San Diego
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
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.
Eligibility
- Age range
- 40–75 years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- Combination ProductHuman induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)
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.
Location
- University of California, San DiegoLa Jolla, California