Transplant restores dopamine producing cells
- Trial ID
- NCT06482268
- 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)
- Goal
- Transplant restores dopamine producing cells
- Phase
- PHASE1
- Status
- RECRUITING
- Sponsor
- University of California, San Diego
- Study Type
- INTERVENTIONAL
- Enrollment
- 7 participants
- Conditions
- PD - Parkinson's Disease
- Interventions
- Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)
Summary For Families
The goal is to restore dopamine-producing cells in the brain to improve motor symptoms and reduce disabling OFF periods for people whose medications are no longer working well. The approach is a one-time surgical transplant of CT1-DAP001, dopaminergic progenitors made from human induced pluripotent stem cells that are intended to mature into dopamine-producing neurons and replace lost cells, and the grafts are given with immunosuppression; participants must be able to take levodopa because a clear levodopa response is required. As a Phase I/II trial the main focus is on safety and early signs of benefit rather than proving long-term effectiveness. Eligible people are ages 40 to 75, have had Parkinson's at least 5 years, experience ON and OFF periods, show at least a 30% improvement with levodopa, and meet specific health, lab, and MRI criteria.
Locations
- University of California, San Diego, La Jolla, California, United States
Frequently Asked Questions
- What is this trial testing?
- This trial is studying Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001). The goal is to restore dopamine-producing cells in the brain to improve motor symptoms and reduce disabling OFF periods for people whose medications are no longer working well. The approach is a one-time surgical transplant of CT1-DAP001, dopaminergic progenitors made from human induced pluripotent stem cells that are intended to mature into dopamine-producing neurons and replace lost cells, and the grafts are given with immunosuppression; participants must be able to take levodopa because a clear levodopa response is required. As a Phase I/II trial the main focus is on safety and early signs of benefit rather than proving long-term effectiveness. Eligible people are ages 40 to 75, have had Parkinson's at least 5 years, experience ON and OFF periods, show at least a 30% improvement with levodopa, and meet specific health, lab, and MRI criteria.
- Who can participate?
- Participants must be between 40 Years and 75 Years.
- Where is this trial located?
- This trial is recruiting at 1 location.
- Does it cost anything to join?
- No. There is no cost to participate. Study-related care and treatment are provided at no charge.
- How long does the trial last?
- This Phase 1 trial is estimated to last approximately 9 months.
- Will I get the real drug or a placebo?
- It depends on the trial design. Some Parkinson's trials are placebo controlled, meaning a portion of participants get an inactive comparison while others get the experimental treatment. Some use crossover designs so everyone eventually receives the active treatment at some point. Observational trials do not use a placebo at all because they are not testing a new treatment. The consent form for any specific trial spells out the design and your odds of being assigned to placebo before you enroll.
- How often will I need to visit the study site?
- Visit frequency varies by trial. Many Parkinson's studies require an in person visit every 4 to 12 weeks during the active treatment phase, with shorter or longer gaps depending on the design. The site coordinator can give you the full visit schedule before you sign anything, so you know what the time commitment looks like.
- Can I leave the trial if I change my mind?
- Yes. You can withdraw from any clinical trial at any time, for any reason, without affecting your standard medical care. Trials are voluntary by law. The team may ask if you are willing to do a brief exit visit so they can collect safety information, but you are not obligated.
- Will travel or parking be reimbursed?
- Many trials reimburse for parking, mileage, and sometimes lodging if the site is far from your home. Reimbursement policies vary by sponsor and site. When you contact the trial team, ask specifically what is covered and how reimbursement is processed.
- Can my spouse or care partner come with me to visits?
- In most cases yes, and it is often encouraged. Care partners can help with notes, questions, and getting home safely after a long visit. Some study assessments do need to happen one on one, but care partners are usually welcome for the rest of the appointment.