Summary

Eligibility
for people ages 40-70 (full criteria)
Location
at La Jolla, California
Dates
study started
completion around
Principal Investigator
by Joseph Ciacci, MD
Headshot of Joseph Ciacci
Joseph Ciacci

Description

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

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)

Details

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.

Keywords

PD - Parkinson's Disease, PD, Parkinson's Disease, ataxia, dopaminergic, dyskinesia,, Parkinson Disease, Dopamine, Dopamine Agonists, Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001), Single-center, open-label, uncontrolled

Eligibility

You can join if…

Open to people ages 40-70

  1. The patient has been diagnosed with PD at least 5 years ago.
  2. The patient has inadequate response to existing drug treatments.
  3. The patient is ≥ 40 years and < 70 years of age at the time of informed consent.
  4. The patient is in stage 2.5 or higher on the Hoehn and Yahr scale at OFF time.
  5. The patient is in stage 3 or lower on the Hoehn and Yahr scale at ON time.
  6. The patient has a L-dopa response of 30% or more.
  7. 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.
  8. The subject is willing to avoid pregnancy using abstinence, highly effective means of birth control, surgical sterility, or menopause.
  9. The subject does not have dyskinesia.
  10. The subject is willing to comply with the protocol-required assessments.

You CAN'T join if...

  1. The presence of any of these features rules out PD. ii. Unequivocal cerebellar abnormalities, such as cerebellar gait, limb ataxia, or cerebellar oculomotor abnormalities iii. Downward vertical supranuclear gaze palsy or selective slowing of downward vertical saccades iv. Diagnosis of probable behavioral variant frontotemporal dementia or primary progressive aphasia, defined according to the MDS criteria (consensus criteria), within the first 5 years of disease v. Parkinsonian features restricted to the lower limbs for more than 3 years. vi. Treatment with a dopamine receptor blocker or a dopamine-depleting agent in a dose and time-course consistent with drug-induced parkinsonism vii. Absence of observable response to high-dose levodopa despite at least moderate severity of disease viii. Unequivocal cortical sensory loss (i.e., graphesthesia, stereognosis with intact primary sensory modalities), clear limb ideomotor apraxia, or progressive aphasia ix. Normal functional neuroimaging of the presynaptic dopaminergic system x. Documentation of an alternative condition known to produce parkinsonism and plausibly connected to the subject's symptoms, or the expert evaluating physician, based on the full diagnostic assessment, feels that an alternative syndrome is more likely than PD

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Location

  • University of California, San Diego accepting new patients
    La Jolla California 92037 United States

Lead Scientist at UCSD

  • Joseph Ciacci, MD
    Dr. Ciacci is the Principal Investigator on several Clinical Trials for Spinal Cord Injury, and Dr. Ciacci is a Clinical Trial Expert in the Sanford Stem Cell Clinical Center Alpha Clinic. Dr. Ciacci's primary are spinal cord injury, tumors of the spine and brain, and complex spinal reconstruction. Dr. Ciacci has extensive experience with neuro-oncology of the spine and brain.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
University of California, San Diego
Links
Parkinson's disease and related disorders (3) Parkinson's disease Pre-clinical study of induced pluripotent stem cell- derived dopaminergic progenitor cells for Parkinson's Disease
ID
NCT06482268
Phase
Phase 1 Parkinson's Disease Research Study
Study Type
Interventional
Participants
Expecting 7 study participants
Last Updated