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

NCT06482268 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 808955
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

PD - Parkinson's Disease

Keywords

Parkinson's Disease; Ataxia; Dopaminergic; Dyskinesia; PD; Neurodegenerative Disease; Brain Disease; CNS; Movement Disorder; Central Nervous System Disease; FDOPA; MRI; Corpus striatum

Outcome Measures

Primary Outcomes (2)

• ACCEPTABILITY

  Assessed by presence or absence of graft expansion (\> 3 cm3) in the brain at 24 months after transplantation

  24 months

• SAFETY

  Incidence and severity of treatment emergent adverse events assessed by graft expansion and size in the corpus striatum.

  24 months

Secondary Outcomes (6)

• QUALITY OF LIFE

  24 months

• ACCURACY

  24 months

• MDS-UPDRS Part III totalscore (at OFF time)

  24 months

• MDS-UPDRS Part III totalscore (at ON time)

  24 months

• Average daily ON duration (with or without dyskinesia) and OFF duration

  24 months

Study Arms (1)

Single-center, open-label, uncontrolled

OTHER

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

Combination Product: Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)

Interventions

Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001) COMBINATION_PRODUCT

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.

Also known as: Investigational imaging - FDOPA & FEPPA, Investigational device - Sunovion needle

Single-center, open-label, uncontrolled

Eligibility Criteria

• Age: 40 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).
• The subject has an inadequate response to drug treatments.
• The subject is ≥ 40 years and ≤ 75 years of age at the time of informed consent.
• The subject has had PD for at least 5 years.
• The subject has both ON and OFF (as demonstrated by the MDS-UPDRS Part III and a symptom diary).
• The subject does not have a debilitating dyskinesia score greater than or equal to 3 on the MDS-UPDRS.
• The subject is in stage 2 or higher on the Hoehn and Yahr scale at OFF time.
• The subject is in stage 3 or lower on the Hoehn and Yahr scale at ON time.
• The subject has an L-dopa response of 30% or more without influence of antiparkinsonian drugs.
• The subject has the following organ functions as determined by laboratory tests at Screening visit:
• Neutrophil count ≥ 2,000/μL
• Platelet count ≥ 5.0 × 104/μL
• AST, ALT ≤ 3.0 × upper limit of normal
• Total bilirubin ≤ 1.5 × upper limit of normal
• 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)

You may not qualify if:

• The subject has an abnormal brain MRI suggestive of brain pathology other than Parkinson's disease.
• Atypical parkinsonism (Parkinsonism-Plus syndrome, secondary parkinsonism, hereditary parkinsonism).
• The subject has clinical indication or diagnosis of abnormal immune function.
• The subject has been diagnosed with a major neurocognitive disorder such as dementia, or is high risk for this.
• The subject has bleeding tendency or abnormal coagulation function as evidenced by platelets \<50 or PT/PTT \> 1.5x normal.
• The subject is HBs antigen-positive, or HBs antibody- or HBc antibody-positive with evidence of HBV-DNA.
• The subject is anti-HIV antibody positive.
• The subject is anti-HTLV-1 antibody-positive.
• The subject has active infection such as hepatitis C or syphilis (STS/TPHA).
• The subject has hypersensitivity or contraindication to tacrolimus, concomitant drugs (e.g., levodopa, carbidopa, MRI contrast), and/or their components.
• Contraindications to general anesthesia as evaluated by subject matter experts.
• 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.
• The subject has any of the following conditions/diseases concurrently:
• Malignant neoplasm
• Epilepsy

Sponsors & Collaborators

• University of California, San Diego: lead
• Sumitomo Pharma America, Inc.: collaborator
• Kyoto University: collaborator
• Sumitomo Pharma Co., Ltd.: collaborator
• CiRA Foundation: collaborator

Study Sites (1)

University of California, San Diego

La Jolla, California, 92037, United States

RECRUITING

Related 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

MeSH Terms

Conditions

Parkinson Disease; Ataxia; Dyskinesias; Neurodegenerative Diseases; Brain Diseases; Movement Disorders; Central Nervous System Diseases

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Nervous System Diseases; Synucleinopathies; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Joseph Ciacci, MD

  University of California, San Diego

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Christian Fulinara

CONTACT

(858) 2494020; chfulinara@health.ucsd.edu

Donna Brusch

CONTACT

(760) 505-6649; dbrusch@health.ucsd.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Program Director/Professor

Model Details: This study will be conducted as a single-center, open-label, uncontrolled study to evaluate the safety of transplantation of CT1-DAP001 as the primary objective, and as a secondary objective, to evaluate efficacy. Seven (7) subjects with PD will be followed up for 24 months after transplantation of approximately 4.2-5.4 × 106 dopaminergic progenitors per side into the bilateral putamen.

Study Record Dates

• First Submitted: June 25, 2024
• First Posted: July 1, 2024
• Study Start: June 1, 2024
• Primary Completion: February 28, 2025
• Study Completion (Estimated): May 1, 2028
• Last Updated: January 29, 2025

Record last verified: 2025-01

Locations

US (1)