Phase 1/2a Study of ANPD001 in Parkinson Disease

NCT06344026 | Enrolling By Invitation Phase 1 DMC FDA Drug FDA Device

• 1 other identifier: ANPD001-02
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 9

Brief Summary

This clinical trial is designed to test the safety and tolerability of injecting ANPD001 cells that will mature into dopamine-producing cells into the brain of participants with Parkinson Disease. All participants will have ANPD001 cells manufactured from their own previously collected cells.

Conditions

Parkinson Disease

Keywords

Autologous; Induced Pluripotent Stem Cells; Cell Therapy; Dopamine; Regenerative Medicine

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of treatment emergent adverse events (Safety and Tolerability)

  incidence and severity of treatment emergent adverse events (TEAE) and serious adverse events (SAE)

  1 year (primary follow up)

Secondary Outcomes (5)

• "ON" time without troublesome dyskinesia

  1 year (primary follow up) and 5 years (long term follow up)

• Post-injection change in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II (Activities of Daily Living - ADL) and Part III (motor score) in the ON state (total score and scores for Parts I-IV)

  1 year (primary follow up) and 5 years (long term follow up)

• Post-injection change in the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score in the practically defined OFF state

  1 year (primary follow up) and 5 years (long term follow up)

• Post-injection change in the 18F-DOPA uptake in the putamen

  1 year (primary follow up) and 5 years (long term follow up)

• Incidence and severity of treatment emergent adverse events during long term follow-up (Continued Safety and Tolerability)

  14 years of long term follow up (4 years in-person visits and 10 additional years follow up via telephone call)

Study Arms (1)

ANPD001

EXPERIMENTAL

Biological: ANPD001; Device: Custom Device

Interventions

ANPD001 BIOLOGICAL

Biologic: ANPD001 is an experimental product derived from the patient's own skin cells converted to induced pluripotent stem cells. The stem cells were differentiated into precursors for brain cells that produce dopamine.

ANPD001

Custom Device DEVICE

A custom device to facilitate slow injection of small volumes of investigational drugs (including cell therapies), under MRI guidance

ANPD001

Eligibility Criteria

• Age: 50 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 50-70 years of age at time of consent in the trial-ready cohort study ANPD001-01
• Diagnosed with Parkinson Disease at least 4 years ago
• Unequivocal motor response to Levodopa

You may not qualify if:

• Prior brain surgery that, in the opinion of the neurologist or neurosurgeon, contraindicates administration of ANPD001
• History of intracranial therapy for PD, including deep brain stimulation (DBS), focused ultrasound (FUS), gene therapy or other biological therapy
• History of cognitive impairment or dementia
• History of clinically significant Dopa Dysregulation syndrome
• History of epilepsy, stroke, multiple sclerosis, poorly controlled or progressive neurological disease (other than PD), or poorly controlled cardiovascular disease
• Inability to temporarily stop anticoagulation or antiplatelet therapy for at least 2 weeks
• History of malignancy (cerebral or systemic) within the prior 5 years, except treated cutaneous squamous or basal cell carcinomas
• Contraindication to MRI and/or use of gadolinium
• Weight \> 300 lbs or Body Mass Index (BMI) \> 35
• Uncontrolled diabetes (HbA1c \> 7.0%) or any other acute or chronic medical condition that would significantly increase the risks of a neurosurgical procedure
• Pregnancy or lactation
• Significant drug-induced dyskinesia (\>2 for any body part on the Abnormal Involuntary Movement Scale \[AIMS\])
• Male or female with reproductive capacity who is unwilling to use barrier contraception for 3 months post-administration of the investigational product
• Unable to comply with the protocol procedures, including frequent and prolonged follow-up assessments
• Any significant issue raised by the neurologist or neurosurgeon

Sponsors & Collaborators

• Aspen Neuroscience: lead
• California Institute for Regenerative Medicine (CIRM): collaborator

Study Sites (9)

University of Arizona - Banner Health

Tucson, Arizona, 85724, United States

Location

Scripps Health

La Jolla, California, 92037, United States

Location

University of California, Los Angeles

Los Angeles, California, 90095, United States

Location

University of California, Irvine

Orange, California, 92868, United States

Location

University of California, San Diego

San Diego, California, 92037, United States

Location

University of California, San Francisco

San Francisco, California, 94143, United States

Location

University of Colorado

Aurora, Colorado, 80045, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Feinstein Institutes

Manhasset, New York, 11030, United States

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Edward D Wirth III, MD, PhD

  Aspen Neuroscience

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open label, dose escalation of bilateral injection of ANPD001 dopaminergic precursor cells to the putamen

Study Record Dates

• First Submitted: March 18, 2024
• First Posted: April 3, 2024
• Study Start: January 23, 2024
• Primary Completion: October 30, 2025
• Study Completion (Estimated): April 30, 2030
• Last Updated: March 20, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (9)