Phase 1/2a Study of ANPD001 in Parkinson Disease (ASPIRO)

Phase 1/2a Dose Escalation Study of ANPD001 in Sporadic Parkinson Disease

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Parkinson Disease
• Intervention / Treatment: Biological: ANPD001; Device: Custom Device

Detailed Description

Participants will undergo a surgical implantation of cells that will mature into dopamine-producing neurons under general anesthesia into a part of the brain where dopamine production is decreased in patients with Parkinson Disease. The effect on Parkinson Disease symptoms, safety and tolerability, and cell survival are assessed for 5 years post-transplant (with MRI and PET imaging scans of the brain). Safety and tolerability are assessed annually for an additional 10 years via telephone call (total follow-up of 15 years)

• Study Type: Interventional
• Enrollment (Estimated): 9
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Tucson, Arizona, United States, 85724
      • University of Arizona - Banner Health
  • California
    • La Jolla, California, United States, 92037
      • Scripps Health
    • Orange, California, United States, 92868
      • University of California, Irvine
    • San Francisco, California, United States, 94143
      • University of California, San Francisco
  • New York
    • Manhasset, New York, United States, 11030
      • Feinstein Institutes

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

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

Exclusion Criteria:

• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: ANPD001

Biological: ANPD001; 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.; Device: Custom Device; A custom device to facilitate slow injection of small volumes of investigational drugs (including cell therapies), under MRI guidance

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
"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)	Part II score range is 0 to 52 with 0 being normal and 30 and above being severe	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	Part III score range is 0 to 132 with 0 being normal and 59 and above being severe	1 year (primary follow up) and 5 years (long term follow up)
Post-injection change in the 18F-DOPA uptake in the putamen	Post-injection change in the 18-fluorodopa uptake in the putamen from baseline via positron emission tomography (PET)	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)

Collaborators and Investigators

• Sponsor: Aspen Neuroscience
• Collaborators: California Institute for Regenerative Medicine (CIRM)
• Investigators: Study Chair: Edward D Wirth III, MD, PhD, Aspen Neuroscience

Study record dates

Study Major Dates

• Study Start (Actual): January 23, 2024
• Primary Completion (Estimated): October 30, 2025
• Study Completion (Estimated): April 30, 2030

Study Registration Dates

• First Submitted: March 18, 2024
• First Submitted That Met QC Criteria: March 27, 2024
• First Posted (Actual): April 3, 2024

Study Record Updates

• Last Update Posted (Actual): April 19, 2024
• Last Update Submitted That Met QC Criteria: April 17, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Cell Therapy; Autologous; Dopamine; Induced Pluripotent Stem Cells; Regenerative Medicine
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease
• Other Study ID Numbers: ANPD001-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: Yes