Clinical trial NCT06344026: Phase 1/2a Study of ANPD001 in Parkinson Disease
Official title: Phase 1/2a Dose Escalation Study of ANPD001 in Sporadic Parkinson Disease
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE1
Conditions: Parkinson Disease
Keywords: Autologous
Induced Pluripotent Stem Cells
Cell Therapy
Dopamine
Regenerative Medicine
Lead sponsor: Aspen Neuroscience
Sponsor class: INDUSTRY
Collaborators: name: California Institute for Regenerative Medicine (CIRM)
First posted: 2024-04-03
Enrollment: 9
Sex: ALL
Minimum age (years): 50
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label, dose escalation of bilateral injection of ANPD001 dopaminergic precursor cells to the putamen
Primary purpose: TREATMENT

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.

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)

Eligibility criteria: 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

Primary outcomes:
measure: Incidence and severity of treatment emergent adverse events (Safety and Tolerability)
timeFrame: 1 year (primary follow up)
Secondary outcomes:
measure: "ON" time without troublesome dyskinesia
timeFrame: 1 year (primary follow up) and 5 years (long term follow up)
measure: 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)
timeFrame: 1 year (primary follow up) and 5 years (long term follow up)
measure: 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
timeFrame: 1 year (primary follow up) and 5 years (long term follow up)
measure: Post-injection change in the 18F-DOPA uptake in the putamen
timeFrame: 1 year (primary follow up) and 5 years (long term follow up)
measure: Incidence and severity of treatment emergent adverse events during long term follow-up (Continued Safety and Tolerability)
timeFrame: 14 years of long term follow up (4 years in-person visits and 10 additional years follow up via telephone call)
Other outcomes:
Locations:
city: Tucson
country: United States
facility: University of Arizona - Banner Health
state: Arizona
city: La Jolla
country: United States
facility: Scripps Health
state: California
city: Los Angeles
country: United States
facility: University of California, Los Angeles
state: California
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: San Diego
country: United States
facility: University of California, San Diego
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Manhasset
country: United States
facility: Feinstein Institutes
state: New York
Study officials:
affiliation: Aspen Neuroscience
name: Edward D Wirth III, MD, PhD
role: STUDY_CHAIR
References: