Phase 1/2a Study of ANPD001 in Parkinson Disease
- Registration Number
- NCT06344026
- Lead Sponsor
- Aspen Neuroscience
- 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)
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 9
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ANPD001 Custom Device - ANPD001 ANPD001 -
- Primary Outcome Measures
Name Time Method Incidence and severity of treatment emergent adverse events (Safety and Tolerability) 1 year (primary follow up) incidence and severity of treatment emergent adverse events (TEAE) and serious adverse events (SAE)
- Secondary Outcome Measures
Name Time Method "ON" time without troublesome dyskinesia 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) 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) Part II score range is 0 to 52 with 0 being normal and 30 and above being severe
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) Part III score range is 0 to 132 with 0 being normal and 59 and above being severe
Post-injection change in the 18F-DOPA uptake in the putamen 1 year (primary follow up) and 5 years (long term follow up) Post-injection change in the 18-fluorodopa uptake in the putamen from baseline via positron emission tomography (PET)
Trial Locations
- Locations (9)
University of Arizona - Banner Health
🇺🇸Tucson, Arizona, United States
Scripps Health
🇺🇸La Jolla, California, United States
University of California, Los Angeles
🇺🇸Los Angeles, California, United States
University of California, Irvine
🇺🇸Orange, California, United States
University of California, San Diego
🇺🇸San Diego, California, United States
University of California, San Francisco
🇺🇸San Francisco, California, United States
University of Colorado
🇺🇸Aurora, Colorado, United States
Emory University
🇺🇸Atlanta, Georgia, United States
Feinstein Institutes
🇺🇸Manhasset, New York, United States