Phase 1/2a Study of ANPD001 in Parkinson Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ANPD001, Custom Device.
- Who it may be relevant to
- Registry conditions: Parkinson Disease. Basic parameters: 40 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Phase 1/2a Dose Escalation Study of ANPD001 in Sporadic Parkinson Disease
Overview
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)
Interventions
- 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
Primary outcome measures
- Incidence and severity of treatment emergent adverse events (Safety and Tolerability) [Time frame: 1 year (primary follow up)]
Secondary outcome measures (5)
- "ON" time without troublesome dyskinesia [Time frame: 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) [Time frame: 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 [Time frame: 1 year (primary follow up) and 5 years (long term follow up)]
- Post-injection change in the 18F-DOPA uptake in the putamen [Time frame: 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) [Time frame: 14 years of long term follow up (4 years in-person visits and 10 additional years follow up via telephone call)]
Eligibility criteria
Inclusion criteria
- Age 40-75 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 / contrast CRT and/or use of contrast agents
- Weight > 300 lbs or Body Mass Index (BMI) > 35
- Newly diagnosed diabetes (HbA1c > 7.0%), uncontrolled diabetes > 8.0% requiring frequent medication changes, 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 (> 3 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 9 centers
- University of Arizona - Banner Health — Tucson
- Scripps Health — La Jolla
- University of California, Los Angeles — Los Angeles
- University of California, Irvine — Orange
- University of California, San Diego — San Diego
- University of California, San Francisco — San Francisco
- University of Colorado — Aurora
- Emory University — Atlanta
- … and 1 more center
Identifiers
NCT: NCT06344026 · ANPD001-02