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Enrolling by Invitation

NCT Number: NCT06344026

Phase 1/2a Study of ANPD001 in 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.

Enrolling by Invitation

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Key information

Conditions

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

Age range

40 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of Arizona - Banner Health, Tucson, Arizona, United States

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About this study

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)

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Custom Device

Device

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

Primary outcomes

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

    Time frame: 1 year (primary follow up)

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

Secondary outcomes

  1. "ON" time without troublesome dyskinesia

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

  2. 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)

    Part II score range is 0 to 52 with 0 being normal and 30 and above being severe

  3. 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)

    Part III score range is 0 to 132 with 0 being normal and 59 and above being severe

  4. 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)

    Post-injection change in the 18-fluorodopa uptake in the putamen from baseline via positron emission tomography (PET)

  5. 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)

Sponsors and collaborators

Lead sponsor

Aspen Neuroscience

Industry

Collaborators

  • California Institute for Regenerative Medicine (CIRM)

Registry information

Official study title

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

Acronym: ASPIRO

Important dates

Study start
2024
Primary completion
2027
Study completion
2030
First posted
Apr 3, 2024
Registry last updated
May 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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