Phase 1/2a Study of ANPD001 in Parkinson Disease

Официальное название

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

Обзор

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.

Подробное описание

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)

Вмешательства

• Биопрепарат 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.
• Устройство Custom Device
  A custom device to facilitate slow injection of small volumes of investigational drugs (including cell therapies), under MRI guidance

Первичные конечные точки

• Incidence and severity of treatment emergent adverse events (Safety and Tolerability) [Срок оценки: 1 year (primary follow up)]

Вторичные конечные точки (5)

• "ON" time without troublesome dyskinesia [Срок оценки: 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) [Срок оценки: 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 [Срок оценки: 1 year (primary follow up) and 5 years (long term follow up)]
• Post-injection change in the 18F-DOPA uptake in the putamen [Срок оценки: 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) [Срок оценки: 14 years of long term follow up (4 years in-person visits and 10 additional years follow up via telephone call)]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 9 центров

• 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
• … и ещё 1 центр

Идентификаторы

NCT: NCT06344026 · ANPD001-02

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗