Phase 1/2a Study of ANPD001 in Parkinson Disease (ASPIRO)

Aspen Neuroscience

Status and phase: Invitation-only; Phase 1

Conditions: Parkinson Disease

Treatments: Device: Custom Device; Biological: ANPD001

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT06344026

ANPD001-02

Details and patient eligibility

About

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.

Full 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)

Enrollment: 9 estimated patients

Sex: All

Ages: 50 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

9 participants in 1 patient group

ANPD001

Experimental group

Treatment:

Biological: ANPD001

Device: Custom Device