Phase 1/2a Study of ANPD001 in Parkinson Disease

Parkinson Disease Clinical Trial

— ASPIRO  

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT06344026
• Other study ID #: ANPD001-02
• Status: Enrolling by invitation
• Phase: Phase 1
• Start date: January 23, 2024
• Est. completion date: April 30, 2030

Study information

• Verified date: April 2024
• Source: Aspen Neuroscience
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Enrolling by invitation
• Enrollment: 9
• Est. completion date: April 30, 2030
• Est. primary completion date: October 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Parkinson Disease

Intervention

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

Locations

Country	Name	City	State
United States	Scripps Health	La Jolla	California
United States	Feinstein Institutes	Manhasset	New York
United States	University of California, Irvine	Orange	California
United States	University of California, San Francisco	San Francisco	California
United States	University of Arizona - Banner Health	Tucson	Arizona

Sponsors (2)

Lead Sponsor	Collaborator
Aspen Neuroscience	California Institute for Regenerative Medicine (CIRM)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and severity of treatment emergent adverse events (Safety and Tolerability)	incidence and severity of treatment emergent adverse events (TEAE) and serious adverse events (SAE)	1 year (primary follow up)
Secondary	"ON" time without troublesome dyskinesia		1 year (primary follow up) and 5 years (long term follow up)
Secondary	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)	Part II score range is 0 to 52 with 0 being normal and 30 and above being severe	1 year (primary follow up) and 5 years (long term follow up)
Secondary	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	Part III score range is 0 to 132 with 0 being normal and 59 and above being severe	1 year (primary follow up) and 5 years (long term follow up)
Secondary	Post-injection change in the 18F-DOPA uptake in the putamen	Post-injection change in the 18-fluorodopa uptake in the putamen from baseline via positron emission tomography (PET)	1 year (primary follow up) and 5 years (long term follow up)
Secondary	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)