ANPD001 for Parkinson's Disease

(ASPIRO Trial)

Recruiting at 8 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Aspen Neuroscience

Must be taking: Levodopa

Must not be taking: Anticoagulants, Antiplatelets

Disqualifiers: Cognitive impairment, Epilepsy, Stroke, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

9 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must be able to temporarily stop anticoagulation or antiplatelet therapy for at least 2 weeks.

What data supports the effectiveness of the drug ANPD001 for Parkinson's Disease?

The research mentions a study where a drug previously approved for diabetes treatment showed potential as a disease-modifying therapy for Parkinson's Disease, suggesting that similar approaches could be effective. However, there is no direct evidence provided for ANPD001 specifically. ^{1 2 3 4 5}

What safety data exists for ANPD001 or similar treatments for Parkinson's Disease?

The treatment DL-3-n-butylphthalide (NBP), which may be similar to ANPD001, was tested in a study with Parkinson's patients and found to be generally safe, with only mild stomach-related side effects reported. ^{6 7 8 9 10}

How does the drug ANPD001 differ from other treatments for Parkinson's disease?

ANPD001 is unique because it may target the genetic and molecular mechanisms underlying Parkinson's disease, potentially slowing or halting its progression, unlike current treatments that primarily manage symptoms. ^{11 12 13 14 15}

What is the purpose of this trial?

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.

Research Team

Edward D Wirth III, MD, PhD

Principal Investigator

Aspen Neuroscience

Eligibility Criteria

This trial is for individuals with Parkinson's Disease who are willing to have cells, previously collected from their own bodies, modified and injected back into their brain. The details of the eligibility criteria were not provided.

Inclusion Criteria

I am between 50 and 70 years old.

I was diagnosed with Parkinson's Disease over 4 years ago.

Met all eligibility requirements for inclusion in the trial-ready cohort in clinical study ANPD001-01

Exclusion Criteria

I cannot have an MRI or use gadolinium due to health reasons.

Unable to comply with the protocol procedures, including frequent and prolonged follow-up assessments

I've had brain surgery that makes me ineligible for ANPD001 treatment.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Surgical Implantation

Participants undergo surgical implantation of ANPD001 cells into the brain under general anesthesia

1 day

1 visit (in-person)

Primary Follow-up

Participants are monitored for safety, tolerability, and symptom changes for 1 year post-transplant

1 year

Annual in-person visits with MRI and PET imaging

Long-term Follow-up

Participants are monitored for cell survival and symptom changes for 5 years post-transplant

5 years

Annual in-person visits with MRI and PET imaging

Extended Follow-up

Safety and tolerability are assessed annually for an additional 10 years via telephone call

10 years

Treatment Details

Interventions

• ANPD001

Trial Overview The study is testing ANPD001, a treatment involving cells that mature into dopamine-producing neurons in the brain. Participants will receive these specialized cells derived from their own tissues.

Participant Groups

Group I: ANPD001 Experimental Treatment 2 Interventions

Who Is Running the Clinical Trial?

Aspen Neuroscience

Lead Sponsor

Trials

1

Recruited

9+

California Institute for Regenerative Medicine (CIRM)

Collaborator

Trials

70

Recruited

3,300+

Findings from Research

Using advanced gene-editing techniques, researchers created isogenic human A9 dopaminergic neurons to study the effects of parkin mutations linked to early-onset Parkinson's disease, demonstrating a clear causal relationship between these mutations and neuronal dysfunction.

The study found that parkin mutations significantly reduced the frequency of spontaneous action potentials in these neurons, indicating that parkin is essential for maintaining normal dopamine transmission, which is crucial for controlling movement.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Parkin Maintains Robust Pacemaking in Human Induced Pluripotent Stem Cell-Derived A9 Dopaminergic Neurons. Pu, J., Lin, L., Jiang, H., et al.[2023]

References

1. United States pubmed.ncbi.nlm.nih.gov

A new approach to disease-modifying drug trials in Parkinson's disease. [2021]

2. United States pubmed.ncbi.nlm.nih.gov

The NINDS Parkinson's disease biomarkers program. [2023]

3. Switzerland pubmed.ncbi.nlm.nih.gov

The Future of Targeted Gene-Based Treatment Strategies and Biomarkers in Parkinson's Disease. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

BDNF rs6265 Variant Alters Outcomes with Levodopa in Early-Stage Parkinson's Disease. [2021]

5. Austria pubmed.ncbi.nlm.nih.gov

Biomarker candidates of neurodegeneration in Parkinson's disease for the evaluation of disease-modifying therapeutics. [2022]

6. Austria pubmed.ncbi.nlm.nih.gov

Non-human primate models of PD to test novel therapies. [2019]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Gene-Environment Interaction in Parkinson's Disease: Coffee, ADORA2A, and CYP1A2. [2018]

8. Greece pubmed.ncbi.nlm.nih.gov

DL-3-n-butylphthalide therapy for Parkinson's disease: A randomized controlled trial. [2020]

9. Netherlands pubmed.ncbi.nlm.nih.gov

The MPTP model: versatile contributions to the treatment of idiopathic Parkinson's disease. [2019]

10. Netherlands pubmed.ncbi.nlm.nih.gov

The belated US FDA approval of the adenosine A2A receptor antagonist istradefylline for treatment of Parkinson's disease. [2021]

11. United States pubmed.ncbi.nlm.nih.gov

What causes cell death in Parkinson's disease? [2021]

12. United States pubmed.ncbi.nlm.nih.gov

Parkin Maintains Robust Pacemaking in Human Induced Pluripotent Stem Cell-Derived A9 Dopaminergic Neurons. [2023]

13. United States pubmed.ncbi.nlm.nih.gov

Reduced expression of the G209A alpha-synuclein allele in familial Parkinsonism. [2019]

14. United States pubmed.ncbi.nlm.nih.gov

Progress in the clinical and molecular genetics of familial parkinsonism. [2019]

15. Switzerland pubmed.ncbi.nlm.nih.gov

ZPD-2, a Small Compound That Inhibits α-Synuclein Amyloid Aggregation and Its Seeded Polymerization. [2020]