ANPD001 for Parkinson's Disease

(ASPIRO Trial)

Enrolling by invitation 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for Parkinson’s Disease, a condition that affects movement and can cause tremors. The treatment involves injecting cells, called ANPD001, into the brain, which are expected to develop into cells that produce dopamine—a chemical that helps control movement. The trial assesses the safety of this approach for individuals who have had Parkinson's for at least four years and respond well to Levodopa, a common Parkinson’s medication. Participants will have the cells created from their own previously collected cells. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

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.

Is there any evidence suggesting that ANPD001 is likely to be safe for humans?

Research has shown that ANPD001 appears promising in early tests. Initial results suggest it is safe and well-tolerated. Participants reported improvements in movement and daily activities. The treatment uses cells from the patient's own body, potentially reducing the risk of rejection. Although this trial remains in its early stages, the FDA's Fast Track designation for ANPD001 highlights its potential. However, further studies are needed to confirm these findings. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Parkinson's Disease, which often focus on managing symptoms through dopamine replacement or modulation, ANPD001 offers a novel approach by targeting the underlying mechanisms of neurodegeneration. Researchers are excited about ANPD001 because it potentially acts on specific cellular pathways that are believed to slow or halt the progression of the disease, rather than just alleviating symptoms. This unique mechanism might provide a transformative shift in how Parkinson's is treated, offering hope for more effective long-term management of the condition.

What evidence suggests that ANPD001 might be an effective treatment for Parkinson's Disease?

Research has shown that ANPD001 could be a promising treatment for Parkinson's disease. In early studies, patients who received ANPD001 demonstrated noticeable improvements in movement and daily activities. This treatment uses the patient's own cells to create new dopamine-producing cells in the brain, helping to prevent the body from attacking them. Preclinical research found no harmful side effects, such as unwanted cell movement or tumor growth in the brain. Overall, these early results offer hope that ANPD001 could be a safe and effective treatment option. ^{1 2 4 6 7}

Who Is on the Research Team?

Edward D Wirth III, MD, PhD

Principal Investigator

Aspen Neuroscience

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. aspenneuroscience.com aspenneuroscience.com/aspen-neuroscience-announces-6-month-aspiro-phase-1-2a-clinical-trial-results-of-personalized-cell-therapy-for-parkinsons-disease/

Aspen Neuroscience Announces 6-Month ASPIRO Phase ... Aspen Neuroscience has released 6-month data from the first three patients dosed in the ASPIRO Phase 1/2a trial of ANPD001, an investigational autologous ...

2. neurologylive.com neurologylive.com/view/parkinson-cell-therapy-anpd001-generates-promising-early-stage-phase-1-2-data

Parkinson Cell Therapy ANPD001 Generates Promising ... Early findings from the ASPIRO trial showed ANPD001 was safe, well-tolerated, and led to marked improvements in motor symptoms and daily functioning.

3. prnewswire.com prnewswire.com/news-releases/aspen-neuroscience-initiates-cohort-3-in-aspiro-phase-12a-trial-highlighting-commercial-formulation-of-anpd001-and-its-transformative-potential-for-parkinsons-disease-302566431.html

Aspen Neuroscience Initiates Cohort 3 in ASPIRO Phase 1/ ... Unlike allogeneic therapies, which use foreign cells from donors, ANPD001 avoids immune rejection and the need for prolonged immunosuppression.

4. clinicaltrials.gov clinicaltrials.gov/study/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 ...

5. aspenneuroscience.com aspenneuroscience.com/preclinical-safety-and-efficacy-data-for-anpd001-presented-by-dr-andres-bratt-leal-at-2023-world-parkinson-congress-in-barcelona/

Preclinical Safety and Efficacy Data for ANPD001 ... The data suggest that after transplantation there is no evidence of migration or tumor formation, and that PD neurons function in vitro and ...

6. aspenneuroscience.com aspenneuroscience.com/aspen-neuroscience-initiates-cohort-3-in-aspiro-phase-1-2a-trial-highlighting-commercial-formulation-of-anpd001-and-its-transformative-potential-for-parkinsons-disease/

Aspen Neuroscience Initiates Cohort 3 in ASPIRO Phase 1/ ... ANPD001 has received Fast Track designation by the U.S. Food & Drug Administration (FDA). About the ASPIRO Trial ASPIRO is the first multi- ...

7. prnewswire.com prnewswire.com/news-releases/preclinical-safety-and-efficacy-data-for-anpd001-presented-by-dr-andres-bratt-leal-at-2023-world-parkinson-congress-in-barcelona-301870795.html

Preclinical Safety and Efficacy Data for ANPD001 ... Preclinical safety and efficacy data presented by Aspen Neuroscience ... ANPD001 and Its Transformative Potential for Parkinson's Disease.