Parkinson DiseasePhase 1ASPIRONCT06344026
Phase 1/2a Study of ANPD001 in Parkinson Disease
A Phase 1 study testing ANPD001 and Custom Device in people with Parkinson Disease, run by Aspen Neuroscience at 9 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 40 to 75 years
- Conditions the study is for
- Parkinson Disease
- What is being tested?
- ANPD001 and Custom Device
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 9 sitesUnited States
- Study dates
- 23 Jan 2024to 30 Apr 2030
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 15Show fewer
- 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
Trial locations and contacts
9 sites in 1 country. Contact the study team or a site near you to ask about taking part.
9 sites in 1 country
United States9 sites
Emory University
Atlanta, Georgia, 30322
University of Colorado
Aurora, Colorado, 80045
Scripps Health
La Jolla, California, 92037
University of California, Los Angeles
Los Angeles, California, 90095
Feinstein Institutes
Manhasset, New York, 11030
University of California, Irvine
Orange, California, 92868
University of California, San Diego
San Diego, California, 92037
University of California, San Francisco
San Francisco, California, 94143
University of Arizona - Banner Health
Tucson, Arizona, 85724
About this study
Official title: Phase 1/2a Dose Escalation Study of ANPD001 in Sporadic 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 participants with Parkinson Disease. All participants will have ANPD001 cells manufactured from their own previously collected cells.
Read the detailed 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)
- Conditions
- Parkinson Disease
- Being tested
- ANPD001, Custom Device
- Keywords
- Autologous, Induced Pluripotent Stem Cells, Cell Therapy, Dopamine, Regenerative Medicine
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANPD001Biological
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.
Custom DeviceDevice
A custom device to facilitate slow injection of small volumes of investigational drugs (including cell therapies), under MRI guidance
| Study group | Role | What they receive |
|---|---|---|
| ANPD001 | Experimental | Not described Receives: Biological: ANPD001; Device: Custom Device |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 1 year (primary follow up)
Secondary outcomes (5)
"ON" time without troublesome dyskinesia
Measured: 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)
Part II score range is 0 to 52 with 0 being normal and 30 and above being severe
Measured: 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
Part III score range is 0 to 132 with 0 being normal and 59 and above being severe
Measured: 1 year (primary follow up) and 5 years (long term follow up)
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)
Measured: 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)
Measured: 14 years of long term follow up (4 years in-person visits and 10 additional years follow up via telephone call)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2024
- Study start23 Jan 2024
- Primary completion30 Mar 2027 (estimated)
- Study completion30 Apr 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open label, dose escalation of bilateral injection of ANPD001 dopaminergic precursor cells to the putamen
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aspen Neuroscience
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Responsible party
- Sponsor
- Investigators
- Revati Shreeniwas, MD, FCCP, Study chair, Aspen Neuroscience
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06344026
- Sponsor's protocol ID
- ANPD001-02
- Acronym
- ASPIRO
- Status last verified
- May 2026
- First submitted
- 18 Mar 2024
- Last updated
- 14 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2a Dose Escalation Study of ANPD001 in Sporadic Parkinson Disease
- ClinicalTrials.gov ID
- NCT06344026
- Acronym / sponsor ID
- ASPIRO / ANPD001-02
- Conditions
- Parkinson Disease
- Keywords
- Autologous, Induced Pluripotent Stem Cells, Cell Therapy, Dopamine, Regenerative Medicine
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Aspen NeuroscienceIndustry (pharma, biotech, devices)
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Study start
- 23 Jan 2024 (actual)
- Primary completion
- 30 Mar 2027 (estimated)
- Study completion
- 30 Apr 2030 (estimated)
- First posted
- 3 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label, dose escalation of bilateral injection of ANPD001 dopaminergic precursor cells to the putamen
| Arm | Type | Receives |
|---|---|---|
| ANPD001 | Experimental | Biological: ANPD001, Device: Custom Device |
Who may qualify
- Ages
- 40 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ANPD001Biologic: 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. | Biological | ANPD001 |
| Custom DeviceA custom device to facilitate slow injection of small volumes of investigational drugs (including cell therapies), under MRI guidance | Device | ANPD001 |
Primary outcome
Incidence and severity of treatment emergent adverse events (Safety and Tolerability)
Time frame: 1 year (primary follow up)
incidence and severity of treatment emergent adverse events (TEAE) and serious adverse events (SAE)
5 secondary outcomes
"ON" time without troublesome dyskinesia
Time frame: 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)
Time frame: 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
Time frame: 1 year (primary follow up) and 5 years (long term follow up)
Post-injection change in the 18F-DOPA uptake in the putamen
Time frame: 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)
Time frame: 14 years of long term follow up (4 years in-person visits and 10 additional years follow up via telephone call)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2026.
- Individual participant dataSharing planned: No
Related trials for Parkinson Disease
Not yet recruitingPhase 1/2
STAR0602 With Nivolumab in Non Small Cell Lung Cancer and in Metastatic Castrate-Resistant Prostate Cancer, and STAR0602 Alone in Thymic Carcinoma
NSCLC, Non-Small Cell Lung Cancer
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
RecruitingPhase 3
A Clinical Study of Sacituzumab Tirumotecan (MK-2870) in Combination With Pembrolizumab (MK-3475) as First-line Maintenance Treatment of Cervical Cancer (MK-2870-036/TroFuse-036/GOG-3123/ENGOT-cx22)
Cervical Cancer
Active, not recruitingPhase 1/2
Clinical Safety and Efficacy of Umbilical Cord Mesenchymal Stromal Cells-derived Exosomes in Patients With Parkinson's Disease
Parkinson Disease (PD)
Active, not recruitingObservational study
Adaptive DBS to Offset STN Dysregulation During Exercise in PD
Parkinson Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Revati Shreeniwas, MD, FCCP study chair
Aspen Neuroscience
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ANPD001 and Custom Device in people with Parkinson Disease, run by Aspen Neuroscience at 9 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 14 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of treatment emergent adverse events (Safety and Tolerability) (time frame: 1 year (primary follow up)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2026.