Parkinson Disease, IdiopathicPhase 1NCT06778265

An Exploratory Clinical Study of UX-DA001 in Subjects With Idiopathic Parkinson's Disease

A Phase 1 study testing UX-DA001 in people with Parkinson Disease, Idiopathic, run by Shanghai UniXell Biotechnology Co., Ltd at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

Search all trials

Trial summary

Who is it for?: Ages 50 to 75 years
Conditions the study is for: Parkinson Disease, Idiopathic
What is being tested?: UX-DA001
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 12 participants
Where: 1 siteChina
Study dates: 1 Mar 2025to Dec 2027
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 50 to 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

The subjects or their legally acceptable representative understand and comply with the study procedures, agree to participate in the clinical trial, and sign the ICF;
Aged between 50-75 years old, male or female;
Subjects diagnosed with idiopathic PD, with a medical history of 5-20 years;
Having received standard anti-PD treatment and been given optimal anti-PD treatment under the guidance of the investigator, but the efficacy has significantly declined;
Good response to levodopa medications; the LCT shows that the maximum improvement rate of the UPDRS part III score exceeds 30%;
The modified H\&Y scale of clinical "OFF" period is ≥ Stage 3 and ≤ Stage 4;

Show all 10Show fewer

Taking a stable dosage of anti-PD medications for at least 4 weeks;
Good physical condition or stable concomitant diseases;
With reliable caregivers who can cooperate to complete the assessment items;
Subjects with good compliance.

Who cannot join

PD Subjects in whom previous genetic testing has found a GBA gene mutation or PD Subjects whom the investigator considers unsuitable for participation in this clinical study due to other gene mutations;
Subjects with the atypical Parkinson's syndrome or secondary Parkinson's syndrome;
Subjects with HIV, HBV, HCV, treponema pallidum (TP) infection, or other active infections;
Subjects infected with HTLV, EBV or CMV whose infection renders their blood cells unsuitable for cell product preparation;
Subjects with a known hereditary disorder;
Subjects with any history of malignancy;

Show all 18Show fewer

Subjects with other serious systemic diseases or functional disorders;
Accompanied by other serious central nervous system diseases or serious cognitive and mental disorders;
Subjects who are currently receiving or have previously received cell therapy or other medicine effecting safety and efficacy assessement;
Subjects whose prior head CT/MRI examinations indicate brain injury, or Subjects with imaging abnormalities in the striatum and other brain regions leading to a significant increase in surgical risk, or Subjects who have previously undergone brain surgery;
Subjects with clinically significant abnormal results in coagulation function, or Subjects who have been using anticoagulants for a long time and cannot discontinue use;
Subjects with a history of severe allergy or hypersensitivity reactions, or a known history of hypersensitivity, or a history of intolerance to the investigational cellular drug or its excipients;
Subjects who have undergone other surgeries within the past six months that the investigator deems may affect this trial, or Subjects who cannot tolerate general anesthesia or stereotactic surgery;
Subjects with contraindications to MRI and PET scans;
Subjects with a history of alcoholism or drug abuse;
Women during pregnancy or lactation;
Subjects who have participated in other interventional clinical trials or similar clinical trials within the past 3 months;
Subjects with other conditions that are not suitable for participation in the clinical study as judged by the investigator.

Trial locations and contacts

1 site in 1 country.

China1 site

Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200025

Other trials in China

About this study

Official title: An Exploratory Clinical Study to Evaluate UX-DA001 Injection (Human Midbrain Dopaminergic Progenitor Cells Injection) in Subjects With Idiopathic Parkinson's Disease

This clinical study is designed to explore the safety and tolerability of UX-DA001. It will also explore if UX-DA001 works to improve motor function in subjects with Parkinson's disease. UX-DA001 manufactured from participant's own cells will differentiate into mature dopaminergic neurons after being transplanted into the brain of the participant.

Read the detailed description

This study is an open-label, multi-center, dose-escalation and dose-expansion exploratory clinical study to evaluate the safety, tolerability, and potential efficacy of UX-DA001 Injection at different dose levels implanted in subjects with idiopathic PD.

Each subject receives only one dose of UX-DA001 for implantation into the putamen bilaterally using stereotactic neurosurgery under general anesthesia. Safety and tolerability of UX-DA001 and its effect on Parkinson's disease symptoms are assessed for 2 years post-treatment.

Conditions: Parkinson Disease, Idiopathic
Being tested: UX-DA001
Keywords: Idiopathic Parkinson´s Disease, autologous iPSC derived mDAPs, Midbrain Dopaminergic Progenitors, Induced pluripotent stem cells, mDAPs, stem cells, iPSC derived product
Medical subject headings: Parkinson Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

UX-DA001Biological
UX-DA001 (Human Midbrain Dopaminergic Progenitor Cells) is used for treating patients with iPD via implanting into bilateral putamina under stereotactic neurosurgery.
Two dose levels will be planned. Each patient only receives one corresponding dose of UX-DA001.

Study groups in NCT06778265
Study group	Role	What they receive
UX-DA001	Experimental	Not described Receives: Biological: UX-DA001

What is being measured

The primary outcome is the main question the study is designed to answer.

The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product
The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product during the surgical treatment period and the 4-week postoperative observation period.
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
Measured: within 4 weeks post surgery
The incidence and severity of AEs/serious adverse events (SAEs)
The incidence and severity of AEs/serious adverse events (SAEs) during the study, including AEs and SAEs during the surgery treatment period, postoperative observation period, and follow-up period. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
Measured: From baseline to 2 years post surgery

Secondary outcomes (7)

18F-FP-CIT uptake using positron emission tomography (PET)
Changes in 18F-FP-CIT uptake using positron emission tomography (PET) from baseline.
Measured: From baseline to 2 years post surgery
The situation of implantation and overgrowth of transplanted cells using cranial MRI
Volume changes of transplanted cells using cranial magnetic resonance imaging (MRI)
Measured: From baseline to 2 years post surgery
Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part III, from baseline.
Minimum score: 0; Maximum score: 132; Higher scores mean a worse outcome.
Measured: From baseline to 2 years post surgery
Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part II, from baseline.
Minimum score: 0; Maximum score: 52; Higher scores mean a worse outcome.
Measured: From baseline to 2 years post surgery
Changes in modified modified Hoehn-Yahr (H&Y) scale from baseline
Grade 0~Grade 5, lower grade means a better outcome
Measured: From baseline to 2 years post surgery
Changes in daily levodopa equivalent dose (LED) from baseline.
Measured: From baseline to 2 years post surgery
Changes in the scores of non-motor symptom scales (NMSS) from baseline.
Minimum score: 0; Maximum score: 960; Higher scores mean a worse outcome.
Measured: From baseline to 2 years post surgery

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Mar 2025Dec 2027

First posted16 Jan 2025
Study start1 Mar 2025
Primary completionDec 2027 (estimated)
Study completionDec 2027 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Parkinson Disease, IdiopathicStudies 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, multi-center, dose-escalation and dose-expansion
Masking: None (open label)
Enrolment: 12 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06778265
Sponsor's protocol ID: UX-DA001-101
Status last verified: Jun 2026
First submitted: 30 Dec 2024
Last updated: 11 Jun 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruitingObservational study
Longitudinal Observation of Neurodegeneration in Parkinson's Disease Using Molecular Imaging and Blood Biomarkers
Idiopathic Parkinson Disease
Not yet recruiting
Non-invasive Vagus Nerve Stimulation for Parkinson's Disease
Idiopathic Parkinson Disease
RecruitingPhase 2
A Phase 2 Study and Open-Label Extension of NEU-411 in Companion Diagnostic-Positive Participants With Early Parkinson's Disease
Parkinson Disease, Parkinson
Recruiting
MediPD: An Online Mediterranean Diet Program for People Living With Parkinson's Disease
Parkinson Disease, Idiopathic
Recruiting
Ginger-PD: Ginger as a Modifier of Ghrelin Levels and Gut Function in Parkinson's Disease
REM Sleep Behavior Disorder, Parkinson s Disease

All Parkinson Disease, Idiopathic trials

Study at a glance

From the registry record, last updated by the sponsor on 11 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Exploratory Clinical Study to Evaluate UX-DA001 Injection (Human Midbrain Dopaminergic Progenitor Cells Injection) in Subjects With Idiopathic Parkinson's Disease
ClinicalTrials.gov ID: NCT06778265
Acronym / sponsor ID: UX-DA001-101
Conditions: Parkinson Disease, Idiopathic
Keywords: Idiopathic Parkinson´s Disease, autologous iPSC derived mDAPs, Midbrain Dopaminergic Progenitors, Induced pluripotent stem cells, mDAPs, stem cells, iPSC derived product
Phase: Phase 1
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 12 participants (estimated)
Lead sponsor: Shanghai UniXell Biotechnology Co., LtdIndustry (pharma, biotech, devices)
Collaborators: Ruijin Hospital
Study start: 1 Mar 2025 (actual)
Primary completion: Dec 2027 (estimated)
Study completion: Dec 2027 (estimated)
First posted: 16 Jan 2025 (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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: open-label, multi-center, dose-escalation and dose-expansion

Arm	Type	Receives
UX-DA001	Experimental	Biological: UX-DA001

Who may qualify

Ages: 50 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (10 listed)

The subjects or their legally acceptable representative understand and comply with the study procedures, agree to participate in the clinical trial, and sign the ICF;
Aged between 50-75 years old, male or female;
Subjects diagnosed with idiopathic PD, with a medical history of 5-20 years;
Having received standard anti-PD treatment and been given optimal anti-PD treatment under the guidance of the investigator, but the efficacy has significantly declined;
Good response to levodopa medications; the LCT shows that the maximum improvement rate of the UPDRS part III score exceeds 30%;

Key exclusion criteria (18 listed)

PD Subjects in whom previous genetic testing has found a GBA gene mutation or PD Subjects whom the investigator considers unsuitable for participation in this clinical study due to other gene mutations;
Subjects with the atypical Parkinson's syndrome or secondary Parkinson's syndrome;
Subjects with HIV, HBV, HCV, treponema pallidum (TP) infection, or other active infections;
Subjects infected with HTLV, EBV or CMV whose infection renders their blood cells unsuitable for cell product preparation;
Subjects with a known hereditary disorder;

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
UX-DA001UX-DA001 (Human Midbrain Dopaminergic Progenitor Cells) is used for treating patients with iPD via implanting into bilateral putamina under stereotactic neurosurgery. Two dose levels will be planned. Each patient only receives one corresponding dose of UX-DA001.	Biological	UX-DA001

Primary outcome

The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product
Time frame: within 4 weeks post surgery
The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product during the surgical treatment period and the 4-week postoperative observation period. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
The incidence and severity of AEs/serious adverse events (SAEs)
Time frame: From baseline to 2 years post surgery
The incidence and severity of AEs/serious adverse events (SAEs) during the study, including AEs and SAEs during the surgery treatment period, postoperative observation period, and follow-up period. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.

7 secondary outcomes

18F-FP-CIT uptake using positron emission tomography (PET)
Time frame: From baseline to 2 years post surgery
The situation of implantation and overgrowth of transplanted cells using cranial MRI
Time frame: From baseline to 2 years post surgery
Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part III, from baseline.
Time frame: From baseline to 2 years post surgery
Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part II, from baseline.
Time frame: From baseline to 2 years post surgery
Changes in modified modified Hoehn-Yahr (H&Y) scale from baseline
Time frame: From baseline to 2 years post surgery
Changes in daily levodopa equivalent dose (LED) from baseline.
Time frame: From baseline to 2 years post surgery
Changes in the scores of non-motor symptom scales (NMSS) from baseline.
Time frame: From baseline to 2 years post surgery

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 11 Jun 2026.
Individual participant dataSharing planned: No

Related trials for Parkinson Disease, Idiopathic

Not yet recruitingObservational study
Longitudinal Observation of Neurodegeneration in Parkinson's Disease Using Molecular Imaging and Blood Biomarkers
Idiopathic Parkinson Disease
Not yet recruiting
Non-invasive Vagus Nerve Stimulation for Parkinson's Disease
Idiopathic Parkinson Disease
RecruitingPhase 2
A Phase 2 Study and Open-Label Extension of NEU-411 in Companion Diagnostic-Positive Participants With Early Parkinson's Disease
Parkinson Disease, Parkinson
Recruiting
MediPD: An Online Mediterranean Diet Program for People Living With Parkinson's Disease
Parkinson Disease, Idiopathic
Recruiting
Ginger-PD: Ginger as a Modifier of Ghrelin Levels and Gut Function in Parkinson's Disease
REM Sleep Behavior Disorder, Parkinson s 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. This study is not recruiting at the moment.

Jun Liu, MD, PhD principal investigator

Ruijin Hospital

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing UX-DA001 in people with Parkinson Disease, Idiopathic, run by Shanghai UniXell Biotechnology Co., Ltd at 1 site in China.

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 10 inclusion and 18 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: under way, not looking for participants, as of the sponsor's last update on 11 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. 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?

The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product (time frame: within 4 weeks post surgery). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06778265.

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 11 Jun 2026.