Parkinson Disease (PD)Phase 1/2NCT07166757
Clinical Study to Evaluate XS411 in Treatment of Early-onset Parkinson's Disease
A Phase 1/2 study testing human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection in people with Parkinson Disease (PD), run by XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd. at 1 site in China.
Trial summary
This study is a Phase I/II clinical study to evaluate the safety, tolerability, and efficacy of XS411 in the treatment of EOPD. The study consists of two phases: Phase I and Phase II. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Parkinson Disease (PD)
- What is being tested?
- human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 1 siteChina
- Study dates
- 22 Sep 2025to 30 Mar 2029
- Phase
- Phase 1/2
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18 years old ≤ age of onset ≤ 50 years old, diagnosed with EOPD (meeting the MDS 2015 clinical diagnostic criteria for Parkinson's disease );
- 18 years old ≤ age at enrollment ≤ 70 years old , male/female;
- Disease duration ≥ 5 years;
- Phase I: the modified Hoehn-Yahr grade in the off-phase is 3-4 (including the critical value) , and the modified Hoehn-Yahr grade in the on-phase is ≤3 ; Phase II: the modified Hoehn-Yahr grade in the off-phase is 2-4 (including the critical value) , and the modified Hoehn-Yahr grade in the on-phase is ≤3;
- Off-period MDS -UPDRS-III score >30;
- Positive L-dopa stress test;
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- The patient is unable to adequately control motor fluctuations even with a stable dose of medication recommended in the "Guidelines for the Treatment of Parkinson's Disease in China (Fourth Edition)" ;
- Patients received stable doses of anti-PD drugs for at least 4 weeks before administration;
- Able to accept surgical anesthesia, suitable for neurosurgery under anesthesia, and able to undergo brain CT /MRI examination;
- Participants agree to postpone any other elective neurosurgery until the completion of the 24-month follow-up study;
- Participants agreed not to participate in any other clinical studies within 24 months after dosing;
- Participants or their legal representatives understand and comply with the research procedures, voluntarily participate and sign the ICF
Who cannot join
- Non-primary PD or Parkinson's superimposed syndromes ;
- Patients are in the late stages of PD and are experiencing severe, disabling peak-dose dyskinesia or biphasic dyskinesia and/or unpredictable or widely fluctuating symptoms;
- Have previously undergone neuronucleotomy, deep brain stimulation (DBS), striatal surgery, extrapyramidal surgery, stereotactic brain surgery, or other brain surgery; or who have undergone other surgical procedures that the investigator determined would affect participation in this study; or who have surgical contraindications
- Patients currently receiving L-dopa intestinal instillation, apomorphine injection, or continuous daily infusion of anti-PD drugs;
- Patients who had used botulinum toxin, phenol, subarachnoid injection of baclofen, or received interventional treatment for dystonia or spasticity within 6 months before medication;
- Have used glucocorticoids or immunosuppressive drugs for a long time within 3 months before the screening visit;
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- Those who have received cell therapy before;
- Patients who received electroconvulsive therapy within 30 days before administration;
- Those who have received or plan to receive vaccines during the trial within 3 months before screening, such as vaccines for novel coronavirus pneumonia (COVID-19), influenza, herpes zoster, and pneumococcal vaccine;
- Those with a history of mental illness who are judged by the researchers to be unsuitable for study participation; or those with severe suicidal ideation currently or within the year before screening or any history of suicide attempts within the past 2 years;
- Those with active epilepsy or currently taking anti-epileptic drugs;
- Those with a history of dementia or severe cognitive impairment; or those with obvious dementia or cognitive impairment at screening; dementia may affect participants' poor compliance, inability to accurately record diaries, and / or inability to sign the ICF;
- Severe anxiety at screening;
- Patients whose previous head CT/MRI examinations showed brain injuries such as brain trauma, cerebral vascular malformation, hydrocephalus, brain tumors, or abnormal brain imaging of the striatum and other brain regions, which significantly increased the surgical risk;
- Those with uncontrolled autoimmune diseases;
- Patients with a history of severe cardiovascular and cerebrovascular diseases;
- Patients with other serious systemic diseases;
- During the screening period, patients have severe arthritis, limp, severe sequelae of stroke, severe osteoporosis, or a history of severe trauma within 1 month (such as hip or lower limb fractures), which may affect the study evaluation as assessed by the researchers;
- Patients with malignant tumors or a history of malignant tumors;
- Patients with active disseminated intravascular coagulation and obvious bleeding tendency within 3 months before signing the ICF, or patients who cannot stop taking antiplatelet drugs or other anticoagulants for at least 10 days before surgery;
- Previous history of abnormal coagulation function;
- Patients with lab result abnormalities during screening include;
- Allergic to the drugs used in the study;
- Are pregnant or breastfeeding, or plan to become pregnant during the study;
- Female participants of fertile potential must have a positive pregnancy test result before dosing; female participants of fertile potential, or male participants who are not sterilized and whose partners are fertile, must not take effective contraceptive measures from the time they sign the ICF until at least 24 months after dosing; female participants must not agree to not donate eggs from the time they sign the ICF until at least 24 months after dosing, and male participants must not agree to not donate sperm from the time they sign the ICF until at least 24 months after dosing;
- Participants who are currently participating in other clinical trials, or have participated in other clinical studies and received intervention treatment within 1 month before administration;
- Those who are considered by the researchers to have poor compliance;
- Any other situation that the researcher deems to jeopardize the safety of the participants or affect the study evaluation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality, 200040
About this study
Official title: A Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Human Allogeneic Induced Pluripotent Stem Cell-derived Dopaminergic Neural Progenitor Cell Injection in the Treatment of Early-onset Parkinson's Disease
This study is a Phase I/II clinical study to evaluate the safety, tolerability, and efficacy of XS411 in the treatment of EOPD. The study consists of two phases: Phase I and Phase II.
Phase I study is planned to be conducted in patients with EOPD, using a single-arm, open-label, traditional " 3+3 " dose-escalation design, aiming to investigate the safety, tolerability and preliminary efficacy of XS411 in the treatment of EOPD and to determine the RP2D.
Read more
Phase I study enrolls 6-12 patients with EOPD. Two dose cohorts (3-6 patients/dose cohort) are planned: 9×10⁶ cells /patient and 1.8 ×10⁷ cells /patient. Each participant will receive a single injection of XS411. Each participant in each dose cohort will be observed for at least 28 days after dosing . If no DLTs occur and the investigator has no other safety concerns for that participant, the next participant in that dose cohort will be enrolled.
Phase II study is planned for patients with EOPD, using a randomized, double-blind, sham-controlled, parallel-group design . The study will investigate the efficacy and safety of XS411 in the treatment of EOPD . Phase II study currently plans to enroll 81 patients with EOPD. The patients will be randomly assigned in a 2:1 ratio to either the experimental or control group. Participants in the experimental group will receive a single injection of XS411 in combination with an immunosuppressant at the RP2D determined during the Phase I dose-escalation phase (which may be adjusted based on the Phase II study results). The control group will receive a sham procedure in combination with an immunosuppressant sham.
- Conditions
- Parkinson Disease (PD)
- Being tested
- human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injectionDrug
5.0×10^7 cells/mL, injection, once, 12 months
| Study group | Role | What they receive |
|---|---|---|
| 9.0×10^6 cells / bilateral putamen | Experimental | Not described Receives: Drug: human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection |
| 1.8×10^7 cells / bilateral putamen | Experimental | Not described Receives: Drug: human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of XS411 cell injection in the treatment of EOPD 28 days after administration
To evaluate the safety and tolerability of XS411 cell injection in the treatment of EOPD through the incidence and severity of AEs/SAEs; the incidence of DLTs ; Vital signs, physical examination, 12-lead ECG, clinical laboratory tests, and head MRI.
AEs occurring during the study will be graded according to CTCAE V5.0. DLT is defined as: (1) any CTCAE grade 3 or 4 AE related to the trial drug (including definitely related, probably related, and possibly related) occurring within 28 days of trial drug administration (such as grade 3 or 4 immune system diseases, infectious diseases, mental illnesses, kidney diseases, etc. related to the trial drug ); (2) Grade 2 AEs cannot be reduced to grade 1 or below within 14 days.
Measured: 28 days after treatment
Secondary outcomes (4)
To check the change on parameters in MDS-UPDRS questionnaire on Day 28, M3, M6, M9 and M12 to evaluate preliminary efficacy of XS411 cell injection.
To evaluate the preliminary efficacy of XS411 cell injection in the treatment of EOPD through Changes from baseline in the scores of parts I, II, III, and IV of the MDS-UPDRS (individual scores, total score, and sum of scores II and III) after administration ;
Measured: 28 days, 3, 6, 9, and 12 months after administration
Evaluation of the improvement of nigrostriatal dopamine transmitter system through PET-CT testing data change compared with baseline in M6 and M12 for 9 partipants
To evaluation of the improvement of nigrostriatal dopamine transmitter system after XS411 cell injection in the treatment of EOPD through 18F-DOPA uptake in the striatum as shown by brain PET-CT at 6 and 12 months after administration compared with baseline.
Measured: 6 and 12 months after administration
To check the change on parameters Hoehn-Yahr (modified) questionnaire on Day 28, M3, M6, M9 and M12 to evaluate preliminary efficacy of XS411 cell injection.
To evaluate the preliminary efficacy of XS411 cell injection in the treatment of EOPD through Change from baseline in the Hoehn-Yahr (modified) Parkinson's disease rating scale score after administration ;
Measured: 28 days, 3, 6, 9, and 12 months after administration
To check the change on parameters on OFF and ON-period change recorded in patient's diary on Day 28, M3, M6, M9 and M12 to evaluate preliminary efficacy of XS411 cell injection.
To evaluate the preliminary efficacy of XS411 cell injection in the treatment of EOPD through Change from baseline in on-period without bothersome dyskinesia; Change from baseline in conscious off-period; Change from baseline in on-period without dyskinesia recorded in Patient's diary
Measured: 28 days, 3, 6, 9, and 12 months after administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2025
- Study start22 Sep 2025
- Primary completion30 Mar 2028 (estimated)
- Study completion30 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson Disease (PD)Studies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07166757
- Sponsor's protocol ID
- XS411-Allo-EOPD-CN1/2-P01
- Status last verified
- Sep 2025
- First submitted
- 6 Aug 2025
- Last updated
- 29 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Human Allogeneic Induced Pluripotent Stem Cell-derived Dopaminergic Neural Progenitor Cell Injection in the Treatment of Early-onset Parkinson's Disease
- ClinicalTrials.gov ID
- NCT07166757
- Acronym / sponsor ID
- XS411-Allo-EOPD-CN1/2-P01
- Conditions
- Parkinson Disease (PD)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 22 Sep 2025 (actual)
- Primary completion
- 30 Mar 2028 (estimated)
- Study completion
- 30 Mar 2029 (estimated)
- First posted
- 10 Sep 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.
This study is a Phase I/II clinical study to evaluate the safety, tolerability, and efficacy of XS411 in the treatment of EOPD. The study consists of two phases: Phase I and Phase II.
Phase I study is planned to be conducted in patients with EOPD, using a single-arm, open-label, traditional " 3+3 " dose-escalation design, aiming to investigate the safety, tolerability and preliminary efficacy of XS411 in the treatment of EOPD and to determine the RP2D.
Phase I study enrolls 6-12 patients with EOPD. Two dose cohorts (3-6 patients/dose cohort) are planned: 9×10⁶ cells /patient and 1.8 ×10⁷ cells /patient. Each participant will receive a single injection of XS411. Each participant in each dose cohort will be observed for at least 28 days after dosing . If no DLTs occur and the investigator has no other safety concerns for that participant, the next participant in that dose cohort will be enrolled.
Phase II study is planned for patients with EOPD, using a randomized, double-blind, sham-controlled, parallel-group design . The study will investigate the efficacy and safety of XS411 in the treatment of EOPD . Phase II study currently plans to enroll 81 patients with EOPD. The patients will be randomly assigned in a 2:1 ratio to either the experimental or control group. Participants in the experimental group will receive a single injection of XS411 in combination with an immunosuppressant at the RP2D determined during the Phase I dose-escalation phase (which may be adjusted based on the Phase II study results). The control group will receive a sham procedure in combination with an immunosuppressant sham.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 9.0×10^6 cells / bilateral putamen | Experimental | Drug: human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection |
| 1.8×10^7 cells / bilateral putamen | Experimental | Drug: human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 18 years old ≤ age of onset ≤ 50 years old, diagnosed with EOPD (meeting the MDS 2015 clinical diagnostic criteria for Parkinson's disease );
- 18 years old ≤ age at enrollment ≤ 70 years old , male/female;
- Disease duration ≥ 5 years;
- Phase I: the modified Hoehn-Yahr grade in the off-phase is 3-4 (including the critical value) , and the modified Hoehn-Yahr grade in the on-phase is ≤3 ; Phase II: the modified Hoehn-Yahr grade in the off-phase is 2-4 (including the critical value) , and the modified Hoehn-Yahr grade in the on-phase is ≤3;
- Off-period MDS -UPDRS-III score >30;
Key exclusion criteria (28 listed)
- Non-primary PD or Parkinson's superimposed syndromes ;
- Patients are in the late stages of PD and are experiencing severe, disabling peak-dose dyskinesia or biphasic dyskinesia and/or unpredictable or widely fluctuating symptoms;
- Have previously undergone neuronucleotomy, deep brain stimulation (DBS), striatal surgery, extrapyramidal surgery, stereotactic brain surgery, or other brain surgery; or who have undergone other surgical procedures that the investigator determined would affect participation in this study; or who have surgical contraindications
- Patients currently receiving L-dopa intestinal instillation, apomorphine injection, or continuous daily infusion of anti-PD drugs;
- Patients who had used botulinum toxin, phenol, subarachnoid injection of baclofen, or received interventional treatment for dystonia or spasticity within 6 months before medication;
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 |
|---|---|---|
| human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection5.0×10^7 cells/mL, injection, once, 12 months | Drug | 1.8×10^7 cells / bilateral putamen, 9.0×10^6 cells / bilateral putamen |
Primary outcome
To evaluate the safety and tolerability of XS411 cell injection in the treatment of EOPD 28 days after administration
Time frame: 28 days after treatment
To evaluate the safety and tolerability of XS411 cell injection in the treatment of EOPD through the incidence and severity of AEs/SAEs; the incidence of DLTs ; Vital signs, physical examination, 12-lead ECG, clinical laboratory tests, and head MRI. AEs occurring during the study will be graded according to CTCAE V5.0. DLT is defined as: (1) any CTCAE grade 3 or 4 AE related to the trial drug (including definitely related, probably related, and possibly related) occurring within 28 days of trial drug administration (such as grade 3 or 4 immune system diseases, infectious diseases, mental illnesses, kidney diseases, etc. related to the trial drug ); (2) Grade 2 AEs cannot be reduced to grade 1 or below within 14 days.
4 secondary outcomes
To check the change on parameters in MDS-UPDRS questionnaire on Day 28, M3, M6, M9 and M12 to evaluate preliminary efficacy of XS411 cell injection.
Time frame: 28 days, 3, 6, 9, and 12 months after administration
Evaluation of the improvement of nigrostriatal dopamine transmitter system through PET-CT testing data change compared with baseline in M6 and M12 for 9 partipants
Time frame: 6 and 12 months after administration
To check the change on parameters Hoehn-Yahr (modified) questionnaire on Day 28, M3, M6, M9 and M12 to evaluate preliminary efficacy of XS411 cell injection.
Time frame: 28 days, 3, 6, 9, and 12 months after administration
To check the change on parameters on OFF and ON-period change recorded in patient's diary on Day 28, M3, M6, M9 and M12 to evaluate preliminary efficacy of XS411 cell injection.
Time frame: 28 days, 3, 6, 9, and 12 months after administration
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 29 Dec 2025.
- Individual participant dataSharing planned: Yes
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection in people with Parkinson Disease (PD), run by XellSmart Bio-Pharmaceutical (Suzhou) 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 12 inclusion and 28 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: looking for participants now, as of the sponsor's last update on 29 Dec 2025. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Contact Michael LEE (+86 21 64027719, CEO@xellsmart.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of XS411 cell injection in the treatment of EOPD 28 days after administration (time frame: 28 days after treatment). 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 29 Dec 2025.