The Safety,Tolerability, Efficacy of XS411 Cell Injection for the Treatment of Primary Parkinson's Disease

NCT07080775 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: XS411-Allo-LOPD-CN1/2-P01
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

Brief Summary

This Phase I clinical trial is designed to evaluate the safety, tolerability of a single dose of XS411 (derived dopaminergic neural progenitor cell injection) in patients with primary Parkinson's disease. This Phase II clinical trial is designed to evaluate the safety, tolerability and Efficacy of XS411 (derived dopaminergic neural progenitor cell injection) in patients with Parkinson's disease.

Conditions

Parkinson's Disease

Outcome Measures

Primary Outcomes (2)

• To evaluate the safety and tolerability of XS411CN in treatment of primary Parkinson's disease

  To evaluate the safety and tolerability of XS411CN in treatment of primary Parkinson's disease through: 1）The occurrence and incidence of adverse events and serious adverse events (adverse event grading criteria are based on NCI-CTCAE V5.0) 28 days after administration of XS411CN; 2) Changes in vital signs, laboratory tests, 12-lead electrocardiogram, etc. compared with baseline after administration of XS411CN;

  28 days after administration of XS411CN

• MDS-UPDRS Part III score during the 'OFF' state

  Change from baseline in MDS-UPDRS Part III (Motor Examination) score during the 'OFF' state at 12 months after administration of XS411 cell injection

  Month 12

Secondary Outcomes (1)

• MDS-UPDRS Part III score during the 'OFF' state

  Baseline, Month 3, Month 6, Month 9

Study Arms (4)

4.5×10^6 cells / bilateral putamen

EXPERIMENTAL

Biological: human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection

9.0×10^6 cells / bilateral putamen

EXPERIMENTAL

Biological: human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection

1.8×10^7 cells / bilateral putamen

EXPERIMENTAL

Biological: human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection

Standard treatment

NO INTERVENTION

Interventions

human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection BIOLOGICAL

5.0×10\^7 cells/mL, injection, single dose

1.8×10^7 cells / bilateral putamen; 4.5×10^6 cells / bilateral putamen; 9.0×10^6 cells / bilateral putamen

Eligibility Criteria

• Age: 50 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, aged between 50 and 75 years old;
• Diagnosed as 'clinically established PD' consistent with MDS clinical Diagnostic Criteria for Parkinson's disease,
• A minimum of 5 years since diagnosis;
• Hoehn- Yahr score during the off-time is 3 to 4;
• The subjects with an average of at least 2.5 hours of "off time" per day;
• Stable anti-PD therapy for at least 1 month before screening;
• Anti-PD treatment was once effective, but the efficacy has declined significantly or drug-induced motor complications have occurred, affecting the quality of life;
• Positive levodopa challenge test (MDS-UPDRS-III score improvement from off- state to on- state \> 30%);
• At least three months before the first administration, the subject should have completed all vaccinations as recommended by local authorities;
• The results of complete blood count meet the following conditions: ANC ≥ 2.0×109 /L, WBC ≥ 4.0×109 /L, PLT≥ 100×109 /L, HGB ≥ 10g/dL
• The patient or his/her legal guardian agree to accept the study and signed the informed consent form in writing.

You may not qualify if:

• Parkinsonian syndrome or secondary Parkinson's disease (Atypical Parkinson's syndrome (Parkinson's plus syndrome, secondary Parkinson's syndrome, hereditary degenerative Parkinson's syndrome);
• Patients are in advanced stages of Parkinson's disease and are experiencing severe, disabling peak-dose dyskinesia or biphasic dyskinesia and/or unpredictable or widely fluctuating symptoms;
• Severe cognitive impairment (MMSE\<24, poor compliance due to dementia, inability to accurately record diaries, and/or inability to sign informed consent;
• Those with a history of severe mental illness, or those with severe suicidal tendencies as measured by the Columbia-Suicide Severity Rating Scale (C-SSRS), or those with suicidal ideation in the past 12 months, or those who have attempted suicide in the past 5 years;
• Patients with severe brain atrophy, space-occupying lesions, hydrocephalus, cerebral vascular malformations as indicated by head CT/MRI, and those with structural abnormalities that may affect transplantation or increase surgical risks as determined by the investigators;
• Currently with active internal bleeding; or arterial puncture at a site that is difficult to stop bleeding within 1 week before screening; or gastrointestinal or urinary bleeding within 3 weeks before screening;
• History of striatal or extrapyramidal surgery, including but not limited to deep brain stimulation (DBS) surgery and globus pallidus lesion;
• Coagulation abnormalities;
• Abnormal liver and kidney function laboratory tests during the screening period;
• The patient is currently receiving or has previously received the following treatments:
• Use of benzodiazepines at a dose level equal to or higher than the recommended dose in Package Insert for more than 4 weeks cumulatively since one month before enrollment, immunosuppressants (except for the immunosuppressants to be used in this study), or antipsychotics within 3 months before treatment;
• Use of botulinum toxin, phenol, subarachnoid baclofen injection, or interventional treatment for dystonia or spasticity within 6 months before treatment;
• Have a history of epilepsy or use anti-epileptic drugs for prevention;
• Received cell therapy within 3 months before screening;
• Contraindications to general anesthesia or stereotactic surgery (such as sleep apnea, chronic obstructive pulmonary disease, etc.), MRI or PET examinations;

Sponsors & Collaborators

• XellSmart Therapeutics (Shanghai) Co., Ltd.: lead
• Beijing Tiantan Hospital: collaborator

Study Sites (1)

Beijing Tiantan Hospital

Beijing, Beijing Municipality, 100070, China

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2025
• First Posted: July 23, 2025
• Study Start: August 5, 2025
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2028
• Last Updated: September 30, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)