The Safety and Tolerability of XS411CN Injection in Treatment of Primary Parkinson's Disease

NCT07080775 | Recruiting Phase 1 FDA Drug

• 1 other identifier: XS411-Allo-LOPD-CN1/2-P01
• Study Type: interventional
• Target: 12
• 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

Conditions

Parkinson's Disease

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (3)

4.5×10^6 cells / bilateral putamen

EXPERIMENTAL

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

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

Interventions

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

5.0×10\^7 cells/mL, injection, once, 12 months

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 Bio-Pharmaceutical (Suzhou) Co., Ltd.: lead
• Beijing Tiantan Hospital: collaborator

Study Sites (1)

Beijing Tiantan Hospital

Beijing, Beijing Municipality, 100070, China

RECRUITING

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

Central Study Contacts

Michael LEE

CONTACT

+86 21 64027719; CEO@xellsmart.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• 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: April 30, 2026
• Study Completion (Estimated): August 30, 2029
• Last Updated: September 11, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)