A Clinical Trial of Parkinson's Disease Treatment by HiPSCs Derived Dopaminergic Neural Precursor Cells

NCT06145711 | Recruiting DMC

• 1 other identifier: XS2022001
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 2

Brief Summary

XellSmart Biomedical (Suzhou) Co., Ltd. has closely collaborated with Shanghai East Hospital(East Hospital Affiliated To Tongji University) , using iPSC seed cells, on the development and establishment of a dopaminergic neural precursor cell production system. This study is conducted under collaboration between Shanghai East Hospital and XellSmart Biomedical (Suzhou) Co., Ltd. It is a distinctive stem cell therapy research targeting Parkinson's disease patients, characterized by the following:

1. 1.The first instance in China of using autologous iPSC-derived subtype-specific dopaminergic neural precursor cells for alternative transplantation therapy in Parkinson's disease.
2. 2.Before the initiation of this study, there was only one reported case around the world involving the use of laboratory-grade autologous iPSC-induced dopaminergic neural precursor cells for Parkinson's disease treatment in an American Caucasian individual. Positive preliminary clinical research results were obtained and published in the New England Journal of Medicine in 2020. Since then, no similar studies have been reported.
3. 3.The use of clinical-grade autologous iPSCs differentiated into dopaminergic neural cells, and treat Parkinson's disease.
4. 4.Evaluating the safety of using autologous iPSC-differentiated dopaminergic neural cells for Parkinson's disease treatment in individuals of Asian descent or Chinese ethnicity.

Conditions

Parkinson's Disease

Keywords

Parkinson's disease;stem cell

Outcome Measures

Primary Outcomes (1)

• 1.Number of participants with adverse event (AE), serious adverse event(SAE) and adverse events of special interest(AESI)

  AE occurring throughout the study period will be evaluated using the CTCAE V5.0 standard. AE/SAE and AESI were evaluated after Ommaya sac installation by laboratory examination, measurement of vital signs, physical examination, and subjects' symptoms to detect new abnormalities and/or deterioration of previous conditions and evaluate the safety of the study. AESI is defined as intracranial infection, hemorrhage, rejection, and edema within 7 days after Ommaya sac installation, as well as acute allergic reactions and ectopic mass formation associated with study therapy. \[Time Frame: 18 Months\]

  18 Months

Secondary Outcomes (6)

• Changes in Unified Parkinson's Disease Rating Scale (UPDRS) in OFF state between the experimental group and the control group at 18 months after the first hiPSCs-DAP treatment

  day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month

• Changes in the Hoehn and Yahr scale

  day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month

• Changes in the Schwab and England score

  day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month

• Changes in the Parkinson's Disease Questionnaire (PDQ-39)

  day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month

• Hamilton Depression Rating Scale (HAM-D)

  day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month

Study Arms (1)

to use cell

EXPERIMENTAL

Other: hiPSC-DAP

Interventions

hiPSC-DAP OTHER

Using hiPSC-DAP for the treatment of the patients of Parkinson's diease

to use cell

Eligibility Criteria

• Age: 30 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The patient or their legally authorized guardian consents to participate in this study and signs the Informed Consent Form (ICF) in writing.
• Patients with primary Parkinson's disease, aged between 30 and 70 years, of any gender, with a disease duration of more than 5 years.
• Hohen-Yahr stage for Parkinson's disease (Appendix V) is between stage 3 and stage 4. Patients with a Hohen-Yahr stage below 3 who refuse other treatments may also be included.
• The MDS-UPDRS-III score for "OFF" periods in Parkinson's disease is greater than 38 points, and the MDS-UPDRS-III score for the two "OFF" periods before surgery is stable, i.e., (high value - low value) / high value is less than or equal to 10%.
• Patients who have stably received anti-Parkinson drug therapy for more than 3 months.
• Previous effective treatment with levodopa, followed by significant drug resistance and wearing-off effect.
• The patient who is in stable condition, well-controlled complications, no contraindications for general anesthesia, no contraindications for stereotactic surgery, and no other conditions that interfere with clinical assessment.
• Head MRI does not reveal structural abnormalities that would affect stem cell transplantation, such as severe brain atrophy or space-occupying lesions.
• Subjects have a caregiver and are able to provide information about their condition before and after stem cell transplantation and can assist the investigator if necessary.

You may not qualify if:

• Patients with atypical Parkinson's disease, such as Parkinson's syndrome or secondary Parkinson's disease.
• Patients with Parkinson's disease with only tremors.
• Patients with severe motor impairment preventing the completion of routine motor tasks.
• Patients with severe neurological deficits caused by other diseases.
• Patients with severe psychiatric symptoms or dementia.
• The Patient who is unwilling or unable to cooperate, incapable of self-assessment, and unable to complete assessments even with the help of a physician.
• Patients who have history of pallidotomy or thalamotomy or deep brain stimulation (DBS) surgery.
• Patients with apomorphine treatment currently .
• Coagulation abnormalities (prothrombin time (PT) or international normalized ratio (INR) \> 1.5×ULN; activated partial thromboplastin time (APTT) \> 1.5×ULN) or receiving anticoagulation therapy.
• Pregnant or lactating female patients and male and female patients who cannot use effective contraception within 1 year after the last study drug use.
• Patients who have participated in other drug or medical device clinical studies within the last 3 months.
• Patients who have used botulinum toxin, phenol, subarachnoid injection of baclofen, or intervention therapy for treating muscle tone disorders or spasticity within the last 6 months.
• Patients who have history of seizures or prophylactic use of antiepileptic drugs.
• Patients who have contraindications for general anesthesia or stereotactic surgery, such as sleep apnea or chronic obstructive pulmonary disease, etc.
• Patients with any other unstable systemic diseases, including active infections, poorly controlled hypertension (systolic blood pressure \>160 mmHg and/or diastolic blood pressure \>100 mmHg after treatment), unstable angina, congestive heart failure (New York Heart Association NYHA class II or above), or metabolic diseases, etc.

Sponsors & Collaborators

• Shanghai East Hospital: lead

Study Sites (2)

Shanghai East Hospital

Shanghai, Shanghai Municipality, 200120, China

RECRUITING

Shanghai East Hospital

Shanghai, Shanghai Municipality, China

NOT YET 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

WU jingwen Shanghai East Hospital

CONTACT

+13810835976; songjiwen001@qq.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2023
• First Posted: November 24, 2023
• Study Start: November 23, 2023
• Primary Completion: December 22, 2025
• Study Completion: December 22, 2025
• Last Updated: October 15, 2024

Record last verified: 2024-10

Locations

CN (2)