Treatment for Parkinson's Disease using Stem Cells

A Clinical Trial of Parkinson's Disease Treatment by Human-induced Pluripotent Stem Cells (hiPSCs) Derived Dopaminergic Neural Precursor Cells

This study is testing if using patients' own stem cells to create new brain cells can help improve symptoms for people with Parkinson's disease.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 3 (estimated)
• Ages: 30 Years to 70 Years
• Sex: All
• Sponsor: Shanghai East Hospital (Academic / other)
• Locations: 2 sites (Shanghai, Shanghai Municipality and 1 other locations)
• Trial ID: NCT06145711 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and efficacy of using autologous induced pluripotent stem cell (iPSC)-derived dopaminergic neural precursor cells for treating Parkinson's disease. Conducted by Shanghai East Hospital in collaboration with XellSmart Biomedical, the trial involves preparing these cells from patients' own iPSCs and injecting them into the brain using stereotactic technology. The study will monitor patients for safety and potential improvements in symptoms over a period of 6 to 12 months, depending on initial outcomes.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 30 to 70 with primary Parkinson's disease, a disease duration of more than 5 years, and specific severity criteria.

Not a fit: Patients with early-stage Parkinson's disease or those who do not meet the inclusion criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients with Parkinson's disease, potentially improving their quality of life.

How similar studies have performed: While there has been limited global experience with similar approaches, preliminary results from a single case in the U.S. suggest potential for success, making this a novel endeavor in China.

Eligibility criteria

Inclusion Criteria:

1. The patient or their legally authorized guardian consents to participate in this study and signs the Informed Consent Form (ICF) in writing.
2. Patients with primary Parkinson's disease, aged between 30 and 70 years, of any gender, with a disease duration of more than 5 years.
3. 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.
4. 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%.
5. Patients who have stably received anti-Parkinson drug therapy for more than 3 months.
6. Previous effective treatment with levodopa, followed by significant drug resistance and wearing-off effect.
7. 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.
8. Head MRI does not reveal structural abnormalities that would affect stem cell transplantation, such as severe brain atrophy or space-occupying lesions.
9. 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.

Exclusion Criteria:

1. Patients with atypical Parkinson's disease, such as Parkinson's syndrome or secondary Parkinson's disease.
2. Patients with Parkinson's disease with only tremors.
3. Patients with severe motor impairment preventing the completion of routine motor tasks.
4. Patients with severe neurological deficits caused by other diseases.
5. Patients with severe psychiatric symptoms or dementia.
6. 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.
7. Patients who have history of pallidotomy or thalamotomy or deep brain stimulation (DBS) surgery.
8. Patients with apomorphine treatment currently .
9. 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.
10. Pregnant or lactating female patients and male and female patients who cannot use effective contraception within 1 year after the last study drug use.
11. Patients who have participated in other drug or medical device clinical studies within the last 3 months.
12. 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.
13. Patients who have history of seizures or prophylactic use of antiepileptic drugs.
14. Patients who have contraindications for general anesthesia or stereotactic surgery, such as sleep apnea or chronic obstructive pulmonary disease, etc.
15. 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.
16. Patients with alcohol or drug abuse.
17. Patients who have used a high dose of benzodiazepines within the last 3 months.
18. Patients with severe cognitive impairment, depression, or behavioral disorders, defined as a Mini-Mental State Examination (MMSE) score less than 26 and a Hamilton Depression Rating Scale (HAMD) score greater than 35.
19. History of any malignant tumors.
20. Patients with hepatitis A, active hepatitis B (HBsAg positive with HBV DNA higher than the upper limit of normal, and excluding hepatitis caused by drugs or other reasons), active hepatitis C (anti-HCV antibody positive with HCV RNA higher than the upper limit of normal), hepatitis E, human immunodeficiency virus (HIV) antibody positive, or syphilis (TP antibody positive).
21. Patients with abnormal liver and kidney function laboratory tests during the screening period, such as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>3×ULN, serum creatinine \>1.5×ULN, or total bilirubin \>1.5×ULN.
22. Other situations in which the investigator believes that the subject is not suitable for inclusion or cannot tolerate stem cell transplantation surgery.
23. PD subjects found to have gene mutations by genetic testing.

Where this trial is running

Shanghai, Shanghai Municipality and 1 other locations

• Shanghai East Hospital — Shanghai, Shanghai Municipality, China (Recruiting)
• Shanghai East Hospital — Shanghai, Shanghai Municipality, China (Not_yet_recruiting)

Study contacts

• Study coordinator: WU jingwen Shanghai East Hospital

Conditions Parkinson's Disease; Parkinson's disease; stem cell