Cell therapy using induced pluripotent stem cells for ALS treatment

Clinical Study of Human Induced Pluripotent Stem Cells Derived Motor Neuron Precursor(iPSC-MNP) Cells for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

This study is testing a new cell therapy using stem cells to see if it can safely help people with ALS feel better.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 3 (estimated)
• Ages: 18 Years to 60 Years
• Sex: All
• Sponsor: Shanghai East Hospital (Academic / other)
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT06765564 on ClinicalTrials.gov

What this trial studies

This clinical study explores the safety and efficacy of using induced pluripotent stem cells (iPSCs) derived motor neuron precursor cells to treat patients with amyotrophic lateral sclerosis (ALS). The trial involves a non-randomized, open-label design with a baseline assessment period, a treatment phase lasting 45 days, and a follow-up period of 6 months. Participants will undergo a series of evaluations and receive immunosuppressants to minimize the risk of graft rejection following cell transplantation. The study aims to gather preliminary data on the safety of this innovative therapy for ALS.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 60 diagnosed with ALS within the last 6 to 24 months who have not responded well to standard treatments.

Not a fit: Patients with severe respiratory compromise or those who have not been diagnosed with ALS will likely not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option that may slow disease progression or improve motor function in ALS patients.

How similar studies have performed: While the use of iPSCs in ALS treatment is a novel approach, similar studies in other neurodegenerative conditions have shown promising results.

Eligibility criteria

Inclusion Criteria:

1. Patients themselves or their legal guardians must consent to undergo this treatment protocol and sign the Informed Consent Form (ICF).
2. Age between 18 and 60 years, inclusive, with no gender restrictions.
3. Diagnosed with ALS according to the World Federation of Neurology criteria, and the initial diagnosis date is between 6 to 24 months before the screening date.
4. Patients who have received standard treatment in the past with poor efficacy or disease progression.
5. Forced Vital Capacity (FVC) should be ≥50%.
6. During any night of the screening period, the total time with peripheral blood oxygen saturation \<90% should not exceed 2%.
7. Patients should be deemed by the investigator to be in good nutritional status, with a Body Mass Index (BMI) ≥18.5.
8. Male patients and their spouses, as well as women of childbearing age, should agree to implement effective contraceptive measures from the time of signing the ICF until one year after the start of treatment.
9. Patients should be able to cooperate in the collection and preservation of medical history data and the visit process.

Exclusion Criteria:

1. Patients with symptoms of neuromuscular weakness but cannot be conclusively determined to have ALS.
2. Patients diagnosed with severe cognitive impairment, clinical dementia, or major psychiatric disorders, including but not limited to schizophrenia, bipolar disorder, or severe depression, according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V).
3. Patients with any disease that impairs nerve or muscle function, such as peripheral neuropathy or metabolic myopathy.
4. Patients with a history of malignant tumors or a previous diagnosis of malignancy.
5. Within the two weeks preceding the screening period, patients who experienced acute active infections requiring treatment with antibiotics, antiviral drugs, or antifungal medications.
6. ALS patients with concomitant respiratory failure.
7. Patients who have previously undergone any allogeneic cell therapy or organ transplantation.
8. Patients who have Participated in other clinical trials within the three months prior to screening.
9. Patients with a history of tracheostomy or those using mechanical ventilatory support.
10. Patients with a documented history of severe allergic reactions to general anesthesia drugs or previous severe allergic reactions for other reasons.
11. Patients with intracranial organic diseases causing increased intracranial pressure.
12. Patients with elevated liver function test results during the screening period, such as total bilirubin \>1.5 times the upper limit of normal (ULN), or aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>3 times ULN.
13. Patients who have abnormal kidney function test results during the screening period, such as serum creatinine \>1.5 mg/dL or an estimated creatinine clearance rate \<60 mL/min calculated by the Cockcroft and Gault formula.
14. Other clinically significant laboratory abnormalities during the screening period.
15. Patients with hepatitis A, active hepatitis B (HBsAg positive and HBV DNA ≥500 IU/ml, excluding drug- or other-caused hepatitis), active hepatitis C (anti-HCV antibody positive and HCV RNA positive), hepatitis E, human immunodeficiency virus (HIV) antibody positive, or syphilis treponemal antibody positive.
16. Patients with impaired consciousness.
17. Coagulation abnormalities (prothrombin time \[PT\] or international normalized ratio \[INR\] \>1.5 times ULN; activated partial thromboplastin time \[APTT\] \>1.5 times ULN) or those currently receiving anticoagulation therapy.
18. Poorly controlled hypertension, with systolic blood pressure \>160 mmHg and/or diastolic blood pressure \>100 mmHg after treatment.
19. Severe diabetes with late complications; patients with other diseases affecting limb mobility (e.g., limping, osteoarthritis, rheumatoid arthritis, gout, etc.).
20. Patients who have undergone surgery or experienced trauma (including fractures) in the past month.
21. Pregnant or breastfeeding women.
22. Patients who, in the opinion of the investigator, have poorly controlled systemic diseases or other conditions that make them unsuitable for participation in this clinical study.

Where this trial is running

Shanghai, Shanghai Municipality

• Shang hai East Hospital — Shanghai, Shanghai Municipality, China (Recruiting)

Conditions ALS