Clinical Study of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell Therapy for Amyotrophic Lateral Sclerosis (ALS)

NCT06765564 | Recruiting DMC

• 1 other identifier: XS2022002
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disease in the human motor system characterized by the selective involvement of spinal cord anterior horn cells, brainstem motor nuclei, and the corticospinal tract. It predominantly presents as concurrent damage to upper and lower motor neurons. Induced pluripotent stem cells (iPSCs) are a type of induced pluripotent stem cell derived from autologous or allogeneic cell sources. They can differentiate into various functional cell types, including specific motor neuron cells. iPSCs are used for stem cell replacement therapy. iPSCs hold significant clinical potential for ALS treatment. The iPSC database with human leukocyte antigen characteristics may represent a promising technology. This technology has the potential to obtain high-quality cell products and reduce the risk of graft rejection. Moreover, human iPSCs have demonstrated a certain degree of efficacy in the transplantation of neural stem/progenitor cells derived from ALS rodent models. The potential mechanisms of iPSC therapy for ALS include: the differentiated motor neuron precursor cells can replace damaged motor neurons, and restore motor conduction function; by secreting neurotrophic factors, they protect neurons; through immune regulation, they inhibit inflammatory reactions, and slow the progression of ALS. Xellsmart Biomedical (Suzhou) Co., Ltd. is developing an injectable solution for ALS treatment using human iPSC-derived motor neuron precursor cells to address the pressing need for ALS therapy.

Conditions

ALS

Outcome Measures

Primary Outcomes (1)

• Number of participants with adverse event (AE) and serious adverse event(SAE)

  AE occurring throughout the study period will be evaluated using the CTCAE V5.0 standard. AE/SAE were evaluated by laboratory examination and imageological examination, measuring method： 1. Head MRI, whole spine MRI, chest CT examination; 2. Changes in vital signs, 12-lead electrocardiogram, ultrasound (thyroid, abdomen, heart, genitourinary system) examination, and neurological signs; 3. Changes in blood routine, urine routine, stool routine, finger end blood oxygen saturation, serum immunoglobulin, cerebrospinal fluid routine, liver function, kidney function, electrolyte, muscle enzyme, lipid, blood glucose, glycated hemoglobin, coagulation function, tumor markers, various hepatitis viruses, treponema lutei antibodies, HIV antibodies and other indicators.

  0、1、3、6 month

Secondary Outcomes (1)

• effective of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell

  0、1、3、6 month

Study Arms (1)

One-arm study

EXPERIMENTAL

Use of experimental drug

Drug: iPSC-MNP

Interventions

iPSC-MNP DRUG

Using iPSC-MNP Cells for the Treatment of ALS Patients

One-arm study

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Patients themselves or their legal guardians must consent to undergo this treatment protocol and sign the Informed Consent Form (ICF).
• Age between 18 and 60 years, inclusive, with no gender restrictions.
• 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.
• Patients who have received standard treatment in the past with poor efficacy or disease progression.
• Forced Vital Capacity (FVC) should be ≥50%.
• During any night of the screening period, the total time with peripheral blood oxygen saturation \<90% should not exceed 2%.
• Patients should be deemed by the investigator to be in good nutritional status, with a Body Mass Index (BMI) ≥18.5.
• 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.
• Patients should be able to cooperate in the collection and preservation of medical history data and the visit process.

You may not qualify if:

• Patients with symptoms of neuromuscular weakness but cannot be conclusively determined to have ALS.
• 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).
• Patients with any disease that impairs nerve or muscle function, such as peripheral neuropathy or metabolic myopathy.
• Patients with a history of malignant tumors or a previous diagnosis of malignancy.
• Within the two weeks preceding the screening period, patients who experienced acute active infections requiring treatment with antibiotics, antiviral drugs, or antifungal medications.
• ALS patients with concomitant respiratory failure.
• Patients who have previously undergone any allogeneic cell therapy or organ transplantation.
• Patients who have Participated in other clinical trials within the three months prior to screening.
• Patients with a history of tracheostomy or those using mechanical ventilatory support.
• Patients with a documented history of severe allergic reactions to general anesthesia drugs or previous severe allergic reactions for other reasons.
• Patients with intracranial organic diseases causing increased intracranial pressure.
• 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.
• 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.
• Other clinically significant laboratory abnormalities during the screening period.
• 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.

Sponsors & Collaborators

• Shanghai East Hospital: lead

Study Sites (1)

Shang hai East Hospital

Shanghai, Shanghai Municipality, China

RECRUITING

Study Design

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

Study Record Dates

• First Submitted: November 13, 2023
• First Posted: January 9, 2025
• Study Start: March 13, 2024
• Primary Completion: March 13, 2025
• Study Completion: May 13, 2025
• Last Updated: January 9, 2025

Record last verified: 2025-01

Locations

CN (1)