Actively Recruiting
Clinical Study of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell Therapy for Amyotrophic Lateral Sclerosis (ALS)
Led by Shanghai East Hospital · Updated on 2025-01-09
3
Participants Needed
1
Research Sites
60 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Clinical Study of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell Therapy for Amyotrophic Lateral Sclerosis (ALS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients or their legal guardians must agree to the treatment and sign the informed consent form.
- Age between 18 and 60 years old, all genders allowed.
- Diagnosed with ALS according to World Federation of Neurology criteria, with diagnosis 6 to 24 months before screening.
- Previous standard treatment with poor response or disease progression.
- Forced Vital Capacity (FVC) of 50% or higher.
- Peripheral blood oxygen saturation below 90% for no more than 2% of any night during screening.
- Good nutritional status with Body Mass Index (BMI) of at least 18.5.
- Male patients and their partners, and women of childbearing age must use effective contraception from consent signing until one year after treatment starts.
- Ability to cooperate with medical history collection and study visits.
You will not qualify if you...
- Symptoms of neuromuscular weakness without confirmed ALS diagnosis.
- Severe cognitive impairment, dementia, or major psychiatric disorders such as schizophrenia, bipolar disorder, or severe depression.
- Diseases affecting nerve or muscle function like peripheral neuropathy or metabolic myopathy.
- History of malignant tumors or previous cancer diagnosis.
- Acute active infections requiring treatment within two weeks before screening.
- ALS patients with respiratory failure.
- Previous allogeneic cell therapy or organ transplantation.
- Participation in other clinical trials within three months before screening.
- History of tracheostomy or use of mechanical ventilators.
- Severe allergic reactions to general anesthesia or other severe allergies.
- Intracranial diseases causing increased pressure in the brain.
- Elevated liver function tests beyond specified limits during screening.
- Abnormal kidney function tests during screening.
- Positive tests for hepatitis A, active hepatitis B or C, hepatitis E, HIV, or syphilis.
- Impaired consciousness.
- Blood clotting abnormalities or current anticoagulation therapy.
- Poorly controlled high blood pressure despite treatment.
- Severe diabetes with complications or other diseases affecting limb movement.
- Recent surgery or trauma within the past month.
- Pregnant or breastfeeding women.
- Other uncontrolled systemic diseases or conditions judged unsuitable by the investigator.
AI-Screening
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Trial Site Locations
Total: 1 location
1
Shang hai East Hospital
Shanghai, Shanghai Municipality, China
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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