Actively Recruiting
Clinical Study of Human Induced Pluripotent Stem Cell Derived Motor Neuron Precursor Cells for Treatment of 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 serious neurodegenerative disease affecting motor neurons, leading to damage of spinal cord cells, brainstem motor nuclei, and corticospinal tracts. Researchers are exploring the use of induced pluripotent stem cells (iPSCs) derived from motor neuron precursor cells as a potential therapy to replace damaged neurons, protect remaining neurons, and slow disease progression. This study focuses on evaluating the safety of this investigational treatment in ALS patients. The study is an open-label, non-randomized clinical trial with three phases: a 7-day baseline period, a 45-day treatment period, and a 6-month follow-up. During baseline, participants undergo assessments and begin oral immunosuppressants for three months. The treatment phase includes surgery to implant a drug pouch followed by four intraventricular injections of iPSC-derived motor neuron precursor cells every two weeks. After each transplantation, safety and efficacy assessments are conducted, including vital signs, laboratory tests, imaging, and functional scales. Participants return for follow-ups at 1, 3, and 6 months after completing stem cell treatments to monitor safety and effectiveness. Throughout the study, researchers track adverse events and serious adverse events, motor function, cognitive status, mood, fatigue, lung function, and neurotrophic factors. The study aims to ensure patient safety while investigating this novel stem cell therapy for ALS over approximately 7.5 months from first treatment to final follow-up.
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
Frequently Asked Questions
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