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Clinical Study of Human Induced Pluripotent Stem Cells Derived Motor Neuron Precursor(iPSC-MNP) Cells for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
Led by Shanghai East Hospital · Updated on 2025-01-09
3
Participants Needed
1
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Amyotrophic lateral sclerosis (ALS) is a serious disease affecting the motor system, involving damage to nerve cells that control movement. Researchers are exploring the use of induced pluripotent stem cells (iPSCs), which can develop into motor neuron precursor cells, as a potential treatment for ALS. This trial aims to evaluate the safety of human iPSC-derived motor neuron precursor cells in people with ALS, addressing the urgent need for new therapies. This open-label clinical trial includes three phases: a 7-day baseline period, a 45-day treatment period, and a 6-month follow-up. During treatment, participants will have a drug pouch surgically implanted, through which they will receive four doses of stem cell injections every two weeks. Each dose consists of 50 million motor neuron precursor cells delivered through the pouch. The trial includes close monitoring for adverse events and assessments using various clinical scales and tests. Participants will undergo detailed assessments before surgery, including physical exams, laboratory tests, and cognitive and mood evaluations. During treatment, vital signs and safety labs will be regularly checked, along with self-assessments using ALS-specific scales. Follow-up visits occur at 1, 3, and 6 months after completing treatment to monitor safety and effectiveness. The study focuses on recording adverse events and evaluating physical function and neurological health over time.
CONDITIONS
Brief 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 consent and sign the informed consent form.
- Age between 18 and 60 years, inclusive.
- Diagnosed with ALS according to World Federation of Neurology criteria, with diagnosis 6 to 24 months before screening.
- Have received standard treatment previously with poor efficacy or disease progression.
- Forced Vital Capacity (FVC) of 50% or higher.
- Peripheral blood oxygen saturation below 90% less than 2% of any night during screening.
- Good nutritional status with Body Mass Index (BMI) of 18.5 or higher.
- Male patients and their partners, and women of childbearing age must agree to effective contraception from consent until one year after treatment starts.
- Ability to cooperate with medical history collection and visit procedures.
You will not qualify if you...
- Uncertain diagnosis of ALS despite neuromuscular weakness symptoms.
- Severe cognitive impairment, clinical dementia, or major psychiatric disorders.
- Diseases impairing 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.
- Respiratory failure related to ALS.
- Previous allogeneic cell therapy or organ transplantation.
- Participation in other clinical trials within three months before screening.
- History of tracheostomy or use of mechanical ventilation.
- Severe allergic reactions to general anesthesia or other causes.
- Intracranial diseases causing increased pressure.
- Elevated liver or kidney function tests beyond specified limits.
- Positive tests for hepatitis A, active hepatitis B or C, hepatitis E, HIV, or syphilis.
- Impaired consciousness.
- Coagulation abnormalities or on anticoagulation therapy.
- Poorly controlled hypertension.
- Severe diabetes with complications or diseases affecting limb mobility.
- Recent surgery or trauma within one month.
- Pregnancy or breastfeeding.
- Other uncontrolled systemic diseases or conditions deemed unsuitable by investigator.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - 7 days
Participants provide informed consent and undergo baseline assessments including medical history, vital signs, physical exams, laboratory and auxiliary tests, and self-assessment of physical condition before surgery.
1 visit for baseline assessments
Duration - 45 days
Participants undergo surgery to install the Sofia drug pouch under general anesthesia followed by intraventricular stem cell transplantation administered four times over 45 days, with monitoring of vital signs, adverse events, and clinical symptoms.
1 surgery visit and 4 stem cell transplantation visits every half month
Duration - 6 months
Participants return for safety and effectiveness assessments at 1 month, 3 months, and 6 months after completing stem cell transplantations to monitor health and treatment impact.
3 visits at 1, 3, and 6 months after treatment
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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