ALSiPSC-MNPNCT06765564
Clinical Study of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell Therapy for Amyotrophic Lateral Sclerosis (ALS)
A study testing iPSC-MNP in people with ALS, run by Shanghai East Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- ALS
- What is being tested?
- iPSC-MNP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteChina
- Study dates
- 13 Mar 2024to 13 May 2025
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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%.
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- 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.
Who cannot join
- 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.
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- 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.
- Patients with impaired consciousness.
- 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.
- Poorly controlled hypertension, with systolic blood pressure >160 mmHg and/or diastolic blood pressure >100 mmHg after treatment.
- Severe diabetes with late complications; patients with other diseases affecting limb mobility (e.g., limping, osteoarthritis, rheumatoid arthritis, gout, etc.).
- Patients who have undergone surgery or experienced trauma (including fractures) in the past month.
- Pregnant or breastfeeding women.
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Shang hai East Hospital
Shanghai, Shanghai Municipality
song jiwen, 13810835976, songjiwen001@qq.com
About this study
Official title: Clinical Study of Human Induced Pluripotent Stem Cells Derived Motor Neuron Precursor(iPSC-MNP) Cells for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
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.
Read more
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.
Read the detailed description
This study is a non-randomized, open-label, multiple-dose, investigator-initiated interventional clinical trial aimed at preliminary exploring the safety of the investigational drug. The study is divided into three phases: a baseline period (7 days), a treatment period (45 days), and a follow-up period (6 months).
Baseline Period: The ALS subjects need to sign the informed consent form for the clinical trial (including the installation surgery for the Sofia drug pouch) and undergo baseline assessments before the transplant surgery to exclude any conditions that would disqualify them from participation. The subjects need to undergo medical history collection, vital signs and physical examinations, laboratory tests, various other auxiliary examinations, and perform self-assessment of their physical condition. The investigator records clinical symptoms, scores and records various scales and tables. [including ALS Functional Rating Scale-Revised (ALSFRS-R), Mini-Mental State Examination (MMSE), Montreal Cognitive Assessment (MoCA), Hamilton Depression Rating Scale (HAMD 17), Hamilton Anxiety Rating Scale (HAMA), Fatigue Severity Scale (FSS)]. Qualified subjects will subsequently take oral immunosuppressants for three months, and the clinical research investigator will adjust the dosage and medication duration based on the subjects' rejection response and tolerability.
Treatment Period: At Day -3 of the treatment period, the surgical expert administers general anesthesia to the subjects and, under the guidance of a robot, installs the Sofia drug pouch. On the first day post-surgery, through head CT and other examinations, adverse events (AEs and SAEs) are assessed. On Treatment Day 0 (i.e., 3 days post-surgery), the first stem cell transplantation begins. Subjects undergo vital sign monitoring, physical examinations, and record self-assessments of their physical condition and clinical symptoms before undergoing intraventricular stem cell transplantation. The investigator injects 2 mL of human iPSC-derived motor neuron precursor cells (5×107 cells) through the subcutaneous drug pouch. Subsequently, the same dose of stem cells is administered through the drug pouch every half month, totaling four times. After the administration, subjects are advised to lie flat for one hour, with simultaneous monitoring of vital signs and blood oxygen saturation, and recording of any possible adverse events (AEs and SAEs). On the third day after each stem cell transplantation treatment, subjects undergo safety assessments (vital signs, physical examinations, laboratory tests, 12-lead electrocardiogram, cerebrospinal fluid routine, ultrasound, and imaging examinations, etc.) and an evaluation of the impact of this study on the efficacy of individual ALS subjects (patient self-assessment of condition and clinical symptoms, scales [ALSFRS-R, MMSE, MoCA, HAMD 17, HAMA, FSS], electrophysiological parameter testing, FVC, and cerebrospinal fluid neurotrophic factor detection).
Follow-up Phase: Subjects return to the clinical research center for safety and effectiveness assessments at 1 month, 3 months, and 6 months after finishing all the stem cell transplantations (i.e., 2.5 months, 4.5 months, and 7.5 months after the first stem cell transplantation). For subjects who do not complete all four stem cell transplantations, the follow-up schedule is adjusted based on the completion time of the stem cell transplantation. For safety reasons, the next subject can only be enrolled after the previous subject has completed the stem cell transplantation and been observed for 6 months without the occurrence of acute or delayed SAEs. The clinical research center should be equipped to handle unexpected acute, severe adverse events, such as allergic reactions and medication-related reactions.
- Conditions
- ALS
- Being tested
- iPSC-MNP
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
iPSC-MNPDrug
Using iPSC-MNP Cells for the Treatment of ALS Patients
| Study group | Role | What they receive |
|---|---|---|
| One-arm study | Experimental | Use of experimental drug Receives: Drug: iPSC-MNP |
What is being measured
The primary outcome is the main question the study is designed to answer.
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：
- Head MRI, whole spine MRI, chest CT examination;
- Changes in vital signs, 12-lead electrocardiogram, ultrasound (thyroid, abdomen, heart, genitourinary system) examination, and neurological signs;
- 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.
Measured: 0、1、3、6 month
Secondary outcomes (1)
effective of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell
ALS functional assessment: Changes in ALSFRS-R scale score; The ALSFRS-R includes 12 different tasks designed to provide a representative picture of the physical abilities most commonly affected by ALS. These tasks include speaking, salivation, swallowing, writing, climbing stairs, turning in bed, walking, dressing and hygiene, and three different measures of respiratory capacity. Each task is rated on a scale of 0 to 4-0 indicates the inability to perform the task, and 4 indicates the ability to perform it without difficulty. These ratings are then summed to yield a total score ranging from 0 to 48, with 0 being the worst and 48 being the best.
Measured: 0、1、3、6 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2025
- Study start13 Mar 2024
- Primary completion13 Mar 2025 (estimated)
- Study completion13 May 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALSStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai East Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06765564
- Sponsor's protocol ID
- XS2022002
- Acronym
- iPSC-MNP
- Status last verified
- Jan 2025
- First submitted
- 13 Nov 2023
- Last updated
- 9 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Human Induced Pluripotent Stem Cells Derived Motor Neuron Precursor(iPSC-MNP) Cells for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT06765564
- Acronym / sponsor ID
- iPSC-MNP / XS2022002
- Conditions
- ALS
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Shanghai East HospitalUniversity, hospital or other
- Study start
- 13 Mar 2024 (actual)
- Primary completion
- 13 Mar 2025 (estimated)
- Study completion
- 13 May 2025 (estimated)
- First posted
- 9 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study is a non-randomized, open-label, multiple-dose, investigator-initiated interventional clinical trial aimed at preliminary exploring the safety of the investigational drug. The study is divided into three phases: a baseline period (7 days), a treatment period (45 days), and a follow-up period (6 months).
Baseline Period: The ALS subjects need to sign the informed consent form for the clinical trial (including the installation surgery for the Sofia drug pouch) and undergo baseline assessments before the transplant surgery to exclude any conditions that would disqualify them from participation. The subjects need to undergo medical history collection, vital signs and physical examinations, laboratory tests, various other auxiliary examinations, and perform self-assessment of their physical condition. The investigator records clinical symptoms, scores and records various scales and tables. [including ALS Functional Rating Scale-Revised (ALSFRS-R), Mini-Mental State Examination (MMSE), Montreal Cognitive Assessment (MoCA), Hamilton Depression Rating Scale (HAMD 17), Hamilton Anxiety Rating Scale (HAMA), Fatigue Severity Scale (FSS)]. Qualified subjects will subsequently take oral immunosuppressants for three months, and the clinical research investigator will adjust the dosage and medication duration based on the subjects' rejection response and tolerability.
Treatment Period: At Day -3 of the treatment period, the surgical expert administers general anesthesia to the subjects and, under the guidance of a robot, installs the Sofia drug pouch. On the first day post-surgery, through head CT and other examinations, adverse events (AEs and SAEs) are assessed. On Treatment Day 0 (i.e., 3 days post-surgery), the first stem cell transplantation begins. Subjects undergo vital sign monitoring, physical examinations, and record self-assessments of their physical condition and clinical symptoms before undergoing intraventricular stem cell transplantation. The investigator injects 2 mL of human iPSC-derived motor neuron precursor cells (5×107 cells) through the subcutaneous drug pouch. Subsequently, the same dose of stem cells is administered through the drug pouch every half month, totaling four times. After the administration, subjects are advised to lie flat for one hour, with simultaneous monitoring of vital signs and blood oxygen saturation, and recording of any possible adverse events (AEs and SAEs). On the third day after each stem cell transplantation treatment, subjects undergo safety assessments (vital signs, physical examinations, laboratory tests, 12-lead electrocardiogram, cerebrospinal fluid routine, ultrasound, and imaging examinations, etc.) and an evaluation of the impact of this study on the efficacy of individual ALS subjects (patient self-assessment of condition and clinical symptoms, scales [ALSFRS-R, MMSE, MoCA, HAMD 17, HAMA, FSS], electrophysiological parameter testing, FVC, and cerebrospinal fluid neurotrophic factor detection).
Follow-up Phase: Subjects return to the clinical research center for safety and effectiveness assessments at 1 month, 3 months, and 6 months after finishing all the stem cell transplantations (i.e., 2.5 months, 4.5 months, and 7.5 months after the first stem cell transplantation). For subjects who do not complete all four stem cell transplantations, the follow-up schedule is adjusted based on the completion time of the stem cell transplantation. For safety reasons, the next subject can only be enrolled after the previous subject has completed the stem cell transplantation and been observed for 6 months without the occurrence of acute or delayed SAEs. The clinical research center should be equipped to handle unexpected acute, severe adverse events, such as allergic reactions and medication-related reactions.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| One-arm study | Experimental | Drug: iPSC-MNPUse of experimental drug |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- 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%.
Key exclusion criteria (22 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| iPSC-MNPUsing iPSC-MNP Cells for the Treatment of ALS Patients | Drug | One-arm study |
Primary outcome
Number of participants with adverse event (AE) and serious adverse event(SAE)
Time frame: 0、1、3、6 month
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.
1 secondary outcome
effective of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell
Time frame: 0、1、3、6 month
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jan 2025.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing iPSC-MNP in people with ALS, run by Shanghai East Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with adverse event (AE) and serious adverse event(SAE) (time frame: 0、1、3、6 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jan 2025.