ALS (Amyotrophic Lateral Sclerosis)NCT07539662
Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
A study testing Mobilization Regimen, Conditioning Regimen and GVHD Prophylaxis in people with ALS (Amyotrophic Lateral Sclerosis), run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive and fatal neurodegenerative disorder. Its global prevalence is approximately 0.73-1.89 per 100,000 individuals. In China, there are about 200,000 ALS patients, with approximately 25,000 new cases diagnosed annually. Read more about this study
- Who is it for?
- Ages 35 to 50 years
- Conditions the study is for
- ALS (Amyotrophic Lateral Sclerosis)
- What is being tested?
- Mobilization Regimen, Conditioning Regimen and GVHD Prophylaxis
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteChina
- Study dates
- 29 Dec 2025to Dec 2027
- 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 35 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patients with a confirmed diagnosis of ALS, with an ALSFRS-R score ≥35 and a maximum vital capacity ≥65%
- Aged >35 years and <50 years, with no gender restrictions
- Karnofsky Performance Status (KPS) score ≥70, and Eastern Cooperative Oncology Group (ECOG) Performance Status ≤2
- Unrelated umbilical cord blood and the recipient demonstrate high-resolution HLA (-A, -B, Cw, DR, DQ) matching at ≥4/6, 5/8, or 7/10 loci, and the post-thaw CD34+ cell count in the cord blood unit is ≥1.2×10⁵/kg (recipient body weight)
- Willing and able to comply with the study procedures and conditions, demonstrating good compliance
- Willing to receive at least two years of treatment and follow-up, with detailed medical records maintained
- The patient and/or their legal guardian voluntarily participate in this clinical trial, sign the informed consent form, and are capable of completing all follow-up assessments as required by the protocol
Who cannot join
- Patients with positive results in the following etiological tests: Human Immunodeficiency Virus (HIV-1/2), Human Cytomegalovirus DNA (HCMV-DNA), Epstein-Barr Virus DNA (EBV-DNA), Hepatitis B (positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B virus DNA (HBV-DNA)), Hepatitis C antibody (HCV-Ab), or Treponema pallidum antibody (TP-Ab)
- Clinically significant active bacterial, viral, fungal, or parasitic infections as judged by the investigator during screening
- Patients who have previously received gene therapy or allogeneic hematopoietic stem cell transplantation
- First-degree relatives with known or suspected familial cancer syndromes (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary nonpolyposis colorectal cancer syndrome, familial adenomatous polyposis, etc.)
- Diagnosis of major psychiatric disorders or predisposition to such conditions that would significantly impair the ability to participate in the clinical study
- History of major organ impairment, including: Liver disorders: Liver function tests showing AST or ALT >3 × ULN; total serum bilirubin >2.5 × ULN; for cases consistent with Gilbert syndrome, total bilirubin >3 × ULN and direct bilirubin >2.5 × ULN; history of hepatic bridging fibrosis, cirrhosis, or active hepatitis; Cardiac disorders: Left ventricular ejection fraction (LVEF) <45% at screening; New York Heart Association (NYHA) Class III or IV congestive heart failure; severe arrhythmias requiring treatment; poorly controlled hypertension (systolic blood pressure >160 mmHg and/or diastolic blood pressure >100 mmHg despite antihypertensive therapy), or prior history of hypertensive emergencies, hypertensive encephalopathy, or unstable angina; history of myocardial infarction, coronary artery bypass graft surgery, peripheral arterial bypass graft implantation, or stent placement within 12 months prior to enrollment; clinically significant valvular disease; calculated eGFR <60 mL/min/1.73m²; Pulmonary function: FEV1/FVC <60% and/or diffusing capacity less than 60% of predicted; clinically significant evidence of pulmonary hypertension requiring medical intervention
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- Uncorrectable coagulation dysfunction or history of severe bleeding disorders
- Any other condition deemed by the physician to render the subject unsuitable for hematopoietic stem cell transplantation
- Known hypersensitivity to the investigational drug or its components
- Participation in or ongoing participation in other interventional clinical studies within 3 months prior to screening
- Vaccination with live vaccines within 6 weeks prior to screening
- Pregnant or breastfeeding women
- History of solid organ transplantation
- Poor compliance of the subject with the study protocol
- Any other condition considered by the investigator as unsuitable for participation in this clinical trial
- Unwillingness of the subject to provide pre-existing valid diagnostic evidence before treatment or to undergo bone marrow, lumbar puncture, blood tests, and other examinations after treatment
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Single-Arm, Open-Label, Single-Center Clinical Study on Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive and fatal neurodegenerative disorder. Its global prevalence is approximately 0.73-1.89 per 100,000 individuals. In China, there are about 200,000 ALS patients, with approximately 25,000 new cases diagnosed annually. Microglia, the resident immune cells of the central nervous system (CNS), rapidly transition from a resting state to a pro-inflammatory phenotype (M1) in ALS. This activation leads to the release of a large number of inflammatory factors (such as TNF-α, IL-1β, IL-6, NO, ROS) and chemokines (such as MCP-1/CCL2), and triggers the NLRP3 inflammasome. Furthermore, systemic immune dysregulation plays a significant role in the pathogenesis of ALS. ALS patients exhibit reduced numbers of regulatory T cells (Tregs), alterations of activated CD8+ T cell infiltrates, and a shift in the helper T cell (Th1/Th2) balance towards the pro-inflammatory Th1 phenotype. In recent years, therapeutic strategies targeting novel pathways such as neuroinflammation, immune dysregulation, and energy metabolism have emerged, including the infusion of Tregs, mesenchymal stem cells (MSCs), and neural stem cells. However, these approaches have still failed to halt disease progression [NCT05695521, NCT03280056, NCT06973629, NCT02290886]. Recent research suggests that hematopoietic stem cell transplantation (HSCT) may disrupt the activation cycle between astrocytes and microglia, alleviate chronic inflammatory states in the CNS, partially mitigate mitochondrial dysfunction, and thereby slow neurodegeneration. Unrelated umbilical cord blood transplantation offers advantages such as low HLA-matching requirements, a lower risk of graft-versus-host disease (GVHD), a potent graft-versus-leukemia (GVL) effect, and immediate availability. Investigators plan to conduct an exploratory clinical trial to evaluate the safety and efficacy of umbilical cord blood transplantation for ALS patients. The preliminary plan is to enroll 8 adult subjects. Following successful neutrophil engraftment (defined as an absolute neutrophil count ≥0.5×10⁹/L for three consecutive days) and confirmation of complete donor chimerism. The trial will focus on assessing transplantation-related complications and patient tolerance. A 3-month post-transplantation follow-up will be conducted for a comprehensive evaluation of safety and efficacy for ALS patients.
Read the detailed description
Please see the detailed description in following content
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Being tested
- Mobilization Regimen, Conditioning Regimen, GVHD Prophylaxis, umbilical cord blood, Rescue
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mobilization RegimenProcedure or surgery
Mobilization and Collection of Recipient's Autologous Peripheral Blood Hematopoietic Stem Cells during the Screening Phase for Cryopreservation and Future Use: G-CSF at 5 µg/kg, administered subcutaneously every 12 hours (q12h). Collection begins when the white blood cell count exceeds 5×10⁹/L and the platelet count exceeds 50×10⁹/L. The collected cells are cryopreserved in liquid nitrogen.
Conditioning RegimenProcedure or surgery
A reduced-intensity myeloablative conditioning regimen consisting of Fludarabine (Flu), Melphalan (Mel), Thiotepa (TT), and Total Marrow Irradiation (TMI) is employed.
GVHD ProphylaxisProcedure or surgery
A combination of Cyclosporine A (CsA) and short-course Mycophenolate Mofetil (MMF) is used.
umbilical cord bloodProcedure or surgery
UCB Infusion: On transplant day 0, the thawed umbilical cord blood unit is infused. Dexamethasone 5 mg is administered intravenously 30 minutes prior to the infusion.
RescueProcedure or surgery
Rescue Protocol for Graft Failure: The cryopreserved autologous peripheral HSCs are reinfused to restore hematopoietic function.
| Study group | Role | What they receive |
|---|---|---|
| ALS group | Experimental | Not described Receives: Procedure: Mobilization Regimen; Procedure: Conditioning Regimen; Procedure: GVHD Prophylaxis; Procedure: umbilical cord blood; Procedure: Rescue |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Survival status of ALS patients after umbilical cord blood transplantation
Measured: 1 year, 3 year, 5 year
LVGI Safety Score
Considering the limited assessment conditions within the transplant isolation unit, this study has modified and simplified the ALSFRS-R scale and incorporated the Inbody score, which provides a comprehensive assessment of muscle mass and body fat mass, to form the "LVGI" safety score. Among the components, VCmax refers to maximum lung capacity; Grip quantifies upper limb strength using a dynamometer, with the Grip Strength-to-Body Weight Index calculated as Grip Strength (kg) / Body Weight (kg) × 100. The results of each assessment are compared. A change within ± 20% in the LSGI score within the first month post-transplantation compared to the pre-transplantation score is considered safe.
Measured: 3 month, 6 month, 1 year
ALS related assessment
Assessment of Disease Functional Progression: The ALSFRS-R scale was used to conduct standardized assessments at baseline (1, 3, 6, 12, 18, and 24 months post-transplantation). Biomarker of Neuroaxonal Injury: Peripheral blood and CSF samples were collected from patients to assess neurofilament light chain (NFL). By analyzing changes in NFL relative to baseline, the dynamic changes in neuroaxonal injury following umbilical cord blood transplantation intervention were objectively evaluated. Imaging Evaluation: MR and PET-CT.
Measured: 3 month, 6 month, 1 year
Secondary outcomes (2)
Assessment of Drug-Induced Liver and Kidney Toxicity
Liver - RUCAM Score: <1: Excluded 1-2: Unlikely 3-5: Possible 6-8: Probable 8: Highly probable Kidney - KDIGO Score: Acute Kidney Injury (AKI) is defined by meeting any one of the following three criteria: ① Increase in serum creatinine (Scr) by ≥0.3 mg/dL (≥26.5 µmol/L) within 48 hours; ② Increase in Scr to >1.5 times the baseline value, which is known or presumed to have occurred within the prior 7 days; ③ Urine output <0.5 mL/(kg·h) for 6 hours.
Measured: up to 24 weeks
Assessment of acute GVHD
Primarily based on the modified Glucksberg criteria and the acute GVHD International Consortium (MAGIC) grading criteria.
Measured: up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2026
- Study start29 Dec 2025
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALS (Amyotrophic Lateral Sclerosis)Studies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- 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
- 8 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07539662
- Sponsor's protocol ID
- IIT2025142
- Status last verified
- Apr 2026
- First submitted
- 9 Feb 2026
- Last updated
- 20 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm, Open-Label, Single-Center Clinical Study on Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT07539662
- Acronym / sponsor ID
- IIT2025142
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 29 Dec 2025 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 20 Apr 2026 (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 rapidly progressive and fatal neurodegenerative disorder. Its global prevalence is approximately 0.73-1.89 per 100,000 individuals. In China, there are about 200,000 ALS patients, with approximately 25,000 new cases diagnosed annually. Microglia, the resident immune cells of the central nervous system (CNS), rapidly transition from a resting state to a pro-inflammatory phenotype (M1) in ALS. This activation leads to the release of a large number of inflammatory factors (such as TNF-α, IL-1β, IL-6, NO, ROS) and chemokines (such as MCP-1/CCL2), and triggers the NLRP3 inflammasome. Furthermore, systemic immune dysregulation plays a significant role in the pathogenesis of ALS. ALS patients exhibit reduced numbers of regulatory T cells (Tregs), alterations of activated CD8+ T cell infiltrates, and a shift in the helper T cell (Th1/Th2) balance towards the pro-inflammatory Th1 phenotype. In recent years, therapeutic strategies targeting novel pathways such as neuroinflammation, immune dysregulation, and energy metabolism have emerged, including the infusion of Tregs, mesenchymal stem cells (MSCs), and neural stem cells. However, these approaches have still failed to halt disease progression [NCT05695521, NCT03280056, NCT06973629, NCT02290886]. Recent research suggests that hematopoietic stem cell transplantation (HSCT) may disrupt the activation cycle between astrocytes and microglia, alleviate chronic inflammatory states in the CNS, partially mitigate mitochondrial dysfunction, and thereby slow neurodegeneration. Unrelated umbilical cord blood transplantation offers advantages such as low HLA-matching requirements, a lower risk of graft-versus-host disease (GVHD), a potent graft-versus-leukemia (GVL) effect, and immediate availability. Investigators plan to conduct an exploratory clinical trial to evaluate the safety and efficacy of umbilical cord blood transplantation for ALS patients. The preliminary plan is to enroll 8 adult subjects. Following successful neutrophil engraftment (defined as an absolute neutrophil count ≥0.5×10⁹/L for three consecutive days) and confirmation of complete donor chimerism. The trial will focus on assessing transplantation-related complications and patient tolerance. A 3-month post-transplantation follow-up will be conducted for a comprehensive evaluation of safety and efficacy for ALS patients.
Detailed description
Please see the detailed description in following content
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALS group | Experimental | Procedure: Mobilization Regimen, Procedure: Conditioning Regimen, Procedure: GVHD Prophylaxis, Procedure: umbilical cord blood, Procedure: Rescue |
Who may qualify
- Ages
- 35 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Patients with a confirmed diagnosis of ALS, with an ALSFRS-R score ≥35 and a maximum vital capacity ≥65%
- Aged >35 years and <50 years, with no gender restrictions
- Karnofsky Performance Status (KPS) score ≥70, and Eastern Cooperative Oncology Group (ECOG) Performance Status ≤2
- Unrelated umbilical cord blood and the recipient demonstrate high-resolution HLA (-A, -B, Cw, DR, DQ) matching at ≥4/6, 5/8, or 7/10 loci, and the post-thaw CD34+ cell count in the cord blood unit is ≥1.2×10⁵/kg (recipient body weight)
- Willing and able to comply with the study procedures and conditions, demonstrating good compliance
Key exclusion criteria (16 listed)
- Patients with positive results in the following etiological tests: Human Immunodeficiency Virus (HIV-1/2), Human Cytomegalovirus DNA (HCMV-DNA), Epstein-Barr Virus DNA (EBV-DNA), Hepatitis B (positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B virus DNA (HBV-DNA)), Hepatitis C antibody (HCV-Ab), or Treponema pallidum antibody (TP-Ab)
- Clinically significant active bacterial, viral, fungal, or parasitic infections as judged by the investigator during screening
- Patients who have previously received gene therapy or allogeneic hematopoietic stem cell transplantation
- First-degree relatives with known or suspected familial cancer syndromes (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary nonpolyposis colorectal cancer syndrome, familial adenomatous polyposis, etc.)
- Diagnosis of major psychiatric disorders or predisposition to such conditions that would significantly impair the ability to participate in the clinical study
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 |
|---|---|---|
| Mobilization RegimenMobilization and Collection of Recipient's Autologous Peripheral Blood Hematopoietic Stem Cells during the Screening Phase for Cryopreservation and Future Use: G-CSF at 5 µg/kg, administered subcutaneously every 12 hours (q12h). Collection begins when the white blood cell count exceeds 5×10⁹/L and the platelet count exceeds 50×10⁹/L. The collected cells are cryopreserved in liquid nitrogen. | Procedure or surgery | ALS group |
| Conditioning RegimenA reduced-intensity myeloablative conditioning regimen consisting of Fludarabine (Flu), Melphalan (Mel), Thiotepa (TT), and Total Marrow Irradiation (TMI) is employed. | Procedure or surgery | ALS group |
| GVHD ProphylaxisA combination of Cyclosporine A (CsA) and short-course Mycophenolate Mofetil (MMF) is used. | Procedure or surgery | ALS group |
| umbilical cord bloodUCB Infusion: On transplant day 0, the thawed umbilical cord blood unit is infused. Dexamethasone 5 mg is administered intravenously 30 minutes prior to the infusion. | Procedure or surgery | ALS group |
| RescueRescue Protocol for Graft Failure: The cryopreserved autologous peripheral HSCs are reinfused to restore hematopoietic function. | Procedure or surgery | ALS group |
Primary outcome
Overall survival
Time frame: 1 year, 3 year, 5 year
Survival status of ALS patients after umbilical cord blood transplantation
LVGI Safety Score
Time frame: 3 month, 6 month, 1 year
Considering the limited assessment conditions within the transplant isolation unit, this study has modified and simplified the ALSFRS-R scale and incorporated the Inbody score, which provides a comprehensive assessment of muscle mass and body fat mass, to form the "LVGI" safety score. Among the components, VCmax refers to maximum lung capacity; Grip quantifies upper limb strength using a dynamometer, with the Grip Strength-to-Body Weight Index calculated as Grip Strength (kg) / Body Weight (kg) × 100. The results of each assessment are compared. A change within ± 20% in the LSGI score within the first month post-transplantation compared to the pre-transplantation score is considered safe.
ALS related assessment
Time frame: 3 month, 6 month, 1 year
Assessment of Disease Functional Progression: The ALSFRS-R scale was used to conduct standardized assessments at baseline (1, 3, 6, 12, 18, and 24 months post-transplantation). Biomarker of Neuroaxonal Injury: Peripheral blood and CSF samples were collected from patients to assess neurofilament light chain (NFL). By analyzing changes in NFL relative to baseline, the dynamic changes in neuroaxonal injury following umbilical cord blood transplantation intervention were objectively evaluated. Imaging Evaluation: MR and PET-CT.
2 secondary outcomes
Assessment of Drug-Induced Liver and Kidney Toxicity
Time frame: up to 24 weeks
Assessment of acute GVHD
Time frame: up to 24 weeks
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 20 Apr 2026.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Mobilization Regimen, Conditioning Regimen and GVHD Prophylaxis in people with ALS (Amyotrophic Lateral Sclerosis), run by Institute of Hematology & Blood Diseases Hospital, China 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 7 inclusion and 16 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: enrolling by invitation, as of the sponsor's last update on 20 Apr 2026. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (time frame: 1 year, 3 year, 5 year). The study lists 3 primary outcomes in all. 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 20 Apr 2026.