A Phase 2 interventional study of Cyclophosphamide and Riluzole in Amyotrophic Lateral Sclerosis, sponsored by Huashan Hospital. Not yet recruiting. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by Huashan Hospital · Phase 2, Interventional, and Treatment
The goal of this clinical trial is to learn whether cyclophosphamide (CTX) combined with standard treatment can slow disease progression in adults with amyotrophic lateral sclerosis (ALS). It will also evaluate the safety and tolerability of CTX. The main questions it aims to answer are:
Researchers will compare CTX combined with standard treatment with standard treatment alone to see whether CTX can slow the progression of ALS.
Participants will:
978 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 277 are open to participants now.
This study's planned enrollment of 60 is above the median of 38 across 662 interventional studies indexed under Amyotrophic Lateral Sclerosis.
Browse Amyotrophic Lateral Sclerosis studies →Huashan Hospital is the lead sponsor of 247 studies on the registry; 104 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Reproductive potential
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Cyclophosphamide · Drug: Riluzole
Drug: Riluzole
Cyclophosphamide will be administered intravenously for 36 weeks in addition to standard treatment with riluzole. During the induction phase, participants will receive a total dose of 2.0 g over approximately 2 weeks in four divided infusions (400 mg, 600 mg, 400 mg, and 600 mg), with an interval of 1-2 days between infusions. During the maintenance phase, cyclophosphamide 1.0 g will be administered intravenously every 4 weeks through Week 36, for a planned cumulative dose of approximately 10.0 g. Hydration, mesna, and antiemetic treatment will be provided as appropriate. Dose delay, dose reduction, or permanent discontinuation will be permitted for treatment-related toxicity according to the study protocol.
Riluzole will be administered orally at a dose of 50 mg twice daily as standard treatment for amyotrophic lateral sclerosis. Participants should be receiving a stable dose before randomization and will continue treatment during the study unless dose modification or discontinuation is required for safety or tolerability reasons.
Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score at Week 48
The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is a 12-item scale used to assess functional impairment in participants with amyotrophic lateral sclerosis. Each item is scored from 0 to 4, yielding a total score ranging from 0 to 48. Higher total scores indicate better functional status, whereas lower scores indicate greater functional impairment. The outcome measure is the change in ALSFRS-R total score from baseline to Week 48, calculated as the Week 48 score minus the baseline score. A more negative change indicates greater functional decline.
Time frame: Baseline to Week 48
Change From Baseline in Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) Score at Week 48
The Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) is a 28-item patient-reported scale used to assess overall disability in participants with amyotrophic lateral sclerosis. Each item is scored from 0 to 2, yielding a raw total score ranging from 0 to 56. The raw score is converted to a linearly weighted normed ROADS score ranging from 0 to 120, with higher scores indicating better functional status and lower scores indicating greater disability. The outcome measure is the change in the normed ROADS score from baseline to Week 48, calculated as the Week 48 score minus the baseline score. A more negative change indicates greater functional decline.
Time frame: Baseline to Week 48
Change From Baseline in Dominant Hand Grip Strength at Week 48
Time frame: Baseline to Week 48
Change From Baseline in Percent-Predicted Forced Vital Capacity (FVC) at Week 48
Forced vital capacity (FVC) will be measured using standardized pulmonary function testing procedures and expressed as a percentage of the predicted value based on appropriate reference equations. The outcome measure is the change in percent-predicted FVC from baseline to Week 48, calculated as the Week 48 value minus the baseline value. A greater decrease indicates greater deterioration in respiratory function.
Time frame: Baseline to Week 48
Change From Baseline in Neurofilament Light Chain (NfL) Levels at Week 48
Time frame: Baseline to Week 48
Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitude at Week 48
Compound muscle action potential (CMAP) amplitude will be assessed using standardized motor nerve conduction studies. CMAP amplitude will be measured in millivolts (mV). The outcome measure is the change in CMAP amplitude from baseline to Week 48, calculated as the Week 48 value minus the baseline value. A greater reduction in CMAP amplitude indicates greater loss of functional motor axons.
Time frame: Baseline to Week 48
Change From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score at Week 48
Time frame: Baseline to Week 48
Time to Death, Enteral Feeding, or Permanent Assisted Ventilation
Time frame: Randomization through Week 48
Change From Baseline in Percent-Predicted Slow Vital Capacity (SVC) at Week 48
Slow vital capacity (SVC) will be measured using standardized pulmonary function testing procedures and expressed as a percentage of the predicted value based on appropriate reference equations. The outcome measure is the change in percent-predicted SVC from baseline to Week 48, calculated as the Week 48 value minus the baseline value. A greater decrease indicates greater deterioration in respiratory function.
Time frame: Baseline to Week 48
Change From Baseline in Motor Evoked Potential (MEP) Amplitude at Week 48
Motor evoked potential (MEP) amplitude will be assessed using standardized transcranial magnetic stimulation and neurophysiological recording procedures. MEP amplitude will be measured in millivolts (mV). The outcome measure is the change in MEP amplitude from baseline to Week 48, calculated as the Week 48 value minus the baseline value.
Time frame: Baseline to Week 48
Change From Baseline in ALSFRS-R Total Score at Week 96
The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is a 12-item scale used to assess functional impairment in participants with amyotrophic lateral sclerosis. Each item is scored from 0 to 4, yielding a total score ranging from 0 to 48. Higher total scores indicate better functional status, whereas lower scores indicate greater functional impairment. The outcome measure is the change in ALSFRS-R total score from baseline to Week 48, calculated as the Week 48 score minus the baseline score. A more negative change indicates greater functional decline.
Time frame: Baseline to Week 96
Change From Baseline in Percent-Predicted Forced Vital Capacity (FVC) at Week 96
Forced vital capacity (FVC) will be measured using standardized pulmonary function testing procedures and expressed as a percentage of the predicted value based on appropriate reference equations. The outcome measure is the change in percent-predicted FVC from baseline to Week 96, calculated as the Week 96 value minus the baseline value. A greater decrease indicates greater deterioration in respiratory function.
Time frame: Baseline to Week 96
Change From Baseline in King's College Clinical Staging System Stage Through Week 96
Disease progression will be assessed using the King's College Clinical Staging System for amyotrophic lateral sclerosis. The system classifies disease progression according to the number of central nervous system regions involved and the occurrence of nutritional or respiratory failure. Stages range from Stage 1 to Stage 4, with Stage 4 subdivided into Stage 4A (nutritional failure) and Stage 4B (respiratory failure). Higher stages indicate more advanced disease. Change in King's College Clinical Stage from baseline through Week 96 will be evaluated.
Time frame: Baseline through Week 96
Change From Baseline in Amyotrophic Lateral Sclerosis Milano-Torino Staging (ALS-MiToS) System Stage Through Week 96
Disease progression will be assessed using the Amyotrophic Lateral Sclerosis Milano-Torino Staging (ALS-MiToS) System, which is based on loss of independence in four functional domains derived from the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R): walking/self-care, swallowing, communicating, and breathing. Stages range from 0 to 5. Stage 0 indicates no loss of independence in any domain; Stages 1 through 4 indicate loss of independence in one to four domains, respectively; and Stage 5 indicates death. Higher stages indicate more advanced disease and greater loss of functional independence. Change in ALS-MiToS stage from baseline through Week 96 will be evaluated.
Time frame: Baseline through Week 96
Time to Death or Permanent Assisted Ventilation Through Week 96
Time frame: Randomization through Week 96
Change From Baseline in Percent-Predicted Slow Vital Capacity (SVC) at Week 96
Slow vital capacity (SVC) will be measured using standardized pulmonary function testing procedures and expressed as a percentage of the predicted value based on appropriate reference equations. The outcome measure is the change in percent-predicted SVC from baseline to Week 96, calculated as the Week 96 value minus the baseline value. A greater decrease indicates greater deterioration in respiratory function.
Time frame: Baseline to Week 48
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Cyclophosphamide
Amyotrophic Lateral Sclerosis →