Efficacy and Safety of Cyclophosphamide in Amyotrophic Lateral Sclerosis

Sponsor
Huashan Hospital
Study ID
NCT07834450
Phase
PHASE2
Status
Not Yet Recruiting

Notify me when recruiting opens

Save your spot on the interest list for this study. We'll keep your details with this study so our team can follow up when recruiting opens.

Not yet recruiting

Add your contact details and location so we can keep your interest tied to this study.

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • Cyclophosphamide — DRUG
    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 — DRUG
    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.

Study Details

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: 1. Does CTX combined with standard treatment reduce the decline in ALS Functional Rating Scale-Revised (ALSFRS-R) scores over 48 weeks compared with standard treatment alone? 2. What medical problems and side effects do participants have while receiving CTX? 3. Are markers of neuroinflammation and immune activity, including TSPO-PET, neurofilament light chain (NfL), upper motor neuron burden, electrophysiological measures, immune cell profiles, and autoantibodies, associated with treatment response? Researchers will compare CTX combined with standard treatment with standard treatment alone to see whether CTX can slow the progression of ALS. Participants will: 1. Be randomly assigned to receive CTX plus standard treatment or standard treatment alone 2. Receive CTX treatment for up to 36 weeks if assigned to the CTX group 3. Visit the study center regularly for clinical assessments, blood tests, lung function tests, electrophysiological tests, and other safety evaluations 4. Complete assessments of physical function, muscle strength, respiratory function, and quality of life 5. Undergo biomarker assessments, including TSPO-PET imaging and NfL testing 6. Be followed for 48 weeks during the main study period and for up to 96 weeks for long-term outcomes and safety

Key Dates

First listed
Sep 22, 2026
Start date
Oct 1, 2026
Status verified
Sep 2026
Primary completion
Oct 30, 2028
Completion
Sep 30, 2029

Study Design

Enrollment
60 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Cyclophosphamide Plus Standard Treatment
  • Active Comparator: Standard Treatment Alone

Primary Outcome Measure

Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score at Week 48 [ Time Frame: Baseline to Week 48 ]

Central Contacts

Related Studies