A Phase II Study of ALT001 in Patients With Amyotrophic Lateral Sclerosis (ALS)

Trial details

Who runs it

Darwin Origin (Hubei) Biopharmaceutical Co., Ltd.

Trial code

NCT07833618

Record updated

22 September 2026

Original description

ALT001 is an allogeneic unrelated human umbilical cord mesenchymal stem cell-derived protein complex under investigation for the treatment of amyotrophic lateral sclerosis (ALS). This is a multicenter, randomized, double-blind, placebo-controlled, Phase 2 dose-ranging study in patients with ALS.Read full original descriptionShow less

A Randomized, Double-blind, Placebo-controlled, Multicenter Phase II Dose-ranging Study to Evaluate the Efficacy and Safety of ALT001 in Patients With Amyotrophic Lateral Sclerosis

ALT001 is an allogeneic unrelated human umbilical cord mesenchymal stem cell-derived protein complex under investigation for the treatment of amyotrophic lateral sclerosis (ALS). This is a multicenter, randomized, double-blind, placebo-controlled, Phase 2 dose-ranging study in patients with ALS.

Approximately 150 participants will be randomized 1:1:1 to receive ALT001 1.0 μg/kg, ALT001 2.0 μg/kg, or matching placebo by intravenous infusion during the 24-week double-blind (DB) treatment period. During the OLE period, the active treatment groups will continue to receive their original dose under blinded conditions, whereas the placebo group will undergo re-randomization in a 1:1 ratio to receive 1.0 or 2.0 μg/kg ALT001.

The primary objective is to evaluate the dose-response relationship and efficacy of ALT001 at 1.0 and 2.0 μg/kg and to determine the recommended dose for subsequent studies. Secondary objectives include assessment of biomarkers related to efficacy and exploration of changes in cytokines, immune function, proteomics, and immunogenicity (anti-drug antibodies, ADA), as well as evaluation of the safety of ALT001.

Inclusion: Participants or their guardians must be able to understand the procedures and methods of this study, communicate adequately with the investigator, be willing to strictly comply with the clinical trial protocol to complete the study, and voluntarily sign a written informed consent form, or have informed consent signed by a legally authorized representative; Age 18 to 75 years (inclusive of both boundaries), of either sex; Diagnosis of definite or probable ALS according to the revised El Escorial criteria of the World Federation of Neurology for amyotrophic lateral sclerosis, including both familial and sporadic ALS patients; Forced vital capacity (FVC) ≥ 50% of predicted at the screening visit; Total ALSFRS-R score ≥ 30 and ≤ 40 prior to enrollment; If receiving riluzole or edaravone treatment before enrollment, participants must have been on a stable dose for ≥ 30 days prior to Day 1 of this study, and must maintain that dose until the final study visit.

Exclusion: History of alcohol dependence or drug abuse within 6 months prior to screening, judged by the investigator to be unsuitable for participation in this study; Other severe and/or uncontrolled major organ or unstable systemic diseases, including but not limited to uncontrolled diabetes, unstable angina, cerebrovascular accident or transient ischemic attack (within 6 months prior to screening), myocardial infarction (within 6 months prior to screening), severe congestive heart failure, uncontrolled hypertension, uncontrolled active infection, severe hepatic, renal or other severe metabolic diseases, or severe gastrointestinal diseases; Pregnant or lactating women, and those planning to conceive during the medication period or within 3 months after stopping the medication; Participated in another interventional clinical study within 4 weeks prior to this study; received stem cell or exosome therapy within 1 year, or previously received ASO drug treatment or gene therapy; Have other known diseases associated with motor neuron dysfunction that may confound or obscure the diagnosis of ALS; Severe autoimmune diseases, myeloproliferative disorders, leukemia or lymphoma, malignant tumors, fractures, or severe scoliosis at screening, which the investigator considers would affect study evaluation; Positive hepatitis B surface antigen (HBsAg) with HBV-DNA > upper limit of normal (if HBsAg is negative, HBV-DNA testing is not required); positive hepatitis C virus (HCV) antibody with HCV-RNA > upper limit of normal (if anti-HCV is negative, HCV-RNA testing is not required); positive human immunodeficiency virus (HIV) antibody; or positive Treponema pallidum test; Judged by the investigator to be unsuitable for participation in this study (e.g., significantly clinically significant abnormalities in physical examination or laboratory tests); Apparent cognitive impairment (MMSE scale: ≤19 for the illiterate group, ≤22 for the primary school group, and ≤26 for the junior high school and above group [>8 years of education]), or other unstable psychiatric disorders that the investigator considers unsuitable for participation (including suicidal intent, untreated major depression, etc.); Continuous use of non-invasive ventilation (NIV), diaphragm pacing system, or invasive ventilation (tracheostomy) at screening.

ClinicalTrials.gov NCT07833618