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.
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.
Have other known diseases associated with motor neuron dysfunction that may confound or obscure the diagnosis of ALS.
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.
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.
Parallel
ALT001 1.0 μg/kg
DrugAllogeneic unrelated human umbilical cord mesenchymal stem cell-derived protein complex; 130 μg/vial (lyophilized powder); reconstituted and administered by intravenous infusion at 1.0 μg/kg body weight.
ALT001 2.0 μg/kg
DrugAllogeneic unrelated human umbilical cord mesenchymal stem cell-derived protein complex; 130 μg/vial (lyophilized powder); reconstituted and administered by intravenous infusion at 2.0 μg/kg body weight.
Placebo
DrugMatching placebo (ALT001 simulant) containing no active ingredient; identical in appearance, packaging, labeling, and post-reconstitution infusion presentation to ALT001.
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score
The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is a validated 12-item clinician-reported scale. Total score ranges from 0 to 48, with higher scores indicating better function and lower scores indicating greater functional impairment. A higher (less negative) change from baseline indicates less functional decline.
ALSFRS-R slope from baseline to Week 24 (double-blind period)
Weekly rate of change in ALSFRS-R total score, calculated as the slope from baseline to Week 24 in the 24-week double-blind period. Higher (less negative) slope indicates slower functional decline.
ALSFRS-R slope from baseline to Week 48 (overall study period)
Weekly rate of change in ALSFRS-R total score from baseline to Week 48, including the double-blind and open-label extension periods.
ALSFRS-R slope from Week 25 to Week 48 (open-label extension period)
The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is a 12-item clinician-reported scale with a total score ranging from 0 to 48; higher scores indicate better function and lower scores indicate greater functional impairment. The single value reported for this outcome is the slope (rate of change) of the ALSFRS-R total score over time during the 24-week open-label extension (OLE) period, estimated by a mixed model for repeated measures using all post-baseline ALSFRS-R assessments at Weeks 25, 29, 33, 37, 41, 45, and 48. The slope is expressed as change in ALSFRS-R total score per month (points/month); a less negative slope indicates slower functional decline. Only this single slope value is reported for this outcome measure.
Assessment of Combined Assessment of Function and Survival (CAFS)
The Combined Assessment of Function and Survival (CAFS) is a single composite outcome that reports ONE value: the CAFS composite rank score. It aggregates two component measurements - (1) survival status and time to death or permanent assisted ventilation, and (2) change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score - using a pre-specified ranking rule: each participant is compared pairwise with every other participant, with time to death or permanent assisted ventilation taking precedence (earlier event = worse outcome) and change from baseline in ALSFRS-R total score used for participants who remain alive and free of permanent assisted ventilation (smaller decline = better outcome). The pairwise comparisons are summed to yield a single unitless CAFS composite rank score; higher scores indicate a better combined function-and-survival outcome. The component measurements are aggregated into this one reported value and are not repo
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