A Phase 2 interventional study of ALT001 1.0 μg/kg and ALT001 2.0 μg/kg in Amyotrophic Lateral Sclerosis (ALS), sponsored by Darwin Origin (Hubei) Biopharmaceutical Co., Ltd.. Not yet recruiting at 1 site in China. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by Darwin Origin (Hubei) Biopharmaceutical Co., Ltd. · Phase 2, Interventional, and Treatment
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.
978 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 277 are open to participants now.
This study's planned enrollment of 150 is above the median of 38 across 662 interventional studies indexed under Amyotrophic Lateral Sclerosis.
Browse Amyotrophic Lateral Sclerosis studies →Darwin Origin (Hubei) Biopharmaceutical Co., Ltd. is the lead sponsor of 2 studies on the registry; 2 are open to participants now.
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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.
Short tests of memory and thinking, each scored out of 30. Lower scores suggest more difficulty; criteria give a range, either to include people with memory problems or to leave them out.
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 “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
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.
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 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.
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.
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.
MMSE and MoCA
Short tests of memory and thinking, each scored out of 30. Lower scores suggest more difficulty; criteria give a range, either to include people with memory problems or to leave them out.
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.
TIA (transient ischaemic attack)
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
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.
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 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.
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.
Drug: ALT001 1.0 μg/kg
Drug: ALT001 2.0 μg/kg
Drug: Placebo
Allogeneic 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.
Allogeneic 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.
Matching 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.
Time frame: Baseline to Week 24 (Week0, Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24 after randomization)
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.
Time frame: Baseline to Week 24(Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24 after randomization)
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.
Time frame: Overall study period：Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24, Week 25, Week 29, Week 33, Week 37, Week 41, Week 45, Week 48 after randomization(48 weeks)
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.
Time frame: Week 25 to Week 48 (Week 25, Week 29, Week 33, Week 37, Week 41, Week 45, Week 48 after randomization)
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
Time frame: Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24, Week 25, Week 29, Week 33, Week 37, Week 41, Week 45, Week 48 after randomization(48 weeks)
Assessment of ventilator-free survival (VAFS)
Time from randomization to the first occurrence of non-invasive ventilation, invasive ventilation (tracheostomy), or death due to any cause, whichever occurs first.
Time frame: Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24, Week 25, Week 29, Week 33, Week 37, Week 41, Week 45, Week 48 after randomization(48 weeks)
Change from baseline in forced vital capacity (FVC) /slow vital capacity (SVC) percent predictedpercent predicted
FVC expressed as percent of predicted normal value, assessed by spirometry. SVC expressed as percent of predicted normal value, assessed by spirometry.
Time frame: Baseline to Week 48 (Week 0，Week 13，Week 24, Week25，Week37，Week48)
Concentration of cytokines in serum (IFN-γ, IL-10, IL-13, IL-1β, IL-2, IL-4, IL-5, IL-6, GRO-α, TNF-α, IL-2)
Serum concentrations of the following cytokines, measured by central laboratory assay: IFN-γ, IL-10, IL-13, IL-1β, IL-2, IL-4, IL-5, IL-6, GRO-α, TNF-α and IL-2. Concentrations are reported in pg/mL for each cytokine. This outcome measure reports laboratory analyte concentrations, not a score on a scale; therefore an unabbreviated scale title, minimum/maximum score, and score direction do not apply.
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Change from baseline in ALSAQ-40 total score
The Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) is a 40-item participant-reported quality-of-life scale; each item is rated on a 5-point scale (0-4). The total score ranges from 0 to 160, with higher scores indicating worse quality of life.
Time frame: Baseline to Week 48 (Week 0，Week 1, Week 13，Week 24, Week25，Week37，Week48)
Change from baseline in ROADS total score
The Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) is a 28-item linearly weighted disability scale; each item is scored from 0 to 2, giving a raw total score range of 0-56 that is linearly transformed to a standardized total score ranging from 0 to 146. Higher scores indicate better preserved function (less disability).
Time frame: Baseline to Week 48 (Week 0，Week 1, Week 13，Week 24, Week25，Week37，Week48)
Change from baseline in modified Ashworth scale score
The Modified Ashworth Scale (MAS) is a clinician-rated scale used to assess muscle tone and spasticity during passive movement of the assessed muscle groups. Muscle tone is graded from 0 (no increase in muscle tone) to 4 (affected part rigid), with grade 1+ indicating a slight increase in tone between grades 1 and 2. The minimum score is 0 and the maximum score is 4 for each assessed muscle, with higher scores indicating greater spasticity (a worse outcome). This outcome measure reports the change from baseline in the Modified Ashworth Scale score; a positive change indicates worsening spasticity (a worse outcome).
Time frame: Baseline to Week 48 (Week 0, Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24, Week 25, Week 29, Week 33, Week 37, Week 41, Week 45, Week 48 after randomization)
Change from baseline in grip strength (kg)
Maximum hand grip strength measured by dynamometry, expressed in kilograms.
Time frame: Baseline to Week 48 (Week 0，Week 13，Week 24, Week25，Week37，Week48 )
Change from baseline in muscle strength (MRC sum score)
Manual muscle testing using the Medical Research Council (MRC) scale summed across specified muscle groups; score ranges from 0 (no contraction) to 5 (normal strength) per muscle, with a higher total sum indicating better strength.
Time frame: Baseline to Week 48 (Week 0，Week 13，Week 24, Week25，Week37，Week48 )
Change from baseline in ALS Functional Staging System stage
ALS Functional Staging System stage, assessed to characterize disease progression.
Time frame: Baseline to Week 48(Week 0，Week 13，Week 24, Week25，Week37，Week48 )
Concentration of neurofilament light chain (NfL) in plasma
Plasma NfL concentration measured by central laboratory assay.
Time frame: Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24, Week 25, Week 29, Week 33, Week 37, Week 41, Week 45, Week 48 after randomization(48 weeks)
Concentration of TAR DNA-binding protein 43 (TDP-43) in plasma
Plasma TDP-43 concentration measured by central laboratory assay.
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Concentration of superoxide dismutase 1 (SOD1) in plasma
Plasma SOD1 concentration measured by central laboratory assay.
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Concentration of immunoglobulins in serum (IgG, IgM, IgA)
Serum concentrations of immunoglobulins measured by central laboratory assay.
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Number and percentage of T-cell subsets in blood (CD3+CD4+, CD3+CD8+, CD28+, CD16+)
Enumeration of T-cell subsets by flow cytometry, including CD3+CD4+/CD3+CD8+ ratio.
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Plasma proteomics profile
Proteomic profiling of plasma samples using a central laboratory platform.
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Incidence of treatment-emergent anti-drug antibodies (ADA)
Number and percentage of participants with treatment-emergent ADA responses to ALT001.
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Incidence of adverse events (AEs) and serious adverse events (SAEs)
Number and percentage of participants with TEAEs and SAEs, graded by NCI CTCAE version 6.0.
Time frame: From informed consent through 28 days after last dose
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Sep 2026; primary completion for Jan 2028, 16 months later.
Of 36 industry-sponsored phase 2 interventional studies under Amyotrophic Lateral Sclerosis, half reached primary completion within 21 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 35. This study's planned 16 months is below that.
Counting every sponsor, half of 73 studies reached it within 24 months; a quarter within 16 and three quarters within 35.
How these figures are worked out, and which studies are left out of them.
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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Amyotrophic Lateral Sclerosis →
Darwin Origin (Hubei) Biopharmaceutical Co., Ltd.