Amyotrophic Lateral Sclerosis (ALS)Phase 2NCT07833618
A Phase II Study of ALT001 in Patients With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing ALT001 1.0 μg/kg and ALT001 2.0 μg/kg in people with Amyotrophic Lateral Sclerosis (ALS), run by Darwin Origin (Hubei) Biopharmaceutical Co., Ltd. at 1 site in China.
Trial summary
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 more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- ALT001 1.0 μg/kg and ALT001 2.0 μg/kg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 1 siteChina
- Study dates
- 1 Sep 2026to 31 Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
- Severe autoimmune diseases, myeloproliferative disorders, leukemia or lymphoma, malignant tumors, fractures, or severe scoliosis at screening, which the investigator considers would affect study evaluation.
- 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.
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- 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.
- 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.
- Judged by the investigator to be unsuitable for participation in this study (e.g., significantly clinically significant abnormalities in physical examination or laboratory tests).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Capital Medical University Affiliated Beijing Tiantan Hospital
Beijing, Beijing Municipality, 100070
Yilong Wang, MD, Principal Investigator, +86 139 1166 6571, yilong528@gmail.com
About this study
Official title: 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.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- ALT001 1.0 μg/kg, ALT001 2.0 μg/kg, Placebo
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALT001 1.0 μg/kgDrug
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.
ALT001 2.0 μg/kgDrug
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.
PlaceboDrug
Matching placebo (ALT001 simulant) containing no active ingredient; identical in appearance, packaging, labeling, and post-reconstitution infusion presentation to ALT001.
| Study group | Role | What they receive |
|---|---|---|
| ALT001 1.0 μg/kg | Experimental | Not described Receives: Drug: ALT001 1.0 μg/kg |
| ALT001 2.0 μg/kg | Experimental | Not described Receives: Drug: ALT001 2.0 μg/kg |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to Week 24 (Week0, Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24 after randomization)
Secondary outcomes (21)
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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From informed consent through 28 days after last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2026
- Study start1 Sep 2026 (estimated)
- Primary completion1 Jan 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Darwin Origin (Hubei) Biopharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07833618
- Sponsor's protocol ID
- ALT001-CN-IND-01002
- Status last verified
- Sep 2026
- First submitted
- 28 Aug 2026
- Last updated
- 22 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07833618
- Acronym / sponsor ID
- ALT001-CN-IND-01002
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Darwin Origin (Hubei) Biopharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2026 (estimated)
- Primary completion
- 1 Jan 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 22 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ALT001 1.0 μg/kg | Experimental | Drug: ALT001 1.0 μg/kg |
| ALT001 2.0 μg/kg | Experimental | Drug: ALT001 2.0 μg/kg |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
- Severe autoimmune diseases, myeloproliferative disorders, leukemia or lymphoma, malignant tumors, fractures, or severe scoliosis at screening, which the investigator considers would affect study evaluation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALT001 1.0 μg/kgAllogeneic 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. | Drug | ALT001 1.0 μg/kg |
| ALT001 2.0 μg/kgAllogeneic 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. | Drug | ALT001 2.0 μg/kg |
| PlaceboMatching placebo (ALT001 simulant) containing no active ingredient; identical in appearance, packaging, labeling, and post-reconstitution infusion presentation to ALT001. | Drug | Placebo |
Primary outcome
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score
Time frame: Baseline to Week 24 (Week0, Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24 after randomization)
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.
21 secondary outcomes
ALSFRS-R slope from baseline to Week 24 (double-blind period)
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)
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)
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)
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 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
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)
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Change from baseline in ALSAQ-40 total score
Time frame: Baseline to Week 48 (Week 0，Week 1, Week 13，Week 24, Week25，Week37，Week48)
Change from baseline in ROADS total score
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
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)
Time frame: Baseline to Week 48 (Week 0，Week 13，Week 24, Week25，Week37，Week48 )
Change from baseline in muscle strength (MRC sum score)
Time frame: Baseline to Week 48 (Week 0，Week 13，Week 24, Week25，Week37，Week48 )
Change from baseline in ALS Functional Staging System stage
Time frame: Baseline to Week 48(Week 0，Week 13，Week 24, Week25，Week37，Week48 )
Concentration of neurofilament light chain (NfL) in plasma
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
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Concentration of superoxide dismutase 1 (SOD1) in plasma
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Concentration of immunoglobulins in serum (IgG, IgM, IgA)
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+)
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Plasma proteomics profile
Time frame: Week 1, Week 13, Week 24, Week 25, Week 37, Week48 after randomization.
Incidence of treatment-emergent anti-drug antibodies (ADA)
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)
Time frame: From informed consent through 28 days after last dose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ALT001 1.0 μg/kg and ALT001 2.0 μg/kg in people with Amyotrophic Lateral Sclerosis (ALS), run by Darwin Origin (Hubei) Biopharmaceutical Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 22 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Pengfei Zhang, Director of Clinical Operations (+86-13911241040, zhangpengfei@darwincell.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score (time frame: Baseline to Week 24 (Week0, Week 1, Week 5, Week 9, Week 13, Week 17, Week 21, Week 24 after randomization)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2026.