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Home › Conditions › Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis

Part of Motor neuron disease

21 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Amyotrophic Lateral Sclerosis

77 trials tracked for Amyotrophic Lateral Sclerosis: 17 in phase 3 or 4 and 11 with published results. The most-cited published study has 915 citations.

77Trials tracked
17Phase 3 & 4
0Recruiting
11With published results
Phase distribution
Phase 3 17 Phase 2 39 Phase 1 2 Other / NA 19
  1. Phase 3 An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS) Completed · 915 cited
  2. Phase 3 Phase 3 Study of Dexpramipexole in ALS Completed · 235 cited
  3. Phase 3 Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From ALS Completed · 137 cited
  4. Phase 3 Arimoclomol in Amyotropic Lateral Sclerosis Completed · 32 cited
  5. Phase 3 Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year Completed · 25 cited
  6. Phase 3 HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose Completed · 21 cited
Show 44 more trials
  1. Phase 3 HEALEY ALS Platform Trial - Regimen D Pridopidine Completed · 20 cited
  2. Phase 3 Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS Completed · 19 cited
  3. Phase 3 HEALEY ALS Platform Trial - Regimen B Verdiperstat Completed · 16 cited
  4. Phase 3 HEALEY ALS Platform Trial - Regimen C CNM-Au8 Completed · 15 cited
  5. Phase 3 Study to Investigate the Efficacy and Safety of FAB122 (Daily Oral Edaravone) in Patients With Amyotrophic Lateral Sclerosis Completed · 1 cited
  6. Phase 3 HEALEY ALS Platform Trial - Regimen A Zilucoplan Completed
  7. Phase 3 HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262 Completed
  8. Phase 3 HEALEY ALS Platform Trial - Regimen G DNL343 Completed
  9. Phase 3 Escitalopram (Lexapro) for Depression MS or ALS Completed
  10. Phase 3 Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS) Trial Completed
  11. Phase 3 Clinical Trial Ceftriaxone in Subjects With ALS Completed
  12. Phase 2 Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211) Completed
  13. Phase 2 Study of AMDX-2011P as a Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy Completed
  14. Phase 2 Treatment Combining Riluzole and IFB-088 in Bulbar Amyotrophic Lateral Sclerosis (TRIALS Protocol) Completed
  15. Phase 2 A Multi-Center Controlled Screening Trial of Safety and Efficacy of Lithium Carbonate in Subjects With Amyotrophic Lateral Sclerosis (ALS) Completed
  16. Phase 2 Clinical Trial of High Dose CoQ10 in ALS Completed
  17. Phase 2 Safety of Urate Elevation in Amyotrophic Lateral Sclerosis (ALS) Completed
  18. Phase 2 Impact of Nuedexta on Bulbar Physiology and Function in ALS Completed
  19. Phase 2 Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis Completed
  20. Phase 2 Clinical Trial of Glatiramer Acetate in Amyotrophic Lateral Sclerosis (ALS) Completed
  21. Phase 2 Safety and Efficacy Study of Creatine and Tamoxifen in Volunteers With Amyotrophic Lateral Sclerosis (ALS) Completed
  22. Phase 2 Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis Completed
  23. Phase 2 Trial of Theracurmin for Patients With Amyotrophic Lateral Sclerosis (ALS) Completed
  24. Phase 2 AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) Completed
  25. Phase 2 Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS) Completed
  26. Phase 2 Therapy in Amyotrophic Lateral Sclerosis (TAME) Completed
  27. Phase 2 3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS) Completed
  28. Phase 2 Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS) Completed
  29. Phase 2 Study of Ozanezumab (GSK1223249) Versus Placebo in the Treatment of Amyotrophic Lateral Sclerosis Completed
  30. Phase 2 Open Label Extension Study of AMX0035 in Patients With ALS Completed
  31. Phase 2 P300 Brain Computer Interface Keyboard to Operate Assistive Technology Completed
  32. Phase 2 Clinical Trial of Ezogabine (Retigabine) in ALS Subjects Completed
  33. Phase 2 A Biomarker Study to Evaluate MN-166 in Subjects With Amyotrophic Literal Sclerosis (ALS) Completed
  34. Phase 2 Clenbuterol on Motor Function in Individuals With Amyotrophic Lateral Sclerosis Completed
  35. Phase 2 Multiple Doses of AT-1501-A201 in Adults With ALS Completed
  36. Phase 2 Gilenya in Amyotrophic Lateral Sclerosis (ALS) Completed
  37. Phase 2 A Trial of Tocilizumab in ALS Subjects Completed
  38. Phase 2 The Effect of RNS60 on ALS Biomarkers Completed
  39. Phase 2 Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis Completed
  40. Phase 2 A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis Completed
  41. Phase 2 Ibudilast (MN-166) in Subjects With Amyotrophic Lateral Sclerosis (ALS) Completed
  42. Phase 2 Efficacy and Safety of Plasma Exchange With Albutein® 5% in Participants With Amyotrophic Lateral Sclerosis Completed
  43. Phase 2 Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) Completed
  44. Phase 2 A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS) Completed

Showing the 50 most-cited and recently-updated of 77 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Amyotrophic Lateral Sclerosis: what the trials found

DNL343 demonstrated no significant difference compared to placebo in ALSFRS-R total scores, CAFS, or respiratory function 1. In a large trial of Dexpramipexole, there were no significant differences in CAFS, mortality at 12 months, or ALSFRS-R scores 3. Similarly, Olesoxime did not show significant differences in failure rates over 18 months or the proportion of patients reaching a specific ALSFRS-R threshold or death 4.

Several other compounds failed to show statistically significant improvements in primary endpoints. Arimoclomol showed no difference in CAFS 5, and Tirasemtiv showed no significant impact on slow vital capacity (SVC), respiratory domain scores, or muscle strength 6. Levosimendan did not significantly affect SVC, CAFS, or time to respiratory events 7. SLS-005, Pridopiren, CNM-Au8, and Zilucoplan also failed to show significant improvements in ALSFRS-R scores, respiratory function, or muscle strength [8, 9, 10, 14]. ABBV-CLS-7262 showed no significant difference in functional scores, respiratory function, or upper limb muscle strength 13.

Regarding specific biomarkers and supportive care, Tofersen significantly reduced CSF levels of total SOD1 protein compared to baseline (p<0.0001), though it did not result in a significant change in ALSFRS-R scores at week 28 2. Insulin-like growth factor, type 1 showed no significant difference in muscle testing rates or survival status 16. Escitalopram was associated with a statistically significant reduction on the Hamilton Depression Scale (p=0.01) 17.

Recent results — preliminary, needs further review

  • AMX0035 demonstrated statistically significant improvements in ALSFRS-R total score change in slope (p=0.0239), time to death (p=0.0475), and the composite of time to hospitalization, death, or death equivalent (p=0.0308) 20. IFB-088 50mg/day did not show significant differences in ALSFRS-R scores, ALS_MITOS staging, or King's College Scale staging 18.

For the clinician treating this condition

  • Most investigated pharmacological interventions (e.g., DNL343, Dexpramipexole, Olesoxime, Arimoclomol, Tirasemtiv, Levosimendan, SLS-005, Pridopidine, CNM-Au8, Zilucoplan) failed to show statistically significant improvements in primary functional outcomes like ALSFRS-R scores or respiratory function.
  • Tofersen demonstrated a significant reduction in CSF SOD1 protein levels (p<0.0001), though this did not translate into a statistically significant change in ALSFRS-R scores at 28 weeks.
  • AMX0035 showed statistically significant improvements in functional progression, survival time, and composite clinical outcomes in a Phase 2 trial.

AI synthesis of 12 cited trials, updated Jun 28, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Meta-analysis links speech features to severity in Parkinsons, cerebral palsy, and ALS
Neurology Meta-analysis
Meta-analysis links speech features to severity in Parkinsons, cerebral palsy, and ALS Speech patterns may help track disease severity in Parkinsons and other conditions
This meta-analysis synthesizes evidence from 890 speakers across 5 languages to evaluate a training-free method for assessing speech degrada…
A new computer method tracks Parkinson's and other disease severity by measuring how specific speech sounds change without needing special t…
medRxiv Apr 19, 2026
Cross-sectional CSF proteomic analysis identifies ALS-specific biomarker signature
Neurology
Cross-sectional CSF proteomic analysis identifies ALS-specific biomarker signature ALS Blood Test Breakthrough Could Speed Diagnosis
This observational cross-sectional study analyzed CSF proteomics in 237 participants to identify ALS-specific biomarkers.
A new five-protein blood test pattern could finally help doctors spot ALS early, before nerve damage becomes too advanced.
medRxiv Apr 19, 2026
Systematic review finds gut microbiome dysbiosis associated with ALS progression
Neurology Meta-analysis
Systematic review finds gut microbiome dysbiosis associated with ALS progression Could gut bacteria changes be linked to ALS, or just happen alongside it?
This systematic review with narrative synthesis examined 61 studies on gut microbiome alterations in ALS.
People with ALS often show reduced gut bacteria diversity, but it is still unclear if these changes cause the disease or simply happen along…
Frontiers Apr 15, 2026
Observational study finds no association between ZFHX3 GGC repeat expansions and ALS risk
Genetics & Precision Medicine
Observational study finds no association between ZFHX3 GGC repeat expansions and ALS risk New DNA Scan Rules Out ALS Link
This observational research article examined ZFHX3 GGC repeat expansions in 5,785 people with ALS and 7,982 healthy controls.
A major genetic test confirms that a specific repeat in your DNA does not cause ALS, even though it can cause a different movement disorder.
medRxiv Apr 15, 2026
REDDI pipeline improves balanced accuracy for classifying MCI, MS, Parkinson's, and ALS using MEG data
Neurology
REDDI pipeline improves balanced accuracy for classifying MCI, MS, Parkinson's, and ALS using MEG data Can a new computer tool help doctors spot brain diseases faster and more accurately?
This study developed a machine learning framework called REDDI that utilizes MEG resting-state data and Riemannian geometry for classifying…
A new computer tool helps doctors distinguish between hard-to-tell-apart brain diseases like Parkinson's and ALS with 13% better accuracy.
medRxiv Apr 15, 2026
Systematic review validates iPSC-derived organoids as models for ALS pathology compared to traditional 2D or animal models
Hematology Sys. Review
Systematic review validates iPSC-derived organoids as models for ALS pathology compared to traditional 2D or animal models Scientists Grow Tiny ALS Brains in Labs — What It Could Mean
This systematic review evaluated in vitro organoid models derived from human-specific neural and neuromuscular subtypes for Amyotrophic Late…
For the first time, scientists can watch ALS unfold in human tissue grown outside the body — which could speed up the search for treatments …
Frontiers Apr 14, 2026
Muscle ultrasonography shows high diagnostic accuracy for fasciculation detection in ALS
Radiology & Imaging Meta-analysis
Muscle ultrasonography shows high diagnostic accuracy for fasciculation detection in ALS A Simple Scan Could Speed Up an ALS Diagnosis
A systematic review and meta-analysis of 13 studies involving 1,176 participants found muscle ultrasonography has high sensitivity (0.87) an…
A common ultrasound machine may help doctors spot a key sign of ALS more quickly, potentially shortening the difficult diagnostic journey.
Apr 11, 2026
FDG-PET reveals dysregulated glucose metabolism as a common feature across late-onset neurodegenerative diseases with unique patterns
Neurology Meta-analysis
FDG-PET reveals dysregulated glucose metabolism as a common feature across late-onset neurodegenerative diseases with unique patterns FDG-PET scans show common brain glucose changes across several late-onset neurodegenerative diseases
This meta-analysis of 5,412 individuals with age-associated neurodegenerative diseases (A2ND) and 3,549 controls utilized FDG-PET to quantif…
FDG-PET scans reveal that dysregulated brain glucose metabolism is a shared feature across late-onset neurodegenerative diseases like Alzhei…
medRxiv Apr 9, 2026
Meta-analysis finds no significant association between statin use and ALS survival outcomes
Neurology Meta-analysis
Meta-analysis finds no significant association between statin use and ALS survival outcomes Do statins help people with ALS live longer? A new analysis finds no clear link
A meta-analysis of 3,739 ALS patients (889 statin users) from six studies found no statistically significant association between statin use …
A new analysis of 3,739 people with ALS found no link between taking statins and living longer.
Frontiers Apr 2, 2026

Questions about Amyotrophic Lateral Sclerosis

Does the way my body functions change differently in amyotrophic lateral sclerosis over time?

Yes, ALS causes progressive changes in motor, respiratory, bulbar, and cognitive functions, with functional decline measurable over time.

Full answer →
How do different treatments for amyotrophic lateral sclerosis compare when looking at many trials together?

A network meta-analysis of 109 trials found that combining cell therapy with neuroprotective agents may slow ALS progression most, while exercise and respiratory interventions also show benefits.

Full answer →
Can a specific protein signature in spinal fluid help identify amyotrophic lateral sclerosis?

Yes, a specific protein signature in spinal fluid can help identify ALS, with a five-protein panel showing high accuracy in distinguishing ALS from healthy controls.

Full answer →
Did the drug Reeldesemtiv help people with amyotrophic lateral sclerosis use their devices better?

No, reldesemtiv did not help people with ALS use devices like wheelchairs or communication aids; a trial found no impact on device use and was stopped early for futility.

Full answer →