52 completed, 18 terminated, 8 recruiting, 8 unknown. 96 Phase 3, 39 Phase 2.
Follow TDP-43 Proteinopathies: new trials by email Literature Review of TDP-43 Proteinopathies trials Research Report on TDP-43 Proteinopathies
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Efficacy and Safety of Tazbentetol in ALS Participants Tazbentetol, placebo - Amyotrophic Lateral Sclerosis | Phase 2 / Phase 3 | Not yet recruiting | Spinogenix | 430 | 2026-10-01 | 2026-09-10 |
| This Study Evaluates the Safety, Target Engagement, and Preliminary Efficacy of Galunisertib (TGF-βR1/ALK5 Inhibitor)Combined With Nerandomilast (PDE4 Inhibitor) in GREM2-positive ALS, a Biomarker-defined Subgroup Hypothesized to Reflect Heightened TGF-β/SMAD-driven Astrocytic and Fibrotic Signaling Galunisertib + Nerandomilast Combination - ALS (Amyotrophic Lateral Sclerosis) | Phase 2 / Phase 3 | Not yet recruiting | Gipfel Life Sciences GmbH | 60 | 2026-06-30 | 2026-01-07 |
| HEALEY ALS Platform Trial - Regimen I NUZ-001 NUZ-001, Matching placebo - Amyotrophic Lateral Sclerosis (ALS) | Phase 2 / Phase 3 | Active not recruiting | Merit E. Cudkowicz, MD | 240 | 2026-02-25 | 2026-07-20 |
| Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS Pridopidine, Placebo - Amyotrophic Lateral Sclerosis | Phase 3 | Recruiting | Prilenia | 500 | 2026-02-01 | 2026-10-27 |
| Efficacy and Safety of Masitinib in Combination With SoC Versus Placebo in the Treatment of ALS Patients Masitinib 4.5 mg/kg/day, Placebo, Riluzole (100 mg) - Amyotrophic Lateral Sclerosis (ALS) | Phase 3 | Not yet recruiting | AB Science | 412 | 2026-01-01 | 2025-10-03 |
| HD-tDCS in Amyotrophic Lateral Sclerosis: A Multicenter Randomized Controlled Trial Active HD-tDCS, Simulated HD-tDCS - Amyotrophic Lateral Sclerosis (ALS) | Phase 2 / Phase 3 | Recruiting | Universidade Federal do Rio Grande do Norte | 80 | 2025-11-28 | 2026-07-20 |
| A Study to Evaluate the Efficacy and Safety of Different Doses of CB03-154 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) Test drug CB03-154 5mg group, Test drug CB03-154 10mg group, Test drug CB03-154 15mg group - ALS (Amyotrophic Lateral Sclerosis) | Phase 2 / Phase 3 | Recruiting | Shanghai Zhimeng Biopharma, Inc. | 240 | 2025-10-30 | 2026-09-22 |
| Study of COYA 302 for the Treatment of ALS COYA 302, Placebo - Amyotrophic Lateral Sclerosis (ALS) | Phase 2 / Phase 3 | Active not recruiting | Coya Therapeutics | 120 | 2025-10-01 | 2026-10-13 |
| Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY) Debamestrocel - MSC-NTF (NurOwn), Placebo - Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis | Phase 3 | Not yet recruiting | Brainstorm-Cell Therapeutics | 200 | 2025-06-30 | 2025-05-22 |
| Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS Acetyl-l-carnitine, Placebo - Amyotrophic Lateral Sclerosis | Phase 2 / Phase 3 | Recruiting | Mario Negri Institute for Pharmacological Research | 246 | 2025-03-26 | 2026-10-06 |
| RAPA-501 Therapy for ALS RAPA-501 Autologous T stem cells - Amyotrophic Lateral Sclerosis | Phase 2 / Phase 3 | Recruiting | Rapa Therapeutics LLC | 41 | 2025-01-02 | 2026-09-29 |
| RolloverTreatment With Triumeq for People With ALS Following the Lighthouse II Trial Abacavir 600mg, Lamivudine 300mg and Dolutegravir 50mg (Triumeq) - Amyotrophic Lateral Sclerosis, ALS | Phase 3 | Terminated | Macquarie University, Australia | 12 | 2024-11-15 | 2025-06-08 |
| Nerve Growth Factor Encapsulated With 2-methacryloyloxyethyl Phosphorylcholine Nanocapsules in the Treatment of Amyotrophic Lateral Sclerosis Nerve Growth Factor - Amyotrophic Lateral Sclerosis | Phase 2 / Phase 3 | Not yet recruiting | Beijing Tiantan Hospital | 60 | 2024-05-01 | 2024-04-30 |
| Continuation Study for Latozinemab Latozinemab - Neurodegenerative Diseases | Phase 3 | Terminated | Alector Inc. | 17 | 2023-12-08 | 2026-01-21 |
| HEALEY ALS Platform Trial - Regimen G DNL343 DNL343, Matching Placebo - Amyotrophic Lateral Sclerosis | Phase 2 / Phase 3 | Completed | Merit E. Cudkowicz, MD | 249 | 2023-05-24 | 2026-01-28 |
| HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262 ABBV-CLS-7262 Dose 1, ABBV-CLS-7262 Dose 2, Matching Placebo - Amyotrophic Lateral Sclerosis | Phase 2 / Phase 3 | Completed | Merit E. Cudkowicz, MD | 310 | 2023-03-23 | 2025-11-14 |
| Study to Investigate the Long-term Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis FAB122 - Amyotrophic Lateral Sclerosis | Phase 3 | Terminated | Ferrer Internacional S.A. | 201 | 2023-03-06 | 2025-03-04 |
| Extension Study Evaluating The Safety And Tolerability of AMX0035 AMX0035 - Amyotrophic Lateral Sclerosis | Phase 3 | Completed | Amylyx Pharmaceuticals Inc. | 352 | 2022-12-29 | 2025-01-13 |
| PET Imaging Tau Accumulation in FTLD and Atypical Alzheimer's Using [18F]-PI-2620 [18F]-PI-2620 - Frontotemporal Lobar Degeneration, Alzheimer Disease | Phase 3 | Recruiting | University of Pennsylvania | 72 | 2022-09-19 | 2026-09-21 |
| A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031) Reldesemtiv - Amyotrophic Lateral Sclerosis | Phase 3 | Terminated | Cytokinetics | 71 | 2022-08-04 | 2024-08-13 |
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Amyotrophic Lateral Sclerosis (90) · Motor Neuron Disease (11) · Frontotemporal Dementia (6) · Alzheimer Disease (3) · Neuromuscular Diseases (3) · Cognitive Dysfunction (2) · Dementia (2) · Multiple Sclerosis (2) · Muscular Atrophy (2) · Neurodegenerative Diseases (2) · Sialorrhea (2) · Anorexia (1) · Brain Diseases (1) · Cachexia (1) · Central Nervous System Diseases (1)
Metabolic Diseases (96) · Nervous System Diseases (96) · Nutritional and Metabolic Diseases (96) · Proteostasis Deficiencies (96) · Central Nervous System Diseases (95) · Neurodegenerative Diseases (94) · Spinal Cord Diseases (90) · Neuromuscular Diseases (87) · Motor Neuron Disease (79) · Mental Disorders (9) · Pathological Conditions, Signs and Symptoms (8) · Brain Diseases (7) · Frontotemporal Lobar Degeneration (6) · Neurocognitive Disorders (6)
Riluzole (9) · Edaravone (6) · Memantine (3) · arimoclomol (3) · masitinib (3) · mecobalamin (3) · pridopidine (3) · sodium phenylbutyrate and taurursodiol (3) · CK-2017357 (2) · Dexpramipexole (2) · Lithium Carbonate (2) · Simendan (2) · abacavir, dolutegravir, and lamivudine drug combination (2) · dextromethorphan - quinidine combination (2) · olesoxime (2)
Merit E. Cudkowicz, MD (9) · Shionogi (5) · Cytokinetics (4) · AB Science (3) · Amylyx Pharmaceuticals Inc. (3) · Assistance Publique - Hôpitaux de Paris (3) · Eisai Co., Ltd. (3) · Alector Inc. (2) · Avanir Pharmaceuticals (2) · Brainstorm-Cell Therapeutics (2) · Ferrer Internacional S.A. (2) · Hoffmann-La Roche (2) · Humanitas Mirasole SpA (2) · Johns Hopkins University (2) · Knopp Biosciences (2)
United States (52) · France (28) · Germany (27) · United Kingdom (27) · Belgium (26) · Canada (26) · Spain (26) · Italy (25) · Netherlands (25) · Sweden (19) · Australia (17) · Ireland (17) · Poland (12) · Portugal (10) · Japan (9)
Merit Cudkowicz, MD (7) · Merit Cudkowicz, MD, MSc (3) · Kazunori Saeki (2) · Merit Cudkowicz (2) · Michael Benatar, MD PhD (2) · Adrian Hepner, M.D. (1) · Albert Ludolph, MD, PhD (1) · Alberto Albanese, MD (1) · Anthony J. Windebank, MD (1) · Carlayne E Jackson, MD (1) · Charles B Simpson, MD (1) · Charles M Wiener, MD (1)
PMID 25982504, PMID 34536404, PMID 40067754, PMID 40067755, PMID 40067821, PMID 14718694, PMID 15505150, PMID 17980667, PMID 19029516, PMID 19682544, PMID 19743466, PMID 20363190, PMID 20839238, PMID 21792308, PMID 21887710, PMID 22674572, PMID 23613806, PMID 24067398, PMID 24819148, PMID 25286015, PMID 25297012, PMID 27751553, PMID 28522181, PMID 28864675, PMID 28872913, PMID 28872914, PMID 28872915, PMID 28872918, PMID 28872920, PMID 29367439, PMID 29402141, PMID 30523161, PMID 30554828, PMID 30578206, PMID 30636701, PMID 30814647, PMID 31719072, PMID 31930364, PMID 32640130, PMID 33853394, PMID 34344312, PMID 34433481, PMID 34457038, PMID 34816762, PMID 35246398, PMID 35532908, PMID 35585556, PMID 35798516, PMID 36008843, PMID 36048877
U01NS049640 (2), 2014 (1), 2015 (1), 2016 (1), 2017 (1), 2018 (1), 2019 (1), 2023 (1), R01NR015226 (1), R01NS042759 (1)