86 completed, 27 recruiting, 22 unknown, 16 terminated. 194 Phase 1, 75 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 |
|---|---|---|---|---|---|---|
| A Multiple Dose Study to Investigate Safety, Tolerability and Pharmacokinetics of TBN Tetramethylpyrazine nitrone (TBN) tablet / Placebo - Healthy | Phase 1 | Not yet recruiting | Guangzhou Magpie Pharmaceuticals Co., Ltd. | 16 | 2028-09-01 | 2026-09-11 |
| Evaluation of Donor-Derived B Cell Infusions in Patients With Amyotrophic Lateral Sclerosis (ALS) B cell infusion - ALS - Amyotrophic Lateral Sclerosis | Phase 1 | Not yet recruiting | Mark Poznansky | 10 | 2027-09-01 | 2026-09-24 |
| Safety Study of XT-150 in Participants With ALS XT-150 - Amyotrophic Lateral Sclerosis (ALS) | Phase 1 | Not yet recruiting | Xalud Therapeutics, Inc. | 8 | 2027-03-01 | 2026-07-29 |
| A Study of Single Ascending Doses of NI112 in Adults With Amyotrophic Lateral Sclerosis (ALS) NI112, Placebo - Amyotrophic Lateral Sclerosis (ALS) | Phase 1 | Not yet recruiting | Sachi Bioworks Inc. | 40 | 2027-01-22 | 2026-10-30 |
| Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Amyotrophic Lateral Sclerosis (ALS) Q-Cells - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Not yet recruiting | Q Therapeutics, Inc. | 30 | 2026-12-01 | 2026-07-08 |
| Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS nL-TARDB-002 - Amyotrophic Lateral Sclerosis (ALS) | Phase 1 / Phase 2 | Enrolling by invitation | n-Lorem Foundation | 1 | 2026-12-01 | 2026-07-14 |
| Pilot Trial Utilizing β-hydroxy-β-methylbutyrate to Lower IGFBP7 Levels in People With ALS β-hydroxy-β-methylbutyrate (HMB) - Amyotrophic Lateral Sclerosis ALS | Phase 1 | Not yet recruiting | Duke University | 100 | 2026-12-01 | 2026-11-04 |
| A Study of LY3873862 in Participants With Sporadic or Familial Amyotrophic Lateral Sclerosis (ALS). LY3873862, CYP450 Drug Cocktail, Placebo - Amyotrophic Lateral Sclerosis, Cytochrome P-450 Enzyme System | Phase 1 | Not yet recruiting | Eli Lilly and Company | 32 | 2026-11-01 | 2026-10-20 |
| A Study to Learn About the Safety of BIIB147 and Its Effects in Adults With Amyotrophic Lateral Sclerosis (ALS) BIIB147, Placebo - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Recruiting | Biogen | 69 | 2026-10-07 | 2026-10-26 |
| Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS) Orelabrutinib - Amyotrophic Lateral Sclerosis (ALS) | Phase 1 | Not yet recruiting | Liu Qing | 12 | 2026-10-01 | 2026-10-15 |
| FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS TRCN-1023, Placebo - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Recruiting | Trace Neuroscience, Inc. | 30 | 2026-08-20 | 2026-10-14 |
| Therapeutic Approach of Repeated Transient Blood-brain Barrier Opening in Amyotrophic Lateral Sclerosis. SonoCloud-4 (SC4) device - Amyotrophic Lateral Sclerosis, Charcot Disease | Phase 1 / Phase 2 | Recruiting | Assistance Publique - Hôpitaux de Paris | 23 | 2026-07-15 | 2026-08-10 |
| MSC-Exosome Therapy for Frontotemporal Dementia Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes （Low-Dose）, Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes （High-Dose）, Placebo Comparator (0.9% NaCl) - Frontotemporal Dementia | Phase 1 / Phase 2 | Not yet recruiting | Ruijin Hospital | 33 | 2026-07-01 | 2026-07-13 |
| AV-1980R (Tau Vaccine) in Preclinical Alzheimer's Disease (TAURUS-1980) AV-1980R 20 µg, AV-1980R 60 µg, AV-1980R 180 µg - Alzheimer Disease, Preclinical Alzheimer's Disease | Phase 1 | Recruiting | Institute for Molecular Medicine | 48 | 2026-06-23 | 2026-08-21 |
| TP04HN106 in the Treatment of Patients With Amyotrophic Lateral Sclerosis TP04HN106, Saline - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Recruiting | Talengen Institute of Life Sciences, Shenzhen, P.R. China. | 60 | 2026-06-04 | 2026-10-02 |
| A Substudy of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis LY4256984 - Amyotrophic Lateral Sclerosis | Phase 1 | Recruiting | Eli Lilly and Company | 32 | 2026-05-14 | 2026-09-22 |
| A Master Protocol (OLMP): A Study of LY4256984 in Participants With Amyotrophic Lateral Sclerosis (ALS) LY4256984 - Amyotrophic Lateral Sclerosis | Phase 1 | Recruiting | Eli Lilly and Company | 32 | 2026-05-14 | 2026-09-22 |
| A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis LTX-002, Placebo (aCSF) - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Recruiting | Leal Therapeutics, Inc | 56 | 2026-04-29 | 2026-08-18 |
| A Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of PLL001 in ALS Patients PLL001 or placebo daily subcutaneous injections - Amyotrophic Lateral Sclerosis, ALS | Phase 1 / Phase 2 | Not yet recruiting | PLL TX AUSTRALIA PTY LTD | 153 | 2026-04-08 | 2026-03-19 |
| Safety and Efficacy of Intrathecal NTF001 Injection in ALS NTF001 Injection - ALS - Amyotrophic Lateral Sclerosis, ALS (Amyotrophic Lateral Sclerosis) | Phase 1 | Active not recruiting | Ruijin Hospital | 12 | 2026-04-01 | 2026-06-01 |
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Amyotrophic Lateral Sclerosis (172) · Frontotemporal Dementia (21) · Motor Neuron Disease (19) · Neurodegenerative Diseases (8) · Alzheimer Disease (7) · Dementia (6) · Neuromuscular Diseases (5) · Pick Disease of the Brain (5) · Parkinson Disease (4) · Primary Progressive Nonfluent Aphasia (4) · Aphasia, Primary Progressive (3) · Frontotemporal Dementia With Motor Neuron Disease (3) · Amyotrophic lateral sclerosis 1 (2) · Central Nervous System Diseases (2) · Cognitive Dysfunction (2)
Metabolic Diseases (194) · Nutritional and Metabolic Diseases (194) · Proteostasis Deficiencies (194) · Central Nervous System Diseases (192) · Nervous System Diseases (192) · Neurodegenerative Diseases (186) · Spinal Cord Diseases (172) · Neuromuscular Diseases (167) · Motor Neuron Disease (153) · Brain Diseases (29) · Mental Disorders (26) · Neurocognitive Disorders (26) · Frontotemporal Lobar Degeneration (22) · Dementia (20)
Dexpramipexole (4) · Copper (3) · Riluzole (3) · AADvac1 (2) · Injections, Spinal (2) · Laminectomy (2) · Rabeprazole (2) · Sodium Chloride (2) · copper (II) diacetyl-di(N(4)-methylthiosemicarbazone) (2) · 2-(4'-(methylamino)phenyl)-6-hydroxybenzothiazole (1) · 4-phenylbutyric acid (1) · 7-(6-fluoropyridin-3-yl)-5H-pyrido(4,3-b)indole (1) · Abatacept (1) · Acamprosate (1) · Acetates (1)
n-Lorem Foundation (7) · Biogen (5) · Knopp Biosciences (5) · Eli Lilly and Company (4) · Newron Sweden AB (4) · Royan Institute (4) · Sanofi (4) · Hanyang University Seoul Hospital (3) · Mayo Clinic (3) · National Institute of Neurological Disorders and Stroke (NINDS) (3) · Phoenix Neurological Associates, LTD (3) · Amylyx Pharmaceuticals Inc. (2) · Assistance Publique - Hôpitaux de Paris (2) · Axon Neuroscience SE (2) · Bennett, James P., Jr., M.D., Ph.D. (2)
United States (94) · Canada (25) · Netherlands (25) · Belgium (14) · United Kingdom (14) · China (11) · Germany (11) · Spain (11) · Australia (10) · South Korea (9) · France (7) · Italy (7) · Poland (6) · Sweden (6) · Iran (5)
Hamid Gourabi, PhD (4) · Nasser Aghdami, MD,PhD (4) · Dominic Rowe, MD (3) · Todd D Levine, MD (3) · Diego Cadavid, MD (2) · Ken Nedd, M.D. (2) · Richard Bedlack, MD, PhD (2) · Seyed Masoud Nabavi, MD (2) · Wojciech Maksymowicz, MD, Prof. (2) · Adam Boxer, MD, PhD (1)
PMID 22415951, PMID 23463272, PMID 30124008, PMID 11050032, PMID 15084121, PMID 17978354, PMID 18270879, PMID 22415942, PMID 22565043, PMID 23300829, PMID 23541756, PMID 24820540, PMID 24841795, PMID 25324884, PMID 25478017, PMID 25503617, PMID 25889343, PMID 25934946, PMID 26746183, PMID 26751635, PMID 27515308, PMID 27581221, PMID 27938483, PMID 27955995, PMID 28674982, PMID 30048006, PMID 30158984, PMID 30355322, PMID 30531015, PMID 31272829, PMID 31629403, PMID 31797084, PMID 32265627, PMID 33031745, PMID 33141172, PMID 34016561, PMID 34558001, PMID 34678870, PMID 34716445, PMID 34719198, PMID 34815790, PMID 35154390, PMID 35260178, PMID 35260846, PMID 35649245, PMID 36008843, PMID 36064599, PMID 36288714, PMID 36467452, PMID 36543877
2023 (6), 2017 (4), 2025 (4), 2016 (1), 2019 (1), R01AG073482 (1), R01AG092949 (1), R21HD054697 (1)