796 completed, 357 recruiting, 233 unknown, 121 terminated. 470 Phase 2, 412 Not applicable, 291 Phase 1, 171 Phase 3, 38 Early Phase 1.
Follow Proteostasis Deficiencies: new trials by email Literature Review of Proteostasis Deficiencies trials Research Report on Proteostasis Deficiencies
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 Randomized Phase 3 Study to Evaluate the Efficacy and Safety of NXC-201 Compared With Daratumumab With Cyclophosphamide, Bortezomib and Dexamethasone (CyBorD) in Newly Diagnosed Systemic AL Amyloidosis NXC-201, Dara-CyBorD - Light Chain (AL) Amyloidosis | Phase 3 | Not yet recruiting | Nexcella Inc. | 260 | 2027-01-01 | 2026-07-16 |
| Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT) INTENT Neural Interface System - Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS) | Not applicable | Recruiting | Johns Hopkins University | 5 | 2027-01-01 | 2026-10-09 |
| 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 |
| Study to Evaluate Change in Disease Activity and Adverse Events of Subcutaneous Etentamig Compared With Daratumumab Plus Cyclophosphamide Plus Bortezomib Plus Dexamethasone (Dara-CyBorD) in Adults With Amyloid Light Chain (AL) Amyloidosis Etentamig, Daratumumab, Cyclophosphamide - Amyloid Light Chain (AL) Amyloidosis | Phase 3 | Not yet recruiting | AbbVie | 370 | 2026-11-29 | 2026-08-28 |
| Brain-Computer Interface Communication Device for People With ALS Cognixion ONE AR-BCI configuration, Cognixion ONE screen-based BCI configuration, Existing eye-gaze speech generating device - Amyotrophic Lateral Sclerosis | Not applicable | Recruiting | Cognixion | 30 | 2026-11-15 | 2026-10-16 |
| A Wearable Sensor Platform for Remote Monitoring of Individuals on the Frontotemporal Dementia Spectrum Frontotemporal Lobar Degeneration (FTLD), Frontotemporal Dementia (FTD) | Not yet recruiting | BioSensics | 60 | 2026-11-01 | 2026-10-13 | |
| Speech-Based Artificial Intelligence for Detection of Dementia in Danish Patients (DetectAI) Mini-mental State Examination, Addenbrooke's Cognitive Examination, Speech Task - Picture Description - Dementia (Diagnosis), Alzheimer Dementia (AD) | Not yet recruiting | Zealand University Hospital | 440 | 2026-11-01 | 2026-09-02 | |
| To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE) Acoramidis (AG10) - Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial | Phase 3 | Not yet recruiting | Eidos Therapeutics, a BridgeBio company | 250 | 2026-11-01 | 2026-08-11 |
| Phase 1a/b Study of GPRC5D CART Therapy in Relapsed/Refractory Multiple Myeloma and Light-chain Amyloidosis GPRC5D CART - Relapsed/Refractory Multiple Myeloma (RRMM), Light-Chain Amyloidosis | Phase 1 | Not yet recruiting | Hadassah Medical Organization | 160 | 2026-11-01 | 2026-10-20 |
| 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 Trial of ALN-6457 in Adult Participants With Symptomatic Prion Disease (PrD) ALN-6457 - Prion Disease | Phase 1 / Phase 2 | Not yet recruiting | Regeneron Pharmaceuticals | 60 | 2026-10-23 | 2026-09-22 |
| A Study to Evaluate the Safety, Tolerability and Pharmacodynamics of NIO752 in Primary Tauopathies NIO752, Placebo - Progressive Supranuclear Palsy, Corticobasal Degeneration | Phase 2 | Not yet recruiting | Novartis Pharmaceuticals | 48 | 2026-10-23 | 2026-10-09 |
| Evaluation of the Feasibility and Efficacy of a Chronic Brain-computer Interface for Speech Rehabilitation in Patients With Locked-in-syndrome (LIS) Chronic speech BCI - Locked in Syndrome, ALS (Amyotrophic Lateral Sclerosis) | Not applicable | Not yet recruiting | University Hospital, Grenoble | 3 | 2026-10-10 | 2026-07-13 |
| 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 | 2026-10-19 |
| iBCI Optimization for Veterans With Paralysis Mobile neural decoding platform (mobile iBCI) - Spinal Cord Injury, Amyotrophic Lateral Sclerosis | Not applicable | Not yet recruiting | VA Office of Research and Development | 2 | 2026-10-02 | 2026-07-27 |
| 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 | 2026-10-01 | 2026-09-08 |
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Amyotrophic Lateral Sclerosis (972) · Amyloidosis (253) · Immunoglobulin Light-chain Amyloidosis (236) · Frontotemporal Dementia (205) · Amyloid Neuropathies, Familial (186) · Alzheimer Disease (166) · Motor Neuron Disease (144) · Multiple Myeloma (125) · Parkinson Disease (91) · Neurodegenerative Diseases (80) · Dementia (75) · Neoplasms, Plasma Cell (68) · Leukemia, Lymphocytic, Chronic, B-Cell (66) · Lymphoma, Non-Hodgkin (63) · Lymphoma, Follicular (62)
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Dexamethasone (70) · Bortezomib (35) · Cyclophosphamide (35) · Melphalan (34) · Calcium Dobesilate (24) · Riluzole (24) · daratumumab (23) · Blood Specimen Collection (20) · Filgrastim (19) · Lenalidomide (16) · Peripheral Blood Stem Cell Transplantation (16) · pomalidomide (13) · Spinal Puncture (11) · Thalidomide (11) · Memantine (10)
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PMID 31353964, PMID 40226119, PMID 25803389, PMID 32107748, PMID 35790423, PMID 36008843, PMID 38065307, PMID 42389794, PMID 16434671, PMID 17980667, PMID 19251627, PMID 21732836, PMID 22415951, PMID 22529995, PMID 23300829, PMID 23463272, PMID 23952636, PMID 24067398, PMID 25982504, PMID 26751635, PMID 27494299, PMID 28550039, PMID 29115008, PMID 29287521, PMID 29972753, PMID 29972757, PMID 30124008, PMID 30558645, PMID 30814647, PMID 31566651, PMID 32499241, PMID 32524297, PMID 32896088, PMID 33070419, PMID 33441454, PMID 33825853, PMID 33827960, PMID 34349004, PMID 34355354, PMID 34396169, PMID 34433481, PMID 34536404, PMID 34923848, PMID 35492244, PMID 35532908, PMID 35577511, PMID 35799473, PMID 36715498, PMID 37254449, PMID 37366170
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