AMX0035

DRUG13 trials

Sponsors

Amylyx Pharmaceuticals Inc., University of Washington

Conditions

ALSAlzheimer DiseaseAmyotrophic Lateral SclerosisAmyotrophic Lateral Sclerosis (ALS)Atypical ParkinsonismAutoimmune DiseasesCentral Nervous System DiseasesCombination Therapy

Phase 1

Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS

Amylyx Pharmaceuticals Inc.ALS

Start: 2021-08-05End: 2023-09-30Target: 14Updated: 2023-07-24

Phase 2

AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)

CompletedNCT03127514

Amylyx Pharmaceuticals Inc.Amyotrophic Lateral Sclerosis, Central Nervous System Diseases, Motor Neuron Disease +5

Start: 2017-06-22End: 2019-11-24Updated: 2024-05-16

Open Label Extension Study of AMX0035 in Patients With ALS

CompletedNCT03488524

Amylyx Pharmaceuticals Inc.ALS, Amyotrophic Lateral Sclerosis

Start: 2018-03-29End: 2021-03-01Updated: 2025-08-13

Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease

CompletedNCT03533257

Amylyx Pharmaceuticals Inc.Alzheimer Disease

Start: 2018-09-14End: 2020-11-06Updated: 2025-03-07

A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)

CompletedNCT04516096

Amylyx Pharmaceuticals Inc.Amyotrophic Lateral Sclerosis

Start: 2020-11-22End: 2023-01-31Updated: 2023-04-07

AMX0035 in Adult Patients With Wolfram Syndrome

Active, not recruitingNCT05676034

Amylyx Pharmaceuticals Inc.Wolfram Syndrome

Start: 2023-03-03End: 2028-05-31Updated: 2026-04-14

AMX0035 and Progressive Supranuclear Palsy

CompletedNCT06122662

Amylyx Pharmaceuticals Inc.Atypical Parkinsonism, Neurodegenerative Diseases, Progressive Supranuclear Palsy +1

Start: 2023-12-21End: 2025-10-21Updated: 2026-04-30

METabolic MODulation to Enhance Insulin Sensitivity and Mitochondrial Function in Type 1 Diabetes (MetMod-T1D)

RecruitingNCT07699380

University of WashingtonAutoimmune Diseases, Combination Therapy, Diabetes Melletus, Type 1 +6

Start: 2026-06-01End: 2029-12-01Target: 60Updated: 2026-07-13

Phase 3

Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment

Active, not recruitingNCT05021536

Amylyx Pharmaceuticals Inc.Amyotrophic Lateral Sclerosis

Start: 2021-10-28End: 2026-01-01Updated: 2024-08-14

Extension Study Evaluating The Safety And Tolerability of AMX0035

CompletedNCT05619783

Amylyx Pharmaceuticals Inc.Amyotrophic Lateral Sclerosis

Start: 2022-12-29End: 2024-10-30Updated: 2025-01-13

A Phase 2b/3 Study of Safety and Efficacy of AMX0035 in Progressive Supranuclear Palsy (ORION)

CompletedCTIS2023-505893-14-00

Amylyx Pharmaceuticals Inc.Progressive Supranuclear Palsy

Start: 2024-06-04End: 2025-08-27Target: 269Updated: 2025-06-27

A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients with Amyotrophic Lateral Sclerosis (ALS)

CompletedCTIS2023-508511-23-00

Amylyx Pharmaceuticals Inc.Amyotrophic Lateral Sclerosis (ALS)

Start: 2021-12-02End: 2025-01-13Target: 526Updated: 2024-08-04

Unknown Phase

An Intermediate Size Expanded Access Protocol of AMX0035 for ALS

Amylyx Pharmaceuticals Inc.Amyotrophic Lateral Sclerosis

Updated: 2022-10-18

Related Papers

Sodium Phenylbutyrate and Tauroursodeoxycholic Acid: A Story of Hope Turned to Disappointment in Amyotrophic Lateral Sclerosis Treatment.

2024-06-2312 citations

Effect of sodium phenylbutyrate and taurursodiol on plasma concentrations of neuroinflammatory biomarkers in amyotrophic lateral sclerosis: results from the CENTAUR trial.

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A phase 1 proof-of-concept study evaluating safety, tolerability, and biological marker responses with combination therapy of CTLA4-Ig and interleukin-2 in amyotrophic lateral sclerosis

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Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis

New England Journal of Medicine2020-09-02503 citations