NCT06122662

AMX0035 and Progressive Supranuclear Palsy

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

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 110 (estimated)
• Sponsor: Amylyx Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 40 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

A35-009 (ORION) is a Phase 2b/3 trial to evaluate the efficacy and safety of AMX0035 in participants with Progressive Supranuclear Palsy (PSP), consisting of randomized, double blind placebo controlled phases, followed by an optional open-label extension phase.

Detailed description

AMX0035 is a fixed dose combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the endoplasmic reticulum (ER) and mitochondria. This clinical trial is designed to demonstrate that AMX0035 is safe and tolerable, and to assess its effect on disease progression as measured by the Progressive Supranuclear Palsy (PSP) Rating Scale (PSPRS) over a 52-week double-blind phase. The Phase 2b and Phase 3 study portions are planned to feature an identical design: a randomized, double-blind, placebo-controlled phase that is followed by an optional open-label extension (OLE) phase. The phase 3 portion of ORION may be initiated based on results of the phase 2b Interim Analysis and/or the Primary Analysis and the totality of data from the Phase 2b study portion.

Conditions

• Progressive Supranuclear Palsy
• PSP
• Neurodegenerative Diseases
• Atypical Parkinsonism

Interventions

Type	Name	Description
DRUG	AMX0035	Proprietary formulation of sodium phenylbutyrate and taurursodiol
OTHER	Placebo	Matching Placebo Comparator

Timeline

• Start date: 2023-12-21
• Primary completion: 2026-04-01
• Completion: 2029-11-30
• First posted: 2023-11-08
• Last updated: 2024-12-20

Locations

62 sites across 5 countries: United States, France, Germany, Italy, Spain

Regulatory

• FDA-regulated drug study