AMX0035 and Progressive Supranuclear Palsy
Research type
Research Study
Full title
A Phase 3 Study of Safety and Efficacy of AMX0035 in Progressive Supranuclear Palsy (ORION).
IRAS ID
1008898
Contact name
Allison Lusis
Contact email
Sponsor organisation
Amylyx Pharmaceuticals, Inc
Clinicaltrials.gov Identifier
Research summary
This clinical research study is designed by the company Amylyx Pharmaceuticals, Inc. Amylyx will support the study by covering the costs of the study and by providing the investigational drug, AMX0035.
PSP is a nervous system disease that affects nerve cells in the brain and spinal cord. PSP worsens over time, causing loss of muscle control and neurological issues. To date, there are no approved treatments for PSP specifically, but some people in the early stage of the disease may benefit from taking medications used to treat Parkinson’s disease.
Approximately 600 participants living with PSP from different study centres in 14 countries are expected to participate in this study.
The main objective of this study is to compare AMX0035 and a product that contains no medicine (placebo) and their ability to slow down the symptoms of PSP.
The main endpoint compares the change over time between AMX0035 and placebo on the development of symptoms of PSP.
This study will be conducted in 2 parts:
Part 1: Participants will be randomly assigned to receive either AMX0035 or a placebo (which will look and taste like AMX0035 but does not contain AMX0035) for 1 year. Three (3) of every 5 participants will receive AMX0035 and 2 of every 5 participants will receive placebo. Participants, the study doctor, and the Sponsor will not know which study drug is assigned during Part 1.
Part 2: Participants may choose to enter Part 2 only if they complete Part 1. Participants in Part 2 will receive AMX0035 for up to 1 year. There is no placebo in Part 2. Participation in Part 2 is not required. Part 2 is an “Open Label Extension.”
Part 1 and Part 2 will assess participant safety. This will include monitoring temperature, heart rate, and blood pressure (vital signs), assessment of the heart rhythm (electrocardiogram; ECG), drawing blood, physical examination, and documentation of any effects that a participant might experience.REC name
Seasonal REC
REC reference
23/LO/1010
Date of REC Opinion
26 Feb 2024
REC opinion
Further Information Unfavourable Opinion