Phase 2

RT001 in Amyotrophic Lateral Sclerosis

Industry trial

In this phase 2 trial, we will study the safety and efficacy of the compound RT001 in people living with ALS. This clinical trial is sponsored by Retrotope Inc.

RT001 in people with ALS

RT001 is made from slightly modified linoleic acid. Linoleic acid is typically part of a normal human diet and is found in most oils. RT001 can protect cells from lipid peroxidation – a process that is believed to cause damage in several neurodegenerative diseases, including ALS. RT001 has previously been shown to be safe when administered to humans. However, RT001 can currently only be prescribed in research settings.

Study overview

This phase 2 trial is sponsored by Retrotope Inc and is a randomised, double-blind, placebo-controlled study. This means that the safety and treatment effects are studied by comparing a group of ALS patients that receive RT001 against a group of patients receiving a placebo drug. A placebo drug looks exactly the same as the RT001, but does not contain any active medicine (a mock treatment). Both treatments need to be ingested orally during breakfast, lunch and dinner. Participants will be randomly assigned to receive RT001 or placebo. Both the participant and the research team do not know who receives a placebo treatment or RT001.

The entire study duration is 28 weeks. During this period you are expected to visit the hospital at least 4 times for examinations. Each visit typically takes around 3 to 4 hours.

Can I participate in this study?

*** Please note that enrolment for this study has now been completed ***

The study intends to recruit 40 participants living with ALS from different countries.

Key eligibility criteria for people with ALS to participate, include:

  • Male or female aged between 20 years to 75 years
  • The onset of ALS is less than 3 years ago
  • Respiratory function (SVC) is at least 70%, without ventilatory support
  • Concurrent treatment with other experimental therapies is not allowed
  • If participants have a feeding tube or if the need for a feeding tube is anticipated within the first 24 weeks after starting the study, they cannot participate

More information

Additional information about the trial can be found on and

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