ORARIALS-01 clinical trial

Phase 3 study investigating the effect of oral arimoclomol on disease progression and survival

ORARIALS-01 is a multicentre, randomised, double-blind, placebo-controlled clinical trial designed to test the efficacy and safety of oral arimoclomol as a potential new therapy for ALS. Arimoclomol is thought to work by amplifying the heat shock response (also known as stress response) to address protein accumulation and dysfunction occurring in people with ALS. 231 patients will be enrolled throughout North America and Europe to test the safety of arimoclomol and study whether it is effective in slowing disease progression. An open label extension trial (ORARIALS-02) is planned for all participants who complete the 76-week treatment period of ORARIALS-01 or who meet pre-defined markers of disease progression.

What does taking part involve?

If you are found eligible to participate, you will be randomly assigned to one of three treatment groups – two groups will receive arimoclomol and one will receive placebo. Riluzole will be given as background treatment to all participants. You will be asked to take treatment (arimoclomol or placebo) in capsule form three times each day at morning, noon and evening. Capsules may be swallowed whole or opened and the contents sprinkled into a liquid or soft food.

Throughout the 18-month period you will be asked to visit the clinic every 2-3 months for routine checks of your symptoms. At some of these visits you will be asked to provide samples of blood, urine and cerebrospinal fluid. A remote visit (telephone call) will take place between the clinic visits. You will also be asked to maintain a diary of your experiences. A mobile app is available to keep you informed of the study schedule and what you need to do before, during and after each visit.

You will need to be accompanied by a caregiver for all clinic visits. If you become unable to travel due to disease progression, you may be assessed in your home.

Study location

London (University College London)

Who can take part?

People living with ALS who:
• are ≥18 years and experienced first signs of weakness within the last 12-18 months, and
• are not participating in another clinical trial or using investigational products, and
• are not pregnant, and
• are not using edaravone.

How do I take part?

If you think you might be eligible, please speak to your neurologist.

More information

For more information about the UK trial, please contact the study co-ordinator Anna Belin (a.bellin@ucl.ac.uk).

Find this trial at clinicaltrials.gov (NCT03491462) or www.clinicaltrialsregister.eu (EudraCT 2018-000137-13).


This trial is funded by Orphazyme A/S. Your participation should not be of financial cost to you, please ask the study doctor about how your expenses will be covered.

Expiry date: N/A

For further information and support contact our MND Connect helpline

mndconnect 0808 802 6262