Complete this brief questionnaire


Are you interested in participating in a Phase II clinical research study of an investigational medication to determine if the treatment may slow down the progression of Amyotrophic Lateral Sclerosis (ALS)?

What is the purpose of the study?

This study will compare an investigational medication (oral tablet) to a placebo to determine if there is a change in disease progression, quality of life and to assess the safety of the investigational medication.    

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What is the length of the study? 

  • Screening (up to 4 weeks) to determine eligibility 
  • If eligible, a 24-week double-blind treatment period (7 visits and 2 phone calls over 24 weeks / 6 months) 
  • Following the double-blind period, you will then enter an open label treatment period (8 visits over 1 ½ years) 
  • Follow-up (1 study visit) 
  • Total length of study is up to 2 years  

What is expected if I qualify and decide to participate?

You will be expected to:

Attend scheduled study visits.

Adhere with daily investigational medication intake: You will be randomly assigned to receive either the investigational medication (2 out of 3 chance) or a placebo (1 out of 3 chance) that you will take by mouth twice daily (morning and evening around the same time each day), in addition to your current treatment for ALS. 

Complete the tests or procedures to be performed as planned including assessments for the safety and tolerability of the investigational medication, pulmonary function tests, muscle strength, questionnaires related to your quality of life, as well as blood and urine sampling

Travel to the study site.  Patient reimbursement for travel, which may include parking, airfare, lodging and meals, may be provided for you and your caregiver.

Can I participate?

The study will enroll 261 patients globally in multiple countries with 80 patients in the US.

Study participants will have a 2 in 3 chance of receiving investigational medication (1 in 3 chance of receiving placebo).  All participants will receive the investigational medication during the open label.

Terms to know:

Double Blind

Neither the patient nor the study doctor will know which treatment the patient is receiving.

Open Label

Both the patient and study doctor will know the treatment assignment, which is the investigational medication.


A placebo is an inactive substance or treatment that looks the same and is given in the way as the investigational medication that is being studied. 


What is an investigational medication?

It is what is being studied to see if patient symptoms improve while taking it.  The drug is not yet approved for general use by regulatory authorities.

What is a clinical research study? 

Clinical research studies are used to show if/how an investigational medication works and to find out if it is safe. They can also be referred to as clinical research trials.  Clinical studies are run by qualified healthcare professionals. The doctors and other healthcare providers are responsible for the study-related care of the people that participate (or enroll) in these studies. Clinical research studies are a mandatory part of the process that leads to health authority review and approval which is required before a drug can be marketed.

Independent committees (called Institutional Review Boards or IRBs) are made up of medical and nonmedical people who also watch over clinical research studies to make sure that the people who enroll are properly and adequately informed and consent to study participation.

What is a Phase II trial?

Larger groups of patients (up to several hundred people) participate in Phase 2 clinical trials. At this stage, researchers study how the investigational drug is tolerated in patients. 

The purpose of Phase 2 trials is to study:

  • Any short-term side effects,
  • What the optimal dose of the investigation drug might be,
  • How many times it should be given, over what period of time. 

    Who can participate in a clinical trial?

    Every clinical trial has a set of criteria that trial participants must meet in order to participate, including agreeing to signing an informed consent form and HIPAA authorization.

    What type of study-related medical care is provided during the study?

    During your in-person research visits, medical care will take place (at no cost to the patient) including but not limited to, physical examination (including check of blood pressure, heart rate and body temperature), blood and urine sampling, study medication dispensation, ALS related tests, questionnaires related to your quality of life, EKG (electrocardiogram).  A full schedule of participation activity will be shared with you at the beginning of the study.

    I speak Spanish, not English. Can I participate in the study?

    Yes, if you let the doctor know, the study site can provide you with translated materials and may be able to provide an interpreter.

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    V1.0 22March2022

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