Frequently Asked Questions and Answers
ABOUT CLINICAL trials
What is a clinical trial?
A clinical trial is a research study required for all new drugs to help us understand whether an investigational medicine is safe and effective for humans.
There are usually several phases of clinical trials; each designed to provide more information on the effects of an investigational medicine. When a trial is in an early phase (often called Phase 1), the clinical trial may involve very few participants or healthy volunteers. As more is learned about the investigational medicine, the size of its trials will typically increase. Later-phase clinical trials (often called Phase 2 and 3) may involve hundreds or thousands of participants.
All clinical trials must follow a strict set of rules that are outlined in the trial's protocol. These rules set specific requirements on which types of participants can be included in the trial and the procedures that must be followed. Because of these rules, it is important that participants who participate carefully adhere to the visit schedule and the instructions of the personnel who are conducting the trials.
WHY ARE CLINICAL TRIALS CONDUCTED?
Clinical trials are conducted to answer research questions about new drugs and to learn if the investigational medicine is safe and effective for humans. The main questions the researchers want to answer are:
- Does this treatment work?
- Does the treatment have side effects?
- Do the benefits of the treatment outweigh the side effects and risks?
- Which participants are most likely to find this treatment helpful?
WHY SHOULD I PARTICIPATE IN A CLINICAL TRIAL?
Participating in a clinical trial is a voluntary opportunity so it is up to you to decide if you would like to be involved.
Many people with serious diseases participate in clinical trials for these reasons:
- They want access to a new type of treatment, even if it is not guaranteed to be as good as or better than available treatments;
- They want to contribute to the development of a new treatment for others with a similar disease.
WHAT IS A PLACEBO?
Many clinical trials use a “placebo” which is an inactive substance that matches the look of the experimental treatment. A placebo is used so participants and doctors will not know which patient received the real experimental treatment. This helps researchers see if the experimental treatment is as good as or better than the placebo. Participants will always be told if they are being asked to participate in a trial with a placebo so the patient can decide if they want to take the chance of not getting the experimental medicine.
WHAT IS INVOLVED IN FORTITUDE-ALS?
FORTITUDE-ALS is enrolling participants with amyotrophic lateral sclerosis (ALS). If you have been diagnosed with ALS within the last two years, you may be eligible to enter the trial. Prior to enrolling in FORTITUDE-ALS, you will need to understand what is involved in the trial by talking to the trial staff and reading the Informed Consent. The Informed Consent outlines the known safety information of this investigational medicine and what is involved in the trial. You will visit the clinical trial site for certain tests to make sure you are eligible to be included. Your doctor and trial team should be available to answer any questions that you might have as you go through the process.
WHAT IS THIS INVESTIGATIONAL MEDICINE?
CK-2127107 is an investigational medicine that is being evaluated for its potential to slow the decline of skeletal muscle function in diseases and conditions associated with muscle weakness or fatigue such as ALS. CK-2127107 binds to and activates a protein found in skeletal muscle that may therefore lead to increased muscle force and increased time to muscle fatigue. Based on how it acts on muscles, CK-2127107 may have an effect in diseases associated with reduced nerve input to the muscle like ALS.
AM I GUARANTEED TO RECEIVE THE INVESTIGATIONAL MEDICINE IF I PARTICIPATE IN FORTITUDE-ALS?
FORTITUDE-ALS is a randomized, placebo-controlled clinical trial. This means that some participants will receive the investigational medicine, CK-2127107, while others will receive a placebo (which does not contain the active drug).
The assignment of participants to each group is done at random, like flipping a coin, but in a way that three times more participants will receive investigational medicine than placebo (3:1 ratio). Neither the patient nor the trial doctor will know the treatment assignment. Participants will be assigned at random to receive either 150 mg, 300 mg or 450 mg of CK-2127107 or placebo.
WHAT SIDE EFFECTS SHOULD I EXPECT?
The Informed Consent will provide details on the safety information on the investigational medicine. Please direct any additional questions to the study doctor.
DO I HAVE TO PAY TO PARTICIPATE IN A CLINICAL TRIAL?
No. The investigational medicine and associated tests, procedures and visits to the site are provided free of charge to clinical trial participants.
WILL I BE COMPENSATED FOR MY TIME PARTICIPATING IN THIS TRIAL?
There may be compensation available to offset travel expenses resulting from trial participation.
HOW DO I GET STARTED?
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