DMD affects about one in 5,000 males at birth, It is the most common type of muscular dystrophy.
Researchers are actively pursuing several exciting strategies in DMD, such as gene therapy, exon skipping, stop codon read-through and gene repair. Human clinical trials are under way for some of these strategies.
The Studies are evaluating an investigational medication of Duchenne Muscular Dystrophy (DMD). These studies will help determine if the investigational medication is safe and effective at curing or slowing the progression of the condition.
A clinical trial is carefully designed to closely monitor patients’ progress as they go through treatment with an investigational drug, product, device or method of treatment that has not been approved by the FDA. Clinical trials help find new ways to treat, prevent and diagnose diseases. Today, all medications prescribed by a doctor must first be tested in clinical trials. Study participants receive close medical supervision and provide valuable feedback on their experiences.
All clinical trials in the United States are reviewed by the FDA and other governing bodies called Institutional Review Boards, whose job is to make sure participants’ rights are fully protected and that participants are not exposed to any unnecessary risks.
Participating in a clinical trial is voluntary. Participants may choose to discontinue participation at any time. Before you enroll in a trial, a doctor or nurse will clearly explain the study procedures and requirements. This is called informed consent. Doctors are not allowed to enroll patients in clinical trials without first ensuring they understand what their involvement in the trial means. Once you enroll, you may end your participation at any time.
When participating in a clinical trial, participants receive study-related medications and medical monitoring at no cost. Most studies reimburse for travel and logistical considerations. It is important to find out the insurance requirements of any study you want to participate in.
There are a number of reasons you or your doctor may want to consider participation in a clinical trial, including:
No. The participant will receive all study-related care, including the investigational medication, at no cost. Insurance is not needed to participate in the study and the participant’s insurance company will not be notified.
Reimbursement for travel expenses may be provided. If pre-qualified, the study team will provide more information.
No. The participant’s doctor does not have to give his or her permission to take part in the study. However, either the participant or the study doctor, with the participant’s permission, may contact the personal doctor to discuss the participant’s participation and progress throughout the study.
An investigational medication is a medication that has not been approved by the appropriate regulatory or health authorities, such as the U.S. Food and Drug Administration (FDA), for prescription use. Investigational medications are yet to be proven safe and effective.
Studies will only use placebos when absolutely necessary. In 99% of clinical trials, if a placebo is used, it is given with standard treatment. You will receive either the best known Duchenne Muscular Dystrophy (DMD) treatment or a new and possibly more effective treatment. Many trials even combine the standard therapy with a new treatment.
All procedures that address a life-threatening illness pose some risk. Your physician and Duchenne Muscular Dystrophy (DMD) treatment team might help you decide if a clinical trial is the best treatment option for you. In muscular dystrophy diagnosis, clinical trials are often viewed as the best treatment option. Keep in mind that all known medications have once gone through the clinical trials process - even aspirin.
Yes, participation is voluntary. The participant may decide to stop or suspend participating at any time. Any participant who decides to stop early should give prior notice to the study team. The study team will ask the participant to return to the study office at least once for a follow-up test to check the participant’s health once the investigational medication has been stopped.
Not at all! Participating in a clinical trial is always voluntary. You will go through a detailed informed consent process and you may always choose to stop participating. Most people like being on a clinical trial because they feel they are being monitored even more closely than before.
To help ensure the clinical trial is ethical and that participants’ rights are protected, Institutional Review Boards (IRB) or Ethics Committees (EC) review and approve study protocols.
The study team respects and protects every participant’s privacy. The study team will not share the participant’s information except when they are required by law. The study team will store the participant’s personal information with codes that do not identify the participant. The Informed Consent Form has more information about the participant’s privacy and you will need to sign it prior being accepted into the study.