Clinical trials play a key role in the discovery and development of new treatments for neuromuscular diseases.
At MDA, we know how critically important clinical trials are to finding treatments and cures, which is why we’ve committed, by 2020, to doubling our research investment in clinical trials and drug development.
Clinical trials are research studies conducted to determine whether a medical strategy, treatment or device is safe and effective for use in humans. Trials are experiments in which novel drugs or treatments are tested in people, sometimes for the very first time. Clinical trials are very different from routine medical care (including prescribed use of FDA-approved drugs), because doctors and researchers don’t know exactly how the treatment or device will affect people.
Some clinical studies are not used to test a treatment or device but instead aim to observe patients in order to better understand a disease. These “observational” studies may be used to determine how to detect or diagnose a disease, or to optimize testing procedures for future trials.
Participating in a trial may help accelerate the development of new treatments, increase knowledge about a disease or even lead to a cure. In addition, trial participants often feel empowered as trials offer another way to fight back against disease. It is important to remember that some trials may provide participants with direct medical benefits, while others may not or may even result in harm to participants.
People of all ages, races, ethnicities and genders can participate. Each study has its own inclusion and exclusion criteria to determine who is an appropriate candidate.
Clinical trials are run at institutions and hospitals by a clinical trial team. Each trial is led by a principal investigator, often a medical doctor, and research teams often include doctors, nurses, study coordinators, social workers and other health care professionals.
As a potential participant, you have a right to receive a written description of the trial specifics and what will be expected of you. You will also have the right to ask questions of the study team until you are comfortable with your understanding of the study, and the right to take time to consider whether participation makes sense for you. You will have every opportunity to ask questions, even after you give consent to participate. Once the study begins, you have a right to withdraw from the study at any time, and the right to learn about new risks or findings as they emerge.
You are required to comply with the study protocol (visits, treatment, keeping notes, etc.). Tell study investigators about any possible side effects, as well as any hospital, ER and outpatient clinic visits. Do not conduct your own experiments, as you may invalidate study results. It’s also important to maintain confidentiality. Sharing impressions of study experiences can bias study investigators and participants and compromise the integrity of the study.
You may discuss any questions or concerns with the study coordinators or principal investigator. The study coordinators and investigators are obligated to maintain your confidentiality and the confidentiality of other participants.
You should not talk about whether you think you are receiving a placebo or drug. However, if you have a concern about the treatment you are receiving, you should always contact the study coordinator or principal investigator.
A placebo, or dummy pill, is an inactive version of the treatment being tested in a clinical trial. Placebos are designed to look exactly like the test treatment, but they lack the active chemical ingredient. Placebos are used to ensure that any measured benefit of the test treatment is due to the active chemical ingredient and not simply to the act of participating in the study.
Not every clinical trial uses a placebo group, but in some studies, a subset of participants will receive a placebo for all or for part of the study. Participants are assigned to either a treatment or placebo group, often by random chance, and are not able to influence their group assignment.
You can withdraw or decide not to participate at any time. If you withdraw from the study, it is very important to contact your study coordinator and inform them of your decision. If you were taking a study medication, the study team likely will request a final visit with you to ensure you are OK. Your health and well-being come first in research.
Some trials do bill insurance. In many cases, treatment received in connection with a clinical trial will be covered. This should be disclosed in the study consent form.
Some studies provide financial support for costs associated with traveling to the clinic or hospital during the trial. Ask the clinical trial team if these resources are available.
Many studies require genetic confirmation of a disease before a person can participate in a clinical trial. Genetic testing can pinpoint an individual’s disease-causing mutation, helping investigators ensure that all people enrolled in the study are similar.
Use this free tool from the National Society of Genetic Counselors to find a genetic counselor for you.
The MDA U.S. Neuromuscular Disease Registry, launched in 2013, is the first comprehensive registry for ALS, BMD, DMD and SMA that collects information from health professionals through MDA’s network of more than 150 Care Centers. This registry serves as a powerful tool to help improve quality of life and survival, facilitate research, and expedite clinical trials.
* Clinical trial information originally written for “Types of Clinical Trials” and “Trial Run: Is Participating in a Clinical Trial Right for Me?” in MDA’s Quest Magazine.
Getting involved in this study was the one thing we could do to make good use of our time. Even if this trial wasn’t going to help Zane, we wanted to make sure it helped other kids in the future.
Download this checklist of questions to ask yourself and your trial coordinator before you begin your clinical trial. As always, your local MDA Care Center staff is available to help you through this process.Download PDF Checklist
Clinical trials are conducted in phases, each designed to answer a specific research question.
Known as "exploratory studies", these trials test a small dose of a new drug in a few people to explore how the drug works and to ensure that it is safe.
In phase 1 “safety studies,” researchers focus on whether an experimental treatment is safe while determining how best to administer the treatment and in what dose.
These studies are designed to assess whether an experimental treatment works. The trial may involve healthy volunteers or people with a targeted condition, and some participants may be randomly assigned to different groups that receive a different dose or treatment protocol.
In this “confirmatory” phase, the number of participants is increased and the experimental drug is tested against a placebo in order to prove that the drug is both effective and better than any existing treatments.
Approved drugs are monitored over a period of time in phase 4 studies. Although these treatments are available for doctors to prescribe outside the phase 4 study, researchers continue to learn more about the treatment from participants in these trials.
In a natural history study, investigators follow a group of people over time who have, or are at risk of developing, a specific medical condition or disease. These studies collect information to determine how the medical condition or disease develops and progresses.
In an observational study, investigators assess health outcomes in groups of participants according to a protocol or research plan. Participants may receive interventions, which can include medical products or procedures as part of their routine medical care.
For questions or one-on-one support for your clinical trial journey, we’re here to help. Give us a call at 800-572-1717 or send us an email using the form below. Your local MDA Care Center will also be able to help.
Our trained specialists are here to provide one-on-one support for every part of your journey. Send a message below or call us at 800-572-1717. If you live outside the U.S., we may be able to connect you to muscular dystrophy groups in your area, but MDA services are only available in the U.S.