• Clinical trials are designed with safety as a primary concern.
• Before a new drug, new dosage, or new combination of treatments for spinal muscular atrophy (SMA) can be studied in a clinical trial, its safety is first assessed in laboratory research.
• Safety is monitored in each phase of a clinical trial, including after a treatment is approved for public use.

This is an exciting time for spinal muscular atrophy research. Numerous clinical trials for SMA drugs are aimed at new, more effective treatments, dosages of existing treatments, and combination treatments. Although clinical studies might offer access to cutting-edge treatments, if you have SMA, you may wonder if clinical trials are really safe for you.

Every clinical trial has numerous precautions in place to ensure it’s as safe as possible. Government rules and regulations are designed to protect the health, safety, and privacy of clinical trial participants. If you’re considering participating in clinical trials studying new SMA treatments, it’s crucial to understand how safeguards are established and to take a look at any potential risks.

Safety Protocols: From Lab to Study

Research for new treatment approaches for SMA starts in labs with studies conducted on cells. Computer models are used to better understand particular aspects of neuromuscular disease. This type of research can take years before study teams begin testing a new drug therapy, an existing drug at a new dosage, or a new combination of drugs. Preclinical research, which takes place before any clinical studies with people, involves testing on cells and animals. Researchers want to see if the treatment works and if there are safety concerns about toxicity.

Study Protocols

In the United States, if a drug, dosage, or combination seems safe in lab research, a study team designs a clinical trial that will involve people. The design of a new clinical trial is a rigorous process that needs approval by the U.S. Food and Drug Administration (FDA) before volunteers can be recruited.

The FDA requires detailed information about a proposed clinical trial, including who is eligible to participate (inclusion criteria), how an investigational drug will be used, and how data will be analyzed for outcome measures, such as improvements in motor function. This process develops protocols — or study plans — to ensure the clinical trial is thorough and will protect the safety of participants.

Institutional Review Boards

Clinical study plans for drugs and medical devices are required to be reviewed by an independent committee called an institutional review board (IRB). IRBs examine the ethics and practices of clinical studies to protect the safety, rights, and well-being of people in clinical research.

Clinical Trial Phases

Clinical trials go through four phases. In each phase, the safety and effectiveness of a drug therapy is carefully monitored and then reviewed in follow-ups. A study can only continue to the next phase if the drug’s safety is proven in the current phase.

Read more about how clinical trials work for SMA.

Informed Consent

Before anyone participates in a clinical trial, they must provide informed consent. This is more than simply requiring a signature. A clinical trial research team is obligated to give potential participants thorough information and time to consider whether or not they want to participate.

With informed consent, you have a right to know how exactly the clinical trial will take place. This includes factors such as:

• The type of drug being tested and its purpose
• The way a drug will be administered
• The potential benefits and risks
• The amount of time involved in the clinical trial
• The ways your privacy will be protected
• Any costs that might be incurred

Before giving consent to participate, you should be given written information about the study, which you should review carefully to determine any questions you may have. In open label studies you will be told exactly which drug is under review. In other studies, the drug name may not be revealed. Enrollment in a clinical trial is completely voluntary, and you have no obligation to participate. You can also leave a clinical trial at any time, for any reason.

Control Groups, Placebos, and Risks

Virtually every drug has a risk of side effects, and participating in a clinical trial for a new therapy carries some risk. If you participate in an SMA clinical trial, you will be carefully monitored for any side effects. If the researchers discover any sign of an unsafe side effect, risk is evaluated and the trial may be stopped. Likewise, if at any time a participant feels discomfort and no longer wants to participate, they can leave the study.

Randomized Controlled Double-Blind Studies

Although there is no guarantee a new drug therapy, new combination of drugs, or new dosage in a clinical trial will work for you, there is a chance that the treatment under study will provide tangible benefits. Many people are motivated to participate in clinical research in order to try a new treatment.

However, some clinical trials for SMA drugs are controlled double-blind studies. In this type of study, participants in the control group may be given a current, existing standard of treatment or a placebo (sugar pill) instead of the new treatment. This helps researchers measure different outcomes between those taking a new drug or dosage and those who are not. “Double-blind” means that neither the researchers nor the participants know who is taking the new treatment. This helps control bias in evaluating results.

With controlled double-blind studies, it’s important to ask whether there’s a chance you may not receive the new treatment. In addition, a clinical trial may require participants to discontinue any current treatments they are using. With this in mind, you should talk to your doctor in detail about any concerns with stopping or switching treatment for a clinical study.

If you are considering participating in a clinical trial or would like to know more about eligibility for clinical trials, talk to your health care team.

Read 5 Reasons To Consider Participating in SMA Clinical Trials.

Talk With Others Who Understand

On mySMAteam, the social network for people with spinal muscular atrophy and their loved ones, more than 1,800 members come together to ask questions, give advice, and share their stories with others who understand life with SMA.

Do you still have questions about the safety of clinical trials? Share your questions of experiences with clinical trials in the comments below, or start a conversation by posting on your Activities page.