What You Need to Know Before Enrolling in a Research Study

The Triangle area’s major hospitals and universities conduct research studies on a global scale, which may be extremely helpful to local families seeking cutting-edge care for their children. But parents who are thinking about enrolling a child in experimental studies or therapies must do some research of their own.

We share the experiences of local families whose children participated in different types of studies as well as advice from experts to help when evaluating a study.

Aiden’s Story: A developmental study

Julie Waddle of Raleigh enrolled her son, Aiden, in an autism study through UNC-Chapel Hill after filling out a questionnaire and being contacted by researchers. The Early Development Project at the UNC Department of Allied Health Science is a developmental study that focuses on early identification and intervention in autism by screening 12-month-old children to determine whether they are at risk for autism or other developmental disabilities. A developmental study is designed to gather information. This study also compares community services intervention to parent-mediated intervention.

Aiden was identified with a speech delay and, beginning at 18 months, participated in assessments every six months. Aiden did not receive services directly as part of the study, but the family was provided a list of services in Wake County.

Having developmental assessments already completed made it easier for Aiden to receive timely assistance, Waddell says. In addition, an occupational therapist working with Aiden helped identify a lactose intolerance. Thanks to these interventions, Aiden will be mainstreamed this year in kindergarten.

“We were so lucky to have gone into this because we have learned so much that will help Aiden and help him become productive and … have good interpersonal relationships,” Waddell says.

As Waddell’s experiences show, parents considering developmental study participation need to be prepared for the possibility of an unexpected diagnosis. Christene Tashjian, project coordinator for the Early Development Project, cautions that “some of the information parents might hear about their child could be uncomfortable if they are not prepared to hear it.”

One tip Waddell has for parents: You should always have access to a child during evaluations or procedures, as through a one-way window, for example.

Adam’s Story: A clinical trial

Adam Willits, 17, of Apex is allergic to peanuts and tree nuts. When Adam was 11, he joined a clinical trial at Duke University Medical Center to determine whether low-dosage exposure to peanuts could reduce sensitivity of allergic individuals.

A clinical trial is a controlled study of a drug or a new type of medical device. Trials usually have some degree of risk and are subject to a review board.

The Duke peanut allergy study required medical appointments every two weeks as well as regular administration of drops and a daily log. As Adam got older, he was not as vigilant about his doses. Debra Willits, Adam’s mother, notes that small cuts from braces may have made dosing more painful. After talking with staff, he decided to withdraw from the trial, especially since he would soon be attending college.

Adam’s experience points to a potential downside to participating in clinical trials: some require a significant commitment over a long period of time. But Willits says the study did reduce her son’s sensitivity to peanuts, although the lasting impact remains unknown.

Other, secondary benefits have been positive as well: Adam learned how to use an Epipen (an auto-injecting device to prevent anaphylactic shock) and, as he entered his teen years, had frank discussions with staff about the potential dangers of sharing beverages with or kissing someone who has consumed nuts. He also learned more about managing his asthma.

Willits believes Adam’s participation in the study was worthwhile, particularly since it has fueled her son’s interest in science and may open the door to a future study.

Megan’s Story: An elective surgical procedure

Karen Hogan’s daughter, Megan, was born with cerebral palsy. She was a candidate for selective dorsal rhizotomy (SDR), an elective procedure that involves cutting some of the sensory nerves from the muscles to the spinal cord, with the goal of improving mobility by relieving muscle spasticity.

Medical procedures, either experimental or elective, can sometimes be part of a clinical trial and entail inherent risks as well as potential benefits. For parents of children with significant medical challenges, careful consideration of both is essential to make an informed decision.

Hogan, who lives in Apex, did her homework regarding the surgery. She researched a surgeon at St. Louis Children’s Hospital who has performed more than 2,000 SDR procedures. She followed some of the surgical patients through Facebook for several years to learn more about outcomes. After years of intense research, the family decided to pursue this option.

“We looked into this [surgery] when Megan was 4,” Hogan says. “There is some controversy about this procedure. It took us this long, until the age of 12, to say, ‘We are doing this.’”

It was a difficult decision, especially when people warn that your daughter may never walk again, Hogan says. The biggest help, she adds, was talking with other parents who have been through the process.

While it will take more time before the full benefits of the procedure are known, Megan is doing well and says her legs feel lighter. 

Evaluating a study

Parents who are thinking about involving their child in any kind of research study should consider a number of factors when making their decision. A well-designed study should follow specific guidelines and clearly state objectives. Detailed consent forms should be part of the process. Consider who is supporting the study and what kind of time commitment is involved, as some studies continue for years.

Parents should also ask how study results will be communicated. “As a parent whose child joins the study, you want to ask, ‘What kind of access do we have to the research findings, and do we control who receives them?’” says Arlene Davis, a bioethicist and director of the Clinical Ethics Consult Services at UNC Hospitals.

Finally, consider your child’s privacy as it pertains to the results of the study. Advancements in genetics have made it crucial to understand not only the type of information you may learn but also whether that information can be used for other purposes. Researchers may collect blood or tissue samples from your child, and there are ethical considerations surrounding the future use of such materials. Privacy can be of particular importance with children because of the length of time this information could be used, Davis says. Also important is whether findings will be part of permanent medical records.

Research studies can be of great benefit to children, but it is important to find the right fit for your child’s needs. Gather information, take a deep breath, and follow your gut to determine which study is best for your child. 

Sidebar: Questions to Consider About Research Studies

Who stands to benefit from the study?
What are the goals of the study?
Are the consent forms thorough?
Will my child be videotaped or photographed? Who will see that information?
How will evaluations and procedures be carried out?
What are the potential risks and benefits?
Will my child receive information about the risks and benefits? (Children older than 7 are sometimes provided information about a study.)

For a detailed list of questions to ask regarding a study, see nhlbi.nih.gov/childrenandclinicalstudies (search Good Questions to Ask).

Carol McGarrahan is a Triangle mother and freelance writer who frequently covers health, science and parenting topics.

Categories: Exceptional Child