Targeted Clinical Drug Trials for Intellectual Disabilities: The Decision-Making Process Open Access

Reines, Victoria Lynne (2013)

Permanent URL: https://etd.library.emory.edu/concern/etds/g158bh485?locale=en
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Abstract

Background. Research and literature has addressed parental decision-making when it comes to prenatal diagnosis for fetuses with genetic conditions resulting in intellectual disabilities. The recent advent of targeted drug trials for intellectual disabilities poses questions about decision-making in the post-natal period and throughout the life span of this vulnerable population that have not yet been explored. Objective. To explore this uncharted territory, we examined factors that influence parental decisions to involve children in a clinical drug trial for Fragile X Syndrome or Down Syndrome. Methods. IRB approval was obtained and 24 parents of individuals with Fragile X Syndrome or Down Syndrome participated in phone interviews. We asked about child involvement in trials and attitudes towards trials and medication. We hypothesized that parents of children with Fragile X Syndrome or Down Syndrome who are more severely affected with debilitating behavioral problems are more inclined to explore or take an interest in clinical drug trial enrollment for their child. Results. We found no evidence for a correlation between a child's behavioral problems or the parent's perception of the child's overall health and a parent's interest in drug trials. Generally, parents were more willing to involve their child in a decision for him/her to take a lifelong medication than to participate in a trial. Limitations. Our participants may not be representative of the target population. Recruitment was primarily done through an academic medical institution so most participants had knowledge or involvement in research. Drug trials for Down Syndrome are new and involve individuals between the ages of 18 and 30; thus, it was difficult to find participants with a child who has been involved in a trial for Down Syndrome. Conclusions. Some of the factors influencing the clinical drug trial and treatment decision-making of this population include: child's behavioral problems, parental attitudes towards drug trials, parental attitudes towards medication, hopes for improvement, concerns, child involvement, and misunderstandings about trials.

Table of Contents

Introduction....................................................................................1

Bioethical Research on Prenatal Diagnosis and Termination Decisions.........2

Fragile X Syndrome...........................................................................4

Down Syndrome...............................................................................6

Clinical Drug Trials............................................................................9

Methods.......................................................................................10

Molecular Laboratory Shadowing........................................................10

Cytogenetics Laboratory Shadowing....................................................10

Original Research: Data Collection......................................................11

Results.........................................................................................12

Demographics.................................................................................12

Health of Child ...............................................................................15

Attitudes Towards Drug Trials............................................................20

Trial and Medication Decision-making Process......................................23

Attitudes Towards Medication............................................................31

Misconceptions/Misunderstandings about Trials....................................32

Discussion...................................................................................32

Health of Child...............................................................................33

Attitudes Towards Drug Trials...........................................................35

Trial and Medication Decision-making Process......................................37

Attitudes Towards Medication............................................................39

Misconceptions/Misunderstandings about Trials.....................................41

Study Limitations.............................................................................42

Concluding Remarks.........................................................................43

References....................................................................................46

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