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Case Study E

Clinical trial of drug for people with Down syndrome

The study

Dr E wants to investigate whether a particular drug will improve the cognitive and learning abilities of people with Down syndrome. He proposes a randomised, double-blind, placebo-controlled study. This means that some of the participants would receive the study drug and some would receive a placebo (for example, a sugar pill). During the trial, neither the participants nor the researchers would know who was receiving the drug. Participants would be required to undergo regular six-hour assessment visits to check their progress.

The study drug has already been tested on people without Down syndrome. That research provided some information about the possible risks and side-effects of the drug, including that, for some participants, it increased the incidence of contemplating suicide. However, there may be other risks or side-effects that have not yet been discovered. In particular, the effects of the drug on people without Down syndrome may be different from those on people with Down syndrome. 

It is not known whether the drug will have the desired effect on cognition or learning (or any other beneficial effects). However, it is likely that even if the drug did lead to an improvement in cognition for the research participants, the effect would be temporary because the drug would not be available to participants after the conclusion of the trial. 

Some adults with Down syndrome may be capable of providing consent if given appropriate support and information. Those consumers could then be enrolled in the study in accordance with ordinary consent principles. Dr E proposes also to enrol participants who are not able to give consent because the effects of the drug on those participants may be different. Dr E proposes to consult with family/whānau/caregivers and, if they express objections, those participants will not be enrolled.


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