Clinical trials – design and results

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This year, two sessions are related to clinical trials. The first, concerns the planning and design of clinical trials. The second is the results of recent trials.

But why should we talk about the planning of trials? Planning is a vital step if we are to find a new treatment for MND. By taking the time and effort to carefully plan trials in new and novel ways, clinical trials can be cheaper and test the true effectiveness of the treatment in a short amount of time with less people involved. By learning from other clinical trials that are currently being planned, as well as the results from trials that had a novel design, researchers can refine the way that trials are planned to speed up the process of moving potentially beneficial drugs from the laboratory to the clinic.

The first talk in the design clinical trials session talked about Phase II clinical trials. Phase II clinical trials are designed to test a number of issues regarding the treatment, including, the safety, tolerability, finding out the ‘right’ dose for it to be effective as well as a number of other issues. Due to the ever expanding list of possible issues to resolve through a Phase II clinical trial, there is no single clinical trial design that can answer every question. This presents clinical trial designers with a dilemma as Phase II trials for MND are often longer and larger than desired or possible – this leads to problems in the design and conduct of later trials.

Getting it right is certainly not an easy task, which is why it is vital that clinical trial designers learn from previous trials and constantly improve how clinical trials are conducted.

The second session provided answers to whether recent treatments that have completed clinical trials are safe or effective.

The results of recent trials for Talampanel and Pioglitazone were discussed. As a summary: Talampanel was found to be safe but not effective; Pioglitazone was found to be potentially unsafe and also did not show effectiveness in altering disease progression.

Although this is disappointing news, by sharing their results with others means that researchers can learn about the design and enrolment of clinical trials in the hope that future trials can learn from the past. Even negative trial results are an answer as to whether a treatment strategy is appropriate and should be further investigated from a different angle.

The last talk of the clinical trials session was regarding a recently completed early safety clinical trial for a new drug (named CK2017357) developed by a pharmaceutical company called Cytokinetics. In the study 67 people with MND took a single dose of either a placebo (dummy drug), small dose, or large dose of the drug on three different days. Neither the patient nor the doctors knew what they were receiving on what day.

This is an extremely novel way to approach a clinical trial, as a direct comparison of safety and preliminary effectiveness can be measured in the same person.

Through the Phase II clinical trial, they identified that within six hours of taking the drug, people had increased muscle strength and endurance which was responsive to the dose given. The drug was also found to be safe. This is positive news, but should be taken with a ‘pinch of salt’, as a much larger future clinical trial using multiple doses is needed to determine the true effectiveness of this drug.