Phase I Clinical Trial Injuries Come During Greater Push for Trial Data Transparency

January 27th, 2016

Earlier this month, one patient died and five were seriously injured in a rare Phase I clinical trial incident in France, where an experimental painkiller was undergoing first-in-human safety testing -- an incident that has researchers around the globe demanding greater insight into the trial's design.

The six patients – men between 28 and 49 years of age – received the drug several times beginning on Jan. 7 and were in good health prior to receiving it. The first patient showed adverse effects three days later and was hospitalized; the others quickly followed. One patient was declared brain dead and died shortly thereafter.

Now, a few weeks after the incident, scientists around the world are in the dark about the test drug and are calling for more details to be released. What is known is that the trial, which was halted immediately after the patients were hospitalized, was to test a drug for the treatment of anxiety and motor disorders associated with Parkinson’s disease, and chronic pain in patients with cancer and other diseases. The non-cannabis-based drug worked by attaching to endocannabinoid receptors in the brain. While France's National Agency for Medicines and Health Products Safety (ANSM) released the clinical trial protocol, researchers say there are many unanswered questions, such as dosages or intervals between patients beginning treatment.

The call for more details about the drug and the clinical trial comes as the movement for greater clinical trial information transparency gains more momentum. The AllTrials movement for trial data transparency, as covered in the October 2015 issue of IRB Advisor, has 600 groups supporting it globally, including 50 in the United States. Pharma giants such as Johnson & Johnson, GlaxoSmithKline, and Bristol-Myers Squibb, have committed to more transparency. And on Jan. 20, the International Committee of Medical Journal Editors (ICMJE) published its proposal for clinical trial data sharing in the Annals of Internal Medicine. The ICJME’s proposal would change how journal editors accept study manuscripts and includes requiring study authors to share deidentified individual patient data underlying the results in the article and requiring authors to create a data-sharing plan (including where the trial data will be housed). ICJME’s proposal is open to public comment until April 18.

While many in the research and IRB field have been cautiously receptive to the idea of greater clinical trial data transparency, some may balk at the proposals from the ICMJE. At what point will data transparency go from not enough to too much information?

Look for more coverage – including IRB industry reactions – of the ICJME’s proposal in the March issue of IRB Advisor.