On ClinicalTrials.gov you will now see that the ACE-031 study is terminated.
While this news is frustrating, it is not unusual in drug development. We all wish it would be a straight path through each phase of the clinical trial process to approval, but that is often not the case. Drug development often has stops and starts, when a SAE (serious adverse event) occurs or questions about dosing arise. This results in days and weeks of discussions about the cause of a particular SAE, whether it is drug related or not, whether it may be dose related or disease related. In trials, all SAEs are reported, whatever happens while an individual is taking the study drug. If only one individual, it is often easy to rule it out as not related to the study drug. If several or many of the study subjects experience the same event, it is up to the sponsor (PI, Biotech, Pharma) to figure out the cause and often the trial is suspended, the SAE’s investigated, and the decision is made to terminate the study. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInfo...
In this particular case, Acceleron/Shire investigated the cause of the “experienced minor nosebleeds, gum bleeding, and/or small dilated blood vessels within the skin, all of which resolved when the study drug was discontinued.” While none of these were labeled as serious, Acceleron/Shire are working with regulatory to gain further understanding of the cause of these events.
Like all government agencies, the FDA uses specific terminology to describe the status of clinical studies. Words such as recruiting, not yet recruiting, clinical hold, suspension, and termination are used to describe a specific study, not programs. The status of a single study does not reflect the sponsor’s decision to continue with a certain program or to a certain disease indication. It is critical that we keep these concepts in our mind as we visit www.clinicaltrials.gov.
Acceleron/Shire is dedicated to Duchenne and the program will continue to move forward. We all wish them godspeed as they continue to advance ACE-031 for Duchenne.