ACE-031 Study Terminated: What Does This Mean?

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.

http://www.fda.gov/ScienceResearch/SpecialTopics/RunningClinicalTri...

 

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.

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Comment by David on July 14, 2011 at 5:30pm
Can anybody summarize what Larry / Shire discussed at the conference?

Staff
Comment by Pat Furlong on June 30, 2011 at 12:15pm

Larry Charnas from Shire will be presenting during the conference.  We will update next week.

Comment by David on June 28, 2011 at 2:06pm

3 months now since termination. Time to give up on ACE-031?

Comment by Jason G on June 9, 2011 at 4:05pm

Michelle, my son does not get nosebleeds.   Numerous serious ones during the three months of the study were definitely related to the drug.   

David,  we've heard nothing from the clinic except that they're waiting to hear something.   

Comment by David on June 9, 2011 at 3:45pm

Has PPMD received any information to suggest when this will be picked back up by Shire / Acceleron? Been nearly two months now ...

Comment by RAKTIM SINGH on April 29, 2011 at 12:43pm
Thanks Pat for the update and clarification. Good to know that Acceleron/SHIRE are focussed on Duchenne.We pary for their SUCCESS.
Comment by cheryl cliff on April 22, 2011 at 10:58am
Pat, Your gratitude to Acceleron/Shire is the same we feel too.  Very relieved they plan to stick with the program to kick Duchenne.  Please let us know more when the opportunity arises. 
Comment by Michelle Scaglione on April 22, 2011 at 2:08am

My son has been getting nosebleeds for years.  My gums bleeds too and I'm not on any medications.  I wonder if people panic and its not even related to the medication.

Michelle


Staff
Comment by Pat Furlong on April 21, 2011 at 11:48pm

Hello Again,  One point of clarification.  In the ACE-031 trial, the nosebleeds and gum bleeding are characterized as AE (adverse events).   Again, this is specific terminology used in clinical studies.   AE are different that SAE.   In a clinical study the AE are adverse events but not serious, as in not requring hospitalization or intervention.    SAE are adverse events requiring hospitalization.

Acceleron/Shire are to be commended.  They are moving forward carefully and thoughfully, investigating the cause of the AE (nosebleeds/gum bleeding) and discussing the design of future studies.  In an earlier blog I talked about my visit to Shire, finding the word 'brave' on every desk and wall.  Brave to be focused on Duchenne (and other rare conditions), working for Brave indivduals with the diagnosis of Duchenne.   I think this press release demonstrates bravery, commitment and resolve to move the ACE-031 program forward.   Their vision is clear -eyes on the prize (treatments for Duchenne) and I, for one, am thankful Acceleron/Shire has a focus on Duchenne.

Comment by cheryl cliff on April 21, 2011 at 7:23pm
I suppose it's too early for the scientists to determine the exact nature of the nosebleeds but is it possible they have some idea?

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