"The boys' mom says her family was preparing to have two sons in wheelchairs until Max was accepted into the clinical trial for eteplirsen at Nationwide Children's Hospital in Columbus, Ohio. He is one of 12 boys in the entire country taking part in the first-of-its-kind study. Austin was not included because he did not meet certain criteria, including his inability to walk.

"The way we explained it to Austin is that his brother is doing this for you and if this drug works, we will make sure you get it, too," Jenn said.

Max is involved in a blind trial, meaning some of the kids were given the drug and some were not. Max's family says they know he's getting a dose given his remarkable success.

"Week 16 Max opened a milk carton at the airport and my husband called me and said, 'Oh my God, he is on the drug,'" Jenn said.

A lot more

http://www.wcax.com/story/19277658/2-vt-brothers-battle-deadly-diso...

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The inability to walk should not be an exclusion criteria. I know the "6 minute walk test" is a proven primary endpoint to show rate of decline. However, DMD effects all skeletal muscle equally and seems logical that there are other physical activities that can prove rate of decline. For example propelling a manual wheelchair, using stationary hand cycle for 6 minute or any activity that maintaining quality of life can be measured.

Having primary endpoints beyond the"6min walk test" can only improve the process of drug approval and give the drug companies larger population of DMD patients for trials.    

To help us all understand the inclusion/exclusion criteria for this trial go to here:http://clinicaltrials.gov/ct2/show/NCT01396239?spons=%22Sarepta+The...

Mind you that these criteria are a combination of what the scientists want & what the FDA insists upon.

 

 

 

PPMD is working hard to establish biomarkers and alternative endpoints, but it is difficult to do anything innovative with US Federal Government.

Let's hope the media attention given to this family will continue to grow and bring more light to the subject.

Does the new legislation allow for changes in endpoint like those described by Jason?



Jason Darienzo said:

The inability to walk should not be an exclusion criteria. I know the "6 minute walk test" is a proven primary endpoint to show rate of decline. However, DMD effects all skeletal muscle equally and seems logical that there are other physical activities that can prove rate of decline. For example propelling a manual wheelchair, using stationary hand cycle for 6 minute or any activity that maintaining quality of life can be measured.

Having primary endpoints beyond the"6min walk test" can only improve the process of drug approval and give the drug companies larger population of DMD patients for trials.    

Not direct answer to your question, but an article I've found very helpful on the subject as background.

http://www.fda.gov/downloads/drugs/scienceresearch/researchareas/ph...

As direct response - YES.

policymed.com has terrific resources in this area. According to their site when describing most recent reauthorization of PDUFA:

The reauthorization discussions yielded agreement on enhancements in several areas:  

  • Review program for NME NDAs and Original BLAs
  • Enhancing Regulatory Science and Expediting Drug Development
    • Promoting Innovation Through Enhanced Communication Between FDA and Sponsors During Drug Development
    • Methods for meta‐analysis
    • Biomarkers and pharmacogenomics
    • Use of patient‐reported outcomes (PROs)
    • Development of drugs for rare diseases
    • Enhancing Benefit‐Risk Assessment
    • Enhancement and Modernization of the FDA Drug Safety System

 

http://www.policymed.com/2012/08/fda-goals-for-the-center-for-drug-...

 

Will the primary and secondary endpoints be enought for FDA approvial for all boys? 

The FDA approval to be sought will include a confirmatory ph-3 clinical, plus consideration for early approval. For the exon deletion as per the ph-2 trial design.

 

Endpoints are established in consultation with the FDA regarding what should be demonstrated to qualify to go forward into a ph-3 clinical.

 

Early approval could also include an open label for all regardless of age, (but still for a specific exon deletion), however this is by no means assured at this time.

 

 

 

 

The FDA has a protocal exception in which a child can be in the trial and safety data is gathered but not efficacy data but the drug company has to agree to allow it.  Unless the drug company agrees to explore this option the is nothng the FDA can do.

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