Joining Jack Announce the Funding of the Isofen 3 Trial
We are delighted to announce that Joining Jack has decided on another breakthrough research project to receive funding from the charity. We, the charity trustees and our scientific advisors believe that this research will help all those with Duchenne and potentially be available in early 2016.
ISOFEN 3 is a phase II clinical trial to investigate whether the combination of two drugs, ibuprofen, (a non steroidal anti-inflammatory agent) and isosorbide dinitrate, (a drug that releases nitric oxide) is capable of slowing the progression of Duchenne Muscular Dystrophy. The study will be carried out in non-ambulant patients affected by DMD, and the rationale of the study and the reasons why it is proposed are the following:
1. The mechanisms by which this drug combination has been established in animal models of dystrophy; essentially the drug combination enhances muscle repair and myogenesis (the formation of muscle tissue), reduces inflammation and adipose tissue deposition, and enhances muscle function. It also prevents the exhaustion of the myogenic stem cell pool.
2. A pilot clinical trial in non-ambulant dystrophic patients affected by Duchenne and Becker Muscular dystrophies showed that the drug combination is safe. The study also showed encouraging results on the efficacy of the drug combination.
3. Two studies in healthy volunteers have led to the definition of the optimal dosage of the drug combination to be used for efficacy testing in patients.
It is important to note that the actions of the drug tested in ISOFEN 3 do not address the genetic cause of the disease, as instead occurs in the case of exon skipping or other proposed gene-therapies, but act downstream on key common pathogenic mechanisms. Because of this, if proven successful in non-ambulant patients, the drug can be easily transferred to ambulant patients.
The study will have a total duration of 24 months of which 6 will be for the enrolment of patients and 18 for the actual trial, in which the primary outcome measure will be the amelioration of the Motor Function Measure scale, a validated scale to assess disease progression in non-ambulant patients. Several other outcome measures will also be assessed as secondary endpoints.
If the drug combination is proven successful it will be authorized for use in patients in a few months after completion of the trial, since this trial is agreed with regulatory Agencies and all preclinical and Phase I studies (healthy volunteers) have already been done.