When will the clinical trial for gene replacement therapy begin? Who will qualify to participate? How many will be selected for the trial?
Answer: The target date for starting the trial is the end of 2015 although this is fully dependent on an approval by the Regulatory Authorities (FDA in the US; EMA in Europe). The protocol is still in the process of being finalized. This includes ongoing detailed discussions with international experts and the Regulatory Authorities. Until these discussions have been completed it is not certain who will qualify for the trial, nor is it certain the number of patients that will be required.
Where or at which hospitals will trials be taking place?
Answer: There will be trial sites in the United States and in Europe. Several locations are under discussion but can't be named until all the necessary contracts are signed.
What kinds of time and travel commitments are involved to be in the trial? Who will cover the costs?
Answer: The time and travel commitment will not be finalized until the protocol is approved. Patients will need to spend a period of time at the hospital where they receive their gene therapy. Audentes will cover the travel, housing, and food for the child and their parent for the trial.
What are the possible risks and side effects?
Answer: Clinical trials carry risks because all the potential effects of a medication are not known. All of the known risks will be included in the informed consent form that parents will sign before starting the trial. The physician conducting the trial will have a full discussion about risks before parents sign the consent form.
If the trial is successful, what is the expected time frame that the treatment might be available to everyone else?
Answer: The timing as to when the medication is available to treat all patients, is dependent on several factors. These include the length of time to enroll the patients in to the study, the actual length of the trial, the data that emerge from the study, and the time it takes regulatory agencies to approve the submission. Likely this will be several years.
What the implications might be for those with CNM and other musculoskeletal diseases?
Answer: Currently, we are solely focused on completing the trials that are planned for MTM as the lead program, and Pompe as the second program. In the future, we may hope to be able to explore additional indications.
Will insurance cover this medication?
Answer: Discussions with insurance companies will not take place until we are closer to approval.
