Delta F 508

Question

Thank you in advance for giving us the latest positive or negative but true results on combined VX 770 and VX 809 clinical trials for example as we read everything and its opposite. It would be good to have regular news because for us, sick or close to sick people with CF, it is the little light that grows for a hope for cure finally. Again thank you

Answer

Hello,
It is impossible for me to give you either positive or negative results on the combined Ivacaftor (VX 770) and Lumacaftor (VX 809) clinical trial in the population of F508del homozygous CF patients; and this for two good reasons:
- Firstly, because no one can claim to have the results of this study which must be carried to its conclusion before being analyzed. I remind you that it is a double-blind, randomized controlled study, meaning that neither the investigator nor the patient knows who is taking the tested product or the placebo;
- Secondly, because like all study investigators, I am held by a confidentiality agreement prohibiting me from giving, on my initiative, any information on the conduct of the study.
I do understand the legitimate impatience of patients and families, but equally the ethics and the scientific rigor require respecting the rules. Failure to do so would have serious consequences to ban or not to conclude the benefit of the tested product and thus deprive patients of the potential benefits of a new drug.
Thank you for your understanding.
Sincerely.
Gilles RAULT, MD, Roscoff CF Center

01.01.2014

1.1.14 Please find detailed information on the progress of ongoing studies under www.clinicaltrials.gov
D. d'Alquen