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Experimental treatment

Ερώτηση
Hello
I’ve got cystic fibrosis with F508del and Q1411X mutations. I was sfor one year and a half under Ataluren experimental-treatment. Yesterday, I was told that the protocol is ending immediately because of a lack of significant results from the clinical trial.
Nevertheless, for me I saw a reduction of exacerbations. I’m extremely disappointed by this news.
This is my question: regarding my mutations could I take part in ongoing clinical trials?
Thank a lot for your answer.
Απάντηση
Hello

To date you can take part in trials for evaluating drug-candidates as anti-infectious or anti-inflammatory ones. Those trials are independent of the type of mutation or in some case are for patients with at least one copy of F508del mutation.

Regarding experimental treatment to restore a functional CFTR protein (like Ataluren for non-sense mutations) there is no ongoing clinical trial for patients carrying F508del and a non-sense mutations (like Q1411X ).

Some clinical trials for the evaluation of new modulators (corrector or potentiator) of CFTR will start before the end of the year. Those trials will be for patients with at least one copy of F508del mutation.

If you want to know trials you can take part in please contact your CF cares center to get information about the research protocols in progress in the center.

Best regards
Anna Ronayette
29.05.2017