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Vertex - results of a phase 2 study for patients heterozygous for F508del
- Question
- Hello
The Vertex company has published results regarding patients heterozygous for the F508del mutation, here is the link: http://investors.vrtx.com/releasedetail.cfm?releaseid=862727.
It seems to me that patients heterozygous for the F508del mutation could have a potential treatment like the one received by patients carrying two copies of the F508del mutation. This is correct? Could they be included in clinical trials? Thank for your reply. Regards.
- Answer
- Hello
In the press release dated on 29 july 2014, we can read information regarding the results of a phase 2 clinical trial in patients carrying one copy of the F508del mutation and a second mutation that is not expected to respond to either Ivacaftor (VX-770) or Lumacafotr (VX-809) alone. 125 patients older than 12 years, received during 8 weeks VX-770 associated to VX-809. But no significant difference, in terms of percentage points for the FEV1, has been shown.
Anna Ronayette
Head of clinical research programs of Vaincre la mucoviscidose
- 15.09.2014
- 15.09.14
Please find even more information under:
ecorn-cf.eu/index.php?id=65&L=8&tx_expertadvice_pi1%5Bshowitem%5D=3111&tx_expertadvice_pi1%5Bsearch%5D=
D. d'Alquen
