809 + 770 Phase 3 announced

B

bigstar

New member
God im so so so frustrated that i cant be in the study. I would qualify but i dont live near a clinic. There is no mention of when it starts! I hope they do it very very soon. Any clues?
 
G

GenH

New member
I just posted this summary from the latest Vertex investor conference in a few facebook groups and thought I would post it here as well:

Current Trials

  • Enrolment is complete in the other gating Kalydeco trial
  • Enrolment is ongoing in the R117H and residual function Kalydeco trials
  • Data from both is expected later this year
  • Dosing has commenced with the G551D 2-5 year old Kalydeco trial
  • Worldwide the above groups are 10-15% of the CF population
  • Data expected for VX661 by June

Phase 3 VX809/Kalydeco trial for people with 2 F508del

  • There are more than 30,000 double F508del patients worldwide
  • In terms of the phase 3 study design that was announced yesterday: The two 24 week trials have the same design, each with 500 patients and 3 groups (600mg of VX809 once a day plus Kalydeco, 400mg of VX809 twice a day plus Kalydeco and placebo). After the trial there is a 96 week open label trial where everyone gets VX809/Kalydeco. Patients need to have two copies of F508del, be aged 12 or older and have an FEV1 between 40-90%. The tablets will contain both VX809 and Kalydeco.
  • The primary endpoint will be relative FEV1% improvement compared to placebo. They are hoping for at least a 5% relative improvement.
  • Depending on the results, it is planned to submit to the EMA and FDA following these trials.
  • Data is expected in 2014.

Other trials

  • The 6-11 year old 24 week study with patients who have two copies of F508del will look at safety and dosage. No time frame was mentioned. This will also have an open label period following the trial.
  • No time frame was mentioned with the 8 week phase 2 study for people with one copy of F508del. This is looking at safety and lung function. They are accelerating the second generation correctors to help the people with one copy of F508del

Results from the phase 2 VX809/Kalydeco trial for people with 2 copies of F508del

  • There was a statistically significant improvement from baseline and placebo at day 56 with the 600mg group (cohort 2). The patients who had a higher level of VX809 in their blood stream (higher exposure) had a better clinical response. There was a 3.4% absolute FEV1 improvement compared to baseline and a 6.7% improvement compared to placebo at day 56.
  • The 400mg twice a day group (cohort 3) had better levels of VX809 in their blood stream. 70% of these patients were in the highest 25% in terms of the levels of VX809 in their blood stream (see the second image). 40% of the 600mg group were in the highest 25%. The mean for both groups was in the top 25%. This demonstrated a relationship between exposure/levels in the blood stream and an improvement in FEV1. There was a 1.9% absolute FEV1 improvement compared to baseline at day 56 (not statistically significant). This trial was very small with only 11 patients so it was not designed to look for FEV1 improvement (designed for safety and dosage).


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http://investors.vrtx.com/eventdetail.cfm?eventid=126065
 
G

GenH

New member
Aboveallislove said:
That's awesome. May I ask which Facebook g routs as I follow a couple and don't see. Thanks!
Click to expand...

I posted this in Kalydeco for Australians and CF Advances Australia, I knew Margot was writing one in the Praying for Success group so I did not post there. I am also a member of Kalydeco Miracles but didn't post this there.

Kalydeco for Australians: www.facebook.com/groups/343431685698805/
CF Advances Australia: www.facebook.com/groups/130421340438389/
Praying for Success with VX770/809: www.facebook.com/groups/153375788050316/
Kalydeco Miracles: www.facebook.com/groups/359954544023650/
 
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