NCT01531673

A Phase 2, Multicenter, Double-Blinded, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 Monotherapy and VX-661/Ivacaftor Cotherapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

Completed Phase 2 Results posted Last updated 14 March 2018

What this trial tests

Phase 2 trial testing VX-661 in Cystic Fibrosis in 194 participants. Completed in 1 March 2014.

Timeline

1 February 2012

Primary endpoint
1 March 2014

1 March 2014

Quick facts

Lead sponsor	Vertex Pharmaceuticals Incorporated
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	194
Start date	1 February 2012
Primary completion	1 March 2014
Estimated completion	1 March 2014
Sites	36 locations across United States, Canada, Germany, United Kingdom

Drugs / interventions tested

• VX-661 — full drug profile →
• Ivacaftor (IVACAFTOR) — full drug profile →
• Placebo matched to VX-661 — full drug profile →
• Placebo matched to ivacaftor

Conditions studied

• Cystic Fibrosis — all drugs for Cystic Fibrosis →

Sponsor

Vertex Pharmaceuticals Incorporated — full company profile →

Who can join

12 and older, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Safety as Determined by Adverse Events (AEs)
  Time frame: Start of study drug through the Follow-up Visit (Up to Day 56)
  An AE is defined as any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the Informed Consent Form is signed. AE includes serious as well as non-serious AEs. Serious Adverse Event (SAE) is any AE
• Change in Sweat Chloride From Baseline Through Study Day 28 for Group 1-5b
  Time frame: Baseline through Day 28
  Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug.
• Change in Sweat Chloride From Baseline Through Study Day 28 for Group 6
  Time frame: Baseline through Day 28
  Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug. As per planned analysis, participants who received placebo in Group 4 and 6 were combined and compared with Group 6.
• Change in Sweat Chloride From Baseline Through Study Day 28 for Group 7
  Time frame: Baseline through Day 28
  Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug.

Sponsor's own description

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) effects of VX-661 alone and when coadministered with ivacaftor in participants with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del-CFTR mutation.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Targeting fibrosis, mechanisms and cilinical trials.
   Zhao M, Wang L, Wang M, Zhou S, et al · · 2022 · cited 352× · PMID 35773269 · DOI 10.1038/s41392-022-01070-3
2. CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine.
   Lopes-Pacheco M. · · 2019 · cited 349× · PMID 32153386 · DOI 10.3389/fphar.2019.01662
3. Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression.
   Veit G, Avramescu RG, Perdomo D, Phuan PW, et al · · 2014 · cited 256× · PMID 25101887 · DOI 10.1126/scitranslmed.3008889
4. Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR.
   Donaldson SH, Pilewski JM, Griese M, Cooke J, et al · · 2018 · cited 133× · PMID 28930490 · DOI 10.1164/rccm.201704-0717oc
5. CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis.
   Lopes-Pacheco M. · · 2016 · cited 108× · PMID 27656143 · DOI 10.3389/fphar.2016.00275
6. State of the Art on Approved Cystic Fibrosis Transmembrane Conductance Regulator (<i>CFTR</i>) Modulators and Triple-Combination Therapy.
   Meoli A, Fainardi V, Deolmi M, Chiopris G, et al · · 2021 · cited 26× · PMID 34577628 · DOI 10.3390/ph14090928
7. Interaction non grata between CFTR's correctors and potentiators.
   Lin WY, Yu YC. · · 2015 · cited 1× · PMID 25992374 · DOI 10.3978/j.issn.2305-5839.2015.01.27