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Drug & research development » Clinical trials » NCT01685801

Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function

Official title: A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 ≥40% Predicted

Clinical Trials gov number: NCT01685801

Click on the tabs to move through the sequence.

Purpose: To evaluate the effect of ivacaftor on lung function in CF patients aged ≥12 years who have phenotypic or molecular evidence of residual CF transmembrane conductance regulator (CFTR) function.

Phase: 2

Type: Interventional, randomized, double-blind, crossover assignment

Study sponsor: Vertex Pharmaceuticals Incorporated

  • Male or female subjects with confirmed diagnosis of CF
  • Clinical evidence of residual CFTR function based on any 1 of the following:

– Clinically documented residual exocrine pancreatic function

– Sweat chloride value ≤80 mmol/L at screening, or

– Age of diagnosis ≥12 years and at least 1 copy of a CFTR mutation associated with residual CFTR function or defective mRNA splicing

  • FEV1 ≥40%
  • Willing to agree to meet the contraception requirements
  • Able to swallow tablets

Intervention
Ivacaftor

Geographical Location
United States

Number of Participants
≤50 (≥12 years)

Primary Endpoint

Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) after 2 weeks of treatment

Secondary Endpoint

At Week 2

  • Change in lung clearance index

At Week 8

  • Absolute change from baseline (BL) in percent predicted FEV1
  • Change from BL in LCI
  • Change from baseline in sweat chloride
  • Change from baseline in weight

At Week 28

  • Safety as determined by adverse events and vital signs

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  • Experimental: Sequence 1 – ivacaftor > placebo > ivacaftor > placebo > ivacaftor (open-label period)

ivacaftor: 150 mg tablet for oral use, administered every 12 hours

  • Experimental: Sequence 2 – ivacaftor > placebo > placebo > ivacaftor > ivacaftor (open-label period)

ivacaftor: 150 mg tablet for oral use, administered every 12 hours

  • Experimental: Sequence 3 – placebo > ivacaftor > ivacaftor > placebo > ivacaftor (open-label period)

placebo > ivacaftor > ivacaftor > placebo > ivacaftor (open-label period)

Drug: ivacaftor
150 mg tablet for oral use, administered every 12 hours
Other Name: Tradename: KALYDECO
Drug: placebo
tablet for oral use, administered every 12 hours

  • Experimental: Sequence 4

placebo > ivacaftor > placebo > ivacaftor > ivacaftor (open-label period)

Drug: ivacaftor
150 mg tablet for oral use, administered every 12 hours
Other Name: Tradename: KALYDECO
Drug: placebo
tablet for oral use, administered every 12 hours

View Trial Results