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Drug & research development » Clinical trials » NCT02141464

Energy Balance and Weight Gain With Ivacaftor Treatment

Official title: Energy Balance and Weight Gain With Ivacaftor Treatment of CFTR Gating Mutations

Clinical Trials gov number: NCT02141464

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Purpose: To determine the mechanism(s) for weight gain in participants whom ivacaftor treatment was initiated based on clinical indications by CF Care Team. Assess energy expenditure, weight gain, body composition, and lung function in 24 CF patients aged ≥6 years with a gating mutation before treatment and after three months treatment with ivacaftor.

Phase: NA

Type: Observational, prospective, cohort

Study sponsor: Children’s Hospital of Philadelphia

  • Cystic fibrosis with one or two CFTR gating mutations
  • A clinical decision has been made for the subject to start ivacaftor treatment
  • In usual state of good health
  • Family and subject commitment to the 3-month study protocol with two, 3-4 day visits to CHOP


Geographical Location
United States

Number of Participants
≤50 (≥6 years)

Primary Endpoint

Significant reduction in resting energy expenditure over 3 months

Secondary Endpoint

Not provided

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Not provided

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