The active ingredient in KALYDECO tablets is ivacaftor.
KALYDECO is classified as a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the G551D mutation.
Limitations of Use
KALYDECO is not effective in patients with CF who are homozygous for the F508del mutation in the CFTR gene and has not been studied in other populations of patients with CF.
Clinical Trials Related to Kalydeco (Ivacaftor)
Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients [Not yet recruiting]
This is a study of the short-term effects of ivacaftor on sweat chloride concentration and
lung function in cystic fibrosis (CF) patients who fall outside current FDA approval. This
new, first of its kind drug is approved for use only in CF patients with the G551D mutation
in whom it safely confers considerable benefits. However, it is highly likely that CF
patients with many other mutations can benefit similarly from this drug, some of whom can be
identified by phenotype or genotype.
We will enroll up to 30 CF subjects with clinical presentations in which there is one or
more signs of residual CF channel function. The signs of residual function include: normal
digestion, concentration of chloride in sweat between 55 and 85, or milder than expected CF
disease in a CF patient with severe gene mutations. The primary outcome measure will be the
difference in sweat chloride concentration measured in subjects on placebo and on ivacaftor.
Secondary outcome measured will be lung function.
Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CTFR Gating Mutation [Recruiting]
The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and
pharmacodynamics (PD), of ivacaftor in children with CF who are 2 through 5 years of age and
have a CFTR gating mutation in at least 1 allele.
This is an open-label study designed to evaluate the safety, PK, PD, and efficacy of
ivacaftor in subjects with CF who are 2 through 5 years of age and have a CFTR gating
mutation in at least 1 allele. Part A is designed to evaluate the safety and PK of
multiple-dose administration of ivacaftor in subjects 2 through 5 years of age and to
confirm the doses for Part B. Part B is designed to evaluate the safety, PK, PD, and
efficacy of ivacaftor in subjects 2 through 5 years of age.
Ivacaftor is currently approved by the US FDA and the EMA under the trade name Kalydeco® for
the treatment of cystic fibrosis in patients 6 years of age and older who have the
Study of VX-661 Alone and in Combination With VX-770 in Subjects Homozygous to the F508del-CFTR Mutation [Recruiting]
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 VX-770 in
subjects with CF who are homozygous for the F508del-CFTR mutation.
Study of Ivacaftor in Subjects With Cystic Fibrosis Who Have the R117H-CFTR Mutation [Recruiting]
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects
with cystic fibrosis (CF) who have the R117H-CFTR mutation.
A Phase 1 Study to Examine the Drug-Drug Interaction of Ciprofloxacin, Itraconazole, and Rifampin on the Combination of Lumacaftor With Ivacaftor in Healthy Adult Subjects [Recruiting]
The purpose of this study is to examine the drug-drug interaction effects of ciprofloxacin,
itraconazole, and rifampin on the pharmacokinetics of lumacaftor in combination with
Reports of Suspected Kalydeco (Ivacaftor) Side Effects
Drug Ineffective (10),
Infective Pulmonary Exacerbation of Cystic Fibrosis (10),
Productive Cough (4),
Abdominal Pain Upper (4),
Chest Pain (4),
Dyspnoea (4), more >>
Page last updated: 2012-08-01