A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C?T or D1152H CFTR Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03068312
• Date of registration: 24/02/2017
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C?T or D1152H CFTR Mutation
• Scientific title: A Randomized, Double-blind, Placebo-controlled, Crossover Study to Evaluate the Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older and Have Either a 3849 + 10KB C?T or D1152H-CFTR Mutation
• Date of first enrolment: July 18, 2017
• Target sample size: 38
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03068312
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Israel

Key inclusion & exclusion criteria

Inclusion Criteria:

- Confirmed diagnosis of CF based on protocol-specified clinical features and at least
one of the following: increased sweat chloride level, identification of 2 CF causing
mutations, or demonstration of abnormal nasal epithelial ion transport.

- A 3849 + 10KB C?T or D1152H mutation on at least 1 CFTR allele.

- FEV1 =40% of predicted and =105% of predicted at screening.

Exclusion Criteria:

- A G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H
mutation.

- History of any illness or any clinical condition that, in the opinion of the
investigator, might confound the results of the study or pose an additional risk in
administering study drug to the subject.

- Ongoing or prior participation in an investigational drug study within 30 days before
the Screening Visit.

- Protocol-specified abnormal laboratory values at the Screening Visit

- For subjects <18 years of age at the Screening Visit, evidence of cataract/lens
opacity determined to be clinically significant by the ophthalmologist or optometrist
during the ophthalmologic examination (OE) at the Screening Visit.

- Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A,
including consumption of certain herbal medications and certain fruit and fruit
juices, within 14 days before Day 1.

- Pregnant, breastfeeding, or planning to become pregnant during the study.

- Sexually active subjects of reproductive potential must be willing to use appropriate
contraception.



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: Ivacaftor

Primary Outcome(s)

Change in Lung Clearance Index 2.5 (LCI2.5) [Time Frame: From baseline through 8 weeks]

Secondary ID(s)

2017-000457-39

VX16-770-127

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 06/01/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03068312