Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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12 December 2020 |
Main ID: |
NCT02971839 |
Date of registration:
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21/11/2016 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
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Scientific title:
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A Phase 2, Randomized, Parallel-Group, Double-Blind, Placebo Controlled Study to Evaluate the Safety and Efficacy of CTP-656 With an Open-Label Active Comparator in Patients With Cystic Fibrosis With CFTR Gating Mutations. |
Date of first enrolment:
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December 2016 |
Target sample size:
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11 |
Recruitment status: |
Terminated |
URL:
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https://clinicaltrials.gov/show/NCT02971839 |
Study type:
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Interventional |
Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- 18 years of age or older
- Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating
mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R.
- Has been stable on Kalydeco therapy for at least 3 months prior to screening
- Has FEV1 = 60% of predicted normal for age, sex, and height at screening and baseline
(Day 1) assessments
- Weighs at least 40 kg at screening
- Patients of either gender and women of childbearing potential must be willing to use a
medically highly effective form of birth control during the treatment period and 30
days after the last dose of study treatment.
Exclusion Criteria:
- Acute upper respiratory infection or lower respiratory infection, pulmonary
exacerbation, or changes in therapy within 4 weeks of study treatment
- Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes
- History of hepatitis C or chronic active hepatitis B infection
- History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or
allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2
years prior to screening
- Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within
2 years prior to Screening
- Abnormal liver function
- History of abnormal renal function
- History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females
- History of solid organ or hematological transplantation
- Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30
days of screening
- Women who are pregnant or lactating, or have plans to become pregnant during the study
or within 1 month following the last dose
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Cystic Fibrosis
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Intervention(s)
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Drug: IVA
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Drug: VX-561
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Drug: Placebo
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Primary Outcome(s)
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Change From Baseline in Sweat Chloride at Day 28
[Time Frame: From baseline at Day 28]
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Secondary Outcome(s)
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Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28
[Time Frame: From baseline at Day 28]
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Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
[Time Frame: From baseline at Day 28]
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Secondary ID(s)
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CP656.2001
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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