Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02971839
• Date of registration: 21/11/2016
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
• Scientific title: 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: December 2016
• Target sample size: 11
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02971839
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

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

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: IVA

Drug: VX-561

Drug: Placebo

Primary Outcome(s)

Change From Baseline in Sweat Chloride at Day 28 [Time Frame: From baseline at Day 28]

Secondary Outcome(s)

Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28 [Time Frame: From baseline at Day 28]

Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 [Time Frame: From baseline at Day 28]

Secondary ID(s)

CP656.2001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 26/08/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02971839