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

• Status: Terminated
• Trial end date: 2017-08-01
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Concert Pharmaceuticals; Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

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