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

Cystic Fibrosis Clinical Trial

Official 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.

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02971839
• Other study ID #: CP656.2001
• Status: Terminated
• Phase: Phase 2
• Start date: December 2016
• Est. completion date: August 2017

Study information

• Verified date: August 2020
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

This is a randomized, parallel-group, double-blind, placebo controlled multicenter study to evaluate the safety and efficacy of CTP-656 in CF patients with CFTR gating mutations, compared to Kalydeco, for a total of 28 days. Subjects will be randomized to receive either double-blind CTP-656 or placebo, or open-label Kalydeco.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 11
• Est. completion date: August 2017
• Est. primary completion date: August 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• VX-561

• Placebo

• IVA

Locations

Country	Name	City	State
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Rush University	Chicago	Illinois
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Indiana University	Indianapolis	Indiana
United States	University of Southern California	Los Angeles	California
United States	University of Miami	Miami	Florida
United States	Atlantic Health	Morristown	New Jersey
United States	Stanford Hospital	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Washington University	Saint Louis	Missouri
United States	New York Medical College	Valhalla	New York
United States	Children's National Health	Washington	District of Columbia
United States	University of Massachusetts	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Sweat Chloride at Day 28		From baseline at Day 28
Secondary	Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28		From baseline at Day 28
Secondary	Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28		From baseline at Day 28