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

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

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.

Key facts

Study ID

NCT02971839

Run by

Vertex Pharmaceuticals Incorporated

People needed

11

Starts

2016-12-01

Expected to finish

2017-08-01

Last updated by the study team

2020-08-26

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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

Where it is running

• University of Southern California — Los Angeles, California, United States
• Stanford Hospital — Palo Alto, California, United States
• Children's National Health — Washington D.C., District of Columbia, United States
• University of Miami — Miami, Florida, United States
• Rush University — Chicago, Illinois, United States
• Indiana University — Indianapolis, Indiana, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Massachusetts — Worcester, Massachusetts, United States
• Washington University — St Louis, Missouri, United States
• Atlantic Health — Morristown, New Jersey, United States
• New York Medical College — Valhalla, New York, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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