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

This study is currently recruiting participants.
See Contacts and Locations
Verified May 2017 by Concert Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Concert Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02971839
First received: November 21, 2016
Last updated: May 30, 2017
Last verified: May 2017
  Purpose
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.

Condition Intervention Phase
Cystic Fibrosis Drug: CTP-656, 20 mg (QD) Drug: CTP-656, 100 mg (QD) Drug: CTP-656, 150 mg (QD) Drug: Placebo (QD) Drug: Kalydeco, 150 mg Tablet (BID; open-label) Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment
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.

Resource links provided by NLM:


Further study details as provided by Concert Pharmaceuticals:

Primary Outcome Measures:
  • Change from baseline in sweat chloride at Day 28 [ Time Frame: 28 days ]

Secondary Outcome Measures:
  • Change from baseline in percent predicted FEV1 at Day 28 [ Time Frame: 28 days ]
  • Change from baseline in CFQ-R Respiratory Domain at Day 28 [ Time Frame: 28 days ]

Estimated Enrollment: 40
Study Start Date: December 2016
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: CTP-656, 20 mg, QD
Oral tablet dosed once-daily for 28 days
Drug: CTP-656, 20 mg (QD)
Experimental: CTP-656, 100 mg, QD
Oral tablet dosed once-daily for 28 days
Drug: CTP-656, 100 mg (QD)
Experimental: CTP-656, 150 mg, QD
Oral tablet dosed once-daily for 28 days
Drug: CTP-656, 150 mg (QD)
Active Comparator: Kalydeco, 150 mg Tablet (open label)
150 mg, oral tablet dosed twice-daily for 28 days
Drug: Kalydeco, 150 mg Tablet (BID; open-label)
Placebo Comparator: Placebo, Oral Tablet, QD
Oral tablet dosed once-daily for 28 days
Drug: Placebo (QD)

Detailed 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.
  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02971839

Contacts
Contact: Vinita Uttamsingh, PhD 781-674-5240

Locations
United States, California
University of Southern California Recruiting
Los Angeles, California, United States
Stanford Hospital Recruiting
Palo Alto, California, United States
United States, District of Columbia
Children's National Health Not yet recruiting
Washington, D.C., District of Columbia, United States
United States, Florida
University of Miami Recruiting
Miami, Florida, United States
United States, Illinois
Rush University Not yet recruiting
Chicago, Illinois, United States
United States, Indiana
Indiana University Recruiting
Indianapolis, Indiana, United States
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States
University of Massachusetts Recruiting
Worcester, Massachusetts, United States
United States, Missouri
Washington University Recruiting
Saint Louis, Missouri, United States
United States, New Jersey
Atlantic Health Recruiting
Morristown, New Jersey, United States
United States, New York
New York Medical College Recruiting
Valhalla, New York, United States
United States, Ohio
Cincinnati Children's Hospital Recruiting
Cincinnati, Ohio, United States
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States
United States, Pennsylvania
Children's Hospital of Philadelphia Not yet recruiting
Philadelphia, Pennsylvania, United States
Sponsors and Collaborators
Concert Pharmaceuticals
Investigators
Study Director: Vinita Uttamsingh, PhD Concert Pharmaceuticals, Inc.
  More Information

Responsible Party: Concert Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02971839     History of Changes
Other Study ID Numbers: CP656.2001
Study First Received: November 21, 2016
Last Updated: May 30, 2017
Individual Participant Data  
Plan to Share IPD: No

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on June 27, 2017