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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02971839
Recruitment Status : Terminated (Decision by Sponsor.)
First Posted : November 23, 2016
Last Update Posted : August 15, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE November 21, 2016
First Posted Date  ICMJE November 23, 2016
Last Update Posted Date August 15, 2018
Actual Study Start Date  ICMJE December 2016
Actual Primary Completion Date August 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 21, 2016)
Change from baseline in sweat chloride at Day 28 [ Time Frame: 28 days ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 21, 2016)
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
Official Title  ICMJE 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.
Brief 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.
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • 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)
Study Arms  ICMJE
  • Experimental: CTP-656, 20 mg, QD
    Oral tablet dosed once-daily for 28 days
    Intervention: Drug: CTP-656, 20 mg (QD)
  • Experimental: CTP-656, 100 mg, QD
    Oral tablet dosed once-daily for 28 days
    Intervention: Drug: CTP-656, 100 mg (QD)
  • Experimental: CTP-656, 150 mg, QD
    Oral tablet dosed once-daily for 28 days
    Intervention: Drug: CTP-656, 150 mg (QD)
  • Active Comparator: Kalydeco, 150 mg Tablet (open label)
    150 mg, oral tablet dosed twice-daily for 28 days
    Intervention: Drug: Kalydeco, 150 mg Tablet (BID; open-label)
  • Placebo Comparator: Placebo, Oral Tablet, QD
    Oral tablet dosed once-daily for 28 days
    Intervention: Drug: Placebo (QD)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: September 6, 2017)
11
Original Estimated Enrollment  ICMJE
 (submitted: November 21, 2016)
40
Actual Study Completion Date  ICMJE August 2017
Actual Primary Completion Date August 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02971839
Other Study ID Numbers  ICMJE CP656.2001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Vertex Pharmaceuticals Incorporated
Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP