Trial record 3 of 8 for:
CTP-656
Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
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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 |
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Recruitment Status :
Terminated
(Decision by Sponsor.)
First Posted : November 23, 2016
Results First Posted : August 26, 2020
Last Update Posted : August 26, 2020
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Sponsor:
Vertex Pharmaceuticals Incorporated
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis | Drug: VX-561 Drug: Placebo Drug: IVA | Phase 2 |
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 : | Interventional (Clinical Trial) |
| Actual Enrollment : | 11 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (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. |
| Actual Study Start Date : | December 2016 |
| Actual Primary Completion Date : | August 2017 |
| Actual Study Completion Date : | August 2017 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
Drug Information available for:
Ivacaftor
| Arm | Intervention/treatment |
|---|---|
| Experimental: VX-561 20 mg |
Drug: VX-561
Other Name: CTP-656 |
| Experimental: VX-561 100 mg |
Drug: VX-561
Other Name: CTP-656 |
| Experimental: VX-561 150 mg |
Drug: VX-561
Other Name: CTP-656 |
| Active Comparator: Ivacaftor |
Drug: IVA
Other Names:
|
| Placebo Comparator: Placebo |
Drug: Placebo |
Primary Outcome Measures :
- Change From Baseline in Sweat Chloride at Day 28 [ Time Frame: From baseline at Day 28 ]
Secondary Outcome Measures :
- Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 [ Time Frame: From baseline at Day 28 ]
- Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28 [ Time Frame: From baseline at Day 28 ]
Information from the National Library of Medicine
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, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02971839
Locations
| United States, California | |
| University of Southern California | |
| Los Angeles, California, United States | |
| Stanford Hospital | |
| Palo Alto, California, United States | |
| United States, District of Columbia | |
| Children's National Health | |
| Washington, District of Columbia, United States | |
| United States, Florida | |
| University of Miami | |
| Miami, Florida, United States | |
| United States, Illinois | |
| Rush University | |
| Chicago, Illinois, United States | |
| United States, Indiana | |
| Indiana University | |
| Indianapolis, Indiana, United States | |
| United States, Massachusetts | |
| Boston Children's Hospital | |
| Boston, Massachusetts, United States | |
| University of Massachusetts | |
| Worcester, Massachusetts, United States | |
| United States, Missouri | |
| Washington University | |
| Saint Louis, Missouri, United States | |
| United States, New Jersey | |
| Atlantic Health | |
| Morristown, New Jersey, United States | |
| United States, New York | |
| New York Medical College | |
| Valhalla, New York, United States | |
| United States, Ohio | |
| Cincinnati Children's Hospital | |
| Cincinnati, Ohio, United States | |
| Nationwide Children's Hospital | |
| Columbus, Ohio, United States | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States | |
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Study Documents (Full-Text)
Documents provided by Vertex Pharmaceuticals Incorporated:
Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol [PDF] October 24, 2016
Statistical Analysis Plan [PDF] September 22, 2017
| Responsible Party: | Vertex Pharmaceuticals Incorporated |
| ClinicalTrials.gov Identifier: | NCT02971839 |
| Other Study ID Numbers: |
CP656.2001 |
| First Posted: | November 23, 2016 Key Record Dates |
| Results First Posted: | August 26, 2020 |
| Last Update Posted: | August 26, 2020 |
| Last Verified: | August 2020 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
Additional relevant MeSH terms:
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Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases |
Genetic Diseases, Inborn Infant, Newborn, Diseases Ivacaftor Chloride Channel Agonists Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |