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A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT03911713
Recruitment Status : Recruiting
First Posted : April 11, 2019
Last Update Posted : September 13, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-561 Drug: IVA Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 88 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Actual Study Start Date : April 17, 2019
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: VX-561
Subjects will be randomized to receive 1 of 4 dose levels of VX-561.
Drug: VX-561
VX-561 tablets for oral administration.
Other Name: CTP-656

Drug: Placebo
Placebo matched to IVA.

Active Comparator: IVA Drug: IVA
150-mg film-coated tablet for oral administration.
Other Names:
  • VX-770
  • Ivacaftor

Drug: Placebo
Placebos matched to VX-561.




Primary Outcome Measures :
  1. Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: From Baseline at Week 12 ]

Secondary Outcome Measures :
  1. Absolute change in sweat chloride concentrations [ Time Frame: From Baseline at Week 12 ]
  2. Maximum observed concentration (Cmax) of VX-561, IVA, and relevant metabolites [ Time Frame: From Baseline up to Week 16 ]
  3. Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-561, IVA, and relevant metabolites [ Time Frame: From Baseline up to Week 16 ]
  4. Observed pre-dose concentration (Ctrough) of VX-561, IVA, and relevant metabolites [ Time Frame: From Baseline up to Week 16 ]
  5. Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From baseline up to Week 16 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
  • On ivacaftor therapy
  • FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • History of clinically significant cirrhosis with or without portal hypertension
  • History of solid organ or hematological transplantation
  • Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply


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): NCT03911713


Contacts
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Contact: Medical Information 6173416777 medicalinfo@vrtx.com

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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03911713     History of Changes
Other Study ID Numbers: VX18-561-101
2018-003970-28 ( EudraCT Number )
First Posted: April 11, 2019    Key Record Dates
Last Update Posted: September 13, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: 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