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A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects With an F508del CFTR Mutation

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: NCT03537651
Recruitment Status : Active, not recruiting
First Posted : May 25, 2018
Last Update Posted : June 11, 2020
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief Summary:
This study will evaluate the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in subjects with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: TEZ Drug: IVA Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 130 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation
Actual Study Start Date : April 25, 2018
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arms and Interventions
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Arm Intervention/treatment
Experimental: TEZ + IVA

Subjects <30 kg will receive 50 mg TEZ/ 75 mg IVA as a FDC tablet in the morning and 75 mg IVA as a mono tablet in the evening.

Subjects >=30 kg will receive 100 mg TEZ/ 150 mg IVA FDC tablet in the morning and 150 mg IVA as a mono tablet in the evening.

 Drug: TEZ
Fixed-dose Combination (FDC) tablet (TEZ/IVA)
Other Name: tezacaftor; VX-661

Drug: IVA
FDC tablet (TEZ/IVA)
Other Name: ivacaftor; VX-770


Outcome Measures
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Primary Outcome Measures :
  1. Safety and tolerability of long-term TEZ/IVA treatment based on adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (up to 28 days after Last Dose at Week 96) ]

Secondary Outcome Measures :
  1. Absolute change in lung clearance index2.5 (LCI2.5) [ Time Frame: from baseline through 96 weeks ]
  2. Absolute change in sweat chloride [ Time Frame: from baseline through 96 weeks ]
  3. Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline through 96 weeks ]
  4. Absolute change in body mass index (BMI) [ Time Frame: from baseline through 96 weeks ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115.
  • Eligible CFTR Mutation.

Exclusion Criteria:

  • Pregnant and nursing females.
  • History of poor compliance with study drug and/or procedures in a previous study as deemed by the investigator.
  • Ongoing participation in another study with investigational drug.

Other protocol defined Inclusion/Exclusion criteria may apply.

Contacts and Locations
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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): NCT03537651


Locations
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03537651    
Other Study ID Numbers: VX17-661-116
2017-002968-40 ( EudraCT Number )
First Posted: May 25, 2018    Key Record Dates
Last Update Posted: June 11, 2020
Last Verified: February 2020

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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