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Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age

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ClinicalTrials.gov Identifier: NCT03633526
Recruitment Status : Recruiting
First Posted : August 16, 2018
Last Update Posted : December 4, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects with F/F or F/MF genotypes.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-659 Drug: TEZ Drug: IVA Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 56 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-659/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age
Actual Study Start Date : August 3, 2018
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Part A: Triple Combination
Subjects will receive 120 mg VX-659/ 50 mg TEZ/ 75 mg IVA as an FDC tablet in the morning and 75 mg IVA as mono tablet in the evening.
Drug: VX-659
Fixed dose combination (FDC) tablet (VX-659/TEZ/IVA)

Drug: TEZ
FDC tablet (VX-659/TEZ/IVA)
Other Name: tezacaftor; VX-661

Drug: IVA
FDC tablet (VX-659/TEZ/IVA) and IVA mono tablet
Other Name: ivacaftor; VX-770

Experimental: Part B: Triple Combination
Subjects will receive VX-659/TEZ/IVA as an FDC tablet in the morning and IVA as mono tablet in the evening, with the dose to be based on the outcome of Part A.
Drug: VX-659
Fixed dose combination (FDC) tablet (VX-659/TEZ/IVA)

Drug: TEZ
FDC tablet (VX-659/TEZ/IVA)
Other Name: tezacaftor; VX-661

Drug: IVA
FDC tablet (VX-659/TEZ/IVA) and IVA mono tablet
Other Name: ivacaftor; VX-770




Primary Outcome Measures :
  1. Part A: Maximum observed concentration (Cmax) of VX-659, TEZ, and IVA [ Time Frame: Day 1 through 15 ]
  2. Part A: Observed pre-dose concentration (Ctrough) of VX-659, TEZ, and IVA [ Time Frame: Day 1 through 15 ]
  3. Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-659, TEZ, and IVA [ Time Frame: Day 1 through 15 ]
  4. Part B: Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (24 Weeks) ]

Secondary Outcome Measures :
  1. Part A: Maximum observed concentration (Cmax) of TEZ and IVA metabolites [ Time Frame: Day 1 through 15 ]
  2. Part A: Observed pre-dose concentration (Ctrough) of TEZ and IVA metabolites [ Time Frame: Day 1 through 15 ]
  3. Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of TEZ and IVA metabolites [ Time Frame: Day 1 through 15 ]
  4. Part A: Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (5 weeks) ]
  5. Part B: Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline through Weeks 12 and 24 ]
  6. Part B: Absolute change in sweat chloride [ Time Frame: from baseline through Weeks 12 and 24 ]
  7. Part B: Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain score [ Time Frame: from baseline through Weeks 12 and 24 ]
  8. Part B: Absolute change in body mass index (BMI) [ Time Frame: from baseline at Week 24 ]
  9. Part B: Absolute change in BMI-for-age-z-score [ Time Frame: from baseline at Week 24 ]
  10. Part B: Absolute change in weight [ Time Frame: from baseline at Week 24 ]
  11. Part B: Absolute change in weight-for-age-z-score [ Time Frame: from baseline at Week 24 ]
  12. Part B: Absolute change in height [ Time Frame: from baseline at Week 24 ]
  13. Part B: Absolute change in height-for-age-z-score [ Time Frame: from baseline at Week 24 ]
  14. Part B: Absolute change in the Modified Facial Hedonic Scale [ Time Frame: from baseline at Week 24 ]
  15. Part B: Ctrough of VX-659, TEZ, IVA, and metabolites [ Time Frame: From Day 1 through Week 24 ]
  16. Part B: Absolute change in lung clearance index2.5 (LCI2.5) [ Time Frame: from baseline through Week 24 ]


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Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Homozygous or heterozygous for F508del mutation (F/F or F/MF genotypes)
  • Forced expiratory volume in 1 second (FEV1) value ≥40% of predicted mean for age, sex, and height.

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status.
  • Solid organ or hematological transplantation.

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): NCT03633526


Contacts
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Contact: Medical Information 6173416777 medicalinfo@vrtx.com

Locations
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United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
United States, Illinois
Ann & Robert Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
United States, Missouri
The Children's Mercy Hospital Recruiting
Kansas City, Missouri, United States, 64108
United States, North Carolina
Clinical Research of Charlotte Recruiting
Charlotte, North Carolina, United States, 28277
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03633526     History of Changes
Other Study ID Numbers: VX18-659-106
2018-001711-67 ( EudraCT Number )
First Posted: August 16, 2018    Key Record Dates
Last Update Posted: December 4, 2018
Last Verified: December 2018

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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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Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases