Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
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| ClinicalTrials.gov Identifier: NCT03691779 |
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Recruitment Status :
Completed
First Posted : October 2, 2018
Last Update Posted : December 17, 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 pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-445, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects 6 through 11 years of age with F/F and F/MF genotypes.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis | Drug: VX-445 Drug: TEZ Drug: IVA | Phase 3 |
Expanded Access : An investigational treatment associated with this study has been approved for sale to the public. More info ...
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 66 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-445/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age |
| Actual Study Start Date : | October 2, 2018 |
| Actual Primary Completion Date : | August 7, 2020 |
| Actual Study Completion Date : | August 7, 2020 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
| Arm | Intervention/treatment |
|---|---|
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Experimental: Part A: Triple Combination
Subjects will receive 100 mg VX-445/ 50 mg TEZ/ 75 mg IVA as an FDC tablet in the morning and 75 mg IVA as mono tablet in the evening.
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Drug: VX-445
Fixed dose combination (FDC) tablet (VX-445/TEZ/IVA) Drug: TEZ FDC tablet (VX-445/TEZ/IVA)
Other Name: tezacaftor; VX-661 Drug: IVA FDC tablet (VX-445/TEZ/IVA) and IVA mono tablet
Other Name: ivacaftor; VX-770 |
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Experimental: Part B: Triple Combination
Subjects will receive VX-445/TEZ/IVA as 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.
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Drug: VX-445
Fixed dose combination (FDC) tablet (VX-445/TEZ/IVA) Drug: TEZ FDC tablet (VX-445/TEZ/IVA)
Other Name: tezacaftor; VX-661 Drug: IVA FDC tablet (VX-445/TEZ/IVA) and IVA mono tablet
Other Name: ivacaftor; VX-770 |
Primary Outcome Measures :
- Part A: Observed pre-dose concentration (Ctrough) of VX-445, TEZ, and IVA [ Time Frame: Day 1 through 15 ]
- Part A: Maximum Observed Concentration (Cmax) of VX-445, TEZ, and IVA [ Time Frame: Day 1 through 15 ]
- Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-445, TEZ, and IVA [ Time Frame: Day 1 through 15 ]
- Part B: Safety and tolerability as assessed by number of subjects with adverse events and serious adverse events [ Time Frame: from baseline through safety follow-up (28 Weeks) ]
Secondary Outcome Measures :
- Part A: Maximum observed concentration (Cmax) of VX-445, TEZ, and IVA metabolites [ Time Frame: from Day 1 through 15 ]
- Part A: Observed pre-dose concentration (Ctrough) of VX-445, TEZ, and IVA metabolites [ Time Frame: from Day 1 through 15 ]
- Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-445, TEZ, and IVA metabolites [ Time Frame: from Day 1 through 15 ]
- 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 (28 Weeks) ]
- Part B: Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline through Week 24 ]
- Part B: Absolute change in sweat chloride [ Time Frame: from baseline through Week 24 ]
- Part B: Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain score [ Time Frame: from baseline through Week 24 ]
- Part B: Absolute change in body mass index (BMI) and BMI for age-z-score [ Time Frame: from baseline at Week 24 ]
- Part B: Absolute change in weight and weight for age-z-score [ Time Frame: from baseline at Week 24 ]
- Part B: Absolute change in height and height for age-z-score [ Time Frame: from baseline at Week 24 ]
- Part B: Absolute change in the Modified Facial Hedonic Scale [ Time Frame: from baseline at Week 24 ]
- Parts B: Ctrough of VX-445, TEZ, IVA, and IVA metabolites [ Time Frame: Day 1 through Week 24 ]
- Part B: Absolute change in lung clearance index2.5 (LCI2.5) [ Time Frame: from baseline through Week 24 ]
- Part B: Number of Pulmonary Exacerbations (PEx) [ Time Frame: from baseline through Week 24 ]
- Part B: Number of CF Related Hospitalizations [ 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): NCT03691779
Locations
Show 21 study locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Vertex Pharmaceuticals Incorporated |
| ClinicalTrials.gov Identifier: | NCT03691779 |
| Other Study ID Numbers: |
VX18-445-106 2018-001695-38 ( EudraCT Number ) |
| First Posted: | October 2, 2018 Key Record Dates |
| Last Update Posted: | December 17, 2020 |
| Last Verified: | September 2020 |
| 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 VX-445 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 |