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Trial record 1 of 2 for:    445-102
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A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

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ClinicalTrials.gov Identifier: NCT03525444
Recruitment Status : Active, not recruiting
First Posted : May 15, 2018
Last Update Posted : December 4, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-445 Drug: TEZ Drug: IVA Drug: Matched Placebos Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 405 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Actual Study Start Date : June 15, 2018
Estimated Primary Completion Date : August 2019
Estimated Study Completion Date : August 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Triple Combination
Subjects will receive 200 mg VX-445/ 100 mg TEZ/ 150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening
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)
Other Name: ivacaftor; VX-770

Placebo Comparator: Placebo Drug: Matched Placebos
Placebo will be used as a comparator.




Primary Outcome Measures :
  1. Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline at Week 4 ]

Secondary Outcome Measures :
  1. Absolute change in ppFEV1 [ Time Frame: from baseline through Week 24 ]
  2. Number of pulmonary exacerbations (PEx) [ Time Frame: from baseline through Week 24 ]
  3. Absolute change in body mass index (BMI) [ Time Frame: from baseline at Week 24 ]
  4. Absolute change in sweat chloride (SwCl) [ Time Frame: from baseline at Week 4 and through Week 24 ]
  5. Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline at Week 4 and through Week 24 ]
  6. Time-to-first PEx [ Time Frame: through Week 24 ]
  7. Absolute change in BMI z-score [ Time Frame: from baseline at Week 24 ]
  8. Absolute change in body weight [ Time Frame: from baseline at Week 24 ]
  9. Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through 4-week safety follow-up (up to 28 weeks) ]
  10. Observed pre-dose concentration (Ctrough) of VX-445, TEZ, M1-TEZ, and IVA [ Time Frame: from Day 1 through Week 16 ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Heterozygous for the F508del mutation (F/MF)
  • Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% 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): NCT03525444


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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03525444     History of Changes
Other Study ID Numbers: VX17-445-102
2018-000183-28 ( EudraCT Number )
First Posted: May 15, 2018    Key Record Dates
Last Update Posted: December 4, 2018
Last Verified: December 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases