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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 : Recruiting
First Posted : May 15, 2018
Last Update Posted : June 21, 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)
Estimated Enrollment : 360 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
  • Glucose-6-phosphate dehydrogenase (G6PD) deficiency
  • 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


Contacts
Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

Locations
United States, Arizona
Banner University of Arizona Medical Center Recruiting
Tucson, Arizona, United States, 85724
United States, Florida
Central Florida Pulmonary Group Recruiting
Orlando, Florida, United States, 32803
United States, Illinois
Northwestern Memorial Hospital Recruiting
Chicago, Illinois, United States, 60611
United States, Kansas
Univesity of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
United States, Massachusetts
Massachusetts General Hospital Cystic Fibrosis Center Recruiting
Boston, Massachusetts, United States, 02114
United States, Minnesota
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55404
United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
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: June 21, 2018
Last Verified: May 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