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)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03525444
• Date of registration: 02/05/2018
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: 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)
• Scientific 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)
• Date of first enrolment: June 15, 2018
• Target sample size: 405
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03525444
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Australia, Austria, Belgium, Canada, Czechia, France, Germany, Greece, Italy, Netherlands, Sweden, United Kingdom, United States

Key inclusion & exclusion 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



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: VX-445/TEZ/IVA

Drug: IVA

Drug: Placebo

Primary Outcome(s)

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [Time Frame: From Baseline at Week 4]

Secondary Outcome(s)

Absolute Change in Body Mass Index (BMI) [Time Frame: From Baseline at Week 24]

Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline [Time Frame: From Baseline at Week 24]

Number of Pulmonary Exacerbations (PEx) [Time Frame: From Baseline through Week 24]

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [Time Frame: From Baseline through Week 24]

Absolute Change in Sweat Chloride [Time Frame: From Baseline at Week 4]

Time-to-first Pulmonary Exacerbation (PEx) [Time Frame: From Baseline through Week 24]

Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score [Time Frame: From Baseline at Week 4]

Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ, M1-TEZ, and IVA [Time Frame: Pre-dose on Week 4, 8, 12, and 16]

Absolute Change in Sweat Chloride (SwCl) [Time Frame: From Baseline through Week 24]

Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 28 weeks)]

Absolute Change in Body Weight [Time Frame: From Baseline at Week 24]

Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score [Time Frame: From Baseline through Week 24]

Secondary ID(s)

VX17-445-102

2018-000183-28

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 19/05/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03525444