A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02951195
• Date of registration: 26/10/2016
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis
• Scientific title: A Phase 2, Randomized, Double Blind, Controlled Study to Evaluate the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis
• Date of first enrolment: November 2016
• Target sample size: 80
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02951195
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Willing and able to comply with scheduled visits, treatment pan, study restrictions,
laboratory tests, contraceptive guidelines, and other study procedures.

- Body weight =35 kg.

- Sweat chloride value = 60 mmol/L from test results obtained during screening.

- Subjects must have an eligible CFTR genotype:

- Cohorts 1A, 1B, 1C: Heterozygous for F508del and a minimal function mutation
known or predicted not to respond to TEZ and/or IVA.

- Cohorts 2A, 2B: Homozygous for F508del.

- Subjects must have an FEV1 =40% and =90% of predicted normal for age, sex, and height
at the Screening Visit.

- Stable CF disease as judged by the investigator.

- Willing to remain on a stable CF medication regimen through the planned end of
treatment or if applicable the Safety Follow-up Visit.

Exclusion Criteria:

- History of any comorbidity that in the opinion of the investigator might confound the
results of the study or pose an additional risk in administering study drug to the
subject.

- History of cirrhosis with portal hypertension.

- Risk factors for Torsade de Pointes.

- History of hemolysis.

- Glucose-6-phosphate dehydrogenase (G6PD) deficiency assessed at Screening.

- Clinically significant abnormal laboratory values at screening.

- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy for pulmonary disease within 28 days before the first dose of study drug.

- Lung infection with organisms associated with a more rapid decline in pulmonary
status.

- An acute illness not related to CF within 14 days before the first dose of study drug.

- A standard digital ECG demonstrating QTc >450 msec at screening.

- History of solid organ or hematological transplantation.

- History or evidence of cataract or lens opacity determined to be clinically
significant by the ophthalmologist or optometrist, based on the ophthalmologic
examination during the Screening Period.

- History of alcohol or drug abuse in the past year, including but not limited to,
cannabis, cocaine, and opiates, as deemed by the investigator.

- Ongoing or prior participation in an investigational drug study with certain
exceptions.

- Use of commercially available CFTR modulator within 14 days before screening (applies
only to Cohorts 1A, 1B, and 1C).

- Pregnant or nursing females: Females of childbearing potential must have a negative
pregnancy test at screening and Day 1.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Ivacaftor

Drug: VX-152

Drug: Placebo for VX-152

Drug: Tezacaftor

Drug: Triple Placebo

Primary Outcome(s)

Safety and Tolerability assessments as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [Time Frame: from baseline up to 8 Weeks]

Secondary Outcome(s)

Maximum observed concentration (Cmax) of VX-152, TEZ, M1-TEZ, IVA, and M1-IVA (µg/mL) [Time Frame: Day 1 through Day 15]

Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [Time Frame: from baseline at Day 15]

Absolute change in sweat chloride concentrations [Time Frame: from baseline at Day 15]

Observed pre-dose concentration (Ctrough) of VX-152, TEZ, M1-TEZ, IVA, and M1-IVA (µg/mL) [Time Frame: Day 1 through Day 15]

Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-152, TEZ, M1-TEZ, IVA, and M1-IVA (µg.h/mL) [Time Frame: Day 1 through Day 15]

Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [Time Frame: from baseline at Day 15]

Relative change in ppFEV1 [Time Frame: from baseline at Day 15]

Secondary ID(s)

VX16-152-102

2016-003049-27

Source(s) of Monetary Support

Please refer to primary and secondary sponsors