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Completed

NCT Number: NCT03768089

Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis

The purpose of this study is to evaluate safety and tolerability of VX-121 in healthy subjects and in subjects with cystic fibrosis (CF).

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Academic Medical Center, Amsterdam, Netherlands

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Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Part A, B, and C: Healthy Volunteers
  • Female subjects must be of non-childbearing potential
  • Between the ages of 18 and 55 years, inclusive
  • Body mass index (BMI) of 18.0 to 32.0 kg/m2, inclusive, and a total body weight >50 kg
  • Part D: Subjects with CF
  • Heterozygous for F508del and an MF mutation (F/MF)
  • FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height
  • Body weight ≥35 kg

Key Exclusion Criteria:

  • Part A, B and C: Healthy Volunteers
  • Any condition possibly affecting drug absorption
  • History of febrile illness or other acute illness within 5 days before the first study drug dose
  • Part D: Subjects with CF
  • History of clinically significant cirrhosis with or without portal hypertension
  • History of solid organ or hematological transplantation
  • Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply

Treatment and study plan

Placebo (matched to VX-121 suspension)

Drug

Placebo matched to VX-121 suspension for oral administration.

VX-121 (Suspension)

Drug

Suspension for oral administration.

TEZ/IVA

Drug

Fixed-dose combination tablet for oral administration.

Other names: VX-661/VX-770, tezacaftor/ivacaftor

IVA

Drug

Tablet for oral administration.

Other names: VX-770, ivacaftor

Placebo (matched to TEZ/IVA)

Drug

Placebo matched to TEZ/IVA for oral administration.

Placebo (matched to IVA)

Drug

Placebo matched to IVA for oral administration.

VX-121 (Tablet)

Drug

Tablet for oral administration.

Placebo (matched to VX-121 tablet)

Drug

Placebo matched to VX-121 tablet for oral administration.

Primary outcomes

  1. Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Time frame: From Day 1 Through Safety Follow-up (up to Day 15 for Part A [except Cohorts A3 and A9], up to Day 26 for Cohort A3, up to Day 34 for Cohort A9, up to Day 20 for Part B, up to Day 24 for Part C and up to Week 9 for Part D)

Secondary outcomes

  1. Part A: Maximum Observed Concentration (Cmax) of VX-121

    Time frame: Cohorts A1-5 (Except A3): Pre-dose up to 240 hours post-dose; Cohorts A3 and A9: Pre-dose up to 168 hours post-dose

  2. Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration (AUC[0-last]) of VX-121

    Time frame: Cohorts A1-5 (Except A3): Pre-dose up to 240 hours post-dose; Cohorts A3 and A9: Pre-dose up to 168 hours post-dose

  3. Part B: Maximum Observed Concentration (Cmax) of VX-121

    Time frame: Day 1, Day 5, and Day 10

  4. Part B: Area Under the Concentration Versus Time Curve During the Dosing Interval (AUCtau) of VX-121

    Time frame: Day 1, Day 5, and Day 10

  5. Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121

    Time frame: Pre-dose at Day 5 and Day 10

  6. Part C: Maximum Observed Concentration (Cmax) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and, IVA and Its Metabolites (M1-IVA and M6-IVA)

    Time frame: Day 1, Day 7, and Day 14

  7. Part C: Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA)

    Time frame: Day 1, Day 7, and Day 14

  8. Part C: Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA)

    Time frame: Pre-dose at Day 7 and Day 14

  9. Part D: Maximum Observed Concentration (Cmax) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA)

    Time frame: Day 1 and Day 15

  10. Part D: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration (AUC[0-last]) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA)

    Time frame: Day 1 and Day 15

  11. Part D: Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA)

    Time frame: Pre-dose at Day 8, Day 15, and Day 29

  12. Part D: Absolute Change in Sweat Chloride (SwCl) Concentrations

    Time frame: From Baseline Through Day 29

    Sweat samples were collected using an approved collection device.

  13. Part D: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

    Time frame: From Baseline Through Day 29

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 1/2 Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis

Important dates

Study start
2018
Primary completion
2019
Study completion
2019
First posted
Dec 7, 2018
Registry last updated
Jul 14, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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