Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis

NCT06154447
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Phase
Phase 1
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Recruitment status
Completed
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Participants
165
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Lead sponsor
Vertex Pharmaceuticals Incorporated
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Start date
2023-12-12
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Number of sites
12
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Summary
The purpose of the study is to evaluate safety, tolerability, and pharmacokinetics of VX-828 and VX-828 in triple combination (TC) with Tezacaftor (TEZ)/ VX-118 or TEZ/ deutivacaftor (D-IVA) in healthy participants and VX-828 in combination with D-IVA with or without TEZ in participants with cystic fibrosis (CF).
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Short title
Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis
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Official title
A Phase 1, Study of VX-828 in Healthy Subjects and in Subjects With Cystic Fibrosis
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Control arm
Placebo
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Registry reports results
No
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Listing at source
Listed
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Recruitment status
Completed
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Phase
Phase 1
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Study type
Interventional
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Completion date
2026-07-10
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First posted
2023-12-04
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Registry last updated
2026-08-10
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Primary completion date
2026-07-10
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Start date
2023-12-12
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Actual participants
165
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Participants
165
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Participant count basis
Actual
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Protocol amendments
6
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Number of sites
12
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Registry sponsor category
Industry
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Lead sponsor
Vertex Pharmaceuticals Incorporated
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Source registry
ClinicalTrials.gov
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Registry ID
NCT06154447
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ClinicalTrials.gov ID
NCT06154447
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Registry record
Open registry record
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Allocation
Randomised
Model
Sequential
Purpose
Treatment
Masking
Quadruple masking
Masked roles
Participants, Care providers, Investigators, Outcome assessors
Sources for study design
clinicaltrials.gov

  1. Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Timeframe: From Signing of Informed Consent Form (ICF) up to Safety Follow Up (Up to Day 67)

  2. Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Timeframe: From Signing of Informed Consent Form (ICF) up to Safety Follow Up (Up to Day 80)

  3. Part D: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Timeframe: From Signing of Informed Consent Form (ICF) up to Safety Follow Up (Up to Day 80)

  4. Part E: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Timeframe: From Signing of Informed Consent Form (ICF) up to End of Study (Up to Day 111)

  5. Part C: Maximum Observed Concentration (Cmax) of VX-828 in Plasma in the Absence and Presence of Itraconazole

    Timeframe: From Day 1 up to Day 71

  6. Part C: Area Under the Concentration Versus Time Curve (AUC) of VX-828 in Plasma in the Absence and Presence of Itraconazole

    Timeframe: From Day 1 up to Day 71

  7. Part C: Maximum Observed Concentration (Cmax) of Midazolam in Plasma in the Absence and Presence of VX-828/TEZ/D-IVA

    Timeframe: From Day 1 up to Day 30

  8. Part C: Area Under the Concentration Versus Time Curve (AUC) of Midazolam in Plasma in the Absence and Presence of VX-828/TEZ/D-IVA

    Timeframe: From Day 1 up to Day 30

  1. Part A: Maximum Observed Concentration (Cmax) of VX-828 in Plasma

    Timeframe: From Day 1 up to Day 67

  2. Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-828 in Plasma

    Timeframe: From Day 1 up to Day 67

  3. Part B: Maximum Observed Concentration (Cmax) of VX-828 at Day 28 in Plasma

    Timeframe: From Day 1 up to Day 80

  4. Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-828 at Day 28 in Plasma

    Timeframe: From Day 1 up to Day 80

  5. Part C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Timeframe: From Signing of Informed Consent Form (ICF) up to Safety Follow Up (Up to Day 82)

  6. Part D: Maximum Observed Concentration (Cmax) of VX-828, TEZ and D-IVA and their Metabolites at Day 28 in Plasma

    Timeframe: Day 28

  7. Part D: Area Under the Concentration Versus Time Curve (AUC) of VX-828, TEZ and D-IVA and their Metabolites at Day 28 in Plasma

    Timeframe: Day 28

  8. Part E: Maximum Observed Concentration (Cmax) of VX-828, TEZ, and D-IVA and their Metabolites in Plasma

    Timeframe: Day 1 and Day 28

  9. Part E: Area Under the Concentration Versus Time Curve (AUC) of VX-828, TEZ, and D-IVA and their Metabolites in Plasma

    Timeframe: Day 28

  10. Part E: Pre-dose Plasma Concentration (Ctrough) of VX-828, TEZ, D-IVA and its Metabolites

    Timeframe: Pre-dose at Day 4, Day 8, Day 15, Day 22, Day 35, Day 49, Day 63, Day 80

  11. Part E: Absolute Change in Sweat Chloride

    Timeframe: From Baseline and At Day 28

Sources for outcomes
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Sex
All
Age groups
Adults, Older adults
Minimum age
18 Years
Healthy volunteers
Yes

Key Inclusion Criteria:

Parts A-D:

  • Participants between the ages of 18 and 55 years
  • Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m^2)
  • A total body weight of more than (>) 50 kg
  • Nonsmoker or ex-smoker for at least 3 months before screening with current nonsmoking status confirmed by urine or blood cotinine at screening
  • Cohort C2 only: Willing to provide a single DNA sample

Part E:

  • Participants 18 years or older
  • Confirmed diagnosis of CF as determined by the investigator
  • A total body weight of more than or equal to (>=) 35 kg
  • Participants must be heterozygous for F508del with a second CFTR allele carrying a minimal function mutation that is not responsive to ELX/TEZ/IVA therapy
  • Participants must have a forced expiratory volume in 1 second (FEV1) of greater than or equal to (≥) 40% of predicted normal for age, sex, and height

Key Exclusion Criteria:

Parts A-D:

  • History of febrile illness or other acute illness within 14 days before the first dose of study drug
  • Any condition possibly affecting drug absorption

Part E:

  • An acute illness not related to CF (e.g., gastroenteritis) within 14 days before the first dose of study drug
  • History of solid organ or hematological transplantation
  • History of clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria will apply.

Sources for eligibility
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  • Joe DiMaggio Cycstic Fibrosis & Pulmonary CenterHollywood, United States
  • AdventHealth Medical Group Pulmonology at Orlando RidgewoodOrlando, United States
  • Altasciences Clinical KansasOverland Park, United States
  • Kentucky Children's HospitalLexington, United States
  • Boston Children's HospitalBoston, United States
  • University of Minnesota -PulmonologyMinneapolis, United States
  • Billings Clinic, Pediatric Pulmonary Dept.Billings, United States
  • New York Medical CollegeHawthorne, United States
  • ProMedica Toledo Children's Hospital & ProMedica Central Physicians, LLCToledo, United States
  • Cook Children's PulmonologyFort Worth, United States
  • University of Utah Hospital - PulmonologySalt Lake City, United States
  • Vermont Lung CenterColchester, United States
Sources for sites
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Related records

Drugs (4)

Lead sponsors (1)

Conditions (1)

Targets (17)