Cystic FibrosisPhase 1NCT06154447
Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis
A Phase 1 study testing VX-828, Itraconazole and Midazolam in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 12 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- VX-828, Itraconazole and Midazolam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 165 participants
- Where
- 12 sitesUnited States
- Study dates
- 12 Dec 2023to 10 Jul 2026
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Billings Clinic, Pediatric Pulmonary Dept.
Billings, Montana, 59101
Boston Children's Hospital
Boston, Massachusetts, 02115
Vermont Lung Center
Colchester, Vermont, 05446
Cook Children's Pulmonology
Fort Worth, Texas, 76104
New York Medical College
Hawthorne, New York, 10532
Joe DiMaggio Cycstic Fibrosis & Pulmonary Center
Hollywood, Florida, 33021
Kentucky Children's Hospital
Lexington, Kentucky, 40508
University of Minnesota -Pulmonology
Minneapolis, Minnesota, 55455
AdventHealth Medical Group Pulmonology at Orlando Ridgewood
Orlando, Florida, 32803
Altasciences Clinical Kansas
Overland Park, Kansas, 66212
University of Utah Hospital - Pulmonology
Salt Lake City, Utah, 84132
ProMedica Toledo Children's Hospital & ProMedica Central Physicians, LLC
Toledo, Ohio, 43606
About this study
Official title: A Phase 1, Study of VX-828 in Healthy Subjects and in Subjects With Cystic Fibrosis
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).
Read the detailed description
This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).
- Conditions
- Cystic Fibrosis
- Being tested
- VX-828, Placebo, Itraconazole, Midazolam, Tezacaftor
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-828Drug
Suspension for Oral Administration
PlaceboDrug
Suspension for Oral Administration
ItraconazoleDrug
Solution for Oral Administration
MidazolamDrug
Syrup for Oral Administration
TezacaftorDrug
Also called TEZ, VX-661
Tablets for Oral Administration
VX-118Drug
Tablets for Oral Administration
PlaceboDrug
Suspension and Tablets for Oral Administration
DeutivacaftorDrug
Also called D-IVA, VX-561
Tablets for Oral Administration
VX-828Drug
Tablets for Oral Administration
| Study group | Role | What they receive |
|---|---|---|
| Part A: Single Ascending Dose (SAD) | Experimental | Participants will be randomized to receive a single dose of different dose levels of VX-828. Receives: Drug: VX-828 |
| Part A: Placebo | Placebo comparator | Participants will be randomized to receive placebo matched to VX-828. Receives: Drug: Placebo |
| Part B: Multiple Ascending Dose (MAD) | Experimental | Participants will be randomized to receive multiple doses of different dose levels of VX-828. The dose levels will be determined based on the data from Part A. Receives: Drug: VX-828 |
| Part B: Placebo | Placebo comparator | Participants will be randomized to receive placebo matched to VX-828. Receives: Drug: Placebo |
| Part C: Drug Drug Interaction | Experimental | Participants will receive a single dose of VX-828, followed by a washout period, Itraconazole administration, and concomitant administration of itraconazole and VX-828; or participants will receive Midazolam followed by VX-828/TEZ/D-IVA administration, and concomitant administration of VX-828/TEZ/D-IVA and Midazolam.
Part C will be an open-label optional cohort. Receives: Drug: VX-828; Drug: Itraconazole; Drug: Midazolam; Drug: Tezacaftor; Drug: Deutivacaftor |
| Part D: VX-828 in combination with TEZ/VX-118 ,TEZ/D-IVA or D-IVA | Experimental | Participants will be randomized to receive VX-828 in combination with TEZ/VX-118, TEZ/D-IVA, or D-IVA. Receives: Drug: VX-828; Drug: Tezacaftor; Drug: VX-118; Drug: Deutivacaftor |
| Part D: Placebo | Placebo comparator | Participants will be randomized to receive placebo matched to VX-828/TEZ/VX-118 or placebo matched to VX-828 in combination with TEZ/D-IVA or D-IVA Receives: Drug: Tezacaftor; Drug: Placebo; Drug: Deutivacaftor |
| Part E: VX-828 in Combination with D-IVA with or without TEZ in CF | Experimental | Participants with cystic fibrosis will receive VX-828 in combination with D-IVA with or without TEZ. Receives: Drug: Tezacaftor; Drug: Deutivacaftor; Drug: VX-828 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From Signing of Informed Consent Form (ICF) up to Safety Follow Up (Up to Day 67)
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From Signing of Informed Consent Form (ICF) up to Safety Follow Up (Up to Day 80)
Part D: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From Signing of Informed Consent Form (ICF) up to Safety Follow Up (Up to Day 80)
Part E: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From Signing of Informed Consent Form (ICF) up to End of Study (Up to Day 111)
Part C: Maximum Observed Concentration (Cmax) of VX-828 in Plasma in the Absence and Presence of Itraconazole
Measured: From Day 1 up to Day 71
Part C: Area Under the Concentration Versus Time Curve (AUC) of VX-828 in Plasma in the Absence and Presence of Itraconazole
Measured: From Day 1 up to Day 71
Part C: Maximum Observed Concentration (Cmax) of Midazolam in Plasma in the Absence and Presence of VX-828/TEZ/D-IVA
Measured: From Day 1 up to Day 30
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
Measured: From Day 1 up to Day 30
Secondary outcomes (11)
Part A: Maximum Observed Concentration (Cmax) of VX-828 in Plasma
Measured: From Day 1 up to Day 67
Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-828 in Plasma
Measured: From Day 1 up to Day 67
Part B: Maximum Observed Concentration (Cmax) of VX-828 at Day 28 in Plasma
Measured: From Day 1 up to Day 80
Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-828 at Day 28 in Plasma
Measured: From Day 1 up to Day 80
Part C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From Signing of Informed Consent Form (ICF) up to Safety Follow Up (Up to Day 82)
Part D: Maximum Observed Concentration (Cmax) of VX-828, TEZ and D-IVA and their Metabolites at Day 28 in Plasma
Measured: Day 28
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
Measured: Day 28
Part E: Maximum Observed Concentration (Cmax) of VX-828, TEZ, and D-IVA and their Metabolites in Plasma
Measured: Day 1 and Day 28
Part E: Area Under the Concentration Versus Time Curve (AUC) of VX-828, TEZ, and D-IVA and their Metabolites in Plasma
Measured: Day 28
Part E: Pre-dose Plasma Concentration (Ctrough) of VX-828, TEZ, D-IVA and its Metabolites
Measured: Pre-dose at Day 4, Day 8, Day 15, Day 22, Day 35, Day 49, Day 63, Day 80
Part E: Absolute Change in Sweat Chloride
Measured: From Baseline and At Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2023
- Study start12 Dec 2023
- Primary completion10 Jul 2026
- Study completion10 Jul 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 165 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06154447
- Sponsor's protocol ID
- VX23-828-001
- Status last verified
- Aug 2026
- First submitted
- 23 Nov 2023
- Last updated
- 10 Aug 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/