Cystic FibrosisPhase 1/2NCT03768089
Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis
A Phase 1/2 study testing VX-121 (Suspension), TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 7 sites in 2 countries.
Trial summary
The purpose of this study is to evaluate safety and tolerability of VX-121 in healthy subjects and in subjects 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-121 (Suspension), TEZ/IVA and IVA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 115 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 20 Mar 2018to 3 May 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- 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
Who cannot join
- 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
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
Netherlands5 sites
Academic Medical Center
Amsterdam
PRA Health Sciences Onderzoekscentrum UMCG
Groningen
UMC St. Radboud
Nijmegen
Erasmus Medical Center
Rotterdam
HagaZiekenhuis van den Haag
The Hague
United Kingdom2 sites
Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital
Birmingham
The Medicines Evaluation Unit
Manchester
About this study
Official title: A Phase 1/2 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).
- Conditions
- Cystic Fibrosis
- Being tested
- Placebo (matched to VX-121 suspension), VX-121 (Suspension), TEZ/IVA, IVA, Placebo (matched to TEZ/IVA)
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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/IVADrug
Also called VX-661/VX-770, tezacaftor/ivacaftor
Fixed-dose combination tablet for oral administration.
IVADrug
Also called VX-770, ivacaftor
Tablet for oral administration.
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.
| Study group | Role | What they receive |
|---|---|---|
| Part A: Pooled Placebo (Cohorts A1-5; Except A3) | Placebo comparator | Participants received single dose of placebo matched to VX-121. Receives: Drug: Placebo (matched to VX-121 suspension) |
| Part A: VX-121 (Cohort A1) | Experimental | Participants received single dose of VX-121 10 milligrams (mg). Receives: Drug: VX-121 (Suspension) |
| Part A: VX-121 (Cohort A2) | Experimental | Participants received single dose of VX-121 20 mg. Receives: Drug: VX-121 (Suspension) |
| Part A: VX-121 (Cohort A3) | Experimental | Participants received single dose of VX-121 5 mg or matched placebo without milk, followed by open label VX-121 5 mg with milk. Receives: Drug: Placebo (matched to VX-121 suspension); Drug: VX-121 (Suspension) |
| Part A: VX-121 (Cohort A4) | Experimental | Participants received single dose of VX-121 40 mg. Receives: Drug: VX-121 (Suspension) |
| Part A: VX-121 (Cohort A5) | Experimental | Participants received single dose of VX-121 60 mg. Receives: Drug: VX-121 (Suspension) |
| Part A: VX-121 (Cohort A9) | Experimental | Participants received single dose of VX-121 10 mg suspension on Day 1, VX-121 10 mg tablet on Day 9, followed by VX-121 10 mg tablet with milk on Day 17. Receives: Drug: VX-121 (Suspension); Drug: VX-121 (Tablet) |
| Part B: Pooled Placebo (Cohorts B1-4) | Placebo comparator | Participants received placebo matched to VX-121 for 10 days. Receives: Drug: Placebo (matched to VX-121 suspension) |
| Part B: VX-121 (Cohort B1) | Experimental | Participants received VX-121 10 mg once daily (qd) for 10 days. Receives: Drug: VX-121 (Suspension) |
| Part B: VX-121 (Cohort B2) | Experimental | Participants received VX-121 20 mg qd for 10 days. Receives: Drug: VX-121 (Suspension) |
| Part B: VX-121 (Cohort B3) | Experimental | Participants received VX-121 40 mg qd for 10 days. Receives: Drug: VX-121 (Suspension) |
| Part B: VX-121 (Cohort B4) | Experimental | Participants received VX-121 60 mg qd for 10 days. Receives: Drug: VX-121 (Suspension) |
| Part C: Pooled Placebo (Cohorts C1-3) | Placebo comparator | Participants received placebo matched to VX-121/TEZ/IVA for 14 days. Receives: Drug: Placebo (matched to VX-121 suspension); Drug: Placebo (matched to TEZ/IVA); Drug: Placebo (matched to IVA) |
| Part C: VX-121 (Cohort C1) | Experimental | Participants received VX-121 10 mg qd/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) for 14 days. Receives: Drug: VX-121 (Suspension); Drug: TEZ/IVA; Drug: IVA |
| Part C: VX-121 (Cohort C2) | Experimental | Participants received VX-121 20 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. Receives: Drug: VX-121 (Suspension); Drug: TEZ/IVA; Drug: IVA |
| Part C: VX-121 (Cohort C3) | Experimental | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. Receives: Drug: VX-121 (Suspension); Drug: TEZ/IVA; Drug: IVA |
| Part D: Placebo | Placebo comparator | Participants received placebo matched to VX-121/TEZ/IVA for 4 weeks. Receives: Drug: Placebo (matched to TEZ/IVA); Drug: Placebo (matched to IVA); Drug: Placebo (matched to VX-121 tablet) |
| Part D: VX-121/TEZ/IVA | Experimental | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks. Receives: Drug: TEZ/IVA; Drug: IVA; Drug: VX-121 (Tablet) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: 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 (13)
Part A: Maximum Observed Concentration (Cmax) of VX-121
Measured: 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
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
Measured: 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
Part B: Maximum Observed Concentration (Cmax) of VX-121
Measured: Day 1, Day 5, and Day 10
Part B: Area Under the Concentration Versus Time Curve During the Dosing Interval (AUCtau) of VX-121
Measured: Day 1, Day 5, and Day 10
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121
Measured: Pre-dose at Day 5 and Day 10
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)
Measured: Day 1, Day 7, and Day 14
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)
Measured: Day 1, Day 7, and Day 14
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)
Measured: Pre-dose at Day 7 and Day 14
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)
Measured: Day 1 and Day 15
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)
Measured: Day 1 and Day 15
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)
Measured: Pre-dose at Day 8, Day 15, and Day 29
Part D: Absolute Change in Sweat Chloride (SwCl) Concentrations
Sweat samples were collected using an approved collection device.
Measured: From Baseline Through Day 29
Part D: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Measured: From Baseline Through Day 29
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2018
- Study start20 Mar 2018
- Primary completion3 May 2019
- Study completion3 May 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 115 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
- NCT03768089
- Sponsor's protocol ID
- VX17-121-001
- Other IDs
- 2018-000126-55, 2018
- Status last verified
- Jun 2022
- First submitted
- 5 Dec 2018
- Last updated
- 14 Jul 2022
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03768089
- Acronym / sponsor ID
- VX17-121-001
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 115 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 20 Mar 2018 (actual)
- Primary completion
- 3 May 2019 (actual)
- Study completion
- 3 May 2019 (actual)
- First posted
- 7 Dec 2018 (actual)
- Results
- Posted 14 Jul 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate safety and tolerability of VX-121 in healthy subjects and in subjects with cystic fibrosis (CF).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 18
| Arm | Type | Receives |
|---|---|---|
| Part A: Pooled Placebo (Cohorts A1-5; Except A3) | Placebo comparator | Drug: Placebo (matched to VX-121 suspension)Participants received single dose of placebo matched to VX-121. |
| Part A: VX-121 (Cohort A1) | Experimental | Drug: VX-121 (Suspension)Participants received single dose of VX-121 10 milligrams (mg). |
| Part A: VX-121 (Cohort A2) | Experimental | Drug: VX-121 (Suspension)Participants received single dose of VX-121 20 mg. |
| Part A: VX-121 (Cohort A3) | Experimental | Drug: Placebo (matched to VX-121 suspension), Drug: VX-121 (Suspension)Participants received single dose of VX-121 5 mg or matched placebo without milk, followed by open label VX-121 5 mg with milk. |
| Part A: VX-121 (Cohort A4) | Experimental | Drug: VX-121 (Suspension)Participants received single dose of VX-121 40 mg. |
| Part A: VX-121 (Cohort A5) | Experimental | Drug: VX-121 (Suspension)Participants received single dose of VX-121 60 mg. |
| Part A: VX-121 (Cohort A9) | Experimental | Drug: VX-121 (Suspension), Drug: VX-121 (Tablet)Participants received single dose of VX-121 10 mg suspension on Day 1, VX-121 10 mg tablet on Day 9, followed by VX-121 10 mg tablet with milk on Day 17. |
| Part B: Pooled Placebo (Cohorts B1-4) | Placebo comparator | Drug: Placebo (matched to VX-121 suspension)Participants received placebo matched to VX-121 for 10 days. |
| Part B: VX-121 (Cohort B1) | Experimental | Drug: VX-121 (Suspension)Participants received VX-121 10 mg once daily (qd) for 10 days. |
| Part B: VX-121 (Cohort B2) | Experimental | Drug: VX-121 (Suspension)Participants received VX-121 20 mg qd for 10 days. |
| Part B: VX-121 (Cohort B3) | Experimental | Drug: VX-121 (Suspension)Participants received VX-121 40 mg qd for 10 days. |
| Part B: VX-121 (Cohort B4) | Experimental | Drug: VX-121 (Suspension)Participants received VX-121 60 mg qd for 10 days. |
| Part C: Pooled Placebo (Cohorts C1-3) | Placebo comparator | Drug: Placebo (matched to VX-121 suspension), Drug: Placebo (matched to TEZ/IVA), Drug: Placebo (matched to IVA)Participants received placebo matched to VX-121/TEZ/IVA for 14 days. |
| Part C: VX-121 (Cohort C1) | Experimental | Drug: VX-121 (Suspension), Drug: TEZ/IVA, Drug: IVAParticipants received VX-121 10 mg qd/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) for 14 days. |
| Part C: VX-121 (Cohort C2) | Experimental | Drug: VX-121 (Suspension), Drug: TEZ/IVA, Drug: IVAParticipants received VX-121 20 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
| Part C: VX-121 (Cohort C3) | Experimental | Drug: VX-121 (Suspension), Drug: TEZ/IVA, Drug: IVAParticipants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
| Part D: Placebo | Placebo comparator | Drug: Placebo (matched to TEZ/IVA), Drug: Placebo (matched to IVA), Drug: Placebo (matched to VX-121 tablet)Participants received placebo matched to VX-121/TEZ/IVA for 4 weeks. |
| Part D: VX-121/TEZ/IVA | Experimental | Drug: TEZ/IVA, Drug: IVA, Drug: VX-121 (Tablet)Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Part A, B, and C: Healthy Volunteers
- Part D: Subjects with CF
Key exclusion criteria (8 listed)
- Part A, B and C: Healthy Volunteers
- Part D: Subjects with CF
- Other protocol defined Inclusion/Exclusion criteria may apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Placebo (matched to VX-121 suspension)Placebo matched to VX-121 suspension for oral administration. | Drug | Part A: Pooled Placebo (Cohorts A1-5; Except A3), Part A: VX-121 (Cohort A3), Part B: Pooled Placebo (Cohorts B1-4), Part C: Pooled Placebo (Cohorts C1-3) |
| VX-121 (Suspension)Suspension for oral administration. | Drug | Part A: VX-121 (Cohort A1), Part A: VX-121 (Cohort A2), Part A: VX-121 (Cohort A3), Part A: VX-121 (Cohort A4), Part A: VX-121 (Cohort A5), Part A: VX-121 (Cohort A9), Part B: VX-121 (Cohort B1), Part B: VX-121 (Cohort B2), Part B: VX-121 (Cohort B3), Part B: VX-121 (Cohort B4), Part C: VX-121 (Cohort C1), Part C: VX-121 (Cohort C2), Part C: VX-121 (Cohort C3) |
| TEZ/IVAAlso: VX-661/VX-770, tezacaftor/ivacaftorFixed-dose combination tablet for oral administration. | Drug | Part C: VX-121 (Cohort C1), Part C: VX-121 (Cohort C2), Part C: VX-121 (Cohort C3), Part D: VX-121/TEZ/IVA |
| IVAAlso: VX-770, ivacaftorTablet for oral administration. | Drug | Part C: VX-121 (Cohort C1), Part C: VX-121 (Cohort C2), Part C: VX-121 (Cohort C3), Part D: VX-121/TEZ/IVA |
| Placebo (matched to TEZ/IVA)Placebo matched to TEZ/IVA for oral administration. | Drug | Part C: Pooled Placebo (Cohorts C1-3), Part D: Placebo |
| Placebo (matched to IVA)Placebo matched to IVA for oral administration. | Drug | Part C: Pooled Placebo (Cohorts C1-3), Part D: Placebo |
| VX-121 (Tablet)Tablet for oral administration. | Drug | Part A: VX-121 (Cohort A9), Part D: VX-121/TEZ/IVA |
| Placebo (matched to VX-121 tablet)Placebo matched to VX-121 tablet for oral administration. | Drug | Part D: Placebo |
Primary outcome
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)
13 secondary outcomes
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
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
Part B: Maximum Observed Concentration (Cmax) of VX-121
Time frame: Day 1, Day 5, and Day 10
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
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121
Time frame: Pre-dose at Day 5 and Day 10
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
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
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
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
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
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
Part D: Absolute Change in Sweat Chloride (SwCl) Concentrations
Time frame: From Baseline Through Day 29
Part D: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: From Baseline Through Day 29
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Cystic Fibrosis
RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VX-121 (Suspension), TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 7 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Netherlands, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Jul 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2022.