Cystic FibrosisPhase 2NCT03911713
A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
A Phase 2 study testing VX-561 and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 48 sites in 7 countries.
Trial summary
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- VX-561 and IVA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 77 participants
- Where
- 48 sitesin 7 countries
- Study dates
- 17 Apr 2019to 20 Aug 2020
- Phase
- Phase 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
- On ivacaftor therapy
- FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height
Who cannot join
- 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
48 sites in 7 countries.
48 sites in 7 countries
United States30 sites
Michigan Medicine
Ann Arbor, Michigan, 48109-5212
Dell Children's Medical Group
Austin, Texas, 78723
Johns Hopkins Hospital
Baltimore, Maryland, 21287
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
Boston, Massachusetts, 02114
Boston Children's Hospital
Boston, Massachusetts, 02115
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27514
Medical University of South Carolina
Charleston, South Carolina, 29425
UC Health Holmes
Cincinnati, Ohio, 45220
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390
National Jewish Health
Denver, Colorado, 80206
Indiana University
Indianapolis, Indiana, 46202
University of Kansas Medical Center
Kansas City, Kansas, 66160
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
University of Kentucky
Lexington, Kentucky, 40536
Miller Children's Hospital / Long Beach Memorial
Long Beach, California, 90806
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792
University of Miami Miller School of Medicine
Miami, Florida, 33136
University of Minnesota
Minneapolis, Minnesota, 55455
Columbia University Medical Center
New York, New York, 10032
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Central Florida Pulmonary Group, PA
Orlando, Florida, 32803
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, 84132
UCSF Gateway Medical Center
San Francisco, California, 94143
Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, 63110
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
Toledo, Ohio, 43606
Banner University of Arizona Medical Center
Tucson, Arizona, 85724
Germany5 sites
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
Erlangen
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen
Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
Halle
Pneumologisches Studienzentrum Muenchen-West
München
University Hospital Wuerzburg
Würzburg
Australia4 sites
The Prince Charles Hospital
Chermside
Alfred Hospital
Melbourne, VIC
Mater Adult Hospital
South Brisbane
Westmead Hospital
Westmead
Ireland4 sites
Beaumont Hospital
Dublin
Cork University Hospital
Dublin
St. Vincent's University Hospital
Dublin
University Hospital Limerick (Adults)
Limerick
United Kingdom3 sites
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow
St. James University Hospital
Leeds
Liverpool Heart and Chest Hospital
Liverpool
About this study
Official title: A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
- Conditions
- Cystic Fibrosis
- Being tested
- VX-561, IVA, Placebo
- 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-561Drug
Also called CTP-656, Deutivacaftor (D-IVA)
VX-561 tablets for oral administration.
IVADrug
Also called VX-770, Ivacaftor
150-mg film-coated tablet for oral administration.
PlaceboDrug
Placebo matched to IVA.
PlaceboDrug
Placebos matched to VX-561.
| Study group | Role | What they receive |
|---|---|---|
| Ivacaftor | Active comparator | Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks. Receives: Drug: IVA; Drug: Placebo |
| VX-561: 25 mg | Experimental | Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks. Receives: Drug: VX-561; Drug: Placebo |
| VX-561: 50 mg | Experimental | Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks. Receives: Drug: VX-561; Drug: Placebo |
| VX-561: 150 mg | Experimental | Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks. Receives: Drug: VX-561; Drug: Placebo |
| VX-561: 250 mg | Experimental | Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks. Receives: Drug: VX-561; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 at Week 12
Secondary outcomes (3)
Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Measured: From Baseline at Week 12
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
Measured: At Week 4
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Baseline up to Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2019
- Study start17 Apr 2019
- Primary completion20 Aug 2020
- Study completion20 Aug 2020
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 77 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
- NCT03911713
- Sponsor's protocol ID
- VX18-561-101
- Other IDs
- 2018-003970-28, 2018
- Status last verified
- Dec 2021
- First submitted
- 9 Apr 2019
- Last updated
- 25 Jan 2022
- Results posted
- Yes, on ClinicalTrials.gov
- 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/independent-research/clinical-trial-data-sharing
Publications
Uluer AZ, MacGregor G, Azevedo P, Indihar V, Keating C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Rubenstein RC, Taylor-Cousar JL, Tullis E, Yonker LM, Chu C, Lam AP, Nair N, Sosnay PR, Tian S, Van Goor F, Viswanathan L, Waltz D, Wang LT, Xi Y, Billings J, Horsley A; VX18-121-101; VX18-561-101 Study Groups. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials. Lancet Respir Med. 2023 Jun;11(6):550-562. doi: 10.1016/S2213-2600(22)00504-5. Epub 2023 Feb 23.
PubMed 36842446
Study at a glance
From the registry record, last updated by the sponsor on 25 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03911713
- Acronym / sponsor ID
- VX18-561-101
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 77 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 17 Apr 2019 (actual)
- Primary completion
- 20 Aug 2020 (actual)
- Study completion
- 20 Aug 2020 (actual)
- First posted
- 11 Apr 2019 (actual)
- Results
- Posted 25 Jan 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 the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Ivacaftor | Active comparator | Drug: IVA, Drug: PlaceboParticipants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks. |
| VX-561: 25 mg | Experimental | Drug: VX-561, Drug: PlaceboParticipants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks. |
| VX-561: 50 mg | Experimental | Drug: VX-561, Drug: PlaceboParticipants received VX-561 50 mg orally qd in the treatment period for 12 weeks. |
| VX-561: 150 mg | Experimental | Drug: VX-561, Drug: PlaceboParticipants received VX-561 150 mg orally qd in the treatment period for 12 weeks. |
| VX-561: 250 mg | Experimental | Drug: VX-561, Drug: PlaceboParticipants received VX-561 250 mg orally qd in the treatment period for 12 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
- On ivacaftor therapy
- FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height
Key exclusion criteria (4 listed)
- 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
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 |
|---|---|---|
| VX-561Also: CTP-656, Deutivacaftor (D-IVA)VX-561 tablets for oral administration. | Drug | VX-561: 150 mg, VX-561: 25 mg, VX-561: 250 mg, VX-561: 50 mg |
| IVAAlso: VX-770, Ivacaftor150-mg film-coated tablet for oral administration. | Drug | Ivacaftor |
| PlaceboPlacebo matched to IVA. | Drug | VX-561: 150 mg, VX-561: 25 mg, VX-561: 250 mg, VX-561: 50 mg |
| PlaceboPlacebos matched to VX-561. | Drug | Ivacaftor |
Primary outcome
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: From Baseline at Week 12
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
3 secondary outcomes
Absolute Change in Sweat Chloride (SwCl)
Time frame: From Baseline at Week 12
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
Time frame: At Week 4
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to Week 16
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jan 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36842446Uluer AZ, MacGregor G, Azevedo P, Indihar V, Keating C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Rubenstein RC, Taylor-Cousar JL, Tullis E, Yonker LM, Chu C, Lam AP, Nair N, Sosnay PR, Tian S, Van Goor F, Viswanathan L, Waltz D, Wang LT, Xi Y, Billings J, Horsley A; VX18-121-101; VX18-561-101 Study Groups. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials. Lancet Respir Med. 2023 Jun;11(6):550-562. doi: 10.1016/S2213-2600(22)00504-5. Epub 2023 Feb 23.
- Individual participant dataSharing planned: No. Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
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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.
48 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VX-561 and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 48 sites in 7 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 3 inclusion and 4 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 25 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 48 sites in United States, Germany, Australia, Ireland, United Kingdom and 2 more countries. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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 25 Jan 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?
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) (time frame: From Baseline at Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03911713, which also lists 1 linked publication.
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 25 Jan 2022.