Cystic FibrosisPhase 2NCT02971839

Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations

A Phase 2 study testing VX-561 and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 14 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation. 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
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 11 participants
Where: 14 sitesUnited States
Study dates: Dec 2016to Aug 2017
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

18 years of age or older
Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R.
Has been stable on Kalydeco therapy for at least 3 months prior to screening
Has FEV1 ≥ 60% of predicted normal for age, sex, and height at screening and baseline (Day 1) assessments
Weighs at least 40 kg at screening
Patients of either gender and women of childbearing potential must be willing to use a medically highly effective form of birth control during the treatment period and 30 days after the last dose of study treatment.

Who cannot join

Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment
Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes
History of hepatitis C or chronic active hepatitis B infection
History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening
Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening
Abnormal liver function

Show all 11Show fewer

History of abnormal renal function
History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females
History of solid organ or hematological transplantation
Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening
Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose

Trial locations and contacts

14 sites in 1 country.

City, postcode or hospital

14 sites in 1 country

United States14 sites

Boston Children's Hospital
Boston, Massachusetts
Rush University
Chicago, Illinois
Cincinnati Children's Hospital
Cincinnati, Ohio
Nationwide Children's Hospital
Columbus, Ohio
Indiana University
Indianapolis, Indiana
University of Southern California
Los Angeles, California
University of Miami
Miami, Florida
Atlantic Health
Morristown, New Jersey
Stanford Hospital
Palo Alto, California
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania
Washington University
St Louis, Missouri
New York Medical College
Valhalla, New York
Children's National Health
Washington D.C., District of Columbia
University of Massachusetts
Worcester, Massachusetts

Other trials in United States

About this study

Official title: A Phase 2, Randomized, Parallel-Group, Double-Blind, Placebo Controlled Study to Evaluate the Safety and Efficacy of CTP-656 With an Open-Label Active Comparator in Patients With Cystic Fibrosis With CFTR Gating Mutations.

This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.

Read the detailed description

This is a randomized, parallel-group, double-blind, placebo controlled multicenter study to evaluate the safety and efficacy of CTP-656 in CF patients with CFTR gating mutations, compared to Kalydeco, for a total of 28 days. Subjects will be randomized to receive either double-blind CTP-656 or placebo, or open-label Kalydeco.

Conditions: Cystic Fibrosis
Being tested: VX-561, Placebo, 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.

VX-561Drug
Also called CTP-656
PlaceboDrug
IVADrug
Also called ivacaftor; VX-770, Kalydeco

Study groups in NCT02971839
Study group	Role	What they receive
VX-561 20 mg	Experimental	Not described Receives: Drug: VX-561
VX-561 100 mg	Experimental	Not described Receives: Drug: VX-561
VX-561 150 mg	Experimental	Not described Receives: Drug: VX-561
Ivacaftor	Active comparator	Not described Receives: Drug: IVA
Placebo	Placebo comparator	Not described Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in Sweat Chloride at Day 28
Measured: From baseline at Day 28

Secondary outcomes (2)

Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
Measured: From baseline at Day 28
Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28
Measured: From baseline at Day 28

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2016Aug 2017

First posted23 Nov 2016 (estimated)
Study startDec 2016
Primary completionAug 2017
Study completionAug 2017

Why it stopped: Decision by Sponsor.

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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 11 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02971839
Sponsor's protocol ID: CP656.2001
Status last verified: Aug 2020
First submitted: 21 Nov 2016
Last updated: 26 Aug 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 26 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Randomized, Parallel-Group, Double-Blind, Placebo Controlled Study to Evaluate the Safety and Efficacy of CTP-656 With an Open-Label Active Comparator in Patients With Cystic Fibrosis With CFTR Gating Mutations.
ClinicalTrials.gov ID: NCT02971839
Acronym / sponsor ID: CP656.2001
Conditions: Cystic Fibrosis
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Decision by Sponsor.
Enrolment: 11 participants (actual)
Lead sponsor: Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
Study start: Dec 2016 (actual)
Primary completion: Aug 2017 (actual)
Study completion: Aug 2017 (actual)
First posted: 23 Nov 2016 (estimated)
Results: Posted 26 Aug 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.

Detailed description

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
VX-561 20 mg	Experimental	Drug: VX-561
VX-561 100 mg	Experimental	Drug: VX-561
VX-561 150 mg	Experimental	Drug: VX-561
Ivacaftor	Active comparator	Drug: IVA
Placebo	Placebo comparator	Drug: Placebo

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (6 listed)

18 years of age or older
Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R.
Has been stable on Kalydeco therapy for at least 3 months prior to screening
Has FEV1 ≥ 60% of predicted normal for age, sex, and height at screening and baseline (Day 1) assessments
Weighs at least 40 kg at screening

Key exclusion criteria (11 listed)

Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment
Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes
History of hepatitis C or chronic active hepatitis B infection
History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening
Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening

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	Drug	VX-561 100 mg, VX-561 150 mg, VX-561 20 mg
Placebo	Drug	Placebo
IVAAlso: ivacaftor; VX-770, Kalydeco	Drug	Ivacaftor

Primary outcome

Change From Baseline in Sweat Chloride at Day 28
Time frame: From baseline at Day 28

2 secondary outcomes

Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
Time frame: From baseline at Day 28
Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28
Time frame: From baseline at Day 28

Results and safety

Results posted to the registry on 26 Aug 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
VX-561 20 mg	2	2
VX-561 100 mg	2	2
VX-561 150 mg	2	2
Placebo	2	2
Ivacaftor	3	3

Primary outcome: Change From Baseline in Sweat Chloride at Day 28

Time frame: From baseline at Day 28

Population analysed: As pre-specified in SAP section 4.13 (Changes in Conduct or Planned Analyses), due to the limited number of participants being enrolled in the study, model-based analyses and summary statistics plan were not performed. Therefore, no efficacy summary is provided for this outcome measure.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Day 1 up to Day 35. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
VX-561 20 mg	0 of 2 (0%)	2 of 2 (100%)	0 of 2
VX-561 100 mg	0 of 2 (0%)	2 of 2 (100%)	0 of 2
VX-561 150 mg	0 of 2 (0%)	1 of 2 (50%)	0 of 2
Placebo	0 of 2 (0%)	2 of 2 (100%)	0 of 2
Ivacaftor	0 of 3 (0%)	1 of 3 (33.3%)	0 of 3

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

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Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
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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.

14 sites in 1 country: 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 14 sites in United States.

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 6 inclusion and 11 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: stopped early, as of the sponsor's last update on 26 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in United States. 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 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 26 Aug 2020). 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?

Change From Baseline in Sweat Chloride at Day 28 (time frame: From baseline at Day 28). 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 NCT02971839.

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 26 Aug 2020.