Cystic FibrosisPhase 2NCT01685801

Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function

A Phase 2 study testing Ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This study is a multiple within participant crossover study to evaluate the effect of ivacaftor on lung function in participants aged 12 years and older with cystic fibrosis (CF) who have phenotypic or molecular evidence of residual CF transmembrane conductance regulator (CFTR) function. Read more about this study

Who is it for?: Ages 12 years and older
Conditions the study is for: Cystic Fibrosis
What is being tested?: Ivacaftor
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 24 participants
Where: 1 siteUnited States
Study dates: Sep 2012to Apr 2014
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 12 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Male or female participants with confirmed diagnosis of CF
Clinical evidence of residual CFTR function based on any 1 of the following: 1) Clinically documented residual exocrine pancreatic function, 2) Sweat chloride value less than equal to (<=) 80 millimole per liter (mmol/L) at screening, or 3) Age of diagnosis greater than equal to (>=) 12 years and at least 1 copy of a CFTR mutation associated with residual CFTR function or defective mRNA splicing
FEV1 >= 40 percent (%)
12 years of age or older
Willing to agree to meet the contraception requirements
Able to swallow tablets

Who cannot join

A copy of any of the following CFTR mutations: G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, or G1349D
Unable to perform spirometry
An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days prior to screening

Trial locations and contacts

1 site in 1 country.

United States1 site

Study site
Denver, Colorado

Other trials in United States

About this study

Official title: A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 ≥40% Predicted

Read the detailed description

CFTR Mutations associated with residual function or defective messenger ribonucleic acid (mRNA) splicing include the following:

R117H, E56K, P67L, D110E, D110H, R117C, R347H, R352Q, A455E, D579G, S945L, L206W, R1070W, F1074L, D1152H, S1235R, D1270N, 2789+5G->A, 3849+10kbC->T, 3272-26A->G, 711+5G->A, 3120G->A, 1811+1.6kbA->G, 711+3A->G, 1898+3A->G, 1898+1G->A, 1717-1G->A, 1717-8G->A, 1342-2A->C, 405+3A->C, 1716G/A 1811+1G->C, 1898+5G->T, 3850-3T->G, IVS14b+5G->A, 1898+1G->T, 4005+2T->C, 621+3A->G, 621+1G->T.

Conditions: Cystic Fibrosis
Being tested: Ivacaftor, Placebo-matched-to-ivacaftor tablet
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

IvacaftorDrug
Also called Kalydeco, VX-770
150 milligram (mg) tablet orally every 12 hours up to 12 weeks.
Placebo-matched-to-ivacaftor tabletDrug
Orally every 12 hours up to 4 weeks.

Study groups in NCT01685801
Study group	Role	What they receive
Ivacaftor, Placebo, Ivacaftor, Placebo (IPIP)	Experimental	During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. Receives: Drug: Ivacaftor; Drug: Placebo-matched-to-ivacaftor tablet
Ivacaftor, Placebo, Placebo, Ivacaftor (IPPI)	Experimental	During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. Receives: Drug: Ivacaftor; Drug: Placebo-matched-to-ivacaftor tablet
Placebo, Ivacaftor, Ivacaftor, Placebo (PIIP)	Experimental	During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. Receives: Drug: Ivacaftor; Drug: Placebo-matched-to-ivacaftor tablet
Placebo, Ivacaftor, Placebo, Ivacaftor (PIPI)	Experimental	During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. Receives: Drug: Ivacaftor; Drug: Placebo-matched-to-ivacaftor tablet

What is being measured

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

Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) After 2 Weeks of Treatment
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment, for overall participants and as per genotype (residual function mutation and mRNA splice site mutation).
Measured: Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms)

Secondary outcomes (6)

Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Lung Clearance Index (LCI) After 2 Weeks of Treatment
LCI is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity [FRC]) required to reduce end-tidal concentration of an inert gas to 1/40th of the starting value. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment, for overall participants and as per genotype (residual function mutation and mRNA splice site mutation).
Measured: Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms)
Open-label Period: Absolute Change From Open-label Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) at Day 57
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation).
Measured: Open-label Baseline, Open-label Day 57
Open-label Period: Absolute Change From Open-label Baseline In Lung Clearance Index (LCI) at Day 57
LCI is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity [FRC]) required to reduce end-tidal concentration of an inert gas to 1/40th of the starting value. Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation).
Measured: Open-label Baseline, Open-label Day 57
Open-label Period: Absolute Change From Study Baseline In Sweat Chloride at Day 57
Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation).
Measured: Study Baseline, Open-label Day 57
Open-label Period: Absolute Change From Open-label Baseline In Weight at Day 57
Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation).
Measured: Open-label Baseline, Open-label Day 57
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Adverse events (AEs) that started (or increased in severity) from first dose of study drug through completion of Follow-up were considered TEAEs, with exception that if an AE started during a Washout Period and was beyond 14 days from last dose date of preceding cycle, AE was considered as a "Washout Period" AE, and hence not TEAE. A TEAE was attributed to treatment in which it started or to the treatment in second cycling period of previous Crossover Period if it started during a Washout Period. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Data was to be reported by drug treatment for double-blind crossover period (Cycle 1 up to Washout Period 2) and open-label period.
Measured: From first dose of study drug through completion of follow-up visit (up to 26 weeks)

Timeline

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

Sep 2012Apr 2014

First posted14 Sep 2012 (estimated)
Study startSep 2012
Primary completionApr 2014
Study completionApr 2014

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: Crossover
Masking: Double blind
Who is masked: Participant, Care provider
Enrolment: 24 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01685801
Sponsor's protocol ID: VX12-770-113
Status last verified: Apr 2015
First submitted: 6 Sep 2012
Last updated: 19 May 2015
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Nick JA, St Clair C, Jones MC, Lan L, Higgins M; VX12-770-113 Study Team. Ivacaftor in cystic fibrosis with residual function: Lung function results from an N-of-1 study. J Cyst Fibros. 2020 Jan;19(1):91-98. doi: 10.1016/j.jcf.2019.09.013. Epub 2019 Nov 26.
PubMed 31784217

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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All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 19 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 ≥40% Predicted
ClinicalTrials.gov ID: NCT01685801
Acronym / sponsor ID: VX12-770-113
Conditions: Cystic Fibrosis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 24 participants (actual)
Lead sponsor: Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
Study start: Sep 2012
Primary completion: Apr 2014 (actual)
Study completion: Apr 2014 (actual)
First posted: 14 Sep 2012 (estimated)
Results: Posted 19 May 2015

Clinical summary

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

Detailed description

CFTR Mutations associated with residual function or defective messenger ribonucleic acid (mRNA) splicing include the following:

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Double blind (participant, care provider)
Number of arms: 4

Arm	Type	Receives
Ivacaftor, Placebo, Ivacaftor, Placebo (IPIP)	Experimental	Drug: Ivacaftor, Drug: Placebo-matched-to-ivacaftor tabletDuring the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor.
Ivacaftor, Placebo, Placebo, Ivacaftor (IPPI)	Experimental	Drug: Ivacaftor, Drug: Placebo-matched-to-ivacaftor tabletDuring the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor.
Placebo, Ivacaftor, Ivacaftor, Placebo (PIIP)	Experimental	Drug: Ivacaftor, Drug: Placebo-matched-to-ivacaftor tabletDuring the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor.
Placebo, Ivacaftor, Placebo, Ivacaftor (PIPI)	Experimental	Drug: Ivacaftor, Drug: Placebo-matched-to-ivacaftor tabletDuring the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor.

Who may qualify

Ages: 12 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (6 listed)

Male or female participants with confirmed diagnosis of CF
Clinical evidence of residual CFTR function based on any 1 of the following: 1) Clinically documented residual exocrine pancreatic function, 2) Sweat chloride value less than equal to (<=) 80 millimole per liter (mmol/L) at screening, or 3) Age of diagnosis greater than equal to (>=) 12 years and at least 1 copy of a CFTR mutation associated with residual CFTR function or defective mRNA splicing
FEV1 >= 40 percent (%)
12 years of age or older
Willing to agree to meet the contraception requirements

Key exclusion criteria (4 listed)

A copy of any of the following CFTR mutations: G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, or G1349D
Unable to perform spirometry
An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days 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
IvacaftorAlso: Kalydeco, VX-770150 milligram (mg) tablet orally every 12 hours up to 12 weeks.	Drug	Ivacaftor, Placebo, Ivacaftor, Placebo (IPIP), Ivacaftor, Placebo, Placebo, Ivacaftor (IPPI), Placebo, Ivacaftor, Ivacaftor, Placebo (PIIP), Placebo, Ivacaftor, Placebo, Ivacaftor (PIPI)
Placebo-matched-to-ivacaftor tabletOrally every 12 hours up to 4 weeks.	Drug	Ivacaftor, Placebo, Ivacaftor, Placebo (IPIP), Ivacaftor, Placebo, Placebo, Ivacaftor (IPPI), Placebo, Ivacaftor, Ivacaftor, Placebo (PIIP), Placebo, Ivacaftor, Placebo, Ivacaftor (PIPI)

Primary outcome

Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) After 2 Weeks of Treatment
Time frame: Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment, for overall participants and as per genotype (residual function mutation and mRNA splice site mutation).

6 secondary outcomes

Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Lung Clearance Index (LCI) After 2 Weeks of Treatment
Time frame: Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms)
Open-label Period: Absolute Change From Open-label Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) at Day 57
Time frame: Open-label Baseline, Open-label Day 57
Open-label Period: Absolute Change From Open-label Baseline In Lung Clearance Index (LCI) at Day 57
Time frame: Open-label Baseline, Open-label Day 57
Open-label Period: Absolute Change From Study Baseline In Sweat Chloride at Day 57
Time frame: Study Baseline, Open-label Day 57
Open-label Period: Absolute Change From Open-label Baseline In Weight at Day 57
Time frame: Open-label Baseline, Open-label Day 57
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: From first dose of study drug through completion of follow-up visit (up to 26 weeks)

Results and safety

Results posted to the registry on 19 May 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Ivacaftor, Placebo, Ivacaftor, Placebo (IPIP)	5	5
Ivacaftor, Placebo, Placebo, Ivacaftor (IPPI)	6	6
Placebo, Ivacaftor, Ivacaftor, Placebo (PIIP)	7	7
Placebo, Ivacaftor, Placebo, Ivacaftor (PIPI)	6	6

Primary outcome: Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) After 2 Weeks of Treatment

Time frame: Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms). Mean (Standard Deviation). Unit: Percent predicted of FEV1

Measure	Cycle 1: Placebo n = 24	Cycle 1: Ivacaftor n = 24	Cycle 2: Placebo n = 23	Cycle 2: Ivacaftor n = 23
Overall (n=24, 24, 23, 23)	0.5828 (3.67326)	2.0898 (4.63833)	0.8495 (4.20641)	3.6868 (6.13466)
mRNA Splice Site(ClassV)Mutations (n=10, 10, 9, 9)	-0.5710 (3.39739)	0.8196 (5.09161)	1.7132 (3.80933)	2.0640 (3.55493)
Residual Function Mutations (n=14, 14, 14, 14)	1.4069 (3.75842)	2.9971 (4.24124)	0.2942 (4.49056)	4.7300 (7.27436)

Cycle 1: Placebo vs Cycle 1: Ivacaftor vs Cycle 2: Placebo vs Cycle 2: Ivacaftor: Posterior Mean: 2.251 (95% CI 0.383 to 4.144)

Population analysed: Full analysis set (FAS) population. Here, number of participants analyzed signifies participant evaluable for this outcome measure and "n" signifies participants who were evaluable for the specified category in each arm, respectively.

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: From first dose of study drug through completion of follow-up visit (up to 26 weeks). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Crossover Double-blind Period: Placebo	0 of 24 (0%)	17 of 24 (70.8%)	Not reported
Crossover Double-blind Period: Ivacaftor	1 of 24 (4.2%)	18 of 24 (75%)	Not reported
Open-label Period: Ivacaftor	0 of 21 (0%)	20 of 21 (95.2%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2015.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 31784217Nick JA, St Clair C, Jones MC, Lan L, Higgins M; VX12-770-113 Study Team. Ivacaftor in cystic fibrosis with residual function: Lung function results from an N-of-1 study. J Cyst Fibros. 2020 Jan;19(1):91-98. doi: 10.1016/j.jcf.2019.09.013. Epub 2019 Nov 26.

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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.

Jerry Nick, MD principal investigator

National Jewish Health

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 1 site 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 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 19 May 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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. 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 19 May 2015). 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?

Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) After 2 Weeks of Treatment (time frame: Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms)). 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 NCT01685801, 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 19 May 2015.