A Phase 3 interventional study of Ivacaftor in Cystic Fibrosis, sponsored by Medical University of South Carolina. Completed. Open to female participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-08-06.
Sponsored by Medical University of South Carolina · Phase 3, Interventional, and Treatment
This postmarketing N of 2 study is designed to evaluate the efficacy and safety of open-label ivacaftor treatment in two sisters with cystic fibrosis and pancreatic sufficiency.
The two sisters have a splicing mutation that is predicted to respond favorably to ivacaftor therapy. In addition to measurement of usual clinical outcomes (i.e. lung function, nutritional status), there is great interest on the impact on nontuberculous mycobacteria (NTM) airways infection.
Subjects will undergo sputum cultures at baseline and monthly during treatment, initially in the absence of anti-NTM therapy but with the intent to treat with antibiotics if there is persistence of the infection in cultures. Other clinical outcomes will include changes in sweat chloride, lung function and weight. Safety measures will include periodic assessment of liver enzymes. All serious and non-serious adverse events will be collected
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 2 is below the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Medical University of South Carolina is the lead sponsor of 852 studies on the registry; 165 are open to participants now.
Of its 128 completed or terminated interventional studies of FDA-regulated products, 101 (79%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment.
Drug: Ivacaftor
Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.
Forced Expiratory Volume
Absolute change in percent predicted in 1 second FEV1 from baseline through week 24
Time frame: 24 weeks
Sputum Results
Achievement of mycobacterial culture conversion (negative culture)
Time frame: 24 weeks
Sweat Chloride
Testing efficacy through gathering absolute change in sweat chloride from baseline through week 24
Time frame: 24 Weeks
| Milestone | Ivacaftor |
|---|---|
| Started | 2 |
| Completed | 2 |
| Not completed | 0 |
Absolute change in percent predicted in 1 second FEV1 from baseline through week 24
| percentage of predicted | Ivacaftor |
|---|---|
| Forced Expiratory Volume | 4 (3 to 5) |
Achievement of mycobacterial culture conversion (negative culture)
| Participants | Ivacaftor |
|---|---|
| Sputum Results | 2 |
Testing efficacy through gathering absolute change in sweat chloride from baseline through week 24
| mmol/L | Ivacaftor |
|---|---|
| Sweat Chloride | -5.7 (-8 to -3.5) |
Collected over 24 weeks. Non-serious events are listed at a 1% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ivacaftor | 0/2 (0%) | 0/2 (0%) | 2/2 (100%) |
| Event | Ivacaftor |
|---|---|
| Sinus congestionInfections and infestations | 2/2 |
| FolliculitisSkin and subcutaneous tissue disorders | 1/2 |
| palpitationsCardiac disorders | 1/2 |
| Age, Continuous(years) | Ivacaftor |
|---|---|
| Mean | 37 (36 to 38) |
| Sex: Female, Male(Participants) | Ivacaftor |
|---|---|
| Female | 2 |
| Male | 0 |
| Ethnicity (NIH/OMB)(Participants) | Ivacaftor |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 2 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Ivacaftor |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 2 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Ivacaftor |
|---|---|
| United States | 2 |
No study locations are listed for this record.
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Plan to share: No
This study is completed, as verified in Jul 2018. You cannot join it, but the record below documents what was studied.
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Medical University of South Carolina