On October 14, 2016, the Cystic Fibrosis Foundati0n (CFF) provided alerts on the following clinical trials. The trial title is a link to expanded information on the CFF web site. Additional information may be found through the ClinicalTrials.gov links included with each alert.
This open-label study tested the effectiveness and safety of two different dose regimens of Tobramycin Inhalation Powder (TIP) for the treatment of Pseudomonas aeruginosa. Participants were randomized to receive either TIP once a day continuously or to receive TIP twice daily intermittently (28 days on-drug followed by 28 days off-drug) for a total of 24 weeks.
This study was not completed due to an inability to recruit participants.
Primary Findings:
Effectiveness: This study was conducted between January 2014 and December 2014. The study enrolled 16 participants into each treatment group (continuous versus intermittent TIP). However, the study was prematurely stopped on November 14, 2014 because recruiting participants became a limitation. Only 31 participants were dosed and only 5 participants completed the planned 24 week study. The primary efficacy endpoint was to determine the difference in lung function (absolute FEV1% predicted) between tobramycin inhalation powder (TIP) taken once daily continuously versus TIP twice daily in 28 day on/off cycles. Changes over time in lung function and difference in lung function between the two treatment groups could not be adequately assessed.
Additional secondary objectives were not evaluated due to the early termination of the study.
Safety: No new safety signals were identified in this study.
These data were provided by the Novartis Clinical Trial Results Website and have not been peer-reviewed.
ClinicalTrials.gov link: https://clinicaltrials.gov/show/NCT02015663
This study tested the safety, tolerability of N6022 and its effects on CFTR biomarkers in people with CF. Participants were randomized to receive either N6022 or placebo intravenously once a day for 7 days. Multiple doses of N6022 were tested. This study demonstrated tolerability of N6022 for doses of up to 40 mg.
Primary Findings:
Effectiveness: In this study, 66 participants were dosed once daily by IV injection for 7 days with one of four doses of N6022 (5 mg: N=10, 10mg: N=9, 20mg: N=9, 40 mg: N=19) or placebo (N=19). The primary objective of this study was to assess the safety and tolerability of intravenously administered N6022. No dose limiting side effects were detected and N6022 was well-tolerated. Additional objectives included assessing the effect of N6022 on lung function and biomarkers of CFTR activity (sweat chloride and nasal potential difference). No beneficial effects on lung function, sweat chloride, or nasal potential difference were identified in this study.
Safety: The overall frequency, severity, and types of adverse events (cardiac and laboratory safety profiles) were similar for all doses (including placebo).
These data are preliminary and have not been peer-reviewed.
ClinicalTrials.gov link: https://clinicaltrials.gov/show/NCT01746784
This open-label study assessed the long term safety of tobramycin inhalation powder (TIP) in people with cystic fibrosis and chronic Pseudomonas aeruginosa lung infection. All study participants received TIP for up to 1 year.
This study looked at the long term safety of tobramycin inhalation powder (TIP) and found that the adverse event frequency and profile were similar to the known TIP safety profile.
Primary Findings:
Effectiveness: This study was conducted between January 16, 2012 and January 13, 2014. The study enrolled 157 participants and all received tobramycin inhalation powder (TIP). Of the 157 participants, 96 completed the study and 61 discontinued. Primary reasons for discontinuation included adverse events (18.5%) and the participant’s decision to withdraw consent (10.8%). The primary objective of this study was to assess the safety of tobramycin inhalation powder (TIP) over 6 cycles of treatment.
Safety: The adverse event frequency and profile were similar to the known TIP safety profile. Of the 96 participants who completed this study, 45 elected to participate in an additional one year extension. All 45 completed the extension and no new safety signals or increases in the frequency of adverse events were identified.
These data were provided by the Novartis Clinical Trial Results Website and have not been peer-reviewed.
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT01519661
Genetic Modifiers of Cystic Fibrosis: Sibling Study (NCT00037778)
The purpose of the study was to find genetic and environmental factors affecting the variability in nutritional outcomes in people with CF.This study required collecting blood samples from siblings and twins with CF and their parents, reviewing medical records of siblings with CF, and the completion of questionnaires about personal medical and family medical history. Clinical data were supplemented by data provided by the CFF Patient Registry.
Study Results: This study collected height and weight data from twins and siblings with CF and found that there are many variables that impact nutritional status.
Primary Findings:
Effectiveness: Height and weight data were collected between 2000 and 2010, for 1,618 twins and siblings with CF. Following the application of inclusion and exclusion criteria, 1,124 participants remained in the study. This study indicates a prominent role for the presence of genetic modifiers influencing nutritional status in addition to CFTR.
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT00037778