April and May 2016 Clinical Trials

The CFF provided information on the following clinical trial:

04/08/2016 – IGNITE: Safety and effectiveness of gallium nitrate in adults with cystic fibrosis

This phase 2 study will look at the safety and effectiveness of a five-day infusion of IV gallium in adults with cystic fibrosis. Researchers will test if the drug is effective by measuring participants’ lung function and testing sputum cultures to see if there is a reduction in Pseudomonas aeruginosa. They will also study how safe gallium is by tracking adverse events and safety labs.

This trial is for people with CF who are chronically infected with P. aeruginosa. It will require blood draws, sputum samples and lung function tests.

05/12/2016 – PEx Pilot Study

The goal of the study is to better understand current treatment practices for pulmonary exacerbations and whether the CF Registry can be used for this type of study.

05/15/2016 – Phase 2 study of VX-371 in people with CF who are currently taking lumacaftor/ivacaftor (Orkambi)

This study will look at the safety and effectiveness of the inhaled drug VX-371 (formerly P-1037) in saline compared to saline alone. This study uses a crossover design, meaning that all participants will receive the study drug and the placebo for part of the study. Researchers will study the safety of VX-371 by tracking adverse events. They will also study the drug’s effectiveness by measuring changes in lung function.

This study is for people with CF who are currently being treated with lumacaftor/ivacaftor (Orkambi). This study may require lung function tests, vision tests, blood draws and/or other measurements.

05/18/2016 – Phase 2 study of N91115 in people with CF who are currently taking ivacaftor

This study will look at the safety and effectiveness of the oral drug N91115 when taken along with ivacaftor (Kalydeco). N91115 is an experimental drug that may stabilize the cystic fibrosis protein. Study participants will receive either N91115 or a placebo. Researchers will test the effectiveness of N91115 by measuring lung function. They will also measure its safety by tracking adverse events.

This study is for people with CF who are currently taking ivacaftor (Kalydeco). This study may require lung function tests, sweat chloride tests, blood draws and/or other measurements.

05/21/2016 – Early Treatment of New Onset of Methicillan Resistant Staphylococcus Aureus (MRSA) Growth in Respiratory Culture – RESULTS

 

STUDY BACKGROUND INFORMATION:

Methicillan-resistant Staphylococcus aureus (MRSA) is one germ that can cause a lung infection in people with CF. MRSA is harder to treat than other forms of the germ Staphylococcus aureus. In both people with CF and people without CF, MRSA can make some people sick and other people can have the germ without getting sick. People with CF who grew MRSA in their respiratory culture for the first time were randomly assigned to either an early eradication protocol group who received treatment because their culture was positive or to an observational group that only received treatment if respiratory symptoms occurred (the current typical approach toward treatment of positive cultures for MRSA).

TRIAL RESULTS:

Primary Efficacy: 45 patients were randomized, 24 to treatment and 21 to observational control. The study was stopped early by the Data Monitoring Committee due to efficacy. The MRSA eradication protocol for newly acquired MRSA demonstrated microbiologic efficacy without safety concerns.
The primary endpoint was met with 82% of participants in the treatment arm (n=22) MRSA negative at day 28 compared to 26% (n=19) in observational arm (adjusted difference: 53%; 95% CI: (23%, 80%); p=<0.001).

Secondary Efficacy: The majority of the participants were compliant with topical antibiotic usage and environmental decontamination. Fifteen out of 24 participants (65%) took at least 80% of their daily doses of both oral antibiotics. Overall, more pulmonary signs and symptoms were experienced by participants in the observational control arm than in the treatment arm over the first 28 days.

Safety: Adverse events (AEs) related to gastrointestinal disorders and skin/subcutaneous tissue disorders were more common in the treatment arm. Overall, the number of AEs did not differ significantly between treatment arms.

These data are preliminary and have not been peer-reviewed.

 

05/21/2016 – Home Monitoring of Lung Function – RESULTS

STUDY BACKGROUND INFORMATION:

This study looked at monitoring lung function and lung symptoms at home as a method to monitor lung health. Study participants were randomly assigned to one of two groups: 1) home monitoring, in which spirometry and respiratory symptoms were recorded daily; evaluated by study center and treatment started or 2) CF education on symptoms to look for with lung infections. Home monitoring information was transmitted by phone modem weekly to the study center and participants in the CF education group contacted the study nurse if they had more respiratory symptoms.

TRIAL RESULTS:

Primary Efficacy: The study enrolled 267 patients, 135 were randomized to the early intervention arm and 132 to the usual care arm. The study was stopped early by the Data Monitoring Committee due to futility and lagging enrollment. The primary analysis results showed no difference in 52 week mean change in FEV1 (Liters) in the early intervention arm relative to the usual care arm (Mean difference = 0.00L, 95% C.I.= [-0.07, 0.07], p-value= 0.991). We could not rule out a beneficial or harmful effect that spans ±70ccâ??s.

Secondary Efficacy: No differences in 52 week mean change in respiratory symptom scores were observed for either the CFQ-R or CFRSD questionnaires. The early intervention arm was associated with a significantly higher risk for time to first PE (Time to first PE Hazard Ratio=1.45, 95% C.I.= [1.09, 1.93]; Time to Subsequent PE Hazard Ratio=1.34, 95% C.I.=[0.95, 1.89]). Adherence to intervention was assessed; 50% of the early intervention arm patients transmitted home monitoring data at least once a week on >80% of their follow-up weeks.

Safety: A higher rate of serious adverse events was observed in the early intervention arm relative to usual care arm (Rate Ratio=1.3, 95% C.I. = [1.05, 1.61]). This higher rate of serious adverse events was anticipated, given the intervention was designed to detect respiratory complications more rapidly than usual care.

These data are preliminary and have not been peer-reviewed.