SNPMiner Trials (Home Page)
Report for SNP rs9984723
Developed by Shray Alag, 2020.
SNP Clinical Trial Gene
There is one clinical trial.
Clinical Trials
1 A Phase III Double-blind, Randomised, Parallel-Group Comparison of the Efficacy and Safety of FP-1201-lyo (Recombinant Human IFN Beta-1a) and Placebo in the Treatment of Patients With Moderate or Severe Acute Respiratory Distress Syndrome
In this study effectiveness and safety of a new drug FP-1201-lyo (recombinant human interferon beta-1a) is compared to placebo. Investigation is conducted with patients who have acute respiratory distress syndrome (ARDS). The new drug is expected to reduce the time which a patient need to be on the ventilator and improve patient's chances of survival. Currently there are no approved drugs for treating moderate or severe ARDS patients.
NCT02622724 Respiratory Distress Syndrome, Adult Drug: Interferon beta-1a Drug: Placebo MeSH:Respiratory Distress Syndrome, Newborn Respiratory Distress Syndrome, Adult Acute Lung Injury Syndrome
A carrier frequency was analysed for a C/T polymorphism (rs9984723) located in the 3'PRIME_UTR/intron region of IFNAR2-gene, which encodes the beta chain for the IFN-alpha/beta receptor and encompasses a regulatory motif for the glucocorticoid receptor.
Primary Outcomes
Description: VFDsurv is a composite measure of all-cause mortality and the number of days free of mechanical ventilation (VFDsurv) within 28 days among survivors. Ventilator-free days (VFDs) correspond to those days when unassisted breathing (UAB) was possible for a complete calendar day. UAB was defined as: spontaneously breathing with face mask, nasal prong oxygen or room air, T-piece breathing, tracheostomy mask breathing, CPAP less than or equal to 5 cmH2O without pressure support or intermittent mandatory ventilation assistance, use of CPAP or BIPAP solely for sleep apnoea management. A patient was reported as ventilator-free after 2 consecutive calendar days of unassisted breathing (a VFD value of 0 is assigned to patients who die without initiating UAB or who require more than 28 days of mechanical ventilation. All patients who die before Day28 are assigned a VFDsurv value of -1).
Measure: Composite Endpoint (VFDsurv; All-cause Mortality and Number of Days Free of Mechanical Ventilation) at Day 28 Time: Day 28Secondary Outcomes
Description: Fatalities, mortality all-causes from randomisation up to Day 28
Measure: Efficacy Endpoint: All-cause Mortality Time: At Day 28Description: All-cause mortality for subjects who died in Intensive Care Units up to Day 28.
Measure: Efficacy Endpoint: Mortality in ICU Time: Up to Day 28Description: This is the number of subjects who died in hospital (i.e. outside of Intensive Care Units) up to Day 28.
Measure: Efficacy Endpoint: Mortality in Hospital Time: Up to Day 28Description: The total number of days free of organ failure by Sequential Organ Failure Assessment (SOFA) methodology. Overall, the median number of days free of organ failure was 0 days in both the treatment groups.
Measure: Other Secondary Efficacy Endpoints: Days Free of Organ Failure Time: Day 28 or last day in intensive care unit [ICU] if patient has left the ICU earlier than Day 28Description: Days without renal support (any renal support was documented). Overall, the median number of days free of renal support was 28 days in the active group and 27 days in the placebo group.
Measure: Other Secondary Efficacy Endpoints: Days Free of Renal Support Time: Day 28Description: Days without vasoactive support. The vasoactive support included catecholamine and non-catecholamine vasopressors, inotropes and vasodilating agents. Overall, the median number of days free of vasoactive support was 20 days in the active group and 21 days in the placebo group.
Measure: Other Secondary Efficacy Endpoints: Days Free of Vasoactive Support Time: Day 28Description: The total number of days free of mechanical ventilation has been derived from the Patient Status report recorded on each day during the 28-day period. Patients who died during this period have been assigned a value of zero. This variable differs from the calculated "Ventilation Free Days (VFD) endpoint, which contribute to the VDFsurv primary efficacy endpoint as it require additional conditions of unassisted breathing (UAB) to be met.
Measure: Other Secondary Efficacy Endpoint: Days Free of Mechanical Ventilation Time: Day 28Description: Number of Intensive Care Unit free days up to Day 28, i.e. the patient is no longer receiving care in ICU. Patients who die during this period have been assigned a value of zero for the number of ICU care free days.
Measure: Other Secondary Efficacy Endpoints: Number of ICU-free Days Time: Day 28Description: Hospitalisation days (including the stay at Intensive Care Units). Patients who died during this period have been assigned a value of 28 for the number of days in ICU or in hospital.
Measure: Other Secondary Efficacy Endpoints: Number of Days in Hospital Time: Day 28Description: Adverse events (AE) up to Day 28. Per protocol, AEs occurring after Day 28 if the investigator considered a causal relationship with the study drug as well as all deaths up to Day 360 were reported. Treatment-emergent AEs (TEAEs) were defined as AEs that begins or worsens in intensity after at least one dose of study drug has been administered. Serious AEs (SAEs) were defined as any untoward medical occurrence that at any dose resulted in death; was life-threatening (immediate risk of death); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; was an important medical event.
Measure: Evaluation of Safety: Adverse Events and Deaths Time: AEs up to Day 28, only related after Day 28 and deaths up to Day 360Description: The immunological response to FP-1201-lyo was assessed by monitoring presence of anti-drug binding antibodies (BAbs) and neutralising antibodies (NAbs) to IFN beta-1a on pre-dose Day 1 and at Day 28, the last day in ICU or early termination (if earlier). The BAbs were determined first, and if present, the NAbs were also determined. Presence of BAbs and NAbs were summarised categorically, using positive/negative classification.
Measure: Efficacy Endpoint: Presence of Neutralising Antibodies to IFN Beta-1a Time: Baseline and Day 28 (or last day in intensive care unit [ICU] or at withdrawal, if earlier)Description: Evaluation of MxA biomarker change from baseline to D14 between treatments arms over time.
Measure: Efficacy Endpoint: Evaluation of Pharmacodynamic (PD) Using Myxovirus Resistance Protein A (MxA) Biomarker Time: From baseline to Day 14Description: Quality of Life was assessed through EQ-5D-3L (EuroQol 5-Dimensions 3-Levels questionnaire). An EQ Visual Analogue Scale (VAS) total score (ranging from 0-100) was measured (0= Worst imaginable health state, 100= Best imaginable health state). The EQ-5D-3L VAS scores are numerically summarised as change from pre-dose (Day 1) to long term follow-up (Day 180 visit).
Measure: Long-term Efficacy Endpoint: Change in Quality of Life From Baseline to Day 180 Time: Change from baseline to Day 180Description: Measuring FEV1 (forced expiratory volume in 1 second) assessed pulmonary function (airway obstruction, bronchoconstriction or bronchodilation). FEV1 is the volume exhaled during the first second of a forced expiratory manoeuvre, starting from the level of total lung capacity.
Measure: Long-term Efficacy Endpoints Relating to Respiratory Functioning (FEV1) Time: Day 180Description: The 6-minute walk test (6MWT) measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes. According to the ERS/ATS technical standard to which the 6MWT was done, the walk test is done twice and the best result is used. It is an evaluation of the global and integrated responses of all systems involved during exercise, including the pulmonary and cardiovascular systems, systemic circulation, peripheral circulation, blood and neuromuscular units and muscle metabolism. The self-paced 6MWT assesses the sub- maximal level of functional capacity and will better reflect the functional exercise level for daily activities. ANOVA parameters estimates (LS Means and 90 % conf.interval) for the overall treatment difference was analysed.
Measure: Long-term Efficacy Endpoints Relating to Neurological Functioning (6MWT) Time: Day 180Other Outcomes
Description: Vital signs were measured supine pre-dose on Day 1 (baseline) and daily up to Day 28 while the patient was in the ICU. The results at baseline and at last observation performed (LOP) were summarized overall by treatment, variable and time point. No statistical analyses were made for vital signs.
Measure: Evaluation of Safety: Vital Signs - Heart Rate Time: From baseline to Last Observation Performed (Day 28 or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Vital signs were measured supine pre-dose on Day 1 (baseline) and daily up to Day 28 while the patient was in the ICU. The results at baseline and at last observation performed (LOP) were summarized overall by treatment, variable and time point. No statistical analyses were made for vital signs.
Measure: Evaluation of Safety: Vital Signs - Body Temperature Time: From baseline to Last Observation Performed (Day 28 or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Vital signs were measured supine pre-dose on Day 1 (baseline) and daily up to Day 28 while the patient was in the ICU. The results at baseline and at last observation performed (LOP) were summarized overall by treatment, variable and time point. No statistical analyses were made for vital signs.
Measure: Evaluation of Safety: Vital Signs - Blood Pressure Time: From baseline to Last Observation Performed (Day 28 or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Physical examination data (covering the major body systems; general appearance, head [ear, nose and throat], cardiovascular, eyes, respiratory, abdomen, urogenital, musculoskeletal, neurological, lymph nodes and skin) were categorized as "normal"; "abnormal, not clinically significant;" "abnormal, clinically significant" or "not done". Physical examinations were performed at Screening, then at the Last day in ICU and Day 28 (Out of ICU) or Early Termination, from which the last observation performed is derived. The changes from baseline to the last observations performed are categorized as "no change", "change clinically significant"; "change not clinically significant", "not done".
Measure: Evaluation of Safety: Physical Examination Time: From baseline to Last Observation Performed (D28 or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Laboratory safety assessments of biochemistry, haematology and urinalysis were performed daily during the stay at ICU. Laboratory data were classified according to normal ranges as out of range (OOR; not clinically significant), OOR (clinically significant), or OOR (clinically significant and an AE). Laboratory test results were summarised by actual results by baseline and last observation period.
Measure: Evaluation of Safety: Laboratory Results Time: From baseline to Day 28 (or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Due to the study terminating early, costs items from hospitals were not collected and pharmacoeconomic analyses could not be done.
Measure: Evaluation of Pharmacoeconomics: Days Free of Organ Failure Time: Day 28 (or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Due to the study terminating early, costs items from hospitals were not collected and pharmacoeconomic analyses could not be done.
Measure: Evaluation of Pharmacoeconomics: Days Free of Renal Support Time: Day 28 (or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Due to the study terminating early, costs items from hospitals were not collected and pharmacoeconomic analyses could not be done.
Measure: Evaluation of Pharmacoeconomics: Days Free of Vasoactive Support Time: Day 28 (or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Due to the study terminating early, costs items from hospitals were not collected and pharmacoeconomic analyses could not be done.
Measure: Evaluation of Pharmacoeconomics: Days Free of Mechanical Ventilation Time: Day 28 (or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Due to the study terminating early, costs items from hospitals were not collected and pharmacoeconomic analyses could not be done.
Measure: Evaluation of Pharmacoeconomics: Number of ICU-free Days Time: Day 28 (or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Due to the study terminating early, costs items from hospitals were not collected and pharmacoeconomic analyses could not be done.
Measure: Evaluation of Pharmacoeconomics: Number of Days in Hospital Time: Day 28 (or last day in ICU if patient has left the ICU earlier than Day 28, or at withdrawal)Description: Composite endpoint including mortality and days free of mechanical ventilation (VFDsurv) within 90 days among survivors. Ventilation Free Survival at Day 90 has been classified as Dead, Alive but on a ventilator and Alive and breathing unassisted
Measure: Exploratory Variables Relating to Efficacy: Composite Endpoint (Mortality and Days Free of Mechanical Ventilation) at Day 90 Time: Within 90 daysDescription: Evaluation of Cluster of differentiation 73 (CD73) change from baseline to D14 between treatments arms over time.
Measure: Change in the Treatment-specific Exploratory Biomarker Cluster of Differentiation 73 (CD73) Concentration Time: From baseline to Day 14Description: Evaluation of potential inflammatory markers (PIMs) change from baseline to D14 between treatments arms over time. Potential biomarkers included IL-1ra, IL-6, FGF basic, IP-10 and TNF-α.
Measure: Changes in Levels of Potential Inflammatory Markers (PIMs) Time: From baseline to Day 14Description: An optional genetic sample was analysed for subjects based on a separate consent. A carrier frequency was analysed for a C/T polymorphism (rs9984723) located in the 3'PRIME_UTR/intron region of IFNAR2-gene, which encodes the beta chain for the IFN-alpha/beta receptor and encompasses a regulatory motif for the glucocorticoid receptor. Biomarker responders were defined by a 3-fold elevation in MxA and a 2-fold in CD73 in comparison to baseline.
Measure: Pharmacogenetic Analysis Time: Anytime from baseline to Day 28Description: Fatalities; as the study was terminated early, the assessment time point for mortality at Day 360 was not reached. Therefore only the overall mortality at study termination is presented.
Measure: Exploratory, Extended Long-term Follow-up: Overall Mortality at Day 360 Time: Day 360 /termination of studyDescription: Quality of Life was assessed through EQ-5D-3L (EuroQol 5-Dimensions 3-Levels questionnaire). An EQ Visual Analogue Scale (VAS) total score (ranging from 0-100) was measured (0= Worst imaginable health state, 100= Best imaginable health state). The EQ-5D-3L VAS scores are numerically summarised as change from pre-dose (Day 1) to extended long term follow-up (Day 360 visit).
Measure: Extended Long-term Follow-up Exploratory Endpoint: Change in Quality of Life From Baseline to Day 360 Time: Change from baseline to Day 360Description: The 6-minute walk test (6MWT) measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes. According to the ERS/ATS technical standard to which the 6MWT was done, the walk test is done twice and the best result is used. It is an evaluation of the global and integrated responses of all systems involved during exercise, including the pulmonary and cardiovascular systems, systemic circulation, peripheral circulation, blood and neuromuscular units and muscle metabolism. The self-paced 6MWT assesses the sub- maximal level of functional capacity and will better reflect the functional exercise level for daily activities. ANOVA parameters estimates (LS Means and 90 % confidence intervals) for the overall treatment difference was analysed.
Measure: Neurological Functioning (6MWT) at Extended Long-term Follow-up Time: Day 360Description: Measuring FEV1 (forced expiratory volume in 1 second) assessed pulmonary function (airway obstruction, bronchoconstriction or bronchodilation). FEV1 is the volume exhaled during the first second of a forced expiratory manoeuvre, starting from the level of total lung capacity.
Measure: Extended Long-term Follow-up Endpoint: Respiratory Functioning (FEV1) at Day 360 Time: Day 360