scholarly journals Rationale and design of the multicentric, double-blind, double-placebo, randomized trial APrepitant versus HYdroxyzine in association with cytoreductive treatments for patients with myeloproliferative neoplasia suffering from Persistent Aquagenic Pruritus. Trial acronym: APHYPAP

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
C. Le Gall-Ianotto ◽  
R. Verdet ◽  
E. Nowak ◽  
L. Le Roux ◽  
A. Gasse ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Myeloproliferative Neoplasms ◽  
Randomized Study ◽  
Primary Objective ◽  
Study Endpoint ◽  
Secondary Outcome ◽  
Primary Study ◽  
Double Blind ◽  
Water Contact ◽  
Number Of Patients

Abstract Background Aquagenic pruritus (AP), an intense sensation of scratching induced after water contact, is the most troublesome aspect of BCR-ABL1-negative myeloproliferative neoplasms (MPNs). Mostly described in polycythemia vera (PV, ~ 40%), it is also present in essential thrombocythemia (ET) and primary myelofibrosis (PMF) (10%). Even if this symptom can decrease or disappear under cytoreductive treatments, 30% of treated MPN patients still persist with a real impact on the quality of life (QoL). Because its pathophysiology is poorly understood, efficient symptomatic treatments of AP are missing. The neuropeptide substance P (SP) plays a crucial role in the induction of pruritus. Several studies showed the efficacy of aprepitant, an antagonist of SP receptor (NK-1R), in the treatment of chronic pruritus but never evaluated in AP. The objectives of APHYPAP are twofold: a clinical aim with the evaluation of the efficacy of two drugs in the treatment of a persistent AP for MPN patients and a biological aim to find clues to elucidate AP pathophysiology. Methods/design A multicentric, double-blind, double-placebo, randomized study will include 80 patients with MPN (PV or ET or PMF) treated since at least 6 months for their hemopathy but suffering from a persistent AP (VAS intensity ≥6/10). Patients will be randomized between aprepitant (80 mg daily) + placebo to match to hydroxyzine OR hydroxyzine (25 mg daily) + placebo to match to aprepitant for 14 days. At D0, baseline information will be collected and drugs dispense. Outcome measures will be assessed at D15, D30, D45, and D60. The primary study endpoint will be the reduction of pruritus intensity below (or equal) at 3/10 on VAS at D15. Secondary outcome measures will include the number of patients with a reduction or cessation of AP at D15 or D60; evaluation of QoL and AP characteristics at D0, D15, D30, D45, and D60 with MPN-SAF and AP questionnaires, respectively; modification of plasmatic concentrations of cytokines and neuropeptides at D0, D15, D30, and D60; and modification of epidermal innervation density and pruriceptor expression at D0 and D15. Discussion The APHYPAP trial will examine the efficacy of aprepitant vs hydroxyzine (reference treatment for AP) to treat persistent AP in MPN patients. The primary objective is to demonstrate the superiority of aprepitant vs hydroxyzine to treat persistent AP of MPN patients. The treatment received will be considered efficient if the AP intensity will be reduced at 3/10 or below on VAS after 14 days of treatment. The results of this study may provide a new treatment option for this troublesome symptom and also give us more insights in the pathophysiology understanding of AP. Trial registration APHYPAP. NCT03808805, first posted: January 18, 2019; last update posted: June 10, 2021. EudraCT 2018-090426-66

scholarly journals Effect of tart cherry juice on risk of gout attacks: protocol for a randomised controlled trial

BMJ Open ◽  
2020 ◽  
Vol 10 (3) ◽  
pp. e035108
Author(s):  
Kirstie Louise Lamb ◽  
Anthony Lynn ◽  
Jean Russell ◽  
Margo E Barker
Keyword(s):  
Research Ethics ◽  
Outcome Measures ◽  
Controlled Trial ◽  
Intervention Group ◽  
Serum Urate ◽  
Secondary Outcome ◽  
Primary Study ◽  
Tart Cherry ◽  
Double Blind ◽  
Cherry Juice

IntroductionGout is a painful form of inflammatory arthritis associated with several comorbidities, particularly cardiovascular disease. Cherries, which are rich in anti-inflammatory and antioxidative bioactive compounds, are proposed to be efficacious in preventing and treating gout, but recommendations to patients are conflicting. Cherry consumption has been demonstrated to lower serum urate levels and inflammation in several small studies. One observational case cross-over study reported that cherry consumption was associated with reduced risk of recurrent gout attacks. This preliminary evidence requires substantiation. The proposed randomised clinical trial aims to test the effect of consumption of tart cherry juice on risk of gout attacks.Methods and analysisThis 12-month, parallel, double-blind, randomised, placebo-controlled trial will recruit 120 individuals (aged 18–80 years) with a clinical diagnosis of gout who have self-reported a gout flare in the previous year. Participants will be randomly assigned to an intervention group, which will receive Montmorency tart cherry juice daily for a 12-month period, or a corresponding placebo group, which will receive a cherry-flavoured placebo drink. The primary study outcome is change in frequency of self-reported gout attacks. Secondary outcome measures include attack intensity, serum urate concentration, fractional excretion of uric acid, biomarkers of inflammation, blood lipids and other markers of cardiovascular risk. Other secondary outcome measures will be changes in physical activity and functional status. Statistical analysis will be conducted on an intention-to-treat basis.Ethics and disseminationThis study has been granted ethical approval by the National Research Ethics Service, Yorkshire and The Humber—Leeds West Research Ethics Committee (ref: 18/SW/0262). Results of the trial will be submitted for publication in a peer-reviewed journal.Trial registration numberNCT03621215.

scholarly journals Early combination therapy with etanercept and methotrexate in JIA patients shortens the time to reach an inactive disease state and remission: results of a double-blind placebo-controlled trial

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Ekaterina Alexeeva ◽  
Gerd Horneff ◽  
Tatyana Dvoryakovskaya ◽  
Rina Denisova ◽  
Irina Nikishina ◽  
...  
Keyword(s):  
Combination Therapy ◽  
Controlled Trial ◽  
Randomized Study ◽  
Primary Objective ◽  
Disease State ◽  
Inactive Disease ◽  
Open Label ◽  
Standard Regimen ◽  
Double Blind ◽  
Early Combination Therapy

Abstract Background Remission is the primary objective of treating juvenile idiopathic arthritis (JIA). It is still debatable whether early intensive treatment is superior in terms of earlier achievement of remission. The aim of this study was to evaluate the effectiveness of early etanercept+methotrexate (ETA+MTX) combination therapy versus step-up MTX monotherapy with ETA added in refractory disease. Methods A multi-centre, double-blind, randomized study in active polyarticular JIA patients treated with either ETA+MTX (n = 35) or placebo+MTX (n = 33) for up to 24 weeks, followed by a 24-week open-label phase. The efficacy endpoints included pedACR30 criteria improvement at week 12, inactive disease at week 24, and remission at week 48. Patients who failed to achieve the endpoints at week 12 or at week 24 escaped to open-label ETA+MTX. Safety was assessed at each visit. Results By intention-to-treat analysis, more patients in the ETA+MTX group reached the pedACR30 response at week 12 (33 (94.3%)) than in the placebo+MTX group (20 (60.6%); p = 0.001). At week 24, comparable percentages of patients reached inactive disease (11 (31.4%) vs 11 (33.3%)). At week 48, 11 (31.4%) and eight (24.2%) patients achieved remission. The median (+/−IQR) times to achieve an inactive disease state in the ETA+MTX and placebo+MTX groups were 24 (14–32) and 32 (24–40) weeks, respectively. Forty-four (74/100 patient-years) adverse events (AEs) were reported, leading to treatment discontinuation in 6 patients. Conclusions Early combination therapy with ETA+MTX proved to be highly effective compared to the standard step-up regimen. Compared to those treated with the standard regimen, more patients treated with a combination of ETA+MTX reached the pedACR30 response and achieved inactive disease and remission more rapidly.

scholarly journals Methotrimeprazine versus haloperidol in palliative care patients with cancer-related nausea: a randomised, double-blind controlled trial

BMJ Open ◽  
2019 ◽  
Vol 9 (9) ◽  
pp. e029942 ◽  
Author(s):  
Janet Rea Hardy ◽  
Helen Skerman ◽  
Jennifer Philip ◽  
Phillip Good ◽  
David C Currow ◽  
...  
Keyword(s):  
Palliative Care ◽  
Outcome Measures ◽  
Controlled Trial ◽  
Response Rates ◽  
Complete Response ◽  
Response To Treatment ◽  
Secondary Outcome ◽  
Double Blind ◽  
Intention To Treat ◽  
Point Reduction

ObjectivesMethotrimeprazine is commonly used for the management of nausea but never tested formally against other drugs used in this setting. The aim was to demonstrate superior antiemetic efficacy.DesignDouble-blind, randomised, controlled trial of methotrimeprazine versus haloperidol.Setting11 palliative care sites in Australia.ParticipantsParticipants were >18 years, had cancer, an average nausea score of ≥3/10 and able to tolerate oral medications. Ineligible patients had acute nausea related to treatment, nausea for which a specific antiemetic was indicated, were about to undergo a procedure or had received either of the study drugs or a change in glucocorticoid dose within the previous 48 hours.InterventionsBased on previous studies, haloperidol was used as the control. Participants were randomised to encapsulated methotrimeprazine 6·25 mg or haloperidol 1·5 mg one time or two times per day and assessed every 24 hours for 72 hours.Main outcome measuresA ≥two-point reduction in nausea score at 72 hours from baseline. Secondary outcome measures were as follows: complete response at 72 hours (end nausea score less than 3), response at 24 and 48 hours, vomiting episodes, use of rescue antiemetics, harms and global impression of change.ResultsResponse to treatment at 72 hours was 75% (44/59) in the haloperidol (H) arm and 63% (36/57) in the methotrimeprazine (M) arm with no difference between groups (intention-to-treat analysis). Complete response rates were 56% (H) and 51% (M). In theper protocolanalysis, there was no difference in response rates: (85% (44/52) (H) and 74% (36/49) (M). Completeper protocolresponse rates were 64% (H) and 59% (M). Toxicity worse than baseline was minimal with a trend towards greater sedation in the methotrimeprazine arm.ConclusionThis study did not demonstrate any difference in response rate between methotrimeprazine and haloperidol in the control of nausea.Trial registration numberACTRN 12615000177550.

Optimizing chair time in infusion centers using intravenous cetirizine premedication for the prevention of hypersensitivity infusion reactions.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e13508-e13508
Author(s):  
Julio Antonio Peguero ◽  
Ahmed Ayad ◽  
Stacia Young-Wesenberg ◽  
Teresa Yang ◽  
Janine North ◽  
...  
Keyword(s):  
Controlled Trial ◽  
Quality Care ◽  
Primary Objective ◽  
Double Blind ◽  
Acute Urticaria ◽  
Number Of Patients ◽  
Study Results ◽  
Secondary Endpoints ◽  
Infusion Reactions ◽  
Anti Cd20

e13508 Background: Oncology infusion centers are increasingly focused on improving operational efficiencies and patient satisfaction, while maintaining quality care. One key component is optimizing chair time, which has been especially important for patient safety during the COVID-19 pandemic to reduce risk of transmission. Many infusions require antihistamine premedication to reduce the risk of hypersensitivity infusion reactions (IRs). The two IV options are IV diphenhydramine and IV cetirizine, which have a quicker onset than oral options and can be administered IV push. In treating acute urticaria, IV cetirizine was shown to be comparable to IV diphenhydramine, with fewer side effects, and it may be effective for preventing IRs with improved chair time. Methods: A randomized, double-blind phase 2 study evaluating premedication with single dose IV cetirizine 10 mg versus IV diphenhydramine 50 mg was conducted in 34 patients receiving paclitaxel, rituximab, its biosimilar or obinutuzumab (first cycle, retreatment after 6 months or with persistent IRs). The primary objective was the incidence of IRs after premedication. Secondary endpoints included sedation due to antihistamines and time to readiness for discharge. Sedation was reported by patients on a scale of 0-4 (0 = none to 4 = extremely severe). No formal statistical analyses were planned given the exploratory nature of the study. Results: Adults primarily with cancer (n = 31 [91%]) were enrolled during the COVID-19 pandemic, from March 25 to November 23, 2020. The median age was 65 and 67 years in the IV cetirizine and diphenhydramine groups, respectively. The number of patients with IRs was 2/17 (11.8%) with IV cetirizine versus 3/17 (17.6%) with IV diphenhydramine. The mean sedation score in the IV cetirizine group compared to the IV diphenhydramine group was lower at all time points, including at discharge (0.1 vs 0.4, respectively). Mean time to discharge was 24 minutes less with IV cetirizine (4.3 hours [1.5]) versus IV diphenhydramine (4.7 hours [1.2]). This difference was greater (30 minutes less) in those ≥65 years of age (4.4 [1.3] vs 4.9 [1.0] hours). Regardless of whether patients received paclitaxel (n = 9) or an anti-CD20 (n = 25), patients had less chair time when premedicated with IV cetirizine. There were fewer treatment-related adverse events (AEs) with IV cetirizine (2 events) than with IV diphenhydramine (4 events). Conclusions: This was the first randomized, controlled trial evaluating IV antihistamine premedication for IRs and chair time. It was shown that IV cetirizine can prevent IRs, with less sedation, fewer related AEs and reduced chair time compared to IV diphenhydramine. This improves infusion center operations and patient experience. Clinical trial information: NCT04189588.

Sutureless versus Stented Bioprostheses for Aortic Valve Replacement: The Randomized PERSIST-AVR Study Design

2018 ◽  
Vol 68 (02) ◽  
pp. 114-123 ◽  
Author(s):  
Roberto Lorusso ◽  
Thierry Folliguet ◽  
Malakh Shrestha ◽  
Bart Meuris ◽  
Arie Pieter Kappetein ◽  
...  
Keyword(s):  
Aortic Valve ◽  
Aortic Valve Replacement ◽  
Valve Replacement ◽  
Severe Aortic Stenosis ◽  
Artery Bypass ◽  
Randomized Study ◽  
Primary Objective ◽  
Surgical Aortic Valve Replacement ◽  
Open Label ◽  
Number Of Patients

Abstract Introduction Sutureless biological valves for surgical aortic valve replacement (SAVR), characterized by the absence of anchoring sutures at the aortic annulus, are gaining popularity because of ease and reproducibility of implant, shorter operating times, and enhancement of minimally invasive approaches. The stentless configuration of the sutureless valve was designed to achieve optimal hemodynamic performance. Materials and Methods PERSIST-AVR (PERceval Sutureless Implant versus STandard Aortic Valve Replacement) is a prospective, randomized, adaptive, open-label, international, postmarket trial (NCT02673697). The primary objective of the trial is to assess the safety and efficacy of the Perceval (LivaNova, London, UK) sutureless bioprosthesis among patients undergoing SAVR in the presence of severe aortic stenosis to demonstrate the noninferiority of Perceval as compared with standard sutured stented bioprosthetic aortic valve as an isolated procedure or combined with coronary artery bypass grafting. Sample size will be determined adaptively through interim analyses performed by an Independent Statistical Unit till a maximum of 1,234 patients, enrolled at ∼60 sites in countries where the device is commercially available. Patients will be followed up for 5 years after implant. The primary end point is the number of patients free from major adverse cardiac and cerebrovascular-related events at 1 year. Additional secondary outcomes will be assessed up to 5 years. Discussion PERSIST-AVR is the first prospective, randomized study comparing in-hospital and postdischarge outcomes in a robust population of patients undergoing SAVR with either the Perceval sutureless bioprosthesis or a conventional sutured stented bioprosthesis up to 5 years.

scholarly journals Efficacy of tofacitinib in reduction of inflammation detected on MRI in patients with Psoriatic ArthritiS presenTing with axial involvement (PASTOR): protocol of a randomised, double-blind, placebo-controlled, multicentre trial

BMJ Open ◽  
2021 ◽  
Vol 11 (11) ◽  
pp. e048647
Author(s):  
Fabian Proft ◽  
Murat Torgutalp ◽  
Burkhard Muche ◽  
Valeria Rios Rodriguez ◽  
Maryna Verba ◽  
...  
Keyword(s):  
Psoriatic Arthritis ◽  
Treatment Period ◽  
Group Treatment ◽  
Activity Index ◽  
Treatment Options ◽  
Study Endpoint ◽  
Primary Study ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Axial Involvement

IntroductionPsoriatic arthritis (PsA) is an inflammatory disease characterised by synovitis, enthesitis, dactylitis and axial involvement. The prevalence of axial involvement ranges from 25% to 70% in this patient group. Treatment recommendations for axial PsA were mainly extrapolated from guidelines for axial spondyloarthritis, and the main treatment options are non-steroidal anti-inflammatory drugs and biological disease-modifying antirheumatic drugs (tumour necrosis factor, IL-17 and IL-23 inhibitors). Tofacitinib was approved for the treatment of PsA and its efficacy on axial inflammation has been demonstrated in a phase II study of ankylosing spondylitis (AS). This prospective study aims to evaluate the efficacy of tofacitinib in reducing inflammation in the sacroiliac joints (SIJs) and spine on MRI in patients with axial disease of their PsA presenting with active axial involvement compatible with axial PsA.Methods and analysesThis is a randomised, double-blind, placebo-controlled, multicentre clinical trial in patients with axial PsA who have evidence of axial involvement, active disease as defined by a Bath AS Disease Activity Index score of ≥4 and active inflammation on MRI of the SIJs and/or spine as assessed by and independent central reader. The study includes a 6-week screening period, a 24-week treatment period, which consist of a 12-week placebo-controlled double-blind treatment period followed by a 12-week active treatment period with tofacitinib for all participants, and a safety follow-up period of 4 weeks. At baseline, 80 subjects shall be randomised (1:1) to receive either tofacitinib or matching placebo for a 12-week double-blind treatment period. At week 12, an MRI of the whole spine and SIJs will be performed to evaluate the primary study endpoint.Ethics and disseminationThe study will be performed according to the ethical principles of the Declaration of Helsinki and the German drug law. The independent ethics committees of each centre approved the ethical, scientific and medical appropriateness of the study before it was conducted.Trial registration numberNCT04062695; ClinicalTrials.gov and EudraCT No: 2018-004254-22; European Union Clinical Trials Register.

Efficacy of Phenazone in the Treatment of Acute Migraine Attacks: A Double-Blind, Placebo-Controlled, Randomized Study

Cephalalgia ◽  
2004 ◽  
Vol 24 (10) ◽  
pp. 888-893 ◽  
Author(s):  
H Göbel ◽  
A Heinze ◽  
U Niederberger ◽  
T Witt ◽  
V Zumbroich
Keyword(s):  
Adverse Events ◽  
Randomized Study ◽  
Controlled Study ◽  
Acute Migraine ◽  
Serious Adverse Events ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Number Of Patients ◽  
Significant Difference ◽  
Main Target

In this study we compared the efficacy of 1000 mg phenazone with that of placebo in the treatment of acute migraine attacks in a randomized double-blind, placebo-controlled study of 208 patients. The main target criterion was the number of patients with a pain reduction from severe or moderate to slight or no pain 2 h after taking the pain medication. The percentage of patients satisfying the main target criterion was 48.6% for phenazone and 27.2% ( P < 0.05) for placebo. Freedom from pain after 2 h was reported by 27.6% with phenazone treatment and 13.6% ( P < 0.05) with placebo. Compared with placebo, the phenazone treatment also resulted in a significant improvement in the associated migraine symptoms of nausea, phonophobia and photophobia. Of patients treated with phenazone 11.4%, and 5.8% of those treated with placebo reported adverse events. There was no significant difference between the groups with regard to numbers of patients with adverse events. No serious adverse events occurred. The results show that phenazone at a dosage of 1000 mg is effective and well tolerated in the treatment of acute migraine attacks.

Vandetanib plus mFOLFOX6 in patients with advanced colorectal cancer (CRC): A randomized, double-blind, placebo-controlled phase II study

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 4084-4084
Author(s):  
T. S. Yang ◽  
D. Y. Oh ◽  
R. Guimbaud ◽  
J. Szanto ◽  
T. Salek ◽  
...  
Keyword(s):  
Tyrosine Kinases ◽  
Day Treatment ◽  
Sensory Neuropathy ◽  
Primary Objective ◽  
Double Blind ◽  
Once Daily ◽  
Assessment Visit ◽  
Number Of Patients ◽  
Peripheral Sensory ◽  
To Receive

4084 Background: Vandetanib is a once-daily oral agent that selectively targets key signaling pathways in cancer by inhibiting VEGF, EGF and RET receptor tyrosine kinases. Methods: Eligible patients with advanced CRC and who had previously progressed after an irinotecan- and fluoropyrimidine-containing regimen were randomized 1:1:1 to receive once-daily oral vandetanib (100 or 300 mg) + modified FOLFOX6 (mFOLFOX6) or placebo + mFOLFOX6; mFOLFOX6 was given as standard 14-day treatment cycles (oxaliplatin 85 mg/m2 2-hr and leucovorin 400 mg/m2 2-hr i.v. infusions, followed by 5- fluorouracil [5-FU] 400 mg/mg2 i.v. bolus and 5-FU 2400 mg/m2 46-hr i.v. infusion). The primary objective was to compare the number of patients with a progression event on or before a mandatory tumor assessment visit at data cut-off (∼4 months after last patient randomized). A progression event was defined as the earliest of objective and/or clinical disease progression, or death from any cause. Results: Between March and November 2007, 104 patients (aged 32–81 years) were randomized to receive study treatment ( Table ). At data cut-off on 8 March 2008, there was a greater % of progression events in the vandetanib 100 mg arm compared with placebo (72% [n=23] versus 65% [n=24]; HR=1.21, 2-sided 80% CI 0.82–1.80; 2-sided P=0.53), and also in the vandetanib 300 mg arm compared with placebo (77% [n=27] versus 65% [n=24]; HR=1.41, 2-sided 80% CI 0.96–2.07; 2-sided P=0.25). All except one patient in each group experienced an adverse event (AE) during the study with diarrhea, nausea, thrombocytopenia, and peripheral sensory neuropathy the most commonly reported AEs ( Table ). Neutropenia was the only CTC grade 4 AE to occur in >1 patient in any group (n=2, vandetanib 100 mg arm; n=0, vandetanib 300 mg arm; n=3, placebo arm). Conclusions: In this study of patients with advanced, previously treated CRC, there was no efficacy benefit for vandetanib (100 or 300 mg) + mFOLFOX6 versus placebo + mFOLFOX6. [Table: see text] [Table: see text]

Bevacizumab biosimilar and reference bevacizumab in subjects with stage IIIB/IV no squamous non-small cell lung cancer (NSCLC) (STELLA study): Results for the primary endpoint in a confirmatory, double-blind, randomized, controlled study.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e21542-e21542
Author(s):  
Susana Millan ◽  
Dmytro Trukhin ◽  
Oleksii Kolesnik ◽  
Elena Poddubskaya ◽  
Andric Zoran ◽  
...  
Keyword(s):  
Primary Endpoint ◽  
Objective Response ◽  
Stage Iiib ◽  
Study Endpoint ◽  
Controlled Study ◽  
Primary Study ◽  
Double Blind ◽  
Study Results ◽  
Significant Difference ◽  
Secondary Endpoints

e21542 Background: MB02 is a proposed biosimilar of the reference bevacizumab. A multinational, double-blind, randomized, parallel group clinical study (STELLA) is undergoing to confirm clinical similarity between MB02 and bevacizumab in patients with stage IIIB/IV no squamous NSCLC. Methods: Subjects were randomized 1:1 to MB02 or bevacizumab (15 mg/kg) plus chemotherapy (paclitaxel [P] 200 mg/m2 and carboplatin [C] AUC6) on Day 1 of every 3-week cycle for 6 cycles (Week 18) followed by MB02/bevacizumab in blinded monotherapy until disease progression, treatment intolerance, death, patient withdrawal or end of study (Week 52). As primary study endpoint, the efficacy by means of the objective response rate (ORR) evaluated by an independent radiological committee (IRC) was compared between arms at Week 18. Secondary endpoints were Progression Free Survival (PFS) and Overall Survival (OS), safety and immunogenicity (assessed at 18 and 52 weeks). Results: 627 patients were randomized: MB02 (n = 315) and bevacizumab (n = 312). Demographic and baseline characteristics were well balanced between arms. The ORR results were comparable for subjects receiving MB02 or bevacizumab plus P/C. A Risk Ratio (RR) of 1.013 (90% CI: -0.037% to 0.059) and a Risk Difference (RD) of 0.011 (90% CI: -0.037% to 0.059), were within the similarity margin predefined by FDA (0.73, 1.36) and EMA (-12%, +12%) respectively. This ORR assessed by IRC was consistent with the investigator assessment criteria. There was no significant difference between arms for secondary efficacy endpoints (PFS/OS) at week 18. Up to primary endpoint cut-off point, the safety assessment showed no significant differences between MB02 and bevacizumab arms (including the immunogenicity assessment) in terms of nature, frequency and severity of the adverse events (AE), being anaemia and hypertension the most common IMP-related AEs, with a RD between treatment groups < 5%. New signals or observable trends were no reported for MB02-treated subjects. Additional information on the secondary endpoints will be available at week 52 (end of monotherapy period). Conclusions: The statistical analysis executed for ORRs confirm the equivalence of MB02 and bevacizumab, supporting the clinical activity of MB02 treatment. MB02 was well tolerated with manageable AEs in patients with Stage IIIB/IV NSCLC. Clinical trial information: NCT03296163.

scholarly journals Montelukast as an Alternative to Low-Dose Inhaled Corticosteroids in the Management of Mild Asthma (The SIMPLE Trial): An Open-Label Effectiveness Trial

2009 ◽  
Vol 16 (suppl a) ◽  
pp. 11A-16A ◽  
Author(s):  
R Andrew McIvor ◽  
Alan Kaplan ◽  
Caroline Koch ◽  
John S Sampalis
Keyword(s):  
Asthma Control ◽  
Outcome Measures ◽  
Low Dose ◽  
Inhaled Corticosteroids ◽  
Symptom Control ◽  
Physician Satisfaction ◽  
Mild Asthma ◽  
Secondary Outcome ◽  
Open Label ◽  
Number Of Patients

OBJECTIVE: To evaluate the effectiveness of montelukast as monotherapy for patients with mild asthma who remain uncontrolled or unsatisfied while on inhaled corticosteroid (ICS) monotherapy.DESIGN: A multicentre, open-label study. Patients (six years of age or older) had ICS therapy discontinued and were treated with orally administered montelukast once daily for six weeks.MAIN OUTCOME MEASURES: The primary outcome measure was the rate at which asthma symptom control was achieved or maintained after six weeks of treatment. The secondary outcome measures were to compare compliance and physician satisfaction, and to further assess the safety and tolerability of montelukast.RESULTS: Of the 534 patients enrolled, 481 (90.1%) completed the study. Mean (± SD) age was 27.8±19.0 years. The number of patients with uncontrolled symptoms decreased from 455 (85.2%) at baseline to 143 (26.8%) at week 6 (P<0.001), and mean Asthma Control Questionnaire score decreased from 1.4±0.8 to 0.6±0.6 (P<0.001), representing a clinically significant improvement. Of the 79 patients with controlled asthma symptoms at baseline, 73.4% maintained asthma control at week 6. Compliance to asthma therapy increased from 41% at baseline for ICS to 88% at week 6 for montelukast (P<0.001). Physician satisfaction with treatment increased from 43% to 85% (P<0.001) and patient satisfaction increased from 45% at baseline to 94% at week 6. No serious adverse events were reported over the course of the study.CONCLUSION: Montelukast is an effective and well-tolerated alternative to ICS treatment in patients with mild asthma who are uncontrolled or unsatisfied with low-dose ICS therapy.

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