Double-blind randomized pilot trial evaluating the efficacy of oral propranolol on infantile haemangiomas in infants < 4 months of age

2013 ◽  
Vol 169 (1) ◽  
pp. 181-183 ◽  
Author(s):  
C. Léauté-Labrèze ◽  
E. Dumas de la Roque ◽  
F. Nacka ◽  
A. Abouelfath ◽  
N. Grenier ◽  
...  
Keyword(s):  
Pilot Trial ◽  
Double Blind ◽  
Oral Propranolol ◽  
Infantile Haemangiomas

A randomised pilot trial of the anti-von Willebrand factor aptamer ARC1779 in patients with type 2b von Willebrand disease

2010 ◽  
Vol 104 (09) ◽  
pp. 563-570 ◽  
Author(s):  
Petra Paulinska ◽  
Petra Jilma-Stohlawetz ◽  
James Gilbert ◽  
Renta Hutabarat ◽  
Paul Knöbl ◽  
...  
Keyword(s):  
Pilot Trial ◽  
Coagulation Factor ◽  
Von Willebrand Disease ◽  
Double Blind ◽  
Clinical Trial Registration ◽  
Maximal Increase ◽  
Double Blind Design ◽  
Von Willebrand

SummaryDesmopressin aggravates thrombocytopenia in type 2B von Willebrand disease (VWF type 2B) by release of large and hyper-adhesive von Wille-brand Factor (VWF) multimers. This pilot study investigated whether the anti-VWF aptamer ARC1779 can prevent desmopressin-induced thrombocytopenia and interferes with the excessive VWF turnover in patients with VWF type 2B. Concentration effect curves of ARC1779 were established for five patients in vitro and two patients with VWF type 2B were treated by infusion of ARC1779, desmopressin, or their combination in a randomised, controlled, double-blind design. ARC1779 concentrations in the range of 1–3 μg/ml blocked free A1 domain binding sites by 90% in vitro. In vivo, desmopressin alone induced a profound (-90%) drop in platelet counts in one of the patients. ARC1779 (4–5 μg/ml) completely inhibited VWF A1 domains and prevented this desmopress-in-induced platelet drop. Desmopressin alone increased VWF antigen two- to three-fold, accompanied by concordant changes in VWF Ristocetin cofactor activity (RCo) and coagulation factor VIII activity. ARC1779 substantially enhanced the desmopressin-induced maximal increase in these parameters, and improved multimer patterns. No treatment related adverse events were observed and no bleeding occurred despite marked thrombocytopenia. These data provide first proof of concept in humans and evidence that ARC1779 is a potent inhibitor of VWF. ARC1779 prevented the rapid consumption of VWF multimers together with agglutinated platelets that occurred in response to desmopressin challenge in patients with VWD type 2B.Clinical Trial registration number: NCT00632242.

scholarly journals Effectiveness and acceptability ofmyo-inositol nutritional supplement in the prevention of gestational diabetes (EMmY): a protocol for a randomised, placebo-controlled, double-blind pilot trial

BMJ Open ◽  
2018 ◽  
Vol 8 (9) ◽  
pp. e022831 ◽  
Author(s):  
Chiamaka Esther Amaefule ◽  
Zoe Drymoussi ◽  
Julie Dodds ◽  
Lorna Sweeney ◽  
Elena Pizzo ◽  
...  
Keyword(s):  
Gestational Diabetes ◽  
Plasma Glucose ◽  
Healthcare Professionals ◽  
Randomised Trial ◽  
Pilot Trial ◽  
Qualitative Evaluation ◽  
Nutritional Supplement ◽  
Cost Data ◽  
Advisory Group ◽  
Double Blind

IntroductionGestational diabetes increases maternal and offspring complications in pregnancy and cardiovascular complications in the long term. The nutritional supplementmyo-inositol may prevent gestational diabetes; however, further evaluation is required, especially in multiethnic high-risk mothers. Our pilot trial onmyo-inositol to prevent gestational diabetes will evaluate trial processes, assess acceptability to mothers and obtain preliminary estimates of effect and cost data prior to a large full-scale trial.Methods and analysisEMmY is a multicentre, placebo-controlled, double-blind, pilot, randomised trial, with qualitative evaluation. We will recruit pregnant women at 12–15+6weeks’ gestation, with gestational diabetes risk factors, from five maternity units in England between 2018 and 2019. We will randomise 200 women to take either 2 g ofmyo-inositol powder (intervention) or placebo, twice daily until delivery. We will assess rates of recruitment, randomisation, adherence to intervention and follow-up. Gestational diabetes will be diagnosed at 24–28 weeks as per the National Institute for Health and Care Excellence (NICE) criteria (fasting plasma glucose: ≥5.6 mmol/L and 2-hour plasma glucose: ≥7.8 mmol/L). We will assess the effects ofmyo-inositol on glycaemic indices at 28 weeks and on other maternal, fetal and neonatal outcomes at postnatal discharge. Qualitative evaluation will explore the acceptability of the trial and the intervention among women and healthcare professionals. Cost data and health-related quality of life measures will be captured. We will summarise feasibility outcomes using standard methods for proportions and other descriptive statistics, and where appropriate, report point estimates of effect sizes (eg, mean differences and relative risks) and associated 95% CIs.Ethics and disseminationEthical approval was obtained through the London Queen Square Research Ethics Committee (17/LO/1741). Study findings will be submitted for publication in peer-reviewed journals. Newsletters will be made available to participants, healthcare professionals and members of Katie’s Team (a patient and public advisory group) to disseminate.Trial registration numberISRCTN48872100.Protocol version and dateVersion 4.0, 15 January 2018.

scholarly journals Regulation of inflammatory biomarkers by intravenous methylprednisolone in pediatric ARDS patients: Results from a double-blind, placebo-controlled randomized pilot trial

Cytokine ◽  
2016 ◽  
Vol 77 ◽  
pp. 63-71 ◽  
Author(s):  
Andreas Schwingshackl ◽  
Dai Kimura ◽  
Cynthia R. Rovnaghi ◽  
Jordy S. Saravia ◽  
Stephania A. Cormier ◽  
...  
Keyword(s):  
Pilot Trial ◽  
Inflammatory Biomarkers ◽  
Intravenous Methylprednisolone ◽  
Double Blind ◽  
Double Blind Placebo

Double-blind pilot trial of desipramine versus fluoxetine in panic patients

Anxiety ◽  
1994 ◽  
Vol 1 (6) ◽  
pp. 287-290 ◽  
Author(s):  
Alexander Bystritsky ◽  
Richard M. Rosen ◽  
Kirk J. Murphy ◽  
Paul Bohn ◽  
Steven A. Keys ◽  
...  
Keyword(s):  
Pilot Trial ◽  
Double Blind
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