- Efficacy and effectiveness of an rVSV-vectored vaccine in preventing Ebola virus disease: final results from the Guinea ring vaccination, open-label, cluster-randomised trial (Ebola Ça Suffit!). [Journal Article]
- LctLancet 2016 Dec 23
- CONCLUSIONS: The results add weight to the interim assessment that rVSV-ZEBOV offers substantial protection against Ebola virus disease, with no cases among vaccinated individuals from day 10 after vaccination in both randomised and non-randomised clusters.
- Enhancing the Scientific Integrity and Safety of Clinical Trials: Recommendations for Data Monitoring Committees. [Journal Article]
- JAMAJAMA 2016 12 13; 316(22):2359-2360
- Data monitoring committees should be used more frequently and appropriately to monitor paediatric clinical trials, particularly those involving neonates. [Editorial]
- APActa Paediatr 2017; 106(1):7-8
- Ambulatory Treatment of Fast Breathing in Young Infants Aged <60 Days: A Double-Blind, Randomized, Placebo-Controlled Equivalence Trial in Low-Income Settlements of Karachi. [Journal Article]
- CIClin Infect Dis 2017 Jan 15; 64(2):184-189
- CONCLUSIONS: This trial failed to show equivalence of placebo to amoxicillin in the management of isolated fast breathing without hypoxemia or other clinical signs of illness in term young infants.
- Multicentre randomised placebo-controlled trial of oral anticoagulation with apixaban in systemic sclerosis-related pulmonary arterial hypertension: the SPHInX study protocol. [Journal Article]
- BOBMJ Open 2016 Dec 08; 6(12):e011028
- Systemic sclerosis (SSc) is a severe and costly multiorgan autoimmune connective tissue disease characterised by vasculopathy and fibrosis. One of the major causes of SSc-related death is pulmonary a...
Systemic sclerosis (SSc) is a severe and costly multiorgan autoimmune connective tissue disease characterised by vasculopathy and fibrosis. One of the major causes of SSc-related death is pulmonary arterial hypertension (PAH), which develops in 12-15% of patients with SSc and accounts for 30-40% of deaths. In situ thrombosis in the small calibre peripheral pulmonary vessels resulting from endothelial dysfunction and an imbalance of anticoagulant and prothrombotic mediators has been implicated in the complex pathophysiology of SSc-related PAH (SSc-PAH), with international clinical guidelines recommending the use of anticoagulants for some types of PAH, such as idiopathic PAH. However, anticoagulation has not become part of standard clinical care for patients with SSc-PAH as only observational evidence exists to support its use. Therefore, we present the rationale and methodology of a phase III randomised controlled trial (RCT) to evaluate the efficacy, safety and cost-effectiveness of anticoagulation in SSc-PAH.
- Northern Manhattan Hispanic Caregiver Intervention Effectiveness Study: protocol of a pragmatic randomised trial comparing the effectiveness of two established interventions for informal caregivers of persons with dementia. [Journal Article]
- BOBMJ Open 2016 Nov 25; 6(11):e014082
- The prevalence of dementia is increasing without a known cure, resulting in an increasing number of informal caregivers. Caring for a person with dementia results in increased stress and depressive s...
The prevalence of dementia is increasing without a known cure, resulting in an increasing number of informal caregivers. Caring for a person with dementia results in increased stress and depressive symptoms. There are several behavioural interventions designed to alleviate stress and depressive symptoms in caregivers of persons with dementia with evidence of efficacy. Two of the best-known interventions are the New York University Caregiver Intervention (NYUCI) and the Resources for Enhancing Alzheimer's Caregivers Health (REACH). The effectiveness of the NYUCI and REACH has never been compared. There is also a paucity of data on which interventions are more effective in Hispanics in New York City. Thus, we proposed the Northern Manhattan Hispanic Caregiver intervention Effectiveness Study (NHiCE), a pragmatic clinical trial designed to compare the effectiveness of adaptations of the NYUCI and the REACH in informal Hispanic caregivers of persons with dementia in New York City.
- Understanding the functions and operations of data monitoring committees: Survey and focus group findings. [Journal Article]
- CTClin Trials 2016 Nov 01; :1740774516679665
- CONCLUSIONS: Findings from the survey and focus groups provide a better understanding of contemporary data monitoring committee operations and insights regarding challenges and best practices. Overall, it was clear that increased training will be needed to prepare the next generation of qualified data monitoring committee members to meet the growing demand. These findings can be used by Clinical Trials Transformation Initiative and others to develop recommendations and tools to improve data monitoring committee operations and the overall quality of trial oversight.
- An Observational Registry of Carotid Endarterectomy and Carotid Artery Stenting in Brazil: Study Protocol. [Journal Article]
- JRJMIR Res Protoc 2016 Nov 23; 5(4):e226
- CONCLUSIONS: The analyses of the primary endpoints (and all others variables of the study) are expected to be published in 2019 in a peer reviewed journal, and results will be presented at scientific meetings, with summary results published online. This study will obtain new data related to the quality of treatment for carotid disease in Brazil at the primary training centers of future vascular surgeons, but the initial data that will be obtained and published (with the outcomes and complications) are restricted to the first 30 days postprocedure. This time restriction limits the comparison of the results that relate to the main goal of treatment, which is to decrease the risk of stroke over 5 years. The purpose of the study group is to continue the monitoring of patient records, and evaluate the follow-up data in the 5 years following the initial evaluation. This study protocol will contribute very significantly to improving the care of patients with carotid disease, in addition to qualifying the level of assistance provided in public university hospitals in the state of São Paulo, Brazil.
- Data and Safety Monitoring Board evaluation and management of a renal adverse event signal in TOPCAT. [Review]
- EJEur J Heart Fail 2016 Nov 21
- Clinical trial Data and Safety Monitoring Boards (DSMBs) have a primary obligation of ensuring study participant safety, while maintaining trial integrity. The role of DSMBs is expanding, and ideally...
Clinical trial Data and Safety Monitoring Boards (DSMBs) have a primary obligation of ensuring study participant safety, while maintaining trial integrity. The role of DSMBs is expanding, and ideally should include post-hoc reporting of deliberative processes related to clinically important safety issues or factors that could impact on future trial designs. We describe how the TOPCAT DSMB detected, investigated, and adjudicated an unexpectedly large renal adverse event signal midway through the trial, and offer general guidelines for dealing with similar unanticipated occurrences in future trials. The detection of a greater than expected incidence of deterioration in renal function, occurring in 6.1% of patients in the spironolactone arm compared with 3.9% in the placebo arm (P = 0.009), led to an in-depth DSMB review of associated study medication withdrawals and adverse events. The trial continued uninterrupted throughout the review, which reached the conclusions that spironolactone-associated renal dysfunction did not compromise overall patient safety or interfere with a perceived efficacy signal. Although no discrete mechanism for the spironolactone-associated renal adverse event signal was identified, likely possibilities are discussed. In clinical trials, DSMBs and co-ordinating centres should have the resources to detect, investigate, and adjudicate unexpected safety issues, with goals of ensuring patient safety and preserving the potential for detection of therapeutic effectiveness. In TOPCAT, spironolactone-associated renal dysfunction emerged as a potentially trial-threatening adverse event and, although clinically important, did not lead to compromise of patient safety, trial interruption, termination, or apparent loss of treatment effectiveness.
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- Challenges in the Diagnosis and Management of Growth Hormone Deficiency in India. [Review]
- IJInt J Endocrinol 2016; 2016:2967578
- In clinical practice, every year approximately 150,000 children are referred with short stature (SS) based on a cut-off of fifth percentile. The most important endocrine and treatable cause of SS is ...
In clinical practice, every year approximately 150,000 children are referred with short stature (SS) based on a cut-off of fifth percentile. The most important endocrine and treatable cause of SS is growth hormone deficiency (GHD). The lack of reliable data on the prevalence of GHD in India limits estimation of the magnitude of this problem. The diagnosis and treatment of GHD are hurdled with various challenges, restricting the availability of growth hormone (GH) therapy to only a very limited segment of the children in India. This review will firstly summarize the gaps and challenges in diagnosis and treatment of GHD based on literature analysis. Subsequently, it presents suggestions from the members at advisory board meetings to overcome these challenges. The advisory board suggested that early initiation of the therapy could better the chances of achieving final adult height within the normal range for the population. Education and awareness about growth disorders among parents, regular training for physicians, and more emphasis on using the Indian growth charts for growth monitoring would help improve the diagnosis and treatment of children with GHD. Availability of an easy-to-use therapy delivery system could also be beneficial in improving adherence and achieving satisfactory outcomes.