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        Down Syndrome Clinical Trial- BTD-001

        Helen Jenny Peter Leonard Downs Richmond HM JA PC MBChB MPH BApplSci (physio) MSc PhD MBBS MRCP(UK) FRACP Principal Research Fellow Head, Child Disability Head, Vaccine Trials Group +61 419 956 946 08 6319 1763 helen.leonard@telethonkids.org.au ...

        Research

        An introduction to clinical trial design

        This manuscript will give a brief overview of clinical trial design including the strengths and limitations of various approaches ...

        Research

        Multi-site validation of a suite of clinical outcome measures for clinical trial readiness in the CDKL5 Deficiency Disorder

        Helen Jenny Peter Leonard Downs Jacoby HM JA PJ MBChB MPH BApplSci (physio) MSc PhD BA (Hons) MSc Principal Research Fellow Head, Child Disability Biostatistician +61 419 956 946 08 6319 1763 helen.leonard@telethonkids.org.au Jenny.Downs@ ...

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        A randomised, controlled clinical trial of narrowband UVB phototherapy for clinically isolated syndrome: The PhoCIS study

        This study provides a basis for further studies to determine if there are any benefits of the therapeutic effects of narrowband UVB radiation on MS progression ...

        Research

        Improving clinical trial readiness to accelerate development of new therapeutics for Rett syndrome

        Rett syndrome is associated with severe functional impairments and many comorbidities, each in urgent need of treatments. Mutations in the MECP2 gene were identified as causing Rett syndrome in 1999. Over the past 20 years there has been an abundance of preclinical research with some studies leading to human clinical trials. ...

        Research

        Continuous Glucose Monitoring Adherence: Lessons from a Clinical Trial to Predict Outpatient Behavior

        Continuous glucose monitoring adherence and patterns of use are individualized ...

        Research

        The ORVAC trial: A clinical trial of a third scheduled dose of Rotarix rotavirus vaccine in Australian Indigenous infants to improve protection against gastroenteritis

        We hypothesised that scheduling an additional (third) dose of oral human rotavirus vaccine for children aged 6 to <12 months would improve protection ...

        Research

        Pragmatic Adaptive Trial for Respiratory Infection in Children (PATRIC) Clinical Registry protocol

        Acute respiratory infections (ARI) are the most common cause of paediatric hospitalisation. There is an urgent need to address ongoing critical knowledge gaps in ARI management. The Pragmatic Adaptive Trial for Respiratory Infections in Children (PATRIC) Clinical Registry will evaluate current treatments and outcomes for ARI in a variety of paediatric patient groups. ...

        Research

        Immunogenicity and Safety of Monovalent Acellular Pertussis Vaccine at Birth: A Randomized Clinical Trial

        The monovalent acellular pertussis vaccine is immunogenic and safe in neonates ...

        Research

        Approaches to prioritising research for clinical trial networks: a scoping review

        Prioritisation of clinical trials ensures that the research conducted meets the needs of stakeholders, makes the best use of resources and avoids duplication. The aim of this review was to identify and critically appraise approaches to research prioritisation applicable to clinical trials, to inform best practice guidelines for clinical trial networks and funders. ...

        Research

        Interferon β-1a ring prophylaxis to reduce household transmission of SARS-CoV-2: a cluster randomised clinical trial

        Accumulating evidence indicates that an early, robust type 1 interferon (IFN) response to SARS-CoV-2 is important in determining COVID-19 outcomes, with an inadequate IFN response associated with disease severity. Our objective was to examine the prophylactic potential of IFN administration to limit viral transmission. ...

        Research

        The ORVAC Trial - A phase IV, double-blind, randomised, placebo-controlled clinical trial to optimise the delivery of RV1 rotavirus vaccine to Northern Territory Aboriginal infants

        Tom Snelling T BMBS DTMH GDipClinEpid PhD FRACP Head, Infectious Disease Implementation 08 6319 1817 tom.snelling@telethonkids.org.au Head, Infectious Disease Implementation Professor Tom Snelling is Head of the Infectious Disease Implementation ...

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