Paediatrics
22 videos · last 6 months shuffled daily, then older videos newest first
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51:58 REMAP-CAP Results Oseltamivir in Critically Ill Influenza Patients
Dr. Srinivas Murthy, Dr. Thomas Hills and Dr. Lindsay Berry discuss on Berry Consultants' In the Interim podcast the REMAP-CAP results finding oseltamivir did not reduce 90-day mortality in critically ill influenza patients.
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10:47 Rinke Riezebos, Princess Máxima Center — Data-Driven Clinical Development
Rinke Riezebos of the Princess Máxima Center for Pediatric Oncology tells The Clinical Trial Vanguard how the center uses data to drive clinical development.
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13:27 Prader-Willi Syndrome: Diazoxide Choline Extended Release Improves Hyperphagia
Evelien Gevers, PhD, MD, of Barts Health NHS Trust on the effect of diazoxide choline extended release on hyperphagia in patients with Prader-Willi syndrome.
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30:58 CAR-T for NMOSD: A Phase 1 Trial
Dr. Michael Levy and Dr. Benjamin Greenberg describe for SRNA an autologous Phase 1 CAR-T study in aquaporin-4-positive NMOSD at Mass General and UT Southwestern, covering eligibility, risks and lessons for neuroimmune research.
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3:04 Mobile Health App Study in Youth With Prediabetes
Talia Hitt, a pediatric endocrinologist at Johns Hopkins Children's Center, discusses her study in which participants aged 13 to 17 with prediabetes used the Prosper mobile health app for lifestyle modification.
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2:19:14 GCP-Mindset on Evidence Standards in European Medical Device Regulation
Andreas Beust talks to Prof. Tom Melvin about whether European medical device regulation gives innovators enough clarity, covering early regulatory advice, randomized trials, registries, real-world evidence, orphan and paediatric devices, and what Europe might take from the US, Japan and the UK.
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44:40 Who Clinical Trials Left Out, and Why It Matters
Pediatrician Dr. Kerry Sease and health equity researcher Dr. Shaniece Criss discuss why women, children and people of color were often left out of clinical trials and how that gap still affects drug dosing and device accuracy, for FOX Carolina News.
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3:27 Gene Therapy Weekly: FOXG1's First Trial Site Is Open
GENEration Hope reports that the FOXG1 gene therapy trial has activated its first clinical site, at Texas Children's Hospital and Baylor College of Medicine.
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3:45 Pediatric Fracture Care: The COMET and DRIFT Trials
Johns Hopkins pediatric orthopaedic surgeon R. Jay Lee, M.D., discusses the multicenter randomized COMET and DRIFT trials, which compare treatment approaches for elbow and wrist fractures in children.
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36:20 Rethinking Child Assent Practices in Research
Dr. David Coker discusses rethinking child assent in research beyond procedural compliance, including regulatory tensions, inclusion of children with disabilities and the role of IRBs, on CITI Program's On Research podcast.
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36:39 Rethinking Trial Design for Rare Diseases
Kim Kushner and Ramin Farhood speak with Marshall Summar, CEO and founder of Uncommon Cures, who has run more than 200 rare disease trials, about rethinking trial design for rare diseases.
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3:41 How to Obtain Orphan Drug Designation
Pace on obtaining Orphan Drug Designation in the US and EU, covering prevalence criteria, incentives such as market exclusivity and tax credits, and the Rare Pediatric Disease Designation program.
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59:30 Kaftrio and the CFTR Modulator Trials in Cystic Fibrosis
Dr Ian Balfour-Lynn, consultant in paediatric respiratory medicine at the Royal Brompton Hospital, reviews the clinical trials that led to licensing of CFTR modulators such as Kaftrio and real-world experience with them, for the Genomics Education Programme.
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1:02:43 Bayesian Borrowing in Clinical Trials
Carl-Fredrik Burman, PhD, of AstraZeneca's statistical innovation team, examines Bayesian dynamic borrowing of control data from earlier trials and borrowing across biomarker-defined subgroups when trials cannot be fully powered, for the ISBS Webinar Series.
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27:20 How to Apply for an EU Orphan Drug Designation
EATRIS and the EMA's Regulatory Science and Innovation Task Force and Orphan Office walk academic medicine developers through applying for an Orphan Drug Designation, from submission in IRIS to protocol assistance and post-designation steps.
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2:46 CHEO on Digital Twins for Under-Recruited Paediatric Trials
Scientists at the CHEO Research Institute are testing whether AI-generated digital twins can let paediatric trials deliver results when recruitment falls short of target.
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54:16 Clinical Trials, EMR and AI: Next Steps
Sharon Nachman of Stony Brook University on using electronic medical records and AI to shorten the delay in licensing drugs for children and pregnant women.
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2:52 Gene Therapy Trial for Inherited Deafness in Children
Zheng-Yi Chen, DPhil, of Mass Eye and Ear and Yilai Shu, MD, of Fudan University's Eye & ENT Hospital explain trial results in which children born deaf, treated in both ears with a gene therapy, gained hearing, speech and sound localisation.
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45:05 Blood Microsampling in Clinical Trials
Trajan and IQVIA discuss how blood microsampling fits decentralized and hybrid trials, covering home self-collection, bioanalytical considerations, kit logistics and training for sites and patients.
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9:35 Sproutel Uses Companion Toys to Help Children With Serious Disease or in Clinical Trials
Sproutel creates tools for children with illness including creating companion toys for children taking part in clinical trials so that the children can communicate better with their caregivers.
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1:21:22 Good Clinical Practice and Ethics with Holly Tabor, PhD
Holly Tabor, director of the Stanford Center for Biomedical Ethics, discusses current and emerging ethical issues in Good Clinical Practice, with case examples and approaches to address them.
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58:18 Conducting Economic Evaluation Alongside Paediatric Clinical Trials
A webinar from the Australian Clinical Trials Alliance's HEAT special interest group on conducting economic evaluation alongside paediatric clinical trials.