Paediatric Clinical Trial Design & Endpoints Workstream

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Paediatric clinical trial design workstream

This Workstream aims to improve the health, quality of care, and outcomes for children with PH worldwide. Members aim to develop and apply novel strategies for the early recognition and initiation of treatment that can reverse, prevent or slow the progression of the disease. 

There are several unique features of paediatric PH which affect:

  • developmental biology of the cardiopulmonary system 
  • mechanisms and timing of vascular injury during susceptible periods of growth and adaptation 
  • differences in genetic/epigenetic mechanisms, maturational changes in vascular function and growth, co-morbidities, and responsiveness to therapeutic strategies 
  • developmental differences in pharmacokinetics and pharmacodynamics, and the potential for adverse effects 
  • maturational changes in right and left ventricular function 

We have a limited understanding of the disease-specific mechanisms underlying paediatric Pulmonary Vascular Disease (PVD), and this is further complicated by factors like the heterogeneity of conditions within PVD, treatments for paediatric patients being extrapolated from adult data, and a lack of well-informed guidelines and quality endpoints for assessing clinical courses and paediatric responses to therapy. 

The Workstream comprises an interactive team of clinicians, scientists, pharma colleagues, regulatory experts, and patient advocates who are working to: 

  • define precise, age-appropriate, disease-specific endpoints and biomarkers 
  • analyse datasets from real-world studies so we can understand long-term outcomes and identify disease severity risk factors 
  • explore innovative methods for paediatric trial design beyond standard MRCTs 
  • develop strategies to better translate data from adult trials to accelerate the care of children 
  • extend novel therapeutic strategies at the interface of clinical and laboratory science to enhance the care and outcomes in paediatric PH. 

The Workstream hosted its first webinar, “Enhancing Drug Development for Paediatric PH: An Integrative Perspective,” in February 2024. 

Moving forward, they have organised eight working groups, each tasked with solving a different challenge: 

  • Enhancing Drug Development for Paediatric PH: an Integrative Perspective 
    Led by: Pegah Nowbakht, Steve Abman, Rolf Berger, Christine Garnett, Sylvia Nikkho, Maria Jesus del Cerro
  • Novel Strategies for Accelerating Drug Development 
    Led by: Maurice Beghetti, Sylvia Nikkho, Christine Garnett 
  • Age-Appropriate Endpoints: Bridging Biomarkers 
    Led by: Maria Jesus del Cerro, Christine Garnett, Allan Everett, Ray Benza 
  • Age-Appropriate Endpoints: Accelerometery 
    Led by: Dunbar Ivy, Rolf Berger, Erika Rosenberg-Berman, Anna Hemnes 
  • Age–Appropriate Endpoints: Imaging and Other Technologies 
    Led by: Shahin Moledina, Jason Woods 
  • Leveraging Registry Data and Real World Data 
    Led by: Rolf Berger, Maria Jesus del Cerro, Monika von Brandenstein 
  • Genetic, Genomic and Proteomic Strategies Enhancing Clinical Care and Outcome 
    Led by: Eric Austin, Megan Griffiths 
  • Underserved Paediatric Indications: BPD-PH 
    Led by: Steve Abman, Anne Hilgendorf, Mary Mullen, Bernard Thebaud


Pegah Nowbakht , Ironwood Pharmaceuticals
Steve Abman , Children’s Hospital Colorado

Supported by

Maria Jesus Del Cerro , Hospital Universitario Ramon y Cajal
Shahin Moledina, GOSH
Christina Garnett , FDA
Sylvia Nikkho , Bayer
Sidra Hoffman , Gossamer Bio