Conducting clinical trials in persons with Down syndrome: summary from the NIH INCLUDE Down syndrome clinical trials readiness working group

Nicole T Baumer; Mara L Becker; George T Capone; Kathleen Egan; Juan Fortea; Benjamin L Handen; Elizabeth Head; James E Hendrix; Ruth Y Litovsky; Andre Strydom; Ignacio E Tapia; Michael S Rafii

doi:10.1186/s11689-022-09435-z

Conducting clinical trials in persons with Down syndrome: summary from the NIH INCLUDE Down syndrome clinical trials readiness working group

J Neurodev Disord. 2022 Mar 23;14(1):22. doi: 10.1186/s11689-022-09435-z.

Authors

Nicole T Baumer¹, Mara L Becker², George T Capone³, Kathleen Egan⁴, Juan Fortea^{5

6

7}, Benjamin L Handen⁸, Elizabeth Head⁹, James E Hendrix¹⁰, Ruth Y Litovsky^{11

12}, Andre Strydom^{13

14}, Ignacio E Tapia¹⁵, Michael S Rafii¹⁶

Affiliations

¹ Department of Neurology, Division of Developmental Medicine, Department of Pediatrics, Boston Children's Hospital, Harvard Medical School, Boston, USA.
² Department of Pediatrics, Duke University School of Medicine, Durham, USA.
³ Department of Pediatrics, Kennedy Krieger Institute, The Johns Hopkins School of Medicine, Baltimore, USA.
⁴ Parent/Advocate, Virginia, USA.
⁵ Sant Pau Memory Unit, Department of Neurology, Hospital de la Santa Creu i Sant Pau, Biomedical Research Institute Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.
⁶ CIBERNED, Madrid, Spain.
⁷ Barcelona Down Medical Center, Fundació Catalana de Síndrome de Down, Barcelona, Spain.
⁸ Department of Psychiatry, University of Pittsburgh, Pittsburgh, USA.
⁹ Department of Pathology & Laboratory Medicine, University of California, Irvine, USA.
¹⁰ LuMind IDSC Foundation, Burlington, USA.
¹¹ Waisman Center, University of Wisconsin, Madison, USA.
¹² Department of Communication Sciences and Disorders, University of Wisconsin, Madison, USA.
¹³ Department of Forensic and Neurodevelopmental Sciences, Institute of Psychiatry, Psychology and Neuroscience, London, UK.
¹⁴ King's College London & South London and the Maudsley NHS Trust, London, UK.
¹⁵ Division of Pulmonary and Sleep Medicine, Children's Hospital of Philadelphia Perelman School of Medicine, University of Pennsylvania, Philadelphia, USA.
¹⁶ Alzheimer's Therapeutic Research Institute, Keck School of Medicine, University of Southern California, Los Angeles, USA. [email protected].

Abstract

The recent National Institute of Health (NIH) INCLUDE (INvestigation of Co-occurring conditions across the Lifespan to Understand Down syndromE) initiative has bolstered capacity for the current increase in clinical trials involving individuals with Down syndrome (DS). This new NIH funding mechanism offers new opportunities to expand and develop novel approaches in engaging and effectively enrolling a broader representation of clinical trials participants addressing current medical issues faced by individuals with DS. To address this opportunity, the NIH assembled leading clinicians, scientists, and representatives of advocacy groups to review existing methods and to identify those areas where new approaches are needed to engage and prepare DS populations for participation in clinical trial research. This paper summarizes the results of the Clinical Trial Readiness Working Group that was part of the INCLUDE Project Workshop: Planning a Virtual Down Syndrome Cohort Across the Lifespan Workshop held virtually September 23 and 24, 2019.

Keywords: Clinical research; Clinical trials; Down syndrome; Intellectual disability; Recruitment; Research engagement.

Conducting clinical trials in persons with Down syndrome: summary from the NIH INCLUDE Down syndrome clinical trials readiness working group

Authors

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Abstract

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Grants and funding