Patient Engagement
Partnerships for CDKL5 Deficiency Disorder
International Foundation for CDKL5 Research
The International Foundation for CDKL5 Research is the leader in CDKL5 patient education, providing guides for families with the information and resources they need to prioritize care for their loved one, while partnering with other CDKL5 organizations globally to adapt these guides to other countries and cultures. They have also funded critical research for expanding our knowledge of CDKL5 deficiency disorder through basic science and clinical research initiatives. They are responsible for establishment and maintenance of the CDKL5 Centers of Excellence within the United States, of which eight currently exist, and they partner with the International CDKL5 Clinical Research Network to support their Clinical Trial Readiness Study.
LouLou Foundation
The Loulou Foundation is a private non-profit foundation based in the United Kingdom that is dedicated to advancing research into the understanding and development of therapeutics for CDKL5 deficiency disorder. They have funded over 60 separate projects across the world to drive this goal, including an online portal to foster new ideas and communication between CDKL5 collaborators around the world: cdkl5forum.org.
CDKL5 Alliance
The goal of the CDKL5 Alliance is to promote cooperation, research and care, and awareness of the condition on a combined front, targeting the medical, scientific and pharmaceutical communities. They are a globally recognized organization responsible for hosting annual conferences focused on the collaborative efforts of management and treatment of CDKL5.
CLINICAL TRIAL
International CDKL5 Clinical Research Network’s Clinical Trial Readiness Study
PI: Heather Olson, MD, MS
Natural History Study — The ICCRN is a collaborative network of researchers in the US and Australia that specialize in CDKL5 Deficiency Disorder (CDD). The goal of this study is to develop appropriate tools to facilitate future clinical trials in CDD, known as clinical outcome measures (COMs). Validated, standardized COMs are necessary to accurately and reproducibly track meaningful changes in clinical trials and can also inform clinical care. Suitable COMs and biomarkers do not currently exist for CDD, and this study will ensure that future clinical trials for CDD more adequately capture the impact of new therapeutics by identifying meaningful changes in the symptoms and quality of life of individuals with CDD.
Patient Advocacy Group Partner: International Foundation for CDKL5 Research (IFCR)