“The TSC Clinical Research Consortium has been the cornerstone of Federally funded research efforts in the US for the past decade. Only through the collaborative research efforts of the TSC clinics and partnership with the TSC Alliance have we begun to understand the complexities of the neurological manifestations of this rare disease. It is an exciting time for the consortium as it expands to include additional TSC clinics across the US. This will facilitate an array of clinical research initiatives that encompass more individuals with TSC and various aspects of their condition.”
Martina Bebin, MD, MPA, University of Alabama at Birmingham
The execution of clinical studies requires extensive planning, cooperation, and collaboration to protect the safety of participants and the integrity of valuable data.
To initiate and implement clinical studies more quickly and effectively, researchers from five TSC Clinics together with the TSC Alliance formed the TSC Clinical Research Consortium in 2012. This team led the implementation of multiple impactful clinical studies, two of which are ongoing, focused on epilepsy, autism, and intellectual disability, primarily in children. These included the PREVeNT, trial which included 13 TSC Clinic sites.
In 2024, the TSC Alliance is coordinating an expansion of the Clinical Research Consortium to foster collaborations leading to clinical studies relevant to individuals with TSC of all ages and all manifestations. Leveraging the large network of TSC Clinics and Centers of Excellence, the consortium can accommodate many types of studies, from healthcare quality improvement studies to double-blind, placebo-controlled trials. We aim to increase the number of impactful clinical studies in all aspects of TSC and all ages from prenatal through adult by convening passionate clinicians and researchers across our clinic network.
Investigators and sponsors are invited to contact Steve Roberds, PhD, at the TSC Alliance at firstname.lastname@example.org to find out how to partner with the TSC Clinical Research Consortium.
- CURRENTLY ENROLLING: Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (TSC-STEPS)
- CURRENTLY ENROLLING: Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
- Preventing Epilepsy using Vigabatrin in Infants with TSC (PREVeNT)
- Early Biomarkers of Autism in Infants with TSC
- Potential EEG Biomarkers and Antiepileptogenic Strategies for Epilepsy in TSC
Thanks to generous donations from the Cowlin Family Fund, the TSC Alliance supported early establishment of the data coordinating center and dedicated clinical coordinator time in 2012 and 2013 to help accelerate launch of the first studies. Clinical studies have been funded primarily by the National Institutes of Health and the Food and Drug Administration with supplemental funding from the TSC Alliance. We are grateful for the ongoing support of the Clinical Research Consortium from the Gerry and Bill Cowlin Foundation, Jim and the late Andrea Maginn, as well as the Foglia Family Foundation and Diane McSwain for their support of the Bcureful® Travel Fund which provides assistance for families to travel to clinical studies.