Tuberous Sclerosis clinical trials at UC Health
6 in progress, 3 open to eligible people

  • Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

    open to eligible people ages 6-65

    The study aims to compare the safety and efficacy of two different strengths of Rapamycin cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA) associated with Tuberous Sclerosis Complex (TSC).

    at UCSD

  • JASPER Early Intervention for Tuberous Sclerosis

    open to eligible people ages 6 months to 40 months

    The investigators are running an intervention study for young children with Tuberous Sclerosis Complex (TSC). The study will include free play-based behavioral intervention administered remotely that may improve social and communication skills in children with TSC. Eligible families will have a child in the age range of 12-36 months, with a diagnosis of TSC. Children with TSC below 12 months may be eligible for an early markers study prior to enrollment in the intervention trial. The intervention will focus on teaching caregivers skills to improve the social and communication outcomes of their children. The content of the intervention will be individually tailored to the child's developmental level. The intervention involves 4 on-site assessment visits, and 12 weekly intervention sessions, administered in-person and remotely. The intervention focuses on improving social-communication and play skills.

    at UCLA

  • Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

    open to eligible people ages up to 6 months

    Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to the onset of their first seizure

    at UCLA

  • Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)

    Sorry, in progress, not accepting new patients

    The purpose of this study is to characterize the developmental phenotype of ASD and ID and identify biomarkers using advanced MRI methodology predictive of ASD and ID presence and severity in pediatric patients with TSC. In addition, this study will be establishing infrastructure for the collection and storage of human bio-specimens, including genetic material, from TSC patients and their family members with ASD.

    at UCLA

  • Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

    Sorry, in progress, not accepting new patients

    The investigators are enrolling 3-12 month old infants with a diagnosis of tuberous sclerosis complex (TSC) for a new study on early markers of autism. The study is looking for early signs for autism in a population (TSC) where autism is common. The goal of this project is to use behavioral testing, MRI and EEG techniques to identify children at risk for developing autism starting at 3 months of age and continuing until 36 months of age. Throughout the study, the investigators will recommend Early Intervention services for any child who shows early signs of autism.

    at UCLA

  • Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

    Sorry, in progress, not accepting new patients

    The purpose of this study is to evaluate the long-term safety in patients with TSC and refractory seizures who are currently receiving everolimus treatment in the Novartis-sponsored EXIST-3 study and who are determined to be benefiting from continued treatment as judged by the investigator at the completion of EXIST-3

    at UCLA UCSF

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