Tuberous Sclerosis clinical trials at UCLA
6 in progress, 2 open to eligible people
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
open to eligible people ages up to 6 months
This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants. This study is supported by research funding from the Office of Orphan Products Division (OOPD) of the US Food and Drug Administration (FDA).
Los Angeles, California and other locations
TSC Biosample Repository and Natural History Database
open to all eligible people
The TSC Biosample Repository collects and stores samples of blood, DNA, and tissues that scientists can request to use in their research. The samples we collect are all linked to clinical data in the TSC Natural History Database. The TSC Natural History Database captures clinical data to document the impact of the disease on a person's health over his or her lifetime. This data may be collected retrospectively or prospectively.
Los Angeles, California and other locations
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 to identify biomarkers using advanced MRI methodology and electrophysiological biomarkers of synaptic function and connectivity predictive of ASD and ID presence and severity in 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.
Los Angeles, California and other locations
Intermediate-Size Expanded Access Protocol (EAP) for LP352
Sorry, not accepting new patients
This is an intermediate-size expanded access program (EAP) study. The purpose of this EAP is to provide continued access to LP352, an investigational drug product being investigated in participants with DEEs. The EAP study will allow continued treatment with LP352 for eligible participants diagnosed with treatment resistant DEEs who successfully completed an LP352 Clinical Trial (Enrollment by Invitation) or an immediate family member who has the exact same gene mutation resulting in the same DEE epilepsy syndrome phenotype or a patient who previously participated in the lorcaserin EAP.
Los Angeles, California and other locations
Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)
Sorry, in progress, not accepting new patients
A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes
Los Angeles, California and other locations
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
Los Angeles, California and other locations
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