Clinical trials: TSC2
TSC1, TSC2
Everolimus for Cancer With TSC1 or TSC2 Mutation
In this research study, the investigators are evaluating the clinical benefit of everolimus in cancer patients with inactivating TSC1 or TSC2 mutations or activating MTOR mutations.
- Conditions
- TSC1, TSC2, Tuberous Sclerosis Complex, MTOR
- Trial name
- NCT02201212
- Testing
- Everolimus
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Dana-Farber Cancer Institute
Solid Tumors, Lung Cancer
A Phase I Study of BKM120 and Everolimus in Advanced Solid Malignancies
This study will assess the safety of combining two agents (everolimus and BKM120) for the treatment of advanced cancer arising from solid organ in patients who are no longer benefiting from or unable to withstand standard…
- Trial name
- NCT01470209
- Testing
- BKM120, Everolimus
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Emory University
Solid Malignancy, Solid Tumor
Everolimus in Patients With Advanced Solid Malignancies With TSC1, TSC2, NF1, NF2, or STK11 Mutations
The purpose of this research study is to look at participants with solid tumor malignancies and specific mutations respond to treatment with everolimus.
- Trial name
- NCT02352844
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Washington University School of Medicine
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Testing MLN0128 (TAK-228) as Potentially Targeted Treatment in Cancers With TSC1 or TSC2 Genetic Changes (MATCH - Subprotocol M)
This phase II MATCH treatment trial tests how well MLN0128 (TAK-228) works in treating patients with cancer that has certain genetic changes called TSC1 or TSC2 mutations.
- Conditions
- Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Refractory Lymphoma, Refractory Malignant Solid Neoplasm
- Trial name
- NCT06390865
- Testing
- Biopsy Procedure, Biospecimen Collection, Computed Tomography
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- National Cancer Institute (NCI)
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.
- Trial name
- EXIST-2 (NCT00790400)
- Testing
- Everolimus (RAD001)
- Where
- 25 sites in United States, Canada and 9 more
- Volunteers
- 118 people
- Sponsor
- Novartis Pharmaceuticals
PEComa, Malignant, TSC1
Expanded Access for ABI-009 in Patients With Advanced PEComa and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation
Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients with a…
- Conditions
- PEComa, Malignant, TSC1, TSC2, mTOR Pathway Abberation
- Trial name
- NCT03817515
- Testing
- ABI-009
- Sponsor
- Aadi Bioscience, Inc.
Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome
Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures
This study will be conducted to evaluate the safety, pharmacokinetics (PK), and efficacy of adjunctive GWP42003-P in participants < 2 years of age with tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS), or Dravet…
- Conditions
- Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
- Trial name
- NCT04485104
- Testing
- GWP42003-P
- Where
- 11 sites in United States, Italy and 1 more
- Volunteers
- 3 people
- Sponsor
- Jazz Pharmaceuticals
Locally Advanced Bladder Urothelial Carcinoma, Metastatic Transitional Cell Carcinoma
Sapanisertib in Treating Patients With Locally Advanced or Metastatic Bladder Cancer With TSC1 and/or TSC2 Mutations
This pilot phase II trial studies how well sapanisertib works in treating patients with bladder cancer that has spread from where it started to nearby tissue or lymph nodes (locally advanced) or other places in the body…
- Conditions
- Locally Advanced Bladder Urothelial Carcinoma, Metastatic Transitional Cell Carcinoma, Metastatic Urothelial Carcinoma, Recurrent Bladder Carcinoma
- Trial name
- NCT03047213
- Testing
- Sapanisertib
- Where
- 20 sites in United States
- Volunteers
- 17 people
- Sponsor
- National Cancer Institute (NCI)
Leiomyosarcoma, Soft-tissue Sarcomas
A Phase I Trial of Combination Gemcitabine and Nab-Sirolimus in Advanced Leiomyosarcomas or Advanced Soft-Tissue Sarcomas With TSC2 or TSC1 Loss-of-function Mutations or Deletions
To find a recommended dose of gemcitabine and nab-sirolimus that can be given in combination to participants with advanced leiomyosarcomas or soft-tissue sarcomas.
- Trial name
- NCT06308419
- Testing
- Gemcitabine, Nab-Sirolimus
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- M.D. Anderson Cancer Center
Tumor, Tumor, Solid
Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)
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
- Conditions
- Tumor, Tumor, Solid, Metastasis, Metastatic Cancer
- Trial name
- NCT05103358
- Testing
- nab-sirolimus
- Where
- 166 sites in United States, Puerto Rico and 1 more
- Volunteers
- 128 people
- Sponsor
- Aadi Bioscience, Inc.