Symptoms and General Pathology

All Conditions

Neoplasms

Nervous System Diseases

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Rare Diseases

Sclerosis

Tuberous Sclerosis

Hamartoma

Neoplasms, Multiple Primary

Syndrome

Neoplastic Syndromes, Hereditary

Congenital Abnormalities

Malformations of Cortical Development

Nervous System Malformations

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Tuberous Sclerosis Complex

Antineoplastic Agents

All Drugs and Chemicals

Anti-Infective Agents

Everolimus

Sirolimus

Temsirolimus

MTOR Inhibitors

Protein Kinase Inhibitors

Enzyme Inhibitors

Immunosuppressive Agents

Immunologic Factors

Everolimus

* Fixed doses orally once a day per each 28 day cycle
* Participants will stay on study as long as they do not progress for a maximum of 24 months.
* Tumor assessments will be performed after every 2 cycles for as long as they are on study.

David Kwiatkowski, MD, PhD

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.

This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called everolimus to learn whether the drug works in treating a specific cancer. "Investigational" means that the drug is being studied. It also means that the FDA (the U.S. Food and Drug Administration) has not yet approved everolimus for your type of cancer.

Everolimus is a drug that may stop cancer cells from growing by blocking an important factor (mTOR) involved in the growth of cells. This drug has been used in treatment for other cancers and is approved by the Food and Drug Administration for treatment of several types of cancer, including renal cell carcinoma. Treatment with this drug has been associated with responses in some patients whose cancers had mutations in TSC1 or TSC2. The investigators think that patients whose tumors have mutations in TSC1 or TSC2 may have a good chance of responding to treatment with drugs like everolimus.

Patients who fulfill eligibility criteria will be entered into the trial.The participant will be given a study drug-dosing calendar for each treatment cycle. Each treatment cycle lasts 28 days (4 weeks), during which time the participant will be taking the study drug orally (by mouth) once daily. The diary will also include special instructions for taking the study drug. In addition to the administration of the study drugs the participant will be asked to return to the clinic at various time points so that additional exams can be performed. These study visits may last as long as 2 hours.

Everolimus for Cancer With TSC1 or TSC2 Mutation

A Phase II Trial of Everolimus for Cancer Patients With Inactivating Mutations in TSC1 or TSC2 or Activating MTOR Mutations

RECIST 1.1 criteria for Objective Response:

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by scan: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

CTCAE v4.0 Toxicity Rate Grade 3 or higher

Novartis Pharmaceuticals

Dana-Farber Cancer Institute

Principal Investigator

National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE v4.0)

Everolimus

* Fixed doses orally once a day per each 28 day cycle
* Participants will stay on study as long as they do not progress for a maximum of 24 months.
* Tumor assessments will be performed after every 2 cycles for as long as they are on study.

Everolimus

Age, Categorical

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

United States

Region of Enrollment

This is ECOG Performance Status 0 is the best, meaning in very good condition. 4 is the worse, meaning in very poor condition.

ECOG Performance Status

Dr. David Kwiatkowski

Objective Response Rate

Duration of Response

Progression-free Survival

Overall Survival

Toxicity Rate

Overall Study

Perivascular Epithelioid Cell Neoplasms

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Anti-Bacterial Agents

Antibiotics, Antitubercular

Clotrimazole

Miconazole

Antifungal Agents

ABI-009

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 Malignancy with Relevant Genetic Mutations or mTOR Pathway Activation

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 Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation

Aadi Bioscience, Inc.

Neoplasm Metastasis

Neoplastic Processes

Patients with pathogenic inactivating TSC1 alterations.

Patients with pathogenic inactivating TSC2 alterations.

Prospective phase 2, open-label, multi-institutional basket trial to determine the efficacy and safety of nab-sirolimus administered by IV infusion to patients

nab-sirolimus

Aadi Bioscience Medical Information

Tempus

Karla Childers

Sherri Arledge, MD

Erika Arguello Vargas

Allan Espinosa Morazan, MD

Terry Thomas

Lyndsay Willmott, MD

Erika Aruello

Alphea Bonds Castro

David Palchak, MD

Kumar Sankhala, MD

Nazeli Livaskani

Jacob Thomas, MD

Nazanin Kavenhfar

Noah Federman, MD

Jericho Rabago

Michael Demeure, MD

Victoria Chua

Erlinda Maria Gordon, MD

Camille Shaffer

Ian Anderson, MD

Khadija Bedar

Kristen Ganjoo, MD

Omkar Marathe, MD

Jennifer Hege

Patrick Eulitt, MD

Staci Williams

Maen Hussein, MD

Patel Chandni

Magali Van den Bergh, MD

Chandni Patel

Barry S Berman, MD

Chandri Park

Justin Elkins

Gaurav Trikha, MD

Robert Doran

Shanna Kennedy

Kamal Sharma, DO

Chandni Park

Praneetha Achanta

Srilata Gundala, MD

Sunil Babu, MD

Chelsea Formato

Ebenezer Kio, MD

Jenelle Larkin

Mark Goldstein, MD

Ava Greenberg

Candace Haddox, MD

Ola Atchison

Dipesh Uprety, MD

Jinan Ali

Sushma Tatineni

Allie Gordon

Brian A Van Tine, MD

Lauren Murphy

David Crockett, MD

Teresa Ryba

Brent Tierney, MD

Audrey Ades

Jill Morrison, MD

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

Study TSC-007 is a prospective phase 2, open-label, multi-institutional basket trial to determine the efficacy and safety profile of nab-sirolimus administered to patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes. Patients will be treated with single agent IV nab-sirolimus until disease progression, or unacceptable toxicity, or until in the opinion of the investigator the patient is no longer benefiting from therapy, or at patient discretion.

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.

ORR based on the proportion of patients with best overall response (BOR) of confirmed partial response (PR) or complete response (CR) from the time of study treatment initiation until disease progression as determined by IRR using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1

Determined for patients with BOR of confirmed CR or PR (by IRR)

BOR of confirmed CR or PR (either of any duration) or stable disease (SD) following study treatment initiation (by IRR)

Time from first dose of study drug to initial measurement of CR or PR, where CR or PR is subsequently confirmed

Number of months from study treatment initiation to the date of disease progression (by IRR) or death due to any cause

Number of months from study treatment initiation to the date of death due to any cause

Changes from baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire v3.0 (EORTC-QOQ-C30) scores

Incidence and severity of treatment-emergent and treatment-related AEs as assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0

Musculoskeletal Diseases

Muscle Cramp

Spasm

Epilepsy

Seizures

Lennox Gastaut Syndrome

Spasms, Infantile

Epilepsies, Myoclonic

Neurologic Manifestations

Epilepsy, Generalized

Brain Diseases

Central Nervous System Diseases

Epileptic Syndromes

Dravet Syndrome

CDKL5 Deficiency Disorder

Lennox-Gastaut Syndrome

West Syndrome

Pharmaceutical Solutions

Anticonvulsants

Cannabidiol

The 52-week treatment period includes a fixed 2-week titration schedule followed by flexible dose optimization.

Day 1: 5 mg/kg/day (2.5 mg/kg twice daily (b.i.d.))

Day 8: 10 mg/kg/day (5 mg/kg b.i.d.)

Day 15 to Week 52: Flexible dosing based on the participant's observed efficacy, safety, and tolerability per the investigator's clinical judgement. Up to a maximum of 20 mg/kg/day (10 mg/kg b.i.d.) for LGS and DS or 25 mg/kg/day (12.5 mg/kg b.i.d.) for TSC, in maximum weekly increments of 5 mg/kg/day (≤ 2.5 mg/kg b.i.d.).

GWP42003-P

Clinical Trial Disclosure & Transparency

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 syndrome (DS).

The study duration will be up to approximately 62 weeks, including a 4-week screening/baseline period, a 52-week dose optimization treatment period (which includes a fixed 2-week titration period followed by flexible dose optimization), a 10-day taper period, and a safety follow-up period (4 weeks after the end-of-taper visit).

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

An Open-label, Single-arm Study to Assess the Safety, Pharmacokinetics, and Efficacy of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Participants With Tuberous Sclerosis Complex (Age 1 Month to < 2 Years of Age), Dravet Syndrome (1 Year to < 2 Years of Age), or Lennox-Gastaut Syndrome (1 Year to < 2 Years of Age) Who Experience Inadequately-controlled Seizures

The CGIC/S is a comprehensive neurodevelopmental assessment that covers the following domains: sensory, motor, cognition, emotional/behavioral health, communication, social, and adaptive functioning. This assessment is a 2-question survey per domain to be completed by the clinician. Individual domain scores will be summed and the total will be reported. Higher scores indicate poor clinical outcome.

The Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) was developed for use in infants and toddlers from 12-months-to-5 years of age and assesses levels of health and well-being. The caregiver will complete the assessment on an electronic device. For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). Higher scores indicate better clinical outcome.

Treatment Responders are defined as participants with ≥ 50% reduction from baseline in caregiver-reported total countable seizures

This endpoint includes the following changes in percentage of seizures:

* \> 25% (increase);
* ≥ 0% to ≤ 25% (increase);
* \> -25% to \< 0% (reduction);
* \> -50% to ≤ -25% (reduction);
* \> -75% to ≤ -50% (reduction);
* ≤ -75% (reduction).

Jazz Pharmaceuticals

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Everolimus for Cancer With TSC1 or TSC2 Mutation	NCT02201212	TSC1 TSC2 Tuberous Sclero... MTOR	Everolimus	18 Years -	Dana-Farber Cancer Institute
Expanded Access for ABI-009 in Patients With Advanced PEComa and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation	NCT03817515	PEComa, Maligna... TSC1 TSC2 mTOR Pathway Ab...	ABI-009	18 Years - 85 Years	Aadi Bioscience, Inc.
Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)	NCT05103358	Tumor Tumor, Solid Metastasis Metastatic Canc... Cancer Cancer Metastat... Tumors Neoplasms Neoplasm Metast... Solid Tumor Advanced Solid ... Advanced Cancer Malignant Solid... Malignant Solid... Malignant Neopl... Malignant Tumor TSC TSC1 TSC2 Metastatic Soli... Metastatic Neop...	nab-sirolimus	12 Years -	Aadi Bioscience, Inc.
Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)	NCT05103358	Tumor Tumor, Solid Metastasis Metastatic Canc... Cancer Cancer Metastat... Tumors Neoplasms Neoplasm Metast... Solid Tumor Advanced Solid ... Advanced Cancer Malignant Solid... Malignant Solid... Malignant Neopl... Malignant Tumor TSC TSC1 TSC2 Metastatic Soli... Metastatic Neop...	nab-sirolimus	12 Years -	Aadi Bioscience, Inc.
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	NCT04485104	Seizure in Part... Seizure in Part... Seizure in Part...	GWP42003-P	1 Month - 23 Months	Jazz Pharmaceuticals

Spots Global Cancer Trial Database for tsc1

Every month we try and update this database with for tsc1 cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for tsc1

Every month we try and update this database with for tsc1 cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for tsc1 cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.