Clinical Trials Logo

Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05103358
Other study ID # TSC-007
Secondary ID
Status Active, not recruiting
Phase Phase 2
First received
Last updated
Start date February 15, 2022
Est. completion date December 31, 2025

Study information

Verified date May 2024
Source Aadi Bioscience, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


Description:

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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 120
Est. completion date December 31, 2025
Est. primary completion date December 31, 2024
Accepts healthy volunteers No
Gender All
Age group 12 Years and older
Eligibility Inclusion Criteria: 1. Patients must have a malignant solid tumor with a pathogenic inactivating TSC1 or TSC2 alteration. Genetic alterations should be identified using NGS in tumor tissue or liquid biopsy). • Patients will be enrolled after the central evaluation of NGS report confirms eligibility. 2. Patients must have solid tumors that are metastatic or locally advanced where surgical resection is not an option or likely to result in severe morbidity. 3. Patients must have received all standard therapies appropriate for their tumor type and stage of disease or, in the opinion of the Investigator, the patient would be unlikely to tolerate or derive clinically meaningful benefit from appropriate standard of care therapy, or the patient has no satisfactory alternative treatments. 4. Patients must have 1 or more measurable target lesions by computed tomography (CT) scan or magnetic resonance imaging (MRI) (RECIST v1.1). 5. Age: 12 years or older. 6. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 or Karnofsky Performance Status (KPS) =80 or Lansky play-performance scale for pediatric patients =80. 7. Adequate liver function: 1. Total bilirubin =1.5 × upper limit of normal (ULN) (unless due to Gilbert's syndrome, then =3 × ULN) 2. Aspartate aminotransferase (AST) =2.5 × ULN (=5 × ULN if attributable to liver metastases) 8. Adequate renal function: creatinine clearance =30 mL/min, Cockcroft-Gault CCr = ((140-age) × weight[kg]) / (72 × SCr[mL/min]) × 0.85, if female 9. Adequate hematologic parameters: 1. Absolute neutrophil count (ANC) =1.0 × 109/L (growth factor support allowed) 2. Platelet count =100,000/mm3 (100 × 109/L) (transfusion and/or growth factor support allowed) 3. Hemoglobin =8.0 g/dL (transfusion and/or growth factor support allowed) 10. Fasting serum triglyceride must be =300 mg/dL; fasting serum cholesterol must be =350 mg/dL. 11. Minimum of 4 weeks since any major surgery, completion of radiation, or completion of prior systemic anticancer therapy, or at least 5 half-lives if the prior therapy is a single agent small-molecule therapeutic, and adequately recovered from the acute toxicities of any prior therapy, including neuropathy, to Grade =1. 12. Male or non-pregnant and non-breastfeeding female: 1. Females of childbearing potential must agree to use effective contraception or abstinence without interruption from 28 days prior to starting investigational product (IP) throughout 3 months after last dose of IP and have a negative serum pregnancy test (beta human chorionic gonadotropin, ß-hCG) result at screening and agree to ongoing pregnancy testing during the course of the study, and after the end of study treatment. A second form of birth control is required even if she has had a tubal ligation. 2. Male patients must agree not to donate sperm and must practice abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study and throughout 3 months after last dose of IP. A second form of birth control is required even if he has undergone a successful vasectomy. 13. The patient or the patient's parent(s) or legal guardian(s) understand(s) and sign(s) the informed consent. 14. Willingness and ability to comply with scheduled visits, laboratory tests, and other study procedures. Exclusion Criteria: 1. Prior treatment with an mTOR inhibitor, including nab-sirolimus. 2. Severe (Grade =3) ongoing infection requiring parenteral or oral anti-infective treatment, either ongoing or completed =7 days prior to enrollment. 3. Patients with primary brain tumors or PEComa. 4. Patients who have any severe and/or uncontrolled medical or psychiatric conditions or other conditions that could affect their participation including: 1. Patients with meningeal carcinomatosis, leptomeningeal carcinomatosis, spinal cord compression, untreated brain metastases or symptomatic or unstable brain metastases. Note: Patients with stable brain metastases (defined as asymptomatic or no requirement for high-dose [defined as dexamethasone 10 mg daily or higher] or increasing dose of systemic corticosteroids) and without imminent need of radiation therapy are eligible. If applicable, patients must have completed brain radiation therapy and recovered adequately from any associated toxicity and/or complications prior to eligibility assessment. For patients who have received prior radiation therapy, post-treatment MRI scan should show no increase in brain lesion size/volume. 2. Unstable angina pectoris, symptomatic congestive heart failure (New York Heart Association, NYHA class III or IV), myocardial infarction =6 months prior to first study treatment, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease. 3. Pre-existing severely impaired lung function. If a patient has a pre-existing pulmonary condition, eligible patients should have a spirometry and diffusing capacity for carbon monoxide (DLCO) that is >50% of the normal predicted value and/or O2 saturation that is >88% at rest on room air (Note: spirometry and pulmonary function tests [PFTs] not required to be performed unless clinically indicated). 4. Nonmalignant medical illnesses that are uncontrolled or whose control may be jeopardized by the treatment with the study therapy. 5. A history of malignancies other than the one under treatment unless the patient is disease-free for more than 5 years from diagnosis. Note, controlled non-melanoma skin cancers, carcinoma in situ of the cervix, resected incidental prostate cancer, certain low grade hematologic malignancies (eg CLL, follicular lymphoma, etc), or other adequately treated carcinoma-in-situ may be eligible, after discussion with the medical monitor. 6. Uncontrolled hypertension (systolic blood pressure =160 mm-Hg and/or diastolic blood pressure =100 mm Hg). 7. Patients with history of interstitial lung disease and/or pneumonitis, or pulmonary hypertension. 8. Individuals with known human immunodeficiency virus (HIV) infection are excluded from this study as combination antiretroviral therapy could potentially result in significant pharmacokinetic interactions. In addition, these individuals are at increased risk of serious infections due to the immunosuppressive effects of mTOR inhibition. 9. Active Hepatitis B or Hepatitis C, with detectable viral load. 5. Regarding concomitant medications with significant CYP3A4 and P-gp interactions, discontinuation of strong inhibitors (eg, ketoconazole, itraconazole, voriconazole, erythromycin, clarithromycin, telithromycin, and others), strong inducers (eg, rifampin, rifabutin), and known CYP3A4 substrates with a narrow therapeutic window (eg, fentanyl, alfentanil, astemizole, cisapride, dihydroergotamine, pimozide, quinidine, or terfenadine) is required at least 5 half lives prior to receiving the first dose of nab-sirolimus, whichever is longer.

Study Design


Related Conditions & MeSH terms

  • Advanced Cancer
  • Advanced Solid Tumor
  • Cancer
  • Cancer Metastatic
  • Malignant Neoplasm
  • Malignant Solid Neoplasm
  • Malignant Solid Tumor
  • Malignant Tumor
  • Metastasis
  • Metastatic Cancer
  • Metastatic Neoplasm
  • Metastatic Solid Tumor
  • Neoplasm Metastasis
  • Neoplasms
  • Solid Tumor
  • TSC
  • TSC1
  • TSC2
  • Tumor
  • Tumor, Solid
  • Tumors

Intervention

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

Locations

Country Name City State
Korea, Republic of Inje University Haeundae Paik Hospital Busan
Korea, Republic of Asan Medical Center Seoul
Korea, Republic of Korea University Guro Hospital Seoul
Korea, Republic of Samsung Medical Center Seoul
Korea, Republic of Seoul National University Hospital Seoul
Korea, Republic of Severance Hospital, Yonsei University Health System Seoul
Puerto Rico Pan Oncology Trials, LLC San Juan
United States Florida Cancer Specialists - North Division Altamonte Springs Florida
United States ThedaCare Appleton Wisconsin
United States PCR Oncology Arroyo Grande California
United States Morehouse School of Medicine Atlanta Georgia
United States Rocky Mountain Cancer Centers Aurora Colorado
United States Our Lady of the Lake Baton Rouge Louisiana
United States New Jersey Cancer Care and Blood Disorders Belleville New Jersey
United States American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders) Bethesda Maryland
United States Nextgen Oncology Beverly Hills California
United States Alabama Oncology Birmingham Alabama
United States Central Care Cancer Center Bolivar Missouri
United States Florida Cancer Specialists - South Division Bonita Springs Florida
United States Dana Farber Cancer Institute Boston Massachusetts
United States Rocky Mountain Cancer Centers Boulder Colorado
United States Florida Cancer Specialists - South Division Bradenton Florida
United States Florida Cancer Specialists - South Division Bradenton Florida
United States Florida Cancer Specialists - North Division Brandon Florida
United States Roswell Park Comprehensive Cancer Center Buffalo New York
United States Aultman Medical Group Canton Ohio
United States Florida Cancer Specialists - South Division Cape Coral Florida
United States Urology of Indiana Carmel Indiana
United States TriHealth Cincinnati Ohio
United States University of Cincinnati (UC) - Cancer Institute Cincinnati Ohio
United States Florida Cancer Specialists - North Division Clearwater Florida
United States Cleveland Clinic Cleveland Ohio
United States Rocky Mountain Cancer Centers Colorado Springs Colorado
United States Mary Crowley Cancer Research Dallas Texas
United States Texas Oncology - DFW Dallas Texas
United States Cancer Specialist - East Daytona Beach Florida
United States Rocky Mountain Cancer Centers Denver Colorado
United States Rocky Mountain Cancer Centers (Williams St) Denver Colorado
United States Barbara Ann Karmanos Cancer Institute Detroit Michigan
United States Sarah Cannon and HCA Research Institute Dickson Tennessee
United States City of Hope Duarte California
United States HOACNY East Syracuse New York
United States Texas Oncology El Paso Texas
United States Englewood Hospital and Medical Center Englewood New Jersey
United States Virginia Cancer Specialists Fairfax Virginia
United States Southcoast Centers for Cancer Care Fairhaven Massachusetts
United States Sanford Health-Fargo Fargo North Dakota
United States Summit Medical Group - NJ Florham Park New Jersey
United States Holy Cross Hospital Fort Lauderdale Florida
United States Florida Cancer Specialists - South Fort Myers Florida
United States Florida Cancer Specialists - South Fort Myers Florida
United States Florida Cancer Specialists South Division Fort Myers Florida
United States Fort Wayne Medical Oncology and Hematology Fort Wayne Indiana
United States Sarah Cannon and HCA Research Institute Franklin Tennessee
United States Frederick Health Frederick Maryland
United States Hematology Oncology Associates of Fredericksburg Fredericksburg Virginia
United States Providence Medical Foundation (Fullerton) Fullerton California
United States Florida Cancer Specialists - North Division Gainesville Florida
United States Sarah Cannon and HCA Research Institute Gallatin Tennessee
United States West Cancer Center Germantown Tennessee
United States Gettysburg-PCSRI Gettysburg Pennsylvania
United States Southeastern Medical Oncology Goldsboro North Carolina
United States Arizona Oncology Associates Goodyear Arizona
United States Goshen Health Goshen Indiana
United States Nebraska Cancer Specialists Grand Island Nebraska
United States Prisma Health Cancer Institute Greenville South Carolina
United States Pontchartrain Hammond Louisiana
United States Hartford Healthcare Hartford Connecticut
United States Sarah Cannon and HCA Research Institute Henderson Tennessee
United States Sarah Cannon and HCA Research Institute Hermitage Tennessee
United States Hope and Healing Cancer Services Hinsdale Illinois
United States Hawaii Cancer Center Honolulu Hawaii
United States MD Anderson Cancer Center Houston Texas
United States Oncology Consultants Houston Texas
United States Arizona Oncology Associates Irving Texas
United States Cayuga Medical Center Ithaca New York
United States Cancer Specialists of North Florida Jacksonville Florida
United States Lumi Research Kingwood Texas
United States Gunderson Health System La Crosse Wisconsin
United States The Oncology Institute of Hope and Innovation Lakeland Florida
United States TOI Florida Lakeland Florida
United States Rocky Mountain Cancer Centers Lakewood Colorado
United States Alliance Cancer Specialists Langhorne Pennsylvania
United States Sparrow Hospital Lansing Michigan
United States Florida Cancer Specialists - North Division Largo Florida
United States Comprehensive Cancer Centers of Nevada Las Vegas Nevada
United States OptumCare Cancer Care-Parent Las Vegas Nevada
United States Sarah Cannon and HCA Research Institute Lebanon Tennessee
United States Florida Cancer Specialists - North Division Lecanto Florida
United States Rocky Mountain Cancer Centers Littleton Colorado
United States Rocky Mountain Cancer Centers Lone Tree Colorado
United States MemorialCare Long Beach California
United States Rocky Mountain Cancer Centers Longmont Colorado
United States UCLA - Jonsson Comprehensive Cancer Center Los Angeles California
United States USC Norris Comprehensive Cancer Center Los Angeles California
United States University of Wisconsin - Carbone Cancer Center Madison Wisconsin
United States Texas Oncology Central-South McAllen Texas
United States Baptist Cancer Center Memphis Tennessee
United States Minnesota Oncology Hematology Minneapolis Minnesota
United States Southern Cancer Center Mobile Alabama
United States Atlantic Health System - Morristown Medical Center Morristown New Jersey
United States Sarah Cannon and HCA Research Institute Murfreesboro Tennessee
United States Providence Medical Foundation (Napa) Napa California
United States Florida Cancer Specialists - South Division Naples Florida
United States Sarah Cannon and HCA Research Institute Nashville Tennessee
United States Sarah Cannon and HCA Research Institute Nashville Tennessee
United States Sarah Cannon and HCA Research Institute Nashville Tennessee
United States Sarah Cannon and HCA Research Institute Nashville Tennessee
United States David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center New York New York
United States Hoag Memorial Hospital Presbyterian Newport Beach California
United States Eastern Connecticut Hematology and Oncology Norwich Connecticut
United States Florida Cancer Specialists - North Division Ocala Florida
United States Ocala Oncology Ocala Florida
United States Community Cancer Trials of Utah Ogden Utah
United States Oklahoma State University (OSU) - Stephenson Cancer Center Oklahoma City Oklahoma
United States Nebraska Methodist Hospital Omaha Nebraska
United States Florida Cancer Specialists - North Division Orange City Florida
United States Florida Cancer Specialists - North Division Orlando Florida
United States Lake Regional Osage Beach Missouri
United States Thomas Jefferson University Philadelphia Pennsylvania
United States Honor Health Phoenix Arizona
United States Florida Cancer Specialists - South Division Port Charlotte Florida
United States Arizona Oncology Associates Prescott Valley Arizona
United States Rocky Mountain Cancer Centers Pueblo Colorado
United States Virginia Urology Richmond Virginia
United States Maryland Oncology Hematology Rockville Maryland
United States Northwest Oncology and Hematology Rolling Meadows Illinois
United States Mosaic Life Care Saint Joseph Missouri
United States Washington University School of Medicine Saint Louis Missouri
United States Florida Cancer Specialists - North Saint Petersburg Florida
United States Florida Cancer Specialists and Research Institute - North Division Saint Petersburg Florida
United States Utah Cancer Specialists Salt Lake City Utah
United States South Texas Accelerated Research Therapeutics (START) San Antonio Texas
United States Sharp HealthCare San Diego California
United States UCSF Helen Diller Family Comprehensive Cancer Center San Francisco California
United States Ridley-Tree Cancer Center Santa Barbara California
United States Sarcoma Oncology Research Center Santa Monica California
United States Providence Medical Foundation Santa Rosa California
United States Florida Cancer Specialists - South Division Sarasota Florida
United States Florida Cancer Specialists - South Division Sarasota Florida
United States University of Washington Cancer Consortium Seattle Washington
United States Sarah Cannon and HCA Research Institute Shelbyville Tennessee
United States Avera Cancer Institute Sioux Falls South Dakota
United States Sanford Health Sioux Falls South Dakota
United States Sarah Cannon and HCA Research Institute Smyrna Tennessee
United States Cancer Care Northwest Spokane Washington
United States Spokane Urology Spokane Washington
United States Oncology Hematology Associates Springfield Missouri
United States Stanford Cancer Center Stanford California
United States Florida Cancer Specialist - East Stuart Florida
United States Northwest Medical Specialties Tacoma Washington
United States Florida Cancer Specialists - North Division Tampa Florida
United States Florida Cancer Specialists - North Division Tavares Florida
United States Rocky Mountain Cancer Centers Thornton Colorado
United States The Toledo Clinic Toledo Ohio
United States Florida Cancer Specialists - North Division Trinity Florida
United States Oklahoma Cancer Specialist Tulsa Oklahoma
United States Texas Oncology Tyler Texas
United States Florida Cancer Specialists - South Division Venice Florida
United States Florida Cancer Specialists - South Division Venice Florida
United States Florida Cancer Specialists - East Vero Beach Florida
United States Florida Cancer Specialists - East Wellington Florida
United States Florida Cancer Specialists - East West Palm Beach Florida
United States The Oncology Institute of Hope & Innovation Whittier California
United States Cancer Care Associates of York - Parent York Pennsylvania
United States Yuma Regional Medical Center Yuma Arizona

Sponsors (1)

Lead Sponsor Collaborator
Aadi Bioscience, Inc.

Countries where clinical trial is conducted

United States,  Korea, Republic of,  Puerto Rico, 

Outcome

Type Measure Description Time frame Safety issue
Primary Overall response rate (ORR) 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 9 months
Secondary Duration of response (DOR) Determined for patients with BOR of confirmed CR or PR (by IRR) 9 months
Secondary Disease control rate BOR of confirmed CR or PR (either of any duration) or stable disease (SD) following study treatment initiation (by IRR) 9 months
Secondary Time to response Time from first dose of study drug to initial measurement of CR or PR, where CR or PR is subsequently confirmed 9 months
Secondary Progression-free survival Number of months from study treatment initiation to the date of disease progression (by IRR) or death due to any cause 9 months
Secondary Overall survival Number of months from study treatment initiation to the date of death due to any cause 24 months
Secondary Patient-reported outcome Changes from baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire v3.0 (EORTC-QOQ-C30) scores 9 months
Secondary Incidence and severity of treatment-emergent and treatment-related adverse events (AEs) 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 9 months
See also
  Status Clinical Trial Phase
Recruiting NCT05346796 - Survivorship Plan HEalth REcord (SPHERE) Implementation Trial N/A
Recruiting NCT05094804 - A Study of OR2805, a Monoclonal Antibody Targeting CD163, Alone and in Combination With Anticancer Agents Phase 1/Phase 2
Completed NCT04867850 - Effect of Behavioral Nudges on Serious Illness Conversation Documentation N/A
Enrolling by invitation NCT04086251 - Remote Electronic Patient Monitoring in Oncology Patients N/A
Completed NCT01285037 - A Study of LY2801653 in Advanced Cancer Phase 1
Completed NCT00680992 - Study of Denosumab in Subjects With Giant Cell Tumor of Bone Phase 2
Completed NCT00062842 - Study of Irinotecan on a Weekly Schedule in Children Phase 1
Active, not recruiting NCT04548063 - Consent Forms in Cancer Research: Examining the Effect of Length on Readability N/A
Completed NCT04337203 - Shared Healthcare Actions and Reflections Electronic Systems in Survivorship N/A
Recruiting NCT04349293 - Ex-vivo Evaluation of the Reactivity of the Immune Infiltrate of Cancers to Treatments With Monoclonal Antibodies Targeting the Immunomodulatory Pathways N/A
Terminated NCT02866851 - Feasibility Study of Monitoring by Web-application on Cytopenia Related to Chemotherapy N/A
Active, not recruiting NCT05304988 - Development and Validation of the EFT for Adolescents With Cancer
Completed NCT04448041 - CRANE Feasibility Study: Nutritional Intervention for Patients Undergoing Cancer Surgery in Low- and Middle-Income Countries
Completed NCT00340522 - Childhood Cancer and Plexiform Neurofibroma Tissue Microarray for Molecular Target Screening and Clinical Drug Development
Recruiting NCT04843891 - Evaluation of PET Probe [64]Cu-Macrin in Cardiovascular Disease, Cancer and Sarcoidosis. Phase 1
Active, not recruiting NCT03844048 - An Extension Study of Venetoclax for Subjects Who Have Completed a Prior Venetoclax Clinical Trial Phase 3
Completed NCT03109041 - Initial Feasibility Study to Treat Resectable Pancreatic Cancer With a Planar LDR Source Phase 1
Completed NCT03167372 - Pilot Comparison of N-of-1 Trials of Light Therapy N/A
Terminated NCT01441115 - ECI301 and Radiation for Advanced or Metastatic Cancer Phase 1
Recruiting NCT06206785 - Resting Energy Expenditure in Palliative Cancer Patients