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Ensayo de canasta de fase 2 de Nab-sirolimus en pacientes con tumores sólidos malignos con alteraciones patogénicas en los genes TSC1 o TSC2 (PRECISION 1)

26 de octubre de 2026 actualizado por: Aadi Bioscience, Inc.

Un ensayo de canasta de etiqueta abierta multicéntrico de fase 2 de Nab-sirolimus para pacientes adultos y adolescentes con tumores sólidos malignos que albergan alteraciones patogénicas de inactivación en los genes TSC1 o TSC2

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

Descripción general del estudio

Estado

Terminado

Condiciones

Intervención / Tratamiento

Descripción detallada

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.

Tipo de estudio

Intervencionista

Inscripción (Actual)

128

Fase

  • Fase 2

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Corea del Sur
      • Busan, Corea del Sur, 48108
        • Inje University Haeundae Paik Hospital
      • Seoul, Corea del Sur, 03080
        • Seoul National University Hospital
      • Seoul, Corea del Sur, 05505
        • Asan Medical Center
      • Seoul, Corea del Sur, 06351
        • Samsung Medical Center
      • Seoul, Corea del Sur, 03722
        • Severance Hospital, Yonsei University Health System
      • Seoul, Corea del Sur, 08308
        • Korea University Guro Hospital
  • Estados Unidos
    • Alabama
      • Birmingham, Alabama, Estados Unidos, 35243
        • Alabama Oncology
      • Mobile, Alabama, Estados Unidos, 36607
        • Southern Cancer Center
    • Arizona
      • Goodyear, Arizona, Estados Unidos, 85395
        • Arizona Oncology Associates
      • Phoenix, Arizona, Estados Unidos, 85016
        • Honor Health
      • Prescott Valley, Arizona, Estados Unidos, 86301
        • Arizona Oncology Associates
      • Yuma, Arizona, Estados Unidos, 85364
        • Yuma Regional Medical Center
    • California
      • Arroyo Grande, California, Estados Unidos, 93420
        • PCR Oncology
      • Beverly Hills, California, Estados Unidos, 90212
        • Nextgen Oncology
      • Duarte, California, Estados Unidos, 91010
        • City of Hope
      • Fullerton, California, Estados Unidos, 92835
        • Providence Medical Foundation (Fullerton)
      • Long Beach, California, Estados Unidos, 90806
        • MemorialCare
      • Los Angeles, California, Estados Unidos, 90033
        • USC Norris Comprehensive Cancer Center
      • Los Angeles, California, Estados Unidos, 90095
        • UCLA - Jonsson Comprehensive Cancer Center
      • Napa, California, Estados Unidos, 94558
        • Providence Medical Foundation (Napa)
      • Newport Beach, California, Estados Unidos, 92663
        • Hoag Memorial Hospital Presbyterian
      • San Diego, California, Estados Unidos, 92123
        • Sharp HealthCare
      • San Francisco, California, Estados Unidos, 94115
        • UCSF Helen Diller Family Comprehensive Cancer Center
      • Santa Barbara, California, Estados Unidos, 93105
        • Ridley-Tree Cancer Center
      • Santa Monica, California, Estados Unidos, 90403
        • Sarcoma Oncology Research Center
      • Santa Rosa, California, Estados Unidos, 95403
        • Providence Medical Foundation
      • Stanford, California, Estados Unidos, 94305
        • Stanford Cancer Center
      • Whittier, California, Estados Unidos, 90602
        • The Oncology Institute of Hope & Innovation
    • Colorado
      • Aurora, Colorado, Estados Unidos, 80012
        • Rocky Mountain Cancer Centers
      • Boulder, Colorado, Estados Unidos, 80303
        • Rocky Mountain Cancer Centers
      • Colorado Springs, Colorado, Estados Unidos, 80907
        • Rocky Mountain Cancer Centers
      • Denver, Colorado, Estados Unidos, 80220
        • Rocky Mountain Cancer Centers
      • Denver, Colorado, Estados Unidos, 80218
        • Rocky Mountain Cancer Centers (Williams St)
      • Lakewood, Colorado, Estados Unidos, 80228
        • Rocky Mountain Cancer Centers
      • Littleton, Colorado, Estados Unidos, 80120
        • Rocky Mountain Cancer Centers
      • Lone Tree, Colorado, Estados Unidos, 80124
        • Rocky Mountain Cancer Centers
      • Longmont, Colorado, Estados Unidos, 80501
        • Rocky Mountain Cancer Centers
      • Pueblo, Colorado, Estados Unidos, 81008
        • Rocky Mountain Cancer Centers
      • Thornton, Colorado, Estados Unidos, 80260
        • Rocky Mountain Cancer Centers
    • Connecticut
      • Hartford, Connecticut, Estados Unidos, 06102
        • Hartford Healthcare
      • Norwich, Connecticut, Estados Unidos, 06360
        • Eastern Connecticut Hematology and Oncology
    • Florida
      • Altamonte Springs, Florida, Estados Unidos, 32701
        • Florida Cancer Specialists - North Division
      • Bonita Springs, Florida, Estados Unidos, 34135
        • Florida Cancer Specialists - South Division
      • Bradenton, Florida, Estados Unidos, 34205
        • Florida Cancer Specialists - South Division
      • Bradenton, Florida, Estados Unidos, 34211
        • Florida Cancer Specialists - South Division
      • Brandon, Florida, Estados Unidos, 33511
        • Florida Cancer Specialists - North Division
      • Cape Coral, Florida, Estados Unidos, 33909
        • Florida Cancer Specialists - South Division
      • Clearwater, Florida, Estados Unidos, 33761
        • Florida Cancer Specialists - North Division
      • Daytona Beach, Florida, Estados Unidos, 32117
        • Cancer Specialist - East
      • Fort Lauderdale, Florida, Estados Unidos, 33308
        • Holy Cross Hospital
      • Fort Myers, Florida, Estados Unidos, 33901
        • Florida Cancer Specialists South Division
      • Fort Myers, Florida, Estados Unidos, 33905
        • Florida Cancer Specialists - South
      • Fort Myers, Florida, Estados Unidos, 33908
        • Florida Cancer Specialists - South
      • Gainesville, Florida, Estados Unidos, 32605
        • Florida Cancer Specialists - North Division
      • Jacksonville, Florida, Estados Unidos, 32256
        • Cancer Specialists of North Florida
      • Lakeland, Florida, Estados Unidos, 33812
        • TOI Florida
      • Lakeland, Florida, Estados Unidos, 33812
        • The Oncology Institute of Hope and Innovation
      • Largo, Florida, Estados Unidos, 33770
        • Florida Cancer Specialists - North Division
      • Lecanto, Florida, Estados Unidos, 34461
        • Florida Cancer Specialists - North Division
      • Naples, Florida, Estados Unidos, 34102
        • Florida Cancer Specialists - South Division
      • Ocala, Florida, Estados Unidos, 34474
        • Ocala Oncology
      • Ocala, Florida, Estados Unidos, 34474
        • Florida Cancer Specialists - North Division
      • Orange City, Florida, Estados Unidos, 32763
        • Florida Cancer Specialists - North Division
      • Orlando, Florida, Estados Unidos, 32806
        • Florida Cancer Specialists - North Division
      • Port Charlotte, Florida, Estados Unidos, 33980
        • Florida Cancer Specialists - South Division
      • Sarasota, Florida, Estados Unidos, 34232
        • Florida Cancer Specialists - South Division
      • Sarasota, Florida, Estados Unidos, 34236
        • Florida Cancer Specialists - South Division
      • St. Petersburg, Florida, Estados Unidos, 33705
        • Florida Cancer Specialists and Research Institute - North Division
      • St. Petersburg, Florida, Estados Unidos, 33707
        • Florida Cancer Specialists - North
      • Stuart, Florida, Estados Unidos, 34994
        • Florida Cancer Specialist - East
      • Tampa, Florida, Estados Unidos, 33607
        • Florida Cancer Specialists - North Division
      • Tavares, Florida, Estados Unidos, 32778
        • Florida Cancer Specialists - North Division
      • Trinity, Florida, Estados Unidos, 34655
        • Florida Cancer Specialists - North Division
      • Venice, Florida, Estados Unidos, 34285
        • Florida Cancer Specialists - South Division
      • Venice, Florida, Estados Unidos, 34292
        • Florida Cancer Specialists - South Division
      • Vero Beach, Florida, Estados Unidos, 32960
        • Florida Cancer Specialists - East
      • Wellington, Florida, Estados Unidos, 33414
        • Florida Cancer Specialists - East
      • West Palm Beach, Florida, Estados Unidos, 33401
        • Florida Cancer Specialists - East
    • Georgia
      • Atlanta, Georgia, Estados Unidos, 30303
        • Morehouse School of Medicine
    • Hawaii
      • Honolulu, Hawaii, Estados Unidos, 96813
        • Hawaii Cancer Center
    • Illinois
      • Hinsdale, Illinois, Estados Unidos, 60521
        • Hope and Healing Cancer Services
      • Rolling Meadows, Illinois, Estados Unidos, 60008
        • Northwest Oncology and Hematology
    • Indiana
      • Carmel, Indiana, Estados Unidos, 46032
        • Urology of Indiana
      • Fort Wayne, Indiana, Estados Unidos, 46804
        • Fort Wayne Medical Oncology and Hematology
      • Goshen, Indiana, Estados Unidos, 46526
        • Goshen Health
    • Louisiana
      • Baton Rouge, Louisiana, Estados Unidos, 70817
        • Our Lady of the Lake
      • Hammond, Louisiana, Estados Unidos, 70403
        • Pontchartrain
    • Maryland
      • Bethesda, Maryland, Estados Unidos, 20817
        • American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)
      • Frederick, Maryland, Estados Unidos, 21702
        • Frederick Health
      • Rockville, Maryland, Estados Unidos, 20850
        • Maryland Oncology Hematology
    • Massachusetts
      • Boston, Massachusetts, Estados Unidos, 02215
        • Dana Farber Cancer Institute
      • Fairhaven, Massachusetts, Estados Unidos, 02719
        • Southcoast Centers for Cancer Care
    • Michigan
      • Detroit, Michigan, Estados Unidos, 48201
        • Barbara Ann Karmanos Cancer Institute
      • Lansing, Michigan, Estados Unidos, 48912
        • Sparrow Hospital
    • Minnesota
      • Minneapolis, Minnesota, Estados Unidos, 55404
        • Minnesota Oncology Hematology
    • Missouri
      • Bolivar, Missouri, Estados Unidos, 65613
        • Central Care Cancer Center
      • Osage Beach, Missouri, Estados Unidos, 65065
        • Lake Regional
      • Saint Joseph, Missouri, Estados Unidos, 64507
        • Mosaic Life Care
      • Springfield, Missouri, Estados Unidos, 65807
        • Oncology Hematology Associates
      • St Louis, Missouri, Estados Unidos, 63110
        • Washington University School of Medicine
    • Nebraska
      • Grand Island, Nebraska, Estados Unidos, 68803
        • Nebraska Cancer Specialists
      • Omaha, Nebraska, Estados Unidos, 68114
        • Nebraska Methodist Hospital
    • Nevada
      • Las Vegas, Nevada, Estados Unidos, 89169
        • Comprehensive Cancer Centers of Nevada
      • Las Vegas, Nevada, Estados Unidos, 89102
        • OptumCare Cancer Care-Parent
    • New Jersey
      • Belleville, New Jersey, Estados Unidos, 07109
        • New Jersey Cancer Care and Blood Disorders
      • Englewood, New Jersey, Estados Unidos, 07631
        • Englewood Hospital and Medical Center
      • Florham Park, New Jersey, Estados Unidos, 07932
        • Summit Medical Group - NJ
      • Morristown, New Jersey, Estados Unidos, 07960
        • Atlantic Health System - Morristown Medical Center
    • New York
      • Buffalo, New York, Estados Unidos, 14263
        • Roswell Park Comprehensive Cancer Center
      • East Syracuse, New York, Estados Unidos, 13057
        • HOACNY
      • Ithaca, New York, Estados Unidos, 14850
        • Cayuga Medical Center
      • New York, New York, Estados Unidos, 10065
        • David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center
    • North Carolina
      • Goldsboro, North Carolina, Estados Unidos, 27534
        • Southeastern Medical Oncology
    • North Dakota
      • Fargo, North Dakota, Estados Unidos, 58102
        • Sanford Health-Fargo
    • Ohio
      • Canton, Ohio, Estados Unidos, 44708
        • Aultman Medical Group
      • Cincinnati, Ohio, Estados Unidos, 45220
        • TriHealth
      • Cincinnati, Ohio, Estados Unidos, 45627
        • University of Cincinnati (UC) - Cancer Institute
      • Cleveland, Ohio, Estados Unidos, 44195
        • Cleveland Clinic
      • Toledo, Ohio, Estados Unidos, 43623
        • The Toledo Clinic
    • Oklahoma
      • Oklahoma City, Oklahoma, Estados Unidos, 73104
        • Oklahoma State University (OSU) - Stephenson Cancer Center
      • Tulsa, Oklahoma, Estados Unidos, 74146
        • Oklahoma Cancer Specialist
    • Pennsylvania
      • Gettysburg, Pennsylvania, Estados Unidos, 17325
        • Gettysburg-PCSRI
      • Langhorne, Pennsylvania, Estados Unidos, 19047
        • Alliance Cancer Specialists
      • Philadelphia, Pennsylvania, Estados Unidos, 19107
        • Thomas Jefferson University
      • York, Pennsylvania, Estados Unidos, 17403
        • Cancer Care Associates of York - Parent
    • South Carolina
      • Greenville, South Carolina, Estados Unidos, 29605
        • Prisma Health Cancer Institute
    • South Dakota
      • Sioux Falls, South Dakota, Estados Unidos, 57105
        • Avera Cancer Institute
      • Sioux Falls, South Dakota, Estados Unidos, 57117
        • Sanford Health
    • Tennessee
      • Dickson, Tennessee, Estados Unidos, 37055
        • Sarah Cannon and HCA Research Institute
      • Franklin, Tennessee, Estados Unidos, 37067
        • Sarah Cannon and HCA Research Institute
      • Gallatin, Tennessee, Estados Unidos, 37066
        • Sarah Cannon and HCA Research Institute
      • Germantown, Tennessee, Estados Unidos, 38138
        • West Cancer Center
      • Henderson, Tennessee, Estados Unidos, 37075
        • Sarah Cannon and HCA Research Institute
      • Hermitage, Tennessee, Estados Unidos, 37076
        • Sarah Cannon and HCA Research Institute
      • Lebanon, Tennessee, Estados Unidos, 37090
        • Sarah Cannon and HCA Research Institute
      • Memphis, Tennessee, Estados Unidos, 38120
        • Baptist Cancer Center
      • Murfreesboro, Tennessee, Estados Unidos, 37129
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Estados Unidos, 37203
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Estados Unidos, 37205
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Estados Unidos, 37207
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Estados Unidos, 37211
        • Sarah Cannon and HCA Research Institute
      • Shelbyville, Tennessee, Estados Unidos, 37160
        • Sarah Cannon and HCA Research Institute
      • Smyrna, Tennessee, Estados Unidos, 37167
        • Sarah Cannon and HCA Research Institute
    • Texas
      • Dallas, Texas, Estados Unidos, 75230
        • Mary Crowley Cancer Research
      • Dallas, Texas, Estados Unidos, 75246
        • Texas Oncology - DFW
      • El Paso, Texas, Estados Unidos, 79915
        • Texas Oncology
      • Houston, Texas, Estados Unidos, 77030
        • MD Anderson Cancer Center
      • Houston, Texas, Estados Unidos, 77030
        • Oncology Consultants
      • Irving, Texas, Estados Unidos, 75063
        • Arizona Oncology Associates
      • Kingwood, Texas, Estados Unidos, 77339
        • Lumi Research
      • McAllen, Texas, Estados Unidos, 78503
        • Texas Oncology Central-South
      • San Antonio, Texas, Estados Unidos, 78229
        • South Texas Accelerated Research Therapeutics (START)
      • Tyler, Texas, Estados Unidos, 75702
        • Texas Oncology
    • Utah
      • Ogden, Utah, Estados Unidos, 84405
        • Community Cancer Trials of Utah
      • Salt Lake City, Utah, Estados Unidos, 84106
        • Utah Cancer Specialists
    • Virginia
      • Fairfax, Virginia, Estados Unidos, 22031
        • Virginia Cancer Specialists
      • Fredericksburg, Virginia, Estados Unidos, 22408
        • Hematology Oncology Associates of Fredericksburg
      • Richmond, Virginia, Estados Unidos, 23235
        • Virginia Urology
    • Washington
      • Seattle, Washington, Estados Unidos, 98109
        • University of Washington Cancer Consortium
      • Spokane, Washington, Estados Unidos, 99216
        • Cancer Care Northwest
      • Spokane, Washington, Estados Unidos, 99202
        • Spokane Urology
      • Tacoma, Washington, Estados Unidos, 98405
        • Northwest Medical Specialties
    • Wisconsin
      • Appleton, Wisconsin, Estados Unidos, 54911
        • ThedaCare
      • La Crosse, Wisconsin, Estados Unidos, 54601
        • Gunderson Health System
      • Madison, Wisconsin, Estados Unidos, 53705
        • University of Wisconsin - Carbone Cancer Center
  • Puerto Rico
    • Puerto Rico
      • San Juan, Puerto Rico, Puerto Rico, 00935
        • Pan Oncology Trials, LLC

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

12 años y mayores (Niño, Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

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, β-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.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación de un solo grupo
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Arm A: Pathogenic inactivating TSC1 alterations

Patients with pathogenic inactivating TSC1 alterations.
Droga: 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

Otros nombres:
  • ABI-009
Experimental: Arm B: Pathogenic inactivating TSC2 alterations

Patients with pathogenic inactivating TSC2 alterations.
Droga: 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

Otros nombres:
  • ABI-009

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Overall Response Rate (ORR)
Periodo de tiempo: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression , up to 30 months.

Based on CT or MRI scans from the time of study treatment initiation until disease progression, overall response rate (ORR) is defined as the proportion of patients with best overall response (BOR) of confirmed partial response (PR) or complete response (CR) in accordance with Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 as determined by independent Radiographic Review (IRR).

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression , up to 30 months.

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Duration of Response (DOR)
Periodo de tiempo: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.

DOR is defined as the number of months from the start of CR or PR (whichever response is recorded first) and subsequently confirmed to the first date of documented progressive disease (PD) or death using CT or MRI scans in accordance with RECIST v1.1.

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.

PD is defined as ≥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.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.
Disease Control Rate
Periodo de tiempo: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression, up to 30 months.

Disease control rate (DCR) is defined as BOR of confirmed CR or PR (either of any duration) or SD ≥12 weeks following study treatment initiation.

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression, up to 30 months.
Time to Response
Periodo de tiempo: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.

Time to response (TTR) is defined as the time from first dose of study drug to initial measurement of CR or PR, where CR or PR is subsequently confirmed.

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.
Progression-free Survival
Periodo de tiempo: From first dose of study treatment to the date of disease progression or death

Number of months from study treatment initiation to the date of disease progression (PD) or death due to any cause PD is defined as ≥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.
From first dose of study treatment to the date of disease progression or death
Overall Survival
Periodo de tiempo: From first dose of study treatment to the date of death

Number of months from study treatment initiation to the date of death due to any cause
From first dose of study treatment to the date of death
Patient-Reported Outcome
Periodo de tiempo: D1 of every cycle up to Cycle 24

Changes from baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QOQ-C30) v3.0 scores EORTC QOQ-C30 includes functional scales (physical, role, cognitive, emotional, social), global health status, symptom scales (fatigue, pain, nausea/vomiting), and other (dyspnoea, appetite loss, insomnia, constipation/diarrhea, financial difficulties). Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); two used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score = better quality of life.
D1 of every cycle up to Cycle 24
Incidence and Severity of Treatment-emergent and Treatment-related Adverse Events (AEs)
Periodo de tiempo: Monitored continuously from signing of Informed Consent until last dose of IP (estimated up to 30 months) plus safety follow-up of 28 days after the last dose.

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
Monitored continuously from signing of Informed Consent until last dose of IP (estimated up to 30 months) plus safety follow-up of 28 days after the last dose.

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Aadi Bioscience, Inc.

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

10 de marzo de 2022

Finalización primaria (Actual)

23 de diciembre de 2024

Finalización del estudio (Actual)

23 de diciembre de 2024

Fechas de registro del estudio

Enviado por primera vez

22 de octubre de 2021

Primero enviado que cumplió con los criterios de control de calidad

22 de octubre de 2021

Publicado por primera vez (Actual)

2 de noviembre de 2021

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

28 de octubre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

26 de octubre de 2026

Última verificación

1 de septiembre de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • TSC-007

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

NO

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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