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Phase-2-Korbstudie mit Nab-Sirolimus bei Patienten mit bösartigen soliden Tumoren mit pathogenen Veränderungen in den TSC1- oder TSC2-Genen (PRECISION 1)

26. Oktober 2026 aktualisiert von: Aadi Bioscience, Inc.

Eine multizentrische Open-Label-Korbstudie der Phase 2 mit Nab-Sirolimus für erwachsene und jugendliche Patienten mit bösartigen soliden Tumoren, die pathogene inaktivierende Veränderungen in TSC1- oder TSC2-Genen beherbergen

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

Studienübersicht

Status

Abgeschlossen

Bedingungen

Intervention / Behandlung

Detaillierte Beschreibung

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.

Studientyp

Interventionell

Einschreibung (Tatsächlich)

128

Phase

  • Phase 2

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienorte

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

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

12 Jahre und älter (Kind, Erwachsene, Älterer Erwachsener)

Akzeptiert gesunde Freiwillige

Nein

Studienberechtigte Geschlechter

Alle

Beschreibung

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.

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Hauptzweck: Behandlung
  • Zuteilung: Nicht randomisiert
  • Interventionsmodell: Einzelgruppenzuweisung
  • Maskierung: Keine (Offenes Etikett)

Waffen und Interventionen

Teilnehmergruppe / Arm
Intervention / Behandlung
Experimental: Arm A: Pathogenic inactivating TSC1 alterations

Patients with pathogenic inactivating TSC1 alterations.
Arzneimittel: 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

Andere Namen:
  • ABI-009
Experimental: Arm B: Pathogenic inactivating TSC2 alterations

Patients with pathogenic inactivating TSC2 alterations.
Arzneimittel: 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

Andere Namen:
  • ABI-009

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Overall Response Rate (ORR)
Zeitfenster: 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.

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Duration of Response (DOR)
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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.

Mitarbeiter und Ermittler

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Sponsor

Aadi Bioscience, Inc.

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Tatsächlich)

10. März 2022

Primärer Abschluss (Tatsächlich)

23. Dezember 2024

Studienabschluss (Tatsächlich)

23. Dezember 2024

Studienanmeldedaten

Zuerst eingereicht

22. Oktober 2021

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

22. Oktober 2021

Zuerst gepostet (Tatsächlich)

2. November 2021

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

28. Oktober 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

26. Oktober 2026

Zuletzt verifiziert

1. September 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Schlüsselwörter

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

  • TSC-007

Plan für individuelle Teilnehmerdaten (IPD)

Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?

NEIN

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Ja

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

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