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Fase 2 Basket Trial van Nab-sirolimus bij patiënten met kwaadaardige solide tumoren met pathogene veranderingen in TSC1- of TSC2-genen (PRECISIE 1)

26 oktober 2026 bijgewerkt door: Aadi Bioscience, Inc.

Een fase 2 multicenter open-label basket-onderzoek met Nab-sirolimus voor volwassen en adolescente patiënten met kwaadaardige solide tumoren met pathogene inactiverende veranderingen in TSC1- of TSC2-genen

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

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

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.

Studietype

Ingrijpend

Inschrijving (Werkelijk)

128

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

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

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

12 jaar en ouder (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

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.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Opdracht voor een enkele groep
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Arm A: Pathogenic inactivating TSC1 alterations

Patients with pathogenic inactivating TSC1 alterations.
Geneesmiddel: 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
Experimenteel: Arm B: Pathogenic inactivating TSC2 alterations

Patients with pathogenic inactivating TSC2 alterations.
Geneesmiddel: 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

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Overall Response Rate (ORR)
Tijdsspanne: 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.

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Duration of Response (DOR)
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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.

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Sponsor

Aadi Bioscience, Inc.

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

10 maart 2022

Primaire voltooiing (Werkelijk)

23 december 2024

Studie voltooiing (Werkelijk)

23 december 2024

Studieregistratiedata

Eerst ingediend

22 oktober 2021

Eerst ingediend dat voldeed aan de QC-criteria

22 oktober 2021

Eerst geplaatst (Werkelijk)

2 november 2021

Updates van studierecords

Laatste update geplaatst (Werkelijk)

28 oktober 2026

Laatste update ingediend die voldeed aan QC-criteria

26 oktober 2026

Laatst geverifieerd

1 september 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • TSC-007

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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