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Fase 2-kurvforsøk med Nab-sirolimus hos pasienter med ondartede solide svulster med patogene endringer i TSC1- eller TSC2-gener (PRESISJON 1)

26. oktober 2026 oppdatert av: Aadi Bioscience, Inc.

En fase 2 multisenter åpen kurvforsøk med Nab-sirolimus for voksne og ungdommer med ondartede solide svulster som har patogene inaktiverende endringer i TSC1- eller TSC2-gener

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

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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

Intervensjonell

Registrering (Faktiske)

128

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

12 år og eldre (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

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.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Arm A: Pathogenic inactivating TSC1 alterations

Patients with pathogenic inactivating TSC1 alterations.
Legemiddel: 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

Andre navn:
  • ABI-009
Eksperimentell: Arm B: Pathogenic inactivating TSC2 alterations

Patients with pathogenic inactivating TSC2 alterations.
Legemiddel: 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

Andre navn:
  • ABI-009

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Overall Response Rate (ORR)
Tidsramme: 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.

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Duration of Response (DOR)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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.

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Aadi Bioscience, Inc.

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

10. mars 2022

Primær fullføring (Faktiske)

23. desember 2024

Studiet fullført (Faktiske)

23. desember 2024

Datoer for studieregistrering

Først innsendt

22. oktober 2021

Først innsendt som oppfylte QC-kriteriene

22. oktober 2021

Først lagt ut (Faktiske)

2. november 2021

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

28. oktober 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

26. oktober 2026

Sist bekreftet

1. september 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • TSC-007

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Abonnere