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Essai de panier de phase 2 sur le nab-sirolimus chez des patients atteints de tumeurs solides malignes présentant des altérations pathogènes des gènes TSC1 ou TSC2 (PRÉCISION 1)

26 octobre 2026 mis à jour par: Aadi Bioscience, Inc.

Un essai multicentrique ouvert de phase 2 sur panier de nab-sirolimus pour les patients adultes et adolescents atteints de tumeurs solides malignes hébergeant des altérations inactivatrices pathogènes des gènes TSC1 ou TSC2

A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes

Aperçu de l'étude

Statut

Complété

Les conditions

Intervention / Traitement

Description détaillée

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.

Type d'étude

Interventionnel

Inscription (Réel)

128

Phase

  • Phase 2

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

12 ans et plus (Enfant, Adulte, Adulte plus âgé)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

Inclusion Criteria:

  1. Patients must have a malignant solid tumor with a pathogenic inactivating TSC1 or TSC2 alteration. Genetic alterations should be identified using NGS in tumor tissue or liquid biopsy).

    • Patients will be enrolled after the central evaluation of NGS report confirms eligibility.

  2. Patients must have solid tumors that are metastatic or locally advanced where surgical resection is not an option or likely to result in severe morbidity.

  3. Patients must have received all standard therapies appropriate for their tumor type and stage of disease or, in the opinion of the Investigator, the patient would be unlikely to tolerate or derive clinically meaningful benefit from appropriate standard of care therapy, or the patient has no satisfactory alternative treatments.

  4. Patients must have 1 or more measurable target lesions by computed tomography (CT) scan or magnetic resonance imaging (MRI) (RECIST v1.1).

  5. Age: 12 years or older.

  6. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 or Karnofsky Performance Status (KPS) ≥80 or Lansky play-performance scale for pediatric patients ≥80.

  7. Adequate liver function:

    1. Total bilirubin ≤1.5 × upper limit of normal (ULN) (unless due to Gilbert's syndrome, then ≤3 × ULN)
    2. Aspartate aminotransferase (AST) ≤2.5 × ULN (≤5 × ULN if attributable to liver metastases)

  8. Adequate renal function: creatinine clearance ≥30 mL/min, Cockcroft-Gault CCr = ((140-age) × weight[kg]) / (72 × SCr[mL/min]) × 0.85, if female

  9. Adequate hematologic parameters:

    1. Absolute neutrophil count (ANC) ≥1.0 × 109/L (growth factor support allowed)
    2. Platelet count ≥100,000/mm3 (100 × 109/L) (transfusion and/or growth factor support allowed)
    3. Hemoglobin ≥8.0 g/dL (transfusion and/or growth factor support allowed)

  10. Fasting serum triglyceride must be ≤300 mg/dL; fasting serum cholesterol must be ≤350 mg/dL.

  11. Minimum of 4 weeks since any major surgery, completion of radiation, or completion of prior systemic anticancer therapy, or at least 5 half-lives if the prior therapy is a single agent small-molecule therapeutic, and adequately recovered from the acute toxicities of any prior therapy, including neuropathy, to Grade ≤1.

  12. Male or non-pregnant and non-breastfeeding female:

    1. Females of childbearing potential must agree to use effective contraception or abstinence without interruption from 28 days prior to starting investigational product (IP) throughout 3 months after last dose of IP and have a negative serum pregnancy test (beta human chorionic gonadotropin, β-hCG) result at screening and agree to ongoing pregnancy testing during the course of the study, and after the end of study treatment. A second form of birth control is required even if she has had a tubal ligation.
    2. Male patients must agree not to donate sperm and must practice abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study and throughout 3 months after last dose of IP. A second form of birth control is required even if he has undergone a successful vasectomy.

  13. The patient or the patient's parent(s) or legal guardian(s) understand(s) and sign(s) the informed consent.

  14. Willingness and ability to comply with scheduled visits, laboratory tests, and other study procedures.

Exclusion Criteria:

  1. Prior treatment with an mTOR inhibitor, including nab-sirolimus.

  2. Severe (Grade ≥3) ongoing infection requiring parenteral or oral anti-infective treatment, either ongoing or completed ≤7 days prior to enrollment.

  3. Patients with primary brain tumors or PEComa.

  4. Patients who have any severe and/or uncontrolled medical or psychiatric conditions or other conditions that could affect their participation including:

    1. Patients with meningeal carcinomatosis, leptomeningeal carcinomatosis, spinal cord compression, untreated brain metastases or symptomatic or unstable brain metastases. Note: Patients with stable brain metastases (defined as asymptomatic or no requirement for high-dose [defined as dexamethasone 10 mg daily or higher] or increasing dose of systemic corticosteroids) and without imminent need of radiation therapy are eligible. If applicable, patients must have completed brain radiation therapy and recovered adequately from any associated toxicity and/or complications prior to eligibility assessment. For patients who have received prior radiation therapy, post-treatment MRI scan should show no increase in brain lesion size/volume.
    2. Unstable angina pectoris, symptomatic congestive heart failure (New York Heart Association, NYHA class III or IV), myocardial infarction ≤6 months prior to first study treatment, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease.
    3. Pre-existing severely impaired lung function. If a patient has a pre-existing pulmonary condition, eligible patients should have a spirometry and diffusing capacity for carbon monoxide (DLCO) that is >50% of the normal predicted value and/or O2 saturation that is >88% at rest on room air (Note: spirometry and pulmonary function tests [PFTs] not required to be performed unless clinically indicated).
    4. Nonmalignant medical illnesses that are uncontrolled or whose control may be jeopardized by the treatment with the study therapy.
    5. A history of malignancies other than the one under treatment unless the patient is disease-free for more than 5 years from diagnosis. Note, controlled non-melanoma skin cancers, carcinoma in situ of the cervix, resected incidental prostate cancer, certain low grade hematologic malignancies (eg CLL, follicular lymphoma, etc), or other adequately treated carcinoma-in-situ may be eligible, after discussion with the medical monitor.
    6. Uncontrolled hypertension (systolic blood pressure ≥160 mm-Hg and/or diastolic blood pressure ≥100 mm Hg).
    7. Patients with history of interstitial lung disease and/or pneumonitis, or pulmonary hypertension.
    8. Individuals with known human immunodeficiency virus (HIV) infection are excluded from this study as combination antiretroviral therapy could potentially result in significant pharmacokinetic interactions. In addition, these individuals are at increased risk of serious infections due to the immunosuppressive effects of mTOR inhibition.
    9. Active Hepatitis B or Hepatitis C, with detectable viral load.

  5. Regarding concomitant medications with significant CYP3A4 and P-gp interactions, discontinuation of strong inhibitors (eg, ketoconazole, itraconazole, voriconazole, erythromycin, clarithromycin, telithromycin, and others), strong inducers (eg, rifampin, rifabutin), and known CYP3A4 substrates with a narrow therapeutic window (eg, fentanyl, alfentanil, astemizole, cisapride, dihydroergotamine, pimozide, quinidine, or terfenadine) is required at least 5 half lives prior to receiving the first dose of nab-sirolimus, whichever is longer.

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Non randomisé
  • Modèle interventionnel: Affectation à un seul groupe
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: Arm A: Pathogenic inactivating TSC1 alterations

Patients with pathogenic inactivating TSC1 alterations.
Médicament: 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

Autres noms:
  • ABI-009
Expérimental: Arm B: Pathogenic inactivating TSC2 alterations

Patients with pathogenic inactivating TSC2 alterations.
Médicament: 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

Autres noms:
  • ABI-009

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Overall Response Rate (ORR)
Délai: 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.

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Duration of Response (DOR)
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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)
Délai: 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.

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Aadi Bioscience, Inc.

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

10 mars 2022

Achèvement primaire (Réel)

23 décembre 2024

Achèvement de l'étude (Réel)

23 décembre 2024

Dates d'inscription aux études

Première soumission

22 octobre 2021

Première soumission répondant aux critères de contrôle qualité

22 octobre 2021

Première publication (Réel)

2 novembre 2021

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

28 octobre 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

26 octobre 2026

Dernière vérification

1 septembre 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • TSC-007

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

NON

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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