Clinical trial Recruiting at 19 sites Phase1; Phase2 Interventional Ages 12 Months to 30

Tegavivint treatment for recurrent or refractory solid tumors in children

Official title: A Phase 1/2 Study of Tegavivint (NSC#826393) in Children, Adolescents, and Young Adults With Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors

Run by Children's Oncology Group · NCT04851119 · Data updated 2026-11-12

This study tests if a new intravenous medication called tegavivint can help children with tough-to-treat solid tumors, like Ewing sarcoma and desmoid tumors, by finding the right dose and seeing how well it works.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Ages12 Months to 30 Years
SexAll
Enrollment147 (estimated)
SponsorChildren's Oncology Group
Research network
Drugs / interventionsdenosumab, chemotherapy, radiation, methotrexate
Locations21 sites (Birmingham, Alabama and 20 other locations)
StatusRecruiting
StartedNovember 8, 2021
Main results expectedJune 30, 2028
Study endsJune 30, 2028
Registry record updatedAugust 25, 2026
Trial IDNCT04851119 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Ideal candidates are pediatric patients aged 12 months to 21 years with recurrent or refractory solid tumors.

Probably not a fit

Patients with tumors that are not recurrent or refractory, or those outside the specified age range, may not benefit from this study.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the safety and effectiveness of tegavivint, an intravenous medication, in treating pediatric patients with recurrent or refractory solid tumors, including various types of cancers such as Ewing sarcoma and desmoid tumors. The study aims to determine the maximum tolerated dose and assess the drug's antitumor activity and side effects. It includes a dose escalation phase to find the optimal dosage and a phase to evaluate the drug's effectiveness against specific tumor types. Additionally, the trial will explore the relationship between tumor characteristics and treatment response.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for children with difficult-to-treat solid tumors.

How similar studies have gone: Other studies have shown promise with similar approaches targeting the Wnt/beta-catenin pathway, suggesting potential for success in this trial.

Who can join: the full rules

Show full inclusion / exclusion criteria
Inclusion Criteria:

* PART A: Patients must be \>= 12 months and =\< 21 years of age at the time of study enrollment
* PART B: Patients must be \>= 12 months and =\< 30 years of age at the time of study enrollment
* Patients with recurrent or refractory solid tumors including non-Hodgkin lymphoma and desmoid tumors are eligible. Patients must have had histologic verification of malignancy at original diagnosis or relapse
* PART A: Patients with relapsed or refractory solid tumors, including patients with non-Hodgkin lymphoma and desmoid tumors
* PART B: Patients with recurrent or refractory Ewing sarcoma, desmoid tumors, osteosarcoma, liver tumors (HCC and hepatoblastoma), Wilms tumor, and tumors with Wnt pathway aberrations. For the Wnt pathway aberrations cohort we will include the most common CTNNB1 mutations (S37F, S45F, T41A, S45P, S33C, S37C, D32Y, S33F, T41I, G34R, G34V, D32N, S33P, G34E, D32G) as well as any loss of function mutations in the APC, Axin2FBXW7, TCF7L2, and RNF43 genes or any gain-of-function mutations in the GSK3B, LRP6, and LGR5 genes. For patients without prior sequencing, immunohistochemistry (IHC), is required. IHC showing strong nuclear beta-catenin staining will be accepted for the following tumor types: colorectal carcinoma, melanoma, endometrial cancer, ovarian cancer, neuroblastoma, non-Hodgkin lymphoma, pancreatic ductal adenocarcinoma, and solid pseudopapillary tumor of the pancreas
* PART A: Patients must have either measurable or evaluable disease. For desmoid tumors, the patient must have disease that the investigator deems unresectable or sufficiently morbid or potentially life-threatening that there is favorable risk/benefit to the patient to participate in the trial
* PART B: Patients must have measurable disease. For desmoid tumors, the patient must have measurable disease that the investigator deems unresectable or sufficiently morbid or potentially life-threatening that there is favorable risk/benefit to the patient to participate in the trial
* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age
* Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately.

  * Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive

    * Solid tumor patients: \>= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea)
    * Non-Hodgkin lymphoma patients

      * A waiting period prior to enrollment is not required for patients receiving standard maintenance chemotherapy (i.e., corticosteroid, vincristine, thioguanine \[6MP\], and/or methotrexate)
      * \>= 14 days must have elapsed after the completion of other cytotoxic therapy, with the exception of hydroxyurea, for patients not receiving standard maintenance therapy
      * NOTE: Cytoreduction with hydroxyurea must be discontinued \>= 24 hours prior to the start of protocol therapy
  * Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or absolute neutrophil counts \[ANC\]): \>= 7 days after the last dose of agent
  * Antibodies: \>= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\< 1
  * Corticosteroids: If used to modify immune adverse events related to prior therapy, \>= 14 days must have elapsed since last dose of corticosteroid
  * Hematopoietic growth factors: \>= 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or 7 days for short acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
  * Interleukins, interferons and cytokines (other than hematopoietic growth factors): \>= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
  * Stem cell Infusions (with or without total-body irradiation \[TBI\]):

    * Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: \>= 84 days after infusion and no evidence of graft versus host disease (GVHD)
    * Autologous stem cell infusion including boost infusion: \>= 42 days.
  * Cellular therapy: \>= 42 days after the completion of any type of cellular therapy (e.g., modified T cells, natural killer \[NK\] cells, dendritic cells, etc.).
  * External beam radiation therapy (XRT)/external beam irradiation including protons: \>= 14 days after local XRT; \>= 150 days after TBI, craniospinal XRT or if radiation to \>= 50% of the pelvis; \>= 42 days if other substantial bone marrow (BM) radiation
  * Radiopharmaceutical therapy (e.g., radiolabeled antibody, iobenguane I-131 \[131I MIBG\]): \>= 42 days after systemically administered radiopharmaceutical therapy
  * Patients must not have received prior exposure to tegavivint
* PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Peripheral absolute neutrophil count (ANC) \>= 1000/uL (within 7 days prior to enrollment)
* PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Platelet count \>= 100,000/uL (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) (within 7 days prior to enrollment)
* PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Hemoglobin \>= 8.0 g/dL at baseline (may receive red blood cell \[RBC\] transfusions) (within 7 days prior to enrollment)
* Patients with known bone marrow metastatic disease will be eligible for study provided they meet blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity. At least 5 of every cohort of 6 patients must be evaluable for hematologic toxicity for the dose-escalation part of the study. If dose-limiting hematologic toxicity is observed, all subsequent patients enrolled on Part A must be evaluable for hematologic toxicity
* Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a creatinine based on age/gender as follows (within 7 days prior to enrollment):

  * Age; maximum serum creatinine
  * Age 1 to \< 2 years; 0.6 mg/dL (male); 0.6 mg/dL (female)
  * Age 2 to \< 6 years; 0.8 mg/dL (male); 0.8 mg/dL (female)
  * Age 6 to \< 10 years; 1 mg/dL (male); 1 mg/dL (female)
  * Age 10 to \< 13 years; 1.2 mg/dL (male); 1.2 mg/dL (female)
  * Age 13 to \< 16 years; 1.5 mg/dL (male); 1.4 mg/dL (female)
  * Age \>= 16 years; 1.7 mg/dL (male); 1.4 mg/dL (female)
* PATIENTS WITH SOLID TUMORS: Bilirubin (sum of conjugated + unconjugated or total) =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment)
* PATIENTS WITH SOLID TUMORS: Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L (within 7 days prior to enrollment)
* PATIENTS WITH SOLID TUMORS: Albumin \>= 2 g/dL (within 7 days prior to enrollment)

Exclusion Criteria:

* Pregnant or breast-feeding women will not be entered on this study because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control, including a medically accepted barrier or contraceptive method (e.g., male or female condom) for the duration of the study. Abstinence is an acceptable method of birth control
* Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, \>= 14 days must have elapsed since last dose of corticosteroid
* Patients who are currently receiving another investigational drug are not eligible
* Patients who are currently receiving other anti-cancer agents are not eligible
* Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
* Patients who are currently receiving drugs that are strong inducers or inhibitors of CYP3A4 are not eligible. Strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to the 1st dose of tegavivint to the end of the study
* Patients who have received bisphosphonates within 4 weeks prior to study enrollment will be excluded
* Patients who have received denosumab within 180 days prior to study enrollment will be excluded
* Patients with primary brain tumors are ineligible
* Patients with known central nervous system (CNS) metastasis, except for craniopharyngeal tumors, will be excluded
* Patients with a known metabolic bone disease (ex: hyperparathyroidism, Paget's disease, osteomalacia) are not eligible
* Patients with a disorder associated with abnormal bone metabolism will be excluded
* Patients with grade \>= 2 hypocalcemia that is not corrected with oral calcium supplementation will be excluded
* Patients with vitamin D \< 20 ng/mL will require supplementation, or will otherwise be excluded. Patients must agree to take vitamin D +/- calcium supplements (if necessary) according to institutional or published guidelines. Additional calcium supplementation is not required if adequate dietary intake can be ascertained
* Patients with pre-existing grade 3 osteoporosis are excluded
* Patients who have an uncontrolled infection are not eligible
* Patients who have received a prior solid organ transplantation are not eligible
* Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible

Where it's running

  • Children's Hospital of Alabama
    Birmingham, Alabama, United States
    Recruiting
  • Children's Hospital Los Angeles
    Los Angeles, California, United States
    Recruiting
  • Children's Hospital of Orange County
    Orange, California, United States
    Recruiting
  • UCSF Medical Center-Mission Bay
    San Francisco, California, United States
    Recruiting
  • Children's Hospital Colorado
    Aurora, Colorado, United States
    Recruiting
  • Children's National Medical Center
    Washington D.C., District of Columbia, United States
    Recruiting
Show 15 more locations
  • Children's Healthcare of Atlanta - Arthur M Blank Hospital
    Atlanta, Georgia, United States
    Recruiting
  • Lurie Children's Hospital-Chicago
    Chicago, Illinois, United States
    Recruiting
  • Riley Hospital for Children
    Indianapolis, Indiana, United States
    Recruiting
  • Dana-Farber Cancer Institute
    Boston, Massachusetts, United States
    Recruiting
  • C S Mott Children's Hospital
    Ann Arbor, Michigan, United States
    Recruiting
  • University of Minnesota/Masonic Cancer Center
    Minneapolis, Minnesota, United States
    Recruiting
  • Washington University School of Medicine
    St Louis, Missouri, United States
    Recruiting
  • NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
    New York, New York, United States
    Recruiting
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio, United States
    Recruiting
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania, United States
    Recruiting
  • Children's Hospital of Pittsburgh of UPMC
    Pittsburgh, Pennsylvania, United States
    Recruiting
  • Saint Jude Children's Research Hospital
    Memphis, Tennessee, United States
    Recruiting
  • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
    Houston, Texas, United States
    Recruiting
  • Lucile Packard Children's Hospital Stanford University
    Palo Alto, California, United States
    Active not recruiting
  • Seattle Children's Hospital
    Seattle, Washington, United States
    Suspended

Study contacts

  • Principal investigator: Sarah B Whittle — Pediatric Early Phase Clinical Trial Network

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.