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Wilms TumorPhase 2NCT05985161

A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors

A Phase 2 study testing Selinexor in people with Wilms Tumor and Rhabdoid Tumor, run by Memorial Sloan Kettering Cancer Center at 16 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

The purpose of this study is to find out whether selinexor is an effective treatment for people who have a relapsed/refractory Wilms tumor, rhabdoid tumor, MPNST, BCOR-driven sarcoma, or another solid tumor that makes a higher than normal amount of XPO1 or has genetic changes that increase the activity of XP01. Read more about this study

Who is it for?
Ages 12 months and older
What is being tested?
Selinexor
Could I receive a placebo?
No, everyone receives an active treatment
How many people
45 participants
Where
16 sitesUnited States
Study dates
1 Aug 2023to 1 Aug 2029
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 months and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Age:
  • Age ≥ 6 at the time of informed consent
  • Age ≥ 2 years to < 6 years at time of informed consent (Refer to Section 4.3): If PK cohort 1 is open, patients in this age range may enroll onto this cohort. If PK cohort 1 has been completed and deemed sufficient to proceed, then such patients may enroll onto the phase 2.
  • Age ≥ 12 months to < 2 years at time of informed consent (Refer to Section 4.3):
  • If PK cohort 2 is open, patients in this age range may enroll onto this cohort. If PK cohort 2 has been completed and deemed sufficient to proceed, then such patients may enroll onto the phase 2.
  • Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
Show all 42Show fewer
  • Performance: Karnofsky ≥ 60% for patients > 16 years of age and Lansky ≥ 60 for patients ≤ 16 years of age.
  • Diagnosis: Patients must enroll into one of the following cohorts:
  • Cohort A: Any type of Wilms tumor or nephroblastoma is eligible for this study provided they meet at least one of these criteria: (1) in their second or greater relapse, (2) refractory or in their first relapse with high risk histology (i.e., any anaplastic or blastemal-type after neoadjuvant chemotherapy), or (3) refractory or in first relapse without high risk histology but after having received chemotherapies other than the initial 4 agents used as current standard of care in the up-front setting for non-high risk cases - specifically vincristine, dactinomycin, doxorubicin, and irinotecan (i.e., any patient who relapses following an initial regimen more intense than EE4A, DD4A, VAD, AVD, or VIVA; for example, those including cyclophosphamide/etoposide - such as Regimen I, M, or MVI - or those additionally including carboplatin - such as Regimens UH-1, UH-2, or UH-3).
  • Cohort B: Any Rhabdoid tumor is eligible for this cohort. This includes, but is not limited to, related subtypes of rhabdoid tumors such as atypical teratoid rhabdoid tumors (ATRT), malignant rhabdoid tumors of the kidney (MRTK), malignant rhabdoid tumors of the soft tissue and liver, small cell undifferentiated hepatoblastomas (SCUH), and small-cell carcinoma of the ovary of hypercalcemic type (SCCOHT). Patients must have failed to respond to at least 1 line of systemic therapy prior to enrollment.
  • Cohort C: Patients with progressive, relapsed, unresectable or metastatic MPNST, are eligible for this cohort. Patients must have failed to respond to at least 1 line of systemic therapy prior to enrollment.
  • Cohort D: Patients must not qualify for Cohorts A, B, or C but have a solid tumor (no hematologic malignancies including lymphoma) for which there is specific evidence that this particular patient's tumor may benefit from selinexor.
  • Patients must have failed to respond to at least 1 line of systemic therapy prior to enrollment. Examples of evidence are listed below. All patients in this cohort require approval of study principal investigator and must provide documentation of specific supporting evidence. i. Tumor XPO1 Dependency: Defined as either Darwin OncoTarget demonstrating XPO1 as aberrantly activated or Darwin OncoTreat demonstrating context-specific tumor checkpoint inversion with Selinexor, both of which must be significant at a -log10 (Bonferroni corrected p-value) of 5 or greater. ii. Tumor XPO1 Activation: Defined as the detection of a gain of function mutation in XPO1, specifically E571K. Additionally, detection of elevated transcriptomic or proteomic expression of XPO1 in the tumor via RNAseq or IHC, respectively, would be considered sufficient for treatment.
  • Preclinical Tumor Testing: Defined as testing of Selinexor on patient derived cell line, organoid, or xenograft models of the patient's tumor (or other related tumors) performed in a laboratory context and for which, in the investigator's opinion, demonstrates promising activity. Testing may include commercial testing as well as academic laboratory testing.
  • Cohort E: Patients must have a solid tumor with an activating genomic alteration (e.g. fusion or internal tandem duplication) involving BCOR. Specific examples of qualifying alterations including BCOR-ITD, BCOR-CCNB3, BCOR-MAML3 and ZC3H7B-BCOR; Other potentially qualifying BCOR alterations require approval of study principal investigator; note that loss of function alterations of BCOR would not qualify.
  • Disease Status: Patients on the phase II portion of the study must have measurable disease whereas patients on the PK cohorts can have either evaluable or measurable disease as measured by the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (Version 1.1).
  • Primary Brain Tumors: Patients with primary brain tumors are eligible and must also have measurable disease for the phase II (as well as evaluable or measurable for the PK cohorts), but this can be defined as at least equal or greater than twice the slice thickness in two perpendicular diameters on MRI OR diffuse leptomeningeal disease OR clear MRI evidence of disease that may not be measurable in two perpendicular diameters OR positive CSF cytology alone.
  • Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and meet minimum washout durations (shown below) from prior therapy.
  • Anti-cancer agents not known to be myelosuppressive: ≥ 7 days
  • Anti-cancer and cytotoxic agents known to be myelosuppressive: ≥ 21 days
  • Immunotherapies (including antibodies, interleukins, interferons, etc.): ≥ 21 days
  • Adoptive cellular therapies (including modified T cells, vaccines, etc.): ≥ 42 days
  • Autologous stem cell infusion (boost, no conditioning): ≥ 21 days
  • Autologous stem cell transplantation (with conditioning): ≥ 42 days
  • Allogeneic bone marrow transplantation: ≥ 84 days
  • Focal external beam radiation (e.g., limited sites of disease): ≥ 14 days
  • Substantial external beam radiation (e.g. whole lung or abdomen): ≥ 42 days
  • Radiopharmaceutical therapy (e.g., radiolabeled antibody or MIBG): ≥ 42 days
  • Hepatic Function: Adequate function (within 14 days prior to C1D1), defined as:
  • Total bilirubin < 1.5 × upper limit of normal (ULN) (except patients with Gilbert's syndrome, who must have a total bilirubin of <3 × ULN)
  • Alanine aminotransferase (ALT) < 3 × ULN
  • Serum albumin ≥ 2 g/dL
  • Renal Function: Adequate function (within 14 days prior to C1D1) defined as a GFR
  • ≥ 50 ml/min/1.73 m2 determined via any of these methods:
  • Nuclear radioisotope
  • 24 hr urine creatinine clearance
  • Serum cystatin c
  • Serum creatinine using the Schwartz formula for estimating creatinine clearance (Schwartz et al. J Peds, 106:522, 1985)
  • Hematologic Function: Adequate function (within 14 days prior to C1D1), defined as:
  • Absolute neutrophil count (ANC) ≥ 1000/mm3
  • Platelet count ≥ 100,000/mm3
  • Note: patients may not receive platelet transfusions nor hematopoietic growth factor support, including granulocyte-colony stimulating factor (e.g. filgrastim) and platelet stimulators (e.g. romiplostim) for at least 7 days prior to demonstrating adequate hematologic function.

Who cannot join

  • Prior Therapy: Has received selinexor or another XPO1 inhibitor previously.
  • Infection: Patients who have an uncontrolled infection are not eligible. Patients on prophylactic antibiotics or with a controlled infection within 1 week prior to C1D1 are acceptable
  • Transplants: Patients who have received allogeneic bone marrow transplant are potentially eligible unless they are being actively treated for GvHD. Patients who have had a prior solid organ transplantation are not eligible.
  • Compliance: Patients who as a result of serious medical, psychiatric, and/or social situation(s), in the opinion of the investigator, may not be able to comply with supportive care, safety monitoring, or any other key requirements of the study protocols are not eligible.
  • Pregnancy and Breast-feeding: 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.
  • Contraception: 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.

Trial locations and contacts

16 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Michael Ortiz, MD contact

1-833-MSK-KIDS

ortizm2@mskcc.org

Julia Glade Bender, MD contact

1-833-MSK-KIDS

16 sites in 1 country

United States16 sites, 16 recruiting

  • Children's Healthcare of Atlanta (Data Collection and Specimen Analysis)

    Atlanta, Georgia, 30322

    Thomas Cash, MD, 404-785-0910

    Recruiting

  • Memorial Sloan Kettering at Basking Ridge (Limited Protocol Activities)

    Basking Ridge, New Jersey, 07920

    Michael Ortiz, MD, 833-675-5437

    Recruiting

  • Dana Farber Cancer Institute (Data Collection Only)

    Boston, Massachusetts, 02115

    Elizabeth Mullen, MD, 617-632-1938

    Recruiting

  • Ann & Robert H. Lurie Children'S Hospital of Chicag

    Chicago, Illinois, 60611

    Elizabeth Sokol, MD, 800-543-7362

    Recruiting

  • Cincinnati Children's Hospital Medical Center (Data collection only)

    Cincinnati, Ohio, 45229

    Brian Turpin, DO, 513-636-3200

    Recruiting

  • Memorial Sloan Kettering Suffolk-Commack (Limited Protocol Activities )

    Commack, New York, 11725

    Michael Ortiz, MD, 833-675-5437

    Recruiting

  • Cook Children's Health Care System (Data Collection Only)

    Fort Worth, Texas, 76104

    Kelly Vallance, MD, MPH, 682-885-4007

    Recruiting

  • Memorial Sloan Kettering Westchester (Limited Protocol Activities)

    Harrison, New York, 10604

    Michael Ortiz, MD, 833-675-5437

    Recruiting

  • Children's Hospital of Los Angeles (Data Collection Only)

    Los Angeles, California, 90027

    Rachana Shah, MD, 323-660-2450

    Recruiting

  • Memorial Sloan Kettering Monmouth (Limited Protocol Activities)

    Middletown, New Jersey, 07748

    Michael Ortiz, MD, 833-675-5437

    Recruiting

  • Memorial Sloan Kettering Bergen (Limited protocol activities)

    Montvale, New Jersey, 07645

    Michael Ortiz, MD, 833-675-5437

    Recruiting

  • Memorial Sloan Kettering Cancer Center (All protocol activites)

    New York, New York, 10065

    Michael Ortiz, MD, 833-675-5437

    Recruiting

  • Stanford Medicine Children's Health (Data Collection Only)

    Palo Alto, California, 94304

    Chelsey Burke, MD, 650-497-8953

    Recruiting

  • Memorial Sloan Kettering Nassau (Limited protocol activities)

    Rockville Centre, New York, 11553

    Michael Ortiz, MD, 833-675-5437

    Recruiting

  • Washington University

    St Louis, Missouri, 63110

    Amy Armstrong, MD, 314-454-6018

    Recruiting

  • Children's National Hospital (Data Collection Only)

    Washington D.C., District of Columbia, 20010

    Aerang Kim, MD, 888-884-2327

    Recruiting

Other trials in United States

About this study

Official title: A Multi-Center Phase II Study of Selinexor in Treating Recurrent or Refractory Wilms Tumor and Other Pediatric Solid Tumors

The purpose of this study is to find out whether selinexor is an effective treatment for people who have a relapsed/refractory Wilms tumor, rhabdoid tumor, MPNST, BCOR-driven sarcoma, or another solid tumor that makes a higher than normal amount of XPO1 or has genetic changes that increase the activity of XP01.

Being tested
Selinexor
Keywords
Wilms Tumor, Rhabdoid Tumor, Malignant Peripheral Nerve Sheath Tumors, MPNST, Nephroblastoma, XPO1, Selinexor, Memorial Sloan Kettering Cancer Center, 22-393, Solid Tumor
Medical subject headings
Wilms Tumor, Rhabdoid Tumor, Neurofibrosarcoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SelinexorDrug

    This phase II study will initially treat all patients, including adult patients, at the pediatric Selinexor RP2D of 35 mg/m2 (maximum dose 100mg/dose) once weekly using a liquid suspension .

Study groups in NCT05985161
Study groupRoleWhat they receive
Cohort A.1 Wilms TumorExperimentalParticipants will have any type of Wilms tumor or nephroblastoma
Receives: Drug: Selinexor
Cohort B.1 Rhabdoid TumorExperimentalParticipants will have any Rhabdoid tumor
Receives: Drug: Selinexor
Cohort C.1 MPNSTExperimentalParticipants will have progressive, relapsed, unresectable or metastatic MPNST
Receives: Drug: Selinexor
Cohort D.1 Other Solid TumorExperimentalParticipants must not qualify for Cohorts A, B, or C but have a solid tumor (no hematologic malignancies including lymphoma) for which there is specific evidence that this particular patient's tumor may benefit from selinexor.
Receives: Drug: Selinexor
Cohort E.1g BCOR driven sarcomaExperimentalPatients must have a solid tumor with an activating genomic alteration (e.g. fusion or internal tandem duplication) involving BCOR. Specific examples of qualifying alterations including BCOR-ITD, BCOR-CCNB3,BCOR-MAML3 and ZC3H7B-BCOR;Other potentially qualifying BCOR alterations require approval of study principal investigator; note that loss of function alterations of BCOR would not qualify.
Receives: Drug: Selinexor

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Response Rate

    To define the antitumor activity of Selinexor in relapsed and refractory Wilms tumor by measuring the Overall Response Rate, defined as Complete Response + Partical Response

    Measured: 6 months following the start of the treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Aug 2023
  2. Study start1 Aug 2023
  3. Primary completion1 Aug 2029 (estimated)
  4. Study completion1 Aug 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
45 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05985161
Sponsor's protocol ID
22-393
Status last verified
Oct 2026
First submitted
2 Aug 2023
Last updated
23 Oct 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Selinexor in people with Wilms Tumor and Rhabdoid Tumor, run by Memorial Sloan Kettering Cancer Center at 16 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 42 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 23 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Michael Ortiz, MD (1-833-MSK-KIDS, ortizm2@mskcc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (time frame: 6 months following the start of the treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05985161.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2026.

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