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Aurora Kinase Inhibitor

Alisertib for Rhabdoid Tumors (SJATRT Trial)

Phase 2

Waitlist Available

Led By Santhosh Upadhyaya, MD

Research Sponsored by St. Jude Children's Research Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Histologic diagnosis of AT/RT or MRT with loss of INI1 or BRG1 expression in tumor cells confirmed by immunohistochemistry, or by molecular confirmation of tumor-specific biallelic SMARCB1/SMARCA4 loss/mutation if INI1/BRG1 immunohistochemistry is not available

Patients with newly diagnosed AT/RT

Must not have

Female participants of childbearing potential cannot be pregnant or breast-feeding

Myocardial infarction within 6 months prior to enrollment or New York Heart Association Class III or IV heart failure

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at the time of tumor assessment (up to 5 years)

Awards & highlights

No Placebo-Only Group

Summary

This trial will test the use of alisertib to treat patients younger than 22 years of age with recurrent or refractory atypical teratoid rhabdoid tumor (AT/RT) or malignant rhabdoid tumor (MRT). Alisertib will be given as a single agent or in combination with other therapy, depending on the patient's age and diagnosis. The trial will also test the use of alisertib in patients with newly diagnosed AT/RT, to see if it can help improve the 3-year progression-free survival rate.

Who is the study for?

This trial is for patients under 22 years old with rhabdoid tumors, including AT/RT or MRT. They must have a confirmed diagnosis and measurable disease or tumor cells in CSF. Participants need proper organ function, no prior alisertib treatment, and a life expectancy over 8 weeks. Females of childbearing age must test negative for pregnancy and agree to birth control use.

What is being tested?

The study tests alisertib's effectiveness on pediatric patients with recurrent or newly diagnosed rhabdoid tumors. It will be given alone or with chemotherapy/radiation depending on the patient's condition and age. The trial aims to measure response rates, progression-free survival (PFS), overall survival (OS), drug toxicity, local/distant failure patterns, and pharmacokinetics.

What are the potential side effects?

Possible side effects include toxicities related to alisertib when used as a single agent or combined with other therapies across multiple courses; these may affect organs like bone marrow, liver, kidneys etc., as well as potential radiation therapy-related toxicities.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My tumor lacks INI1 or BRG1, confirmed by tests.

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I have been recently diagnosed with AT/RT.

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I have been newly diagnosed with AT/RT and have cancer outside the brain.

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My cancer has returned or worsened and can be seen on scans.

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My kidney, liver, and bone marrow are functioning well.

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I was diagnosed before my 22nd birthday.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not pregnant or breastfeeding.

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I have not had a heart attack in the last 6 months and do not have severe heart failure.

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I currently have an infection that is not under control.

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I do not have stomach or bowel problems that affect medication absorption.

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I need to take medication regularly for stomach acid or digestion.

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I have a condition causing excessive daytime sleepiness.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at the time of tumor assessment (up to 5 years)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at the time of tumor assessment (up to 5 years)

This trial's timeline: 3 weeks for screening, Varies for treatment, and at the time of tumor assessment (up to 5 years) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

1-year progression free survival rate (stratum B2)

1-year progression free survival rate (stratum C2)

3-year progression free survival rate (stratum B1)

+4 more

Secondary study objectives

1-year progression-free survival (PFS) by stratum A1 and A2

5-year Overall survival (OS) rate in patients with newly diagnosed AT/RT (strata B1, B2, B3, C1, C2)

5-year Progression-free survival (PFS) rate in patients with newly diagnosed AT/RT (strata B1, B2, B3, C1, C2)

+2 more

Side effects data

From 2013 Phase 3 trial • 397 Patients • NCT00004054

78%

Menopausal symptoms

60%

Urinary frequency

52%

Impotence

47%

Late RT Toxicity: Bladder: NOS

39%

Diarrhea NOS

27%

Late RT Toxicity: Bowel: NOS

26%

Fatigue

26%

Late RT Toxicity: Other GU: NOS

21%

Proctitis NOS

21%

Dysuria

14%

Libido decreased

14%

Dermatitis radiation NOS

12%

Late RT Toxicity: Other GI: NOS

12%

Late RT Toxicity: Other: NOS

12%

Hemoglobin decreased

12%

Alanine aminotransferase increased

Gynaecomastia

Urinary retention

Pain-other

Aspartate aminotransferase increased

Rectal bleeding

Constipation

Leukopenia NOS

Edema NOS

Hematuria present

Renal/GU-Other

Arthralgia

Blood creatinine increased

Dyspnea NOS

Dermatitis exfoliative NOS

Depression NEC

Nausea

Hyperglycemia NOS

Lymphopenia

Myalgia

Peripheral sensory neuropathy

Platelet count decreased

Hypocalcemia

Hyponatremia

Blood albumin decreased

Stomatitis

Weight decreased

Anorexia

Hematologic-Other

Neutropenia

Alopecia

100%

80%

60%

40%

20%

Study treatment Arm

Hormones and RT

Hormones and RT Plus Chemotherapy

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: (C) Alisertib, chemotherapy, radiation therapyExperimental Treatment6 Interventions

Stratum C: Children ≥36 months old with newly diagnosed AT/RT. Participants with synchronous extraneural AT/RT (Stratum D4) will also be treated as those assigned to Stratum C. Interventions: Craniospinal radiation therapy; followed by consolidation chemotherapy using alisertib, vincristine, cisplatin (or carboplatin), cyclophosphamide; followed by maintenance alisertib, surgical resection, if indicated.

Group II: (B) Alisertib, chemotherapy, radiation therapyExperimental Treatment10 Interventions

Stratum B: Children \< 36 months old with newly diagnosed AT/RT. AT/RT those with synchronous extraneural AT/RT (Stratum D1) may also be treated on this arm. Interventions: * B1 or D1: Induction chemotherapy using methotrexate, vincristine, cisplatin (or carboplatin), cyclophosphamide; followed by focal radiation therapy; followed by induction therapy using alisertib, vincristine, cisplatin (or carboplatin), cyclophosphamide; followed by maintenance alisertib. Those \<12 months who are not ready for focal radiation therapy will receive consolidation chemotherapy using alisertib, cyclophosphamide, carboplatin and etoposide while RT is delayed. Surgical resection, if indicated. * B2, B3, D2 or D3: Induction chemotherapy using alisertib, vincristine, cisplatin (or carboplatin), cyclophosphamide; followed by consolidation with topotecan and cyclophosphamide or optional craniospinal irradiation; followed by maintenance alisertib. Surgical resection, if indicated.

Group III: (A) Alisertib aloneExperimental Treatment2 Interventions

Stratum A: Patients with recurrent/progressive AT/RT or extra-CNS malignant rhabdoid tumors (MRT). Interventions: alisertib, 35 cycles of 3 weeks each (up to 105 weeks). Surgical resection, if indicated.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Surgical resection

2021

Completed Phase 2

~1740

Radiation therapy

2013

Completed Phase 3

~2850