Actively Recruiting

Phase 2
Age: 1Year - 30Years
All Genders
NCT04469530

Sirolimus in Combination With Metronomic Chemotherapy in Children With High-Risk Solid Tumors

Led by Emory University · Updated on 2026-02-27

55

Participants Needed

5

Research Sites

437 weeks

Total Duration

On this page

Sponsors

E

Emory University

Lead Sponsor

P

PeachBowl LegACy Fund

Collaborating Sponsor

AI-Summary

What this Trial Is About

The primary objective of this study is to improve the 2-year progression-free survival in children with high-risk solid tumors who are administered a maintenance regimen with continuous sirolimus administered on a backbone of metronomic chemotherapy following the completion of "standard" therapy, as compared to high-risk solid tumor patients treated with observation alone following completion of "standard" therapy.

CONDITIONS

Official Title

Sirolimus in Combination With Metronomic Chemotherapy in Children With High-Risk Solid Tumors

Who Can Participate

Age: 1Year - 30Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age between 12 months and 30 years at enrollment
  • Diagnosis of high-risk pediatric extracranial solid tumor in complete remission or with minimal imaging abnormalities after initial therapy or relapse treatment
  • Specific tumor types include metastatic or unresectable osteosarcoma, metastatic Ewing or Ewing-like sarcoma, high-risk rhabdomyosarcoma, metastatic non-rhabdomyosarcoma soft tissue sarcoma, desmoplastic small round cell tumor, malignant rhabdoid tumor, or other high-risk tumors approved by study chair
  • No primary central nervous system tumors or lymphomas
  • Histologic confirmation of malignancy at diagnosis or relapse
  • Karnofsky score ≥ 50% for patients >16 years or Lansky score ≥ 50% for patients ≤16 years
  • Full recovery from acute non-blood-related toxic effects of prior cancer therapy and stable or improving chronic toxic effects
  • Adequate bone marrow function with ANC ≥ 750/μL and platelets ≥ 50,000/μL without recent transfusions
  • Adequate kidney function by creatinine clearance or GFR ≥ 70 ml/min/1.73 m2 or age/gender-based creatinine values
  • Adequate liver function with bilirubin ≤ 2 times upper limit and AST/ALT ≤ 225 U/L
  • Serum triglycerides ≤ 300 mg/dL and cholesterol ≤ 300 mg/dL
  • Random blood glucose ≤ 1.5 times upper limit for age
  • Adequate lung function if clinically indicated; normal oxygen saturation if no symptoms
Not Eligible

You will not qualify if you...

  • Pregnant or breastfeeding women; pregnancy tests required for post-menarchal girls
  • Males and females of reproductive potential must use effective contraception during and for 3 months after treatment
  • Receiving corticosteroids must be on stable or decreasing dose for prior 7 days
  • Currently taking enzyme-inducing anticonvulsants
  • Taking potent CYP3A4 inducers or inhibitors
  • Receiving other investigational drugs or anti-cancer agents
  • Presence of uncontrolled infection
  • Enrolled in other clinical trials for upfront or relapse therapy if in second remission
  • Unable to comply with safety monitoring requirements as judged by investigator

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 5 locations

1

Aflac Cancer & Blood Disorders Centers

Atlanta, Georgia, United States, 30329

Actively Recruiting

2

Children's Healthcare of Atlanta

Atlanta, Georgia, United States, 30329

Actively Recruiting

3

Washington University School of Medicine

St Louis, Missouri, United States, 63110

Actively Recruiting

4

Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center (UPMC)

Pittsburgh, Pennsylvania, United States, 15224

Actively Recruiting

5

Texas Children's Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

K

Kathryn Sutton, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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