Actively Recruiting

Phase 1
Phase 2
Age: 12Months - 30Years
All Genders
NCT04901702

Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma

Led by St. Jude Children's Research Hospital · Updated on 2026-05-08

90

Participants Needed

10

Research Sites

290 weeks

Total Duration

On this page

Sponsors

S

St. Jude Children's Research Hospital

Lead Sponsor

P

Pfizer

Collaborating Sponsor

AI-Summary

What this Trial Is About

The phase I portion of this study is designed for children or adolescents and young adults (AYA) with a diagnosis of a solid tumor that has recurred (come back after treatment) or is refractory (never completely went away). The trial will test 2 combinations of therapy and participants will be randomly assigned to either Arm A or Arm B. The purpose of the phase I study is to determine the highest tolerable doses of the combinations of treatment given in each Arm. In Arm A, children and AYAs with recurrent or refractory solid tumors will receive 2 medications called Onivyde and talazoparib. Onivyde works by damaging the DNA of the cancer cell and talazoparib works by blocking the repair of the DNA once the cancer cell is damaged. By damaging the tumor DNA and blocking the repair, the cancer cells may die. In Arm B, children and AYAs with recurrent or refractory solid tumors will receive 2 medications called Onivyde and temozolomide. Both of these medications work by damaging the DNA of the cancer call which may cause the tumor(s) to die. Once the highest doses are reached in Arm A and Arm B, then "expansion Arms" will open. An expansion arm treats more children and AYAs with recurrent or refractory solid tumors at the highest doses achieved in the phase I study. The goal of the expansion arms is to see if the tumors go away in children and AYAs with recurrent or refractory solid tumors. There will be 3 "expansion Arms". In Arm A1, children and AYAs with recurrent or refractory solid tumors (excluding Ewing sarcoma) will receive Onivyde and talazoparib. In Arm A2, children and AYAs with recurrent or refractory solid tumors, whose tumors have a problem with repairing DNA (identified by their doctor), will receive Onivyde and talazoparib. In Arm B1, children and AYAs with recurrent or refractory solid tumors (excluding Ewing sarcoma) will receive Onivyde and temozolomide. Once the highest doses of medications used in Arm A and Arm B are determined, then a phase II study will open for children or young adults with Ewing sarcoma that has recurred or is refractory following treatment received after the initial diagnosis. The trial will test the same 2 combinations of therapy in Arm A and Arm B. In the phase II, a participant with Ewing sarcoma will be randomly assigned to receive the treatment given on either Arm A or Arm B.

CONDITIONS

Official Title

Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma

Who Can Participate

Age: 12Months - 30Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients must be over 12 months and under 30 years old at enrollment.
  • Phase I: Patients with refractory or recurrent non-CNS solid tumors with histologic verification are eligible.
  • Patients eligible for expansion cohort A2 must have defects in DNA repair genes confirmed by prior testing.
  • Phase II: Patients with refractory or recurrent Ewing sarcoma after first-line therapy.
  • Histologic diagnosis of Ewing sarcoma with specific genetic markers is required.
  • Patients must have measurable or evaluable disease.
  • Performance score above 50% (Karnofsky for >16 years, Lansky for <16 years).
  • Prior irinotecan- or temozolomide-based therapy allowed in phase I; prior PARP inhibitors other than talazoparib allowed.
  • Phase II patients must have adequate organ and bone marrow function as defined by lab values.
  • Patients must have recovered from acute effects of prior treatments.
  • Post-menarchal females must have a negative pregnancy test and agree to ongoing testing.
  • Participants of reproductive potential must agree to use effective contraception during the study.
Not Eligible

You will not qualify if you...

  • Pregnant or breastfeeding individuals are not eligible.
  • Participants unwilling to use two forms of birth control during and after treatment.
  • Patients who donate sperm or eggs during and shortly after the study treatment period are excluded.
  • Females not of childbearing potential include those surgically sterile.
  • Patients with ongoing severe toxicities or conditions preventing treatment continuation are excluded.

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Trial Site Locations

Total: 10 locations

1

Lucille Packard Children's Hospital Stanford

Palo Alto, California, United States, 94304

Actively Recruiting

2

Children's Hospital Colorado

Aurora, Colorado, United States, 80045

Actively Recruiting

3

Children's National Medical Center

Washington D.C., District of Columbia, United States, 20010

Actively Recruiting

4

Children's Healthcare of Atlanta/Emory University School of Medicine

Atlanta, Georgia, United States, 30322

Not Yet Recruiting

5

Children's Hospital and Clinics of Minn

Minneapolis, Minnesota, United States, 55404

Actively Recruiting

6

St. Jude Children's Research Hospital

Memphis, Tennessee, United States, 38105

Actively Recruiting

7

Texas Children's Hospital/ Baylor College of Medicine

Houston, Texas, United States, 77030

Active, Not Recruiting

8

The Hospital for Sick Children

Toronto, Ontario, Canada

Actively Recruiting

9

CHU Sainte-Justine

Montreal, Canada

Actively Recruiting

10

BC Children's Hospital Research Institute

Vancouver, Canada

Not Yet Recruiting

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Research Team

S

Sara Federico, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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