Children's National

Eligibility

Eligible Ages

Over 8 Years

Eligible Genders

All

Accepts Healthy Volunteers

No

Criteria

1. Willing and able to provide written IRB/IEC-approved Informed Consent. For patients <
18 years of age, their parents or legal guardians must sign a written informed
consent. Assent, when appropriate, will be obtained according to institutional
guidelines.

2. Have histologically or cytologically confirmed diagnosis of Ewing sarcoma (including
ESFT) with relapsed or refractory disease. Patients with metastatic disease who had
standard chemotherapy at the time of diagnosis Pathology reports and slides or blocks
should be available for review or additional testing. If not available, site must
discuss with Sponsor.

3. Measurable disease according to RECIST version 1.1. Measurable disease can be verified
from a previously documented computed tomography (CT) scan or MRI as long as no
anti-cancer treatments have been administered in the interim.

4. Must have a central venous catheter in place prior to initiating infusion of study
drug.

5. Prior cancer therapy:

Patients may have received no more than 5 prior systemic regimens. At the time of
treatment initiation, at least 2 weeks or 5 half-lives, whichever is longer, must have
elapsed since prior cytotoxic chemotherapy. At least 7 days must have elapsed since
completion of any prior non-cytotoxic cancer therapy.

6. Prior radiotherapy is allowed If ≥ 4 weeks has elapsed for radiation therapy (RT); ≥ 6
months must have elapsed if prior total body irradiation, craniospinal RT or if > 50%
radiation of the pelvis; > 6 weeks must have elapsed if other substantial bone marrow
radiation. Patients who have received brain irradiation must have completed whole
brain radiotherapy and/or gamma knife at least 4 weeks prior to enrollment.

7. Stem Cell Transplant or Rescue without TBI: No evidence of active graft vs. host
disease and ≥ 3 months must have elapsed since transplant.

8. Patients with controlled asymptomatic CNS involvement are allowed (see Concomitant
Medications). Patients not requiring steroids or requiring steroids at stable dose (≤
4 mg/day dexamethasone or equivalent) for at least 2 weeks are eligible.

9. Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer
therapy to NCI CTCAE (Version 4.03) Grade < 1. Details can be provided by Sponsor.

10. Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤ 2 in patients ≥17
years old; or Karnofsky/Lansky >50 in patients <16 years old.

11. Life expectancy of at least 3 months.