Clinical Trial Details
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NCT03206021 : Treatment of Children With Recurrent Refractory Brain/Solid Tumors and Recurrent Ependymoma
PhasePhase 1
AgesMin: 1 Year Max: 18 Years
Inclusion Criteria:

1. Greater than the age of 1 year and under age 18 at the time of study enrolment

2. Recurrent or refractory solid tumor (Phase I), or recurrent or refractory ependymoma
(Phase Ib)

3. Tissue from diagnosis or resection prior to registration must be available (either
flash frozen tissue or an FFPE (formalin fixed paraffin embedded) block)

4. Previous therapy with carboplatin will be permitted

5. Failed first line treatment (surgery, radiation therapy or chemotherapy).

6. Be at least 4 weeks from the completion of myelosuppressive chemotherapy and/or
biologic agents before starting day 1 of this study treatment

7. Be at least 3 months post hematopoetic stem cell rescue following myeloablative
therapy before starting day 1 of this study treatment

8. Must have visible disease on imaging. Resection of visible disease is permitted while
on study after two cycles including achievement of a gross total resection. If a
resection is performed while on study, fresh frozen tissue should be submitted for

9. Concurrent medications will be limited to supportive medications including
anti-emetics, steroids, analgesics and non-enzyme inducing anticonvulsants. Inducers
of the P450 system will not be permitted. Other concurrent medications require
approval of the study sponsor.

10. Ability of the parent and/or child to understand and the willingness to sign a written
informed consent document

11. Karnofsky = 50 for patients > 16 years of age and Lansky = 50 for patients = 16 years
of age (See Appendix I for the Karnofsky-Lansky Scores). Patients who are unable to
walk because of paralysis, but who are up in a wheelchair, will be considered
ambulatory for the purpose of assessing the performance score. Patients with posterior
fossa syndrome/cerebellar mutism demonstrating clear improvement post-surgically can
be enrolled based on physician discretion.

12. Adequate hepatic, renal, marrow and cardiac function as defined below within 14 days
prior to cycle 1 day 1:

- Serum creatinine within normal institutional limits or creatinine clearance
greater than 60mL/min

- Serum bilirubin <1.5 times upper limit of institutional normal. Higher levels are
acceptable if these can be attributed to active hemolysis or ineffective

- AST, ALT and Alkaline Phosphatase <2.5 times upper limit of institutional normal.
If liver metastases are present, then <5 times upper limit of normal is

- Normal cardiac function with a normal QTc interval at screening ECG

- Adequate marrow function defined below within 14 days prior to cycle 1 day 1:

- Leukocytes greater than or equal to 3000 x10 to the power of 6/L

- Absolute neutrophil count greater than or equal to 1 x10 to the power of 9/L

- Platelets greater than or equal to 100 x10 to the power of 9/L

- Hemoglobin greater than or equal to 10g/dL (may be transfused).

Exclusion Criteria:

1. Female patient who is pregnant or breast feeding (Lactating females must agree not to
breast feed while taking azacitidine) or with childbearing potential and not willing
to use a double method of contraception up to 3 months after the end of study
treatment. Male patient who is not willing to use a barrier method of contraception up
to 6 months after the end of study treatment.

2. Patients may not be receiving any other investigational agents with the exception of
Carboplatin within 30 days prior to day 1 of protocol treatment

3. Prior therapy with a DNA demethylase inhibitor

4. Evidence of cardiac toxicity (shortening fraction below 28%; shortening fraction
measures and ratios the change in the diameter of the left ventricle between the
contracted and relaxed states)

5. Abnormal coagulation parameters (PT >15 seconds, PTT>40 seconds, and/or INR >1.5)

6. Significant active cardiac disease within the previous 6 months including:

- NYHA class 3 or 4 CHF

- Unstable angina

- Myocardial infarction

7. Known or suspected hypersensitivity to azacitidine or mannitol carboplatin

8. Previous carboplatin exposure is not an exclusion criteria but previous allergic
reaction to carboplatin will exclude enrolment.

9. Patient must not require use of enzyme inducing anticonvulsants; patients who are
receiving an enzyme inducing anticonvulsant must be able to switch to a non-enzyme
inducing anticonvulsant such as Levetiracetam, Clobazam, Lacosamide, Valproate or
Topiramate at least 2 weeks prior to study enrolment.

10. Uncontrolled systemic fungal, bacterial or viral infection (defined as ongoing
signs/symptoms related the infection without improvement despite appropriate
antibiotics, antiviral therapy and/or other treatment)

11. Active viral infection with HIV or hepatitis type B or C Patients with advanced
malignant hepatic tumors
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