A Study of Pomalidomide Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors

Inclusion Criteria

• Subject is 1 to < 21 years of age at the time of signing the Informed Consent Form/Informed Assent Form (ICF/IAF).
• Subject (when applicable, parental/legal representative) must understand and voluntarily sign an ICF/IAF prior to any study-related assessments/procedures being conducted.
• Subject has received at least one prior standard therapy (or generally accepted upfront therapy if no standard exists) and have no known curative therapy.
• Subject has a diagnosis of high-grade glioma, medulloblastoma, ependymoma or diffuse intrinsic pontine glioma (DIPG) that is recurrent or progressive. Subjects with neurofibromatosis type 1 (NF-1) associated tumors are eligible if they meet all other eligibility criteria.
• Subject has histological verification of tumor either at the time of diagnosis or recurrence. Subjects with DIPG are exempt from histologic verification if they have typical magnetic resonance imaging (MRI) findings of DIPG
• Subject has measurable disease defined as a tumor that is measurable in 2 perpendicular diameters on MRI. For a lesion to be considered measurable, it must be at least twice the slice thickness on MRI (ie, visible on 2 or more axial slices)
• To document the degree of tumor at study baseline, the following scan(s) must be obtained:
• A brain MRI with and without contrast (ie, gadolinium) and a spine MRI with contrast within 21 days prior to first dose of study treatment. For subjects on steroids, baseline MRI scans must be performed while on stable or decreasing dose of steroids for at least 5 days.
• Subject has Karnofsky (age ≥ 16 years) or Lansky (age < 16 years) performance status score ≥ 50 at screening
• Subject has adequate bone marrow function defined as:
• Peripheral absolute neutrophil count (ANC) ≥ 1000/mm³
• Platelet count ≥ 100, 000/mm³ (transfusion independent defined as no platelet transfusion within 7 days and recovery from nadir)
• Hemoglobin ≥ 8 g/dL (red blood cell [RBC] transfusion is allowed)
• Subject has adequate renal function defined as:
• Serum creatinine based on age/gender calculated using the Schwartz formula, or a 24-hour creatinine clearance or radioisotope glomerular filtration rate (GFR) (radioisotope or iothalamate) ≥ 70 mL/min/1.73 m².
• Subject has adequate liver function defined as:
• Total bilirubin ≤ 1.5 X upper limit of normal (ULN) for current age (≤ 3 X ULN if increase in bilirubin is attributable to Gilbert's Syndrome)
• Alanine aminotransferase (ALT) (SPGT) is ≤ 3 X ULN for age
• Serum albumin ≥ 3 g/dL
• Subject has adequate pulmonary function defined as:
• No evidence of dyspnea at rest
• A pulse oximetry ≥ 93%
• Subject has recovered from clinically significant acute treatment related toxicities from all prior therapies. Recovery is defined as a toxicity Grade ≤ 2 (common terminology criteria for adverse events [CTCAE] v. 4.03).
• Subject has no significant worsening in clinical status for a minimum of 7 days prior to first dose of study drug.
• Subject (and when applicable, with parental/legal representative) is willing and able to adhere to the study visit schedule and other protocol requirements.
• Females of Childbearing Potential (FCBP) and male subjects who have reached puberty (and when applicable, with parental/legal representative) must agree to undergo physician-approved reproductive education and discuss the side effects of the study therapy on reproduction.
• Females of childbearing potential must agree and meet the following conditions below:
• Medically supervised (ie, performed in a clinic) pregnancy testing, including those who commit to true abstinence. Two pregnancy tests must be conducted prior to starting pomalidomide. The first pregnancy test must be performed 10 to 14 days prior to the start of pomalidomide and the second pregnancy test must be performed within 24 hours prior to starting pomalidomide. Females of childbearing potential with regular or no menstrual cycles must al