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Protocol Details

CC-4047-BRN-001: A Phase 2 Clinical Study of Pomalidomide (CC-4047) Monotherapy for Children and Young Adults with Recurrent or Progressive Primary Brain Tumors

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Cancer Institute (NCI)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 3
Max Age: 20

Referral Letter Required


Population Exclusion(s)



Blood-Brain Barrier;
IMiDs Compounds

Recruitment Keyword(s)



Malignant Glioma;
Brain Tumor, Recurrent;
Medulloblastoma, Childhood;
Neurofibromatosis Type 1;

Investigational Drug(s)

Pomalidomide (CC-4047)

Investigational Device(s)



Drug: Pomalidomide (CC-4047)

Supporting Site

National Cancer Institute


Pomalidomide is a drug to treat cancer. This kind of drug can affect the way the immune system works and how tumor cells grow. The study drug may block or change some substances that help brain tumors survive and grow. It may also help the immune system fight the tumor.


To test pomalidomide for the treatment of brain tumors and see if it is helpful for children and young people whose tumor came back or kept growing after previous treatment.


People ages 3 21 with certain brain tumors


Participants will be screened with:

-Medical history

-Blood and urine tests

-Physical exam

-Brain and nervous system assessment.

-Some will have a magnetic resonance imaging (MRI) scan. They will lie in a machine that takes pictures of the brain and spine. A solution will be injected to get better pictures.

The study will be done in 28-day cycles. Participants may have up to 24 cycles.

Participants will take up to 3 pills of the study drug each day for the first 21 days of each cycle.

Throughout the study:

-Participants will repeat the screening tests.

-They will be told about the risks of the study drug.

-They may have cerebrospinal fluid collected by a needle in the spine.

Participants will write in a diary all the doses of study drug they take.

Participants will have follow-up for 28 days after they stop taking the study drug. They will repeat most of the screening tests. Then they will have a phone call or visit every 3 months for up to a few years.

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- Age greater than or equal to 3 to less than 21 years at the time of enrollment.

- Must weight greater than or equal to 12 kilograms

- Must have a diagnosis of high-grade glioma, medulloblastoma, ependymoma or DIPG and have received at least one prior standard therapy (or generally accepted upfront therapy if no standard exists) and have no known curative therapy options. Patients with neurofibromatosis type 1 (NF-1) associated tumors are eligible if they meet all other eligibility criteria.

- Subject has measurable disease and histological verification of tumor either at the time of diagnosis or recurrence. Subjects with DIPG are exempt from histologic verification if they have typical MRI findings of DIPG (ie, hypo- or isointense on T1-weighted imaging, hyperintense on fluid-attenuated inversion recovery (FLAIR) or T2-weighted imaging, epicenter in the pons, greater than 50% of pons involved)

- Must have performance status greater than or equal to 50% (Karnofsky if patient greater than or equal to 16 years of age or Lansky if patient <16 years of age).

- Must have acceptable major organ function reflected in laboratory evaluation of bone marrow, liver function tests, renal function tests and pulmonary function.

- Females of childbearing potential (FCBP) must not be pregnant or breast feeding; and both males who have reached puberty and females (FCBP) must agree to undergo approved reproductive education, and must follow study specific requirements for contraception in the Pregnancy Prevention Program.


- Patients with history of non-central line related thrombosis, more than one central-line related thrombosis or known coagulopathy, or have first degree family member with known hereditary coagulopathy, or is actively on anticoagulation therapy.

- Has had major surgery within 21 days from first dose of therapy or have had prior treatment with pomalidomide. All other wash-out periods must be met.

- Patients who have received known moderate to potent CYP1A2 inhibitor within 14 days or 5 half-lives of the first dose of study treatment.

- Patients with untreated or uncontrolled infection, or active infectious hepatitis (type A, B, or C), or chronic carrier of Hepatitis C.

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Not Provided

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Principal Investigator

Referral Contact

For more information:

John W. Glod, M.D.
National Cancer Institute (NCI)
NIHBC 10 - CRC BG RM 1-3750
(240) 760-6194

Andrea (Andy) F. Gillespie, R.N.
National Cancer Institute (NCI)
National Institutes of Health
Building 10
Room 84DD
10 Center Drive
Bethesda, Maryland 20892
(240) 760-6185

NCI Referral Office
National Institute of Health Clinical Center (CC), 9000 Rockville Pike, Bethesda, Maryland 20892, United States: NCI Clinical Trials Referral Office

Clinical Trials Number:


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