Recurrent or progressive primary brain tumors in one of four primary brain tumor types: high-grade glioma, medulloblastoma, ependymoma and diffuse intrinsic pontine glioma (DIPG
Conditions
Sponsors
Celgene Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject is 1 to < 21 years of age at the time of signing the ICF/IAF. 2. Subject must understand and voluntarily sign an ICF/IAF prior to any study-related assessments/procedures being conducted. 3. Subject has received at least one prior standard therapy (or generally accepted upfront therapy if no standard exists) and have no known curative therapy. 4. Subject has a diagnosis of high-grade glioma, medulloblastoma, ependymoma or DIPG that is recurrent or progressive. 5. Subject has histological verification of tumor either at the time of diagnosis or recurrence. 6. Subject has measurable disease, defined as a tumor that is measurable in two perpendicular diameters on MRI. 7. To document the degree of tumor through scan; 8. Subject has Karnofsky (age = 16 years) or Lansky (age < 16 years) performance status score = 50 at screening (Appendix D) 9. Subject has adequate bone marrow function; 10. Subject has adequate renal function; 11. Subject has adequate liver function; 12. Subject has adequate pulmonary function; 13. Subject has recovered from clinically significant acute treatment related toxicities from all prior therapies. 14. Subject has no significant worsening in clinical status for a minimum of 7 days prior to first dose of study drug; 15. Subject (and when applicable, with parental/legal representative) is willing and able to adhere to the study visit schedule and other protocol requirements. 16. Females of Childbearing Potential (FCBP), Female Children of Childbearing Potential (FCCBP), 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. 17. All subjects and/or parents/legal representative must have an understanding that pomalidomide could have a potential teratogenic risk. Female children of childbearing potential, defined as females who have achieved menarche and/or breast development in Tanner Stage 2 or greater and have not undergone a hysterectomy or bilateral oophorectomy and FCBP, defined as a sexually mature woman who has not undergone a hysterectomy or bilateral oophorectomy and has not been naturally postmenopausal for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) must agree and meet the following conditions below: -Medically supervised pregnancy tests with a sensitivity of at least 25 mIU/mL must be conducted in FCCBP/FCBP, 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. The subject may not receive pomalidomide until the Investigator has verified that the results of these pregnancy tests performed on Cycle 1, Day 1 are negative. Female children of childbearing potential/females of childbearing potential with regular or no menstrual cycles must agree to have pregnancy tests weekly for the first 28 days study participation, every 28 days while on study, at study treatment discontinuation visit, and at Day 28 following pomalidomide discontinuation. If menstrual cycles are irregular, the pregnancy testing must occur weekly for the first 28 days of study participation and then every 14 days while on study, at study treatment d
Exclusion criteria
Exclusion criteria: 1. Subject has a history of non-central line related thrombosis (arterial or venous), more than one prior central-line related thrombosis or known coagulopathy. 2. Subject has first degree family member with a known hereditary coagulopathy. 3. Subject is actively on anticoagulation therapy. 4. Subject has had major (per Investigator discretion) surgery, with the exception of tumor resection, within 21 days from first dose of study drug. 5. Subject has previously received (presence of any of the following will exclude a subject from enrollment): -Any prior treatment with pomalidomide. Subjects who have prior treatment with other IMiDs (thalidomide, lenalidomide) ARE eligible if they meet all other eligibility criteria and did not have allergic reactions or other “significant toxicity” per Investigator discretion associated with lenalidomide or thalidomide use. -Myelosuppressive chemotherapy, immunotherapy, or any investigational agent: = 28 days (= 42 days if a nitrosourea) prior to screening. -Biological (anti-neoplastic) therapy: = 7 days prior to screening. -Immunomodulatory therapy: = 28 days prior to screening. -Monoclonal antibody treatment and agents with known prolonged half-lives: < 3 half-lives have elapsed or = 28 days prior to screening, whichever is longer. -Prior radiation: .Cranial irradiation, total body irradiation (TBI), or = 50% radiation of pelvis: = 3 months prior to screening. .Focal irradiation: = 6 weeks prior to screening. .Local palliative radiation therapy (small port): < 4 weeks prior to screening. -Bone marrow transplant: .Presence of graft versus host disease (GVHD) . < 6 months since allogeneic bone marrow transplant prior to screening. . < 3 months since autologous bone marrow/stem cell transplant prior to screening. . < 3 months since stem cell transplant (SCT) or Rescue without TBI with no evidence of GVHD prior to screening. - Radioisotopes: fluorothymidine (18FLT) = 72 hours prior to first dose of study drug 8. Subject is pregnant, breast-feeding or lactating.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To identify potential tumor type(s) for further development by establishing the preliminary efficacy of pomalidomide in children and young adults with recurrent or progressive primary brain tumors within four distinct tumor types.;Secondary Objective: To evaluate the safety of pomalidomide within the study populations. To estimate the long-term efficacy of pomalidomide treatment.;Primary end point(s): Objective response and long-term stable disease rate;Timepoint(s) of evaluation of this end point: Cycle 1, Day 1 to completion of Cycle 6 (completion of Cycle 3 for DIPG) | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: 1. Cycle 1, Day 1 of Treatment Period to end of Follow-up Period 2. Cycle 1, Day 1 to completion of Cycle 6 (completion of Cycle 3 for DIPG) 3. Cycle 1, Day 1 of Treatment Period to end of Follow-up Period 4. Cycle 1, Day 1 of Treatment Period to end of Follow-up Period 5. Cycle 1, Day 1 of Treatment Period to end of Follow-up Period 6. Signing of informed consent form (ICF)/informed assent form (IAF) until the end of Follow-up Period;Secondary end point(s): 1. Objective response rate 2. Long-term stable disease rate 3. Duration of response 4. Progression-free survival 5. Overall Survival 6. Safety | — |
Countries
France, Italy, Spain, United Kingdom, United States
Contacts
Public ContactClinicalTrialDisclosure
Celgene Corporation
Outcome results
None listed