Infusion of 5-Azacytidine (5-AZA) Into the Fourth Ventricle in Children With Recurrent Posterior Fossa Ependymoma (5-AZA)
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|ClinicalTrials.gov Identifier: NCT02940483|
Recruitment Status : Completed
First Posted : October 21, 2016
Last Update Posted : November 30, 2018
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|Condition or disease||Intervention/treatment||Phase|
|Brain Tumor Recurrent||Drug: 5-Azacytidine||Early Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Infusion of 5-Azacytidine (5-AZA) Into the Fourth Ventricle or Resection Cavity in Children With Recurrent Posterior Fossa Ependymoma: A Pilot Study|
|Actual Study Start Date :||February 1, 2017|
|Actual Primary Completion Date :||November 28, 2018|
|Actual Study Completion Date :||November 28, 2018|
Experimental: 5-Azacytidine Infusion
12 infusions (once a week) into implanted fourth ventricle catheter/Ommaya reservoir following surgical catheter placement into fourth ventricle.
5-Azacytidine 10 mg into the fourth ventricle of the brain via the Ommaya reservoir 1 day a week for 12 weeks.
Other Name: Vidaza, Mylosar
- Number of patients with grade 3 through grade 5 new neurological adverse events that are related to study drug, graded according to NCI CTCAE Version 4.0 [ Time Frame: 4 months ]New neurological deficit defined as new cranial neuropathy, nystagmus, change in mental status, motor deficit or cerebellar finding (ataxia, dysmetria, dysdiadochokinesis) that is attributed by treating physicians to intraventricular 5-Azacytidine infusions. Primary endpoint for basis of safety monitoring is acute toxicity occurring at any time within 30 days of receiving the intraventricular 5-Azacytidine infusion. Rate of acute toxicity monitored using Bayesian method of Thall and Sung. Method of Kaplan and Meier used to estimate unadjusted distributions o time to a neurological deficit and progression free survival time and summary statistic of all variable computed and tabulated.
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|Ages Eligible for Study:||1 Year to 21 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Diagnosis: Patients with histologically verified ependymoma, with recurrence or progression involving anywhere in the brain and/or spine. To be eligible, patients' disease must have originated in the posterior fossa of the brain.
- Patient must have either measurable or evaluable tumor as assessed by MRI of the brain and total spine.
- An implanted catheter in the fourth ventricle or posterior fossa tumor cavity attached to a ventricular access device or agreement to have one placed.
- A minimum of 7 days between last dose of systemic chemotherapy and/or radiation therapy and first infusion of 5-Azacytidine into fourth ventricle.
- Life expectancy of at least 12 weeks in the opinion of the PI
- Lansky score of 50 or greater if ≤16 years of age or Karnofsky score of 50 or greater if > 16 years of age.
- Existing neurological deficits must have been stable for a minimum of 1 week prior to study enrollment.
- Patients must have recovered from the acute toxic effects of all prior anticancer chemotherapy
- Adequate bone marrow function defined by peripheral absolute neutrophil count (ANC) ≥ 500/µL, platelet count ≥ 50,000/ µL (transfusion independent), and hemoglobin ≥9.0 gm/dL (may receive RBC transfusions)
- Patient or patient's legal representative, parent(s), or guardian able to provide written informed consent.
- Enrolled in another treatment protocol
- Has received another investigational or chemotherapy agent or radiation therapy within 7 days prior to 5-Azacytidine infusion into the fourth ventricle.
- Evidence of untreated infection
- Pregnant of lactating women
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02940483
|United States, Texas|
|UTHealth & Children's Memorial Hermann Hospital|
|Houston, Texas, United States, 77030|
|Principal Investigator:||David I Sandberg, M.D.||UTHealth|
|Responsible Party:||David Ilan Sandberg, Director of Pediatric Neurosurgery, Professor Pediatric Surgery and Neurosurgery, The University of Texas Health Science Center, Houston|
|Other Study ID Numbers:||
|First Posted:||October 21, 2016 Key Record Dates|
|Last Update Posted:||November 30, 2018|
|Last Verified:||November 2018|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Molecular Mechanisms of Pharmacological Action