New treatment for children with recurrent brain tumors
Official title: A Phase II Study of Metronomic and Targeted Anti-angiogenesis Therapy for Children With Recurrent/Progressive Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors
Run by Medical University of Vienna · NCT01356290 · Data updated 2026-11-12
This study is testing a new treatment combining an intravenous drug with five oral medications to see if it can help children with recurring brain tumors live longer and feel better.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Ages | Any age to 19 Years |
| Sex | All |
| Enrollment | 232 (estimated) |
| Sponsor | Medical University of Vienna Academic / other |
| Drugs / interventions | cyclophosphamide, bevacizumab, chemotherapy |
| Locations | 22 sites (Chicago, Illinois and 21 other locations) |
| Status | Recruiting |
| Started | April 2014 |
| Main results expected | April 2030 |
| Study ends | April 2030 |
| Registry record updated | October 13, 2026 |
| Trial ID | NCT01356290 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are children aged 0 to 19 years with relapsed or progressive medulloblastoma, ependymoma, or ATRT.
✕Probably not a fit
Patients with active infections, VP-shunt dependency, or those who are pregnant or breastfeeding may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This clinical trial evaluates the effectiveness of antiangiogenic therapy using biweekly intravenous bevacizumab combined with five oral medications for children with relapsed medulloblastoma, ependymoma, and ATRT. The approach aims to target both tumor and endothelial cells while minimizing toxicity through a metronomic schedule. The study seeks to provide alternative treatment options for these patients, who currently have limited curative therapies available, with the goal of prolonging survival and maintaining a good quality of life.
Why it matters
Potential benefit: If successful, this treatment could significantly improve survival rates and quality of life for children with recurrent brain tumors.
How similar studies have gone: Other studies have shown promise with antiangiogenic therapies in treating solid tumors, suggesting potential for success in this approach.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion criteria for patients * Stratum I: Relapsed or progressive medulloblastoma - completed * Stratum II: Relapsed or progressive ependymoma (at least one site of untreated recurrent disease) * Stratum III: Relapsed or progressive ATRT (at least one site of untreated recurrent disease) * Stratum IV: Relapsed or progressive medulloblastoma (at least one site of untreated recurrent disease) * Stratum V: Relapsed or progressive CNS tumor of various histologies or patients with exclusion criteria or adult patients (explorative) * Histological confirmation at diagnosis or relapse Stratum IV: Confirmation of the medulloblastoma group by methylation; IDAT (Intensity Data; raw data of methylation array) * Female or male, aged from 0 to \<20 years (at time of original diagnosis) * Participants must have normal organ and bone marrow function (ALT \<5x institutional upper limit of normal, creatinine \<1.5x institutional upper limit of normal for age, WBC \>1000/mm3, platelets \> 20,000/mm3. Patients with values less than WBC 2000/mm3 or platelets 50,000/mm3 will require initiation of treatment with etoposide and cyclophosphamide at a lower starting dose as defined within the protocol * Karnofsky performance status ≥50. For infants and children less than 12 years of age, the Lansky play scale ≥50% will be used * Written informed consent of patients and / or legal guardian Exclusion criteria for patients * VP- or subdural peritoneal shunt dependency (can be included in Stratum V) * Prior treatment with temozolomide/irinotecan (can be included in Stratum V) * Active infection, pregnancy or breast feeding * Treatment for current relapse (surgery may be performed before MEMMAT treatment; patients with sites of disease not irradiated are still eligible for the protocol) * Known hypersensitivity to any of the drugs in the protocol * Active peptic ulcer * Any significant cardiovascular disease not controlled by standard therapy e.g. systemic hypertension * Anticipation of the need for major elective surgery during the course of the study treatment * Any disease or condition that contraindicates the use of the study medication/treatment or places the patient at an unacceptable risk of experiencing treatment-related complications * Non-healing surgical wound * A bone fracture that has not satisfactorily healed
Where it's running
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Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States Recruiting -
Dell Children's Medical Group SFC-HEM/ONC
Austin, Texas, United States Recruiting -
Medical University of Graz
Graz, Austria Recruiting -
Medical University of Innsbruck
Innsbruck, Austria Recruiting -
Kepler Universitätsklinikum Med Campus IV
Linz, Austria Recruiting -
Salzburger Universitätsklinikum
Salzburg, Austria Recruiting
Show 16 more locations
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Medical University of Vienna
Vienna, Austria Recruiting -
University Hospital Brno
Brno, Czechia Recruiting -
University hospital Rigshospitalet
Copenhagen, Denmark Recruiting -
Centre Léon Bérard
Lyon, France Recruiting -
Onkologisk-hematologisk seksjon Barneklinikken Haukeland universitetssjukehus
Bergen, Bergen, Norway Recruiting -
Hospital Infantil Universitario Nino Jesus
Madrid, Spain Recruiting -
Sahlgrenska Universitetssjukhuset
Gothenburg, Sweden Recruiting -
Universitetssjukhuset Linköping
Linköping, Sweden Recruiting -
Skånes universitetssjukhus
Lund, Sweden Recruiting -
Karolinska University Hospital
Stockholm, Sweden Recruiting -
Norrlands Universitetssjukhus
Umeå, Sweden Recruiting -
Akademiska sjukhuset
Uppsala, Sweden Recruiting -
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States Terminated -
Dana-Farber Cancer Institute and Boston Children's Hospital
Boston, Massachusetts, United States Terminated -
Motol University Hospital Prague
Prague, Czechia Withdrawn -
Centre Oscar Lambret
Lille, France Terminated
Study contacts
- Principal investigator: Andreas Peyrl, MD — Medical University of Vienna
- Study coordinator: Andreas Peyrl, MD
- Email: andreas.peyrl@meduniwien.ac.at
- Phone: +43 1 40400
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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TP53 gene therapy (SGT-53) with nivolumab and focused radiation for children with recurrent brain tumors
This study tests a TP53 gene therapy called SGT-53 combined with the immune drug nivolumab and focused radiation in children whose brain tumors have come back, grown, or not responded to prior treatment.
-
Neuro-navigation-guided focused ultrasound with nivolumab for relapsed or progressive high-grade brain tumors in children
This study tests whether using focused ultrasound to open the blood-brain barrier and give nivolumab is safe and feasible for kids with relapsed or progressive DMG/DIPG or other high-grade brain tumors.
-
Combining Lutathera® and Olaparib for treating recurrent solid tumors in children and adolescents
This study is testing if combining Lutathera® with olaparib can help children and teenagers with hard-to-treat solid tumors that have certain receptors.
-
B7-H3 CAR T cells for recurrent brain tumors in kids and young adults
This early-phase trial tests a personalized CAR T-cell therapy called B7-H3 CAR T for children and young adults with recurrent or progressive brain tumors.
-
Liothyronine with BIT chemotherapy for relapsed medulloblastoma in kids and young adults
This study tests Liothyronine (L-T3) with a BIT chemotherapy regimen in children and young adults whose medulloblastoma has come back or progressed after standard treatment.
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Registry for relapsed or progressive SHH medulloblastoma with U1 mutation
This registry collects medical records and biological samples from people aged 3–50 with relapsed or progressive SHH medulloblastoma to test for U1 mutations and create a biobank for research.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.