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MEPENDAX: Phase I/II study of axitinib (Inlyta®) and oral metronomic etoposide for pediatric children and AYA with refractory/relapsing medulloblastoma and ependymoma
EU CTIS ID: 2023-509585-38-00
What this study is testing
First stage (Escalation part): to determine the MTD of axitinib when combined to a fixed dose of oral metronomic etoposide Second stage (Extension part): to estimate the efficacy of axitinib when used at the dose determined during the escalating part of the study, in combination with the fixed dose of oral metronomic etoposide in terms of progression-free survival (PFS), in children and AYA with refractory / relapsing medulloblastoma or ependymoma.
- Phase I and Phase II (Integrated)- Other
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Histologically proven diagnosis of ependymoma or medulloblastoma.
- Able to take oral treatments
- Adequate organ function: Hematologic criteria: - Peripheral absolute neutrophil count (ANC) ≥ 1000/mm3 (unsupported) - Platelet count ≥ 100,000/mm3 (unsupported) - Hemoglobin ≥ 8.0 g/dL (transfusion is allowed) Cardiac function : - Shortening fraction (SF) >29% and left ventricular ejection fraction (LVEF) ≥50% at baseline, as determined by echocardiography (mandatory only for patients who have received cardiotoxic therapy). Renal and hepatic function: - Serum creatinine < 1.5 x upper limit of normal (ULN) for age - Total bilirubin < 1.5 x ULN - Alanine aminotransferase (ALT)/ Aspartate aminotransferase (AST)/ < 2.5 x ULN
- Able to comply with scheduled follow-up and with management of toxicity.
- Females of child bearing potential must have a negative serum pregnancy test within 7 days prior to initiation of treatment.
- Sexually active patients must agree to use adequate and appropriate contraception (in accordance with Clinical Trials Facilitation and Coordination Group (CTFG) recommendations) while on study drug and for 6 months after stopping the study drug.
You likely can't join if
- Chemotherapy within 21 days of day 1 from the start of study treatment. This period can be shortened in the case of treatment with vincristine (2 weeks) and extended to 6 weeks in the case of treatment with nitrosureas. The period is set to 5 half-lives in the case of targeted therapies or metronomic chemotherapy. The period is set to 2 weeks after bevacizumab administration.
- Bleeding disorder
- Inability to undergo medical monitoring of the trial for geographic, social or psychological reasons Known hypersensitivity to any study drug or component of the formulation.
- Hypersensitivity to any study drug or component of the formulation.
- Absence of effective contraception in patients of childbearing age
- Pregnant or nursing (lactating) females.
See the full eligibility criteria
Who can join
- Histologically proven diagnosis of ependymoma or medulloblastoma.
- Able to take oral treatments
- Adequate organ function: Hematologic criteria: - Peripheral absolute neutrophil count (ANC) ≥ 1000/mm3 (unsupported) - Platelet count ≥ 100,000/mm3 (unsupported) - Hemoglobin ≥ 8.0 g/dL (transfusion is allowed) Cardiac function : - Shortening fraction (SF) >29% and left ventricular ejection fraction (LVEF) ≥50% at baseline, as determined by echocardiography (mandatory only for patients who have received cardiotoxic therapy). Renal and hepatic function: - Serum creatinine < 1.5 x upper limit of normal (ULN) for age - Total bilirubin < 1.5 x ULN - Alanine aminotransferase (ALT)/ Aspartate aminotransferase (AST)/ < 2.5 x ULN
- Able to comply with scheduled follow-up and with management of toxicity.
- Females of child bearing potential must have a negative serum pregnancy test within 7 days prior to initiation of treatment.
- Sexually active patients must agree to use adequate and appropriate contraception (in accordance with Clinical Trials Facilitation and Coordination Group (CTFG) recommendations) while on study drug and for 6 months after stopping the study drug.
- Patient able to comfortably swallow capsules.
- Written informed consent from parents/legal representative, patient, and age-appropriate assent before any study-specific screening procedures are conducted according to local, regional or national guidelines.
- Patient affiliated to a social security regimen or beneficiary of the same according to local requirements.
- Methyloma classification performed or available material for methyloma analysis
- Confirmed progressive or refractory disease despite standard therapy, or for which no effective standard therapy exists
- Male and female subjects with > 4 to ≤ 25 years of age at inclusion
- Weight > 20 kg
- Evaluable target lesion(s) according to RAPNO
- Performance status: Karnofsky performance status (for patients >12 years of age) or Lansky Play score (for patients ≤12 years of age) ≥ 70%. Patients who are unable to walk because of paralysis or stable neurological disability, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
- Life expectancy ≥ 3 months
- No known allergy to any of the compounds in the experimental treatment
What rules you out
- Chemotherapy within 21 days of day 1 from the start of study treatment. This period can be shortened in the case of treatment with vincristine (2 weeks) and extended to 6 weeks in the case of treatment with nitrosureas. The period is set to 5 half-lives in the case of targeted therapies or metronomic chemotherapy. The period is set to 2 weeks after bevacizumab administration.
- Bleeding disorder
- Inability to undergo medical monitoring of the trial for geographic, social or psychological reasons Known hypersensitivity to any study drug or component of the formulation.
- Hypersensitivity to any study drug or component of the formulation.
- Absence of effective contraception in patients of childbearing age
- Pregnant or nursing (lactating) females.
- Evidence of > Grade 1 recent CNS hemorrhage on the baseline MRI scan
- Impairment of gastrointestinal (GI) function or GI disease that may significantly alter drug absorption of oral drugs (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, or malabsorption syndrome).
- Clinically significant, uncontrolled heart disease (including history of any cardiac arrhythmias, e.g., ventricular, supraventricular, nodal arrhythmias, or conduction abnormality within 12 months of screening)
- Known active viral hepatitis or known human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
- Known congenital immune-deficiency
- Presence of any NCI-CTCAE v5 grade ≥ 2 treatment-related extra-hematological toxicity with the exception of alopecia, ototoxicity or peripheral neuropathy.
- Radiotherapy within the 2 months preceding D1 of the start of study treatment. Palliative RT on a non-target lesion is allowed up to 1 weeks before beginning of treatment.
- Invasive procedure/surgery during the last 15 days.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.