NCT06485908Not yet recruitingPhase 1/2
Axitinib and Oral Metronomic Etoposide for Pediatric Children and AYA Refractory/Relapsing Medulloblastoma and Ependymoma
MEPENDAX: Phase I/II Study of Axitinib (Inlyta®) and Oral Metronomic Etoposide for Pediatric Children and AYA Refractory/Relapsing Medulloblastoma and Ependymoma
Source: ClinicalTrials.gov · Last updated 3 Jul 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
It is an open multicentric phase I/II trial with axitinib (Inlyta®) and metronomic delivery of etoposide for children, adolescent and young adults (AYA) with refractory/ relapsing solid tumors. It is a two-stage trial: First stage: To determine the Maximum Tolerated Dose (MTD) of the combination of axitinib and oral metronomic etoposide for patient with medulloblastoma or ependymoma Second stage: Extension cohort evaluating the preliminary efficacy at the recommended dose for the phase II (RDP2) of the combination. The 2nd stage will start after a meeting of independent data monitoring committee (IDMC). Two cohorts of 9 patients with ependymoma and medulloblastoma Patients treated at first stage won't be included in the second stage.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 40 (estimated)
- First submitted
- 25 Jun 2024
- Start date
- Oct 2024
- Primary completion date
- Apr 2026
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Diseases
- Childhood cancers
- NCT number
- NCT06485908
- EU CT number
- 2023-509585-38-00
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 40
- From Oct 2024 to the primary completion date, Apr 2026
- 7 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Who the study is looking for
- Histologically proven diagnosis of ependymoma or medulloblastoma
- 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
- 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.
- Non-inclusion Criteria
- 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. Evidence of \> Grade 1 recent CNS hemorrhage on the baseline MRI or 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.
- Presence of any NCI-CTCAE v5 grade ≥ 2 treatment-related extra-hematological toxicity with the exception of alopecia, ototoxicity or peripheral neuropathy.
- Known congenital immunodeficiency.
- 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.
- Major surgery within 21 days of the first dose. Gastrostomy, ventriculo-peritoneal shunt, endoscopic ventriculostomy, tumor biopsy and insertion of central venous access devices are not considered major surgery, but for these procedures, a 48 hour interval must be maintained before the first dose of the investigational drug is administered.
- Bleeding disorder.
- 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 hypersensitivity to any study drug or component of the formulation.
- Absence of effective contraception in patients of childbearing age (see appendix 3)
- Pregnant or nursing (lactating) females.
- Patients with galactose intolerance, lactase deficiency or glucose or galactose malabsorption syndrome (rare hereditary diseases).
- Severe infections requiring parenteral antibiotic therapy.
- Inability to undergo medical monitoring of the trial for geographic, social or psychological reasons.
Age in the registry: 4 years to 25 years · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Centre Hospitalier Universitaire D'Angers Coordinator of this site? Confirm its status | Angers | France | Recruiting |
| Centre hospitalier Universitaire de Bordeaux Coordinator of this site? Confirm its status | Bordeaux | France | Recruiting |
| Hospices Civils de Lyon Coordinator of this site? Confirm its status | Lyon | France | Recruiting |
| Centre Hospitalier Regional De Marseille Coordinator of this site? Confirm its status | Marseille | France | Recruiting |
| Institut Curie Coordinator of this site? Confirm its status | Paris | France | Recruiting |
| Les Hopitaux Universitaires De Strasbourg Coordinator of this site? Confirm its status | Strasbourg Cedex 2 | France | Recruiting |
| CHRU De Nancy Coordinator of this site? Confirm its status | Vandoeuvre Les Nancy | France | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.