Идёт набор NCT04696029

DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma

Фаза II С лечением Medulloblastoma

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Difluoromethylornithine.
Кому может быть актуально: Состояния в реестре: Medulloblastoma. Базовые параметры: до 21 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США, Канада
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

Обзор

Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma.

Подробное описание

In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 2500 mg/m2 BID on each day of study.

Subjects will be evaluated in 3 Cohorts:

Cohort 1: Molecular High Risk Medulloblastoma Cohort 2: Molecular Very High Risk Medulloblastoma Cohort 3: Relapsed/Refractory Medulloblastoma

A total of 118 subjects across all cohorts will be enrolled to ensure that there will be 107 evaluable subjects (32-39 per cohort)

Вмешательства

Препарат Difluoromethylornithine
DFMO (difluoromethylornithine is an inhibitor of ornithine decarboxylase (ODC) designated chemically as 2-(difluoromethyl)-DL-ornithine monohydrochloride monohydrate. The dosage form to be used in this study is provided as a convex tablet containing 192 mg eflornithine (equivalent to 250 mg of eflornithine HCl, monohydrate). The tablets are packaged and sealed in opaque white HDPE bottles, and each bottle contains 100 tablets. The DMFO tablets are supplied by USWorldMeds (USWM). The tablets ar

Первичные конечные точки

Number of participants with event free survival (EFS) during study [Срок оценки: 2 years plus 5 years follow up]

Вторичные конечные точки (4)

Length of time that participants experience Overall Survival (OS) [Срок оценки: 7 years]
Determine the Overall Response Rate (ORR) of Participants using Modified RANO Criteria [Срок оценки: 2 years]
Number of Participants with Adverse Events as a Measure of Safety and Tolerability [Срок оценки: 2 years plus 30 days]
Determine amount of DFMO in the CSF at 3 hours post dose [Срок оценки: 2 years]

Критерии участия

Критерии включения

Age: 0-21 years of age at diagnosis
Pathology All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either Nanostring or methylation profiling.

Cohort 1- Molecular High Risk:

Metastatic non-MYC amplified Group 3
Metastatic Group 4
Metastatic non-WNT/non-SHH (Must be non-MYC amplified)

Cohort 2- Molecular Very High Risk

Metastatic OR MYCN amplified OR TP53 mutant non-infant (>3 yrs) SHH
MYC amplified Group 3
Non-WNT, non-SHH infant (< 3 yrs)

Cohort 3: Relapsed/Refractory Medulloblastoma

Pre-enrollment tumor survey:

Prior to enrollment on this study, a determination of mandatory disease staging must be performed:

Tumor imaging studies including: Brain and spine MRI
Lumbar Puncture only if previously positive
Bone Marrow aspiration/biopsy only if previously positive
This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to first dose of study drug, but must be done within a maximum of 4 weeks before first dose of study drug.
Disease Status: Subjects must have no evidence of disease, or stable\* residual nonbulky\*\* disease.

\*Stable residual disease defined as non-progression over 2 separate imaging studies at least 6 weeks apart

\*\*Non-bulky disease defined as maximal cross-sectional area < 3cm\^2 at enrollment. Patients with leptomeningeal disease are allowed to participate on study.

Timing from prior therapy:

Enrollment (first dose of DFMO) no later than 60 days after last dose of conventional chemotherapy. Patients who have undergone high dose chemotherapy (HDCT) with autologous stem cell transplantation (SCT) are eligible if more than 45 days have elapsed since date of last SCT.

Patients must have a Lansky or Karnofsky Performance Scale score of ≥ 50% (see Appendix II) and patients must have a life expectancy of ≥ 2 months.
All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to first dose of study drug unless otherwise indicated below.
Patients must have adequate organ functions at the time of registration:

Hematological: Hematological recovery as defined by ANC ≥750/μL, platelets ≥30 (non-transfused x 7 days)
Liver: Adequate liver function as defined by AST and ALT <10x upper limit of normal
Renal: Adequate renal function defined as (perform one of the following): Creatinine clearance or radioisotope GFR ≥ 70 mL/min/1.73 m2 or a serum creatinine based on age/gender
Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding.
Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or patients' legal representative).

Критерии исключения

BSA of <0.25 m2
Metastatic disease outside of CNS
Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет