Panoramica

Idoneità

Dettagli del Disegno

Bracci e Interventi

Misure di Esito

Siti dello Studio

Fonte: NCT04696029

Reclutamento in corso

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

Interventistico

Fase 2

Stanford University

Sponsor: Giselle Sholler

Ultimo Aggiornamento: 28 aprile 2026

Avviso - Le informazioni provengono da registri pubblici e potrebbero non riflettere i cambiamenti in tempo reale presso il centro locale.

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.

Mostra Descrizione Dettagliata

Categoria di Trattamento

Altri Farmaci

Mutazioni richieste

Nessuno

Studio Iniziato

29 marzo 2021

Ultimo Aggiornamento dello Sponsor

28 aprile 2026

Completamento Stimato

1 marzo 2028

Contatto dello studio

Nome:BCC Enroll

Email:BCCEnroll@pennstatehealth.psu.edu

Telefono:7175310003

Idoneità

Criteri di Inclusione

1. Age: 0-21 years of age at diagnosis

2. 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

3. 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.

4. 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.

5. 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.

6. 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.

7. 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.

8. 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

9. 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.

10. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or patients' legal representative).

Criteri di Esclusione

1. BSA of <0.25 m2

2. Metastatic disease outside of CNS

3. Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment

4. Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.

5. 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.

6. 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.

7. 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.

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Dettagli del Disegno

AllocazioneN/A

Modello di InterventoSingle Group Assignment

MascheramentoNone (Open Label)

Numero da Arruolare118

Bracci e Interventi

Bracci

Interventi

Tipo: Experimental

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

Interventi:

Difluoromethylornithine

Misure di Esito

Misure di Esito Primarie

Number of participants with event free survival (EFS) during study2 years plus 5 years follow up

o To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in preventing relapse in patients with molecular high risk and very high risk medulloblastoma, and relapsed/refractory medulloblastoma based upon the 2-year progression-free survival rate (PFS) compared to relevant historical controls.

Misure di Esito Secondarie

Length of time that participants experience Overall Survival (OS)7 years

o To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in patients with molecular high risk and very high risk medulloblastoma, and relapsed/refractory medulloblastoma based upon overall survival

Determine the Overall Response Rate (ORR) of Participants using Modified RANO Criteria2 years

To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in patients with molecular high risk and very high risk medulloblastoma, and relapsed/refractory medulloblastoma based upon Response Rate for patients with non-bulky residual disease present.

Number of Participants with Adverse Events as a Measure of Safety and Tolerability2 years plus 30 days

To develop a complete safety and tolerability profile of difluoromethylornithine (DFMO) in pediatric and young adult subjects with medulloblastoma.

Determine amount of DFMO in the CSF at 3 hours post dose2 years

o To measure CSF penetration after DFMO administration in pediatric subjects with medulloblastoma

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Siti dello Studio

Questo studio ha 24 siti

Stanford University

Stanford, California, US

Nome:Stefania U Chirita

Email:schirita@stanford.edu

In fase di reclutamento

Stanford, California, 94305 US

Stanford University

In fase di reclutamento

Hackensack, New Jersey, 07601 US

Hackensack University Medical Center

In fase di reclutamento

Charleston, South Carolina, 29425 US

Medical University of South Carolina

In fase di reclutamento

Oklahoma City, Oklahoma, 73104 US

University of Oklahoma

In fase di reclutamento

Miami, Florida, 33155 US

Nicklaus Children's Hospital

In fase di reclutamento

Orlando, Florida, 32806 US

Arnold Palmer Hospital for Children

In fase di reclutamento

Kansas City, Missouri, 64108 US

Children's Mercy Hospitals and Clinics

In fase di reclutamento

Hartford, Connecticut, 06106 US

Connecticut Children's Hospital

In fase di reclutamento

San Diego, California, 92123 US

Rady Children's Hospital

In fase di reclutamento

Little Rock, Arkansas, 72202 US

Arkansas Children's Hospital

In fase di reclutamento

Honolulu, Hawaii, 96813 US

Kapiolani Medical Center for Women and Children

In fase di reclutamento

Charlotte, North Carolina, 28204 US

Levine Children's Hospital

In fase di reclutamento

Hershey, Pennsylvania, 17033 US

Penn State Milton S. Hershey Medical Center and Children's Hospital

In fase di reclutamento

Austin, Texas, 78723 US

Dell Children's Blood and Cancer Center

In fase di reclutamento

Providence, Rhode Island, 02903 US

Hasbro Children's Hospital

In fase di reclutamento

Oakland, California, 94609 US

UCSF Benioff Children's Hospital Oakland

In fase di reclutamento

Tampa, Florida, 33607 US

St. Joseph's Children's Hospital

In fase di reclutamento

Portland, Oregon, 97227 US

Randall Children's Hospital

In fase di reclutamento

Lexington, Kentucky, 40502 US

Kentucky Children's Hospital

In fase di reclutamento

St Louis, Missouri, 63104 US

Cardinal Glennon Children's Medical Center

In fase di reclutamento

St. Petersburg, Florida, 33701 US

All Children's Hospital Johns Hopkins Medicine

In fase di reclutamento

Chicago, Illinois, 60453 US

Advocate Aurora Research Institute

In fase di reclutamento

Louisville, Kentucky, 40202 US

Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine

In fase di reclutamento

Québec, Quebec, QC G1V 4W6 CA

CHUQ

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