Neuroblastoma Maintenance Therapy Trial

NCT02679144 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: NMTRC014
• Study Type: interventional
• Target: 441
• Locations: 2 countries
• Sites: 49

Brief Summary

Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence.

Conditions

Neuroblastoma

Outcome Measures

Primary Outcomes (1)

• Number of participants with event free survival (EFS) during study.

  2 years

Secondary Outcomes (6)

• Length of time that participants experience Overall Survival (OS)

  7 years

• Number of Participants with Adverse Events as a Measure of Safety and Tolerability

  2 years

• Peak Plasma Concentration (Cmax)

  1 year

• Area under the plasma concentration versus time curve (AUC)

  1 year

• Time to reach Peak Plasma Concentration (Tmax)

  1 year

Study Arms (1)

Difluoromethylornithine (DFMO)

EXPERIMENTAL

Subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study.

Drug: Difluoromethylornithine (DFMO)

Interventions

Difluoromethylornithine (DFMO) DRUG

Subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study.

Also known as: eflornithine

Difluoromethylornithine (DFMO)

Eligibility Criteria

• Age: 1 Year - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• All patients must have a pathologically confirmed diagnosis of neuroblastoma, \< 30.99 years of age and classified as high risk at the time of diagnosis. Exception: patients who are initially diagnosed as non-high-risk neuroblastoma, but later converted (and/or relapsed) to high risk neuroblastoma are also eligible.
• All patients must be in complete remission (CR):
• No evidence of residual disease on scan
• No evidence of disease metastatic to bone marrow.
• Specific Criteria by Stratum:
• Stratum 1/1B: All patients must have completed standard upfront therapy that replicates treatment which patients who were enrolled on ANBL0032 received, including:
• intensive induction chemotherapy and (if feasible) resection of primary tumor, followed by: consolidation with high-dose chemotherapy with stem cell transplant and radiotherapy, followed by: immunotherapy with Ch14.18/IL-2/GM-CSF (dinutuximab) and retinoic acid;.
• All subjects on Stratum 1/B must have also met the following criteria:
• A pre-transplant disease status evaluation that met International Neuroblastoma Response Criteria (INRC) for CR (complete response), VGPR (very good partial response), or PR (partial response) for primary site, soft tissue metastases and bone metastases. Patients who meet those criteria must also meet the protocol-specified criteria for bone marrow response prior to transplant as outlined below: No more than 10% tumor involvement (based on total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy.
• Stratum 2: Neuroblastoma that is in first complete remission following standard upfront therapy different from that described for Stratum 1.
• Stratum 3: Neuroblastoma that failed to have a response of at least PR following induction chemotherapy and surgical resection of the primary tumor, but that has achieved CR following additional therapy.
• Stratum 4: Patients who have achieved a second or subsequent CR following relapse(s).
• Pre-enrollment tumor survey: Prior to enrollment on this study, a determination of mandatory disease staging must be performed:
• Tumor imaging studies including
• Bilateral bone marrow aspirates and biopsy

You may not qualify if:

• BSA (Body Surface Area) of \<0.25 m2.
• 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 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.

Sponsors & Collaborators

• Giselle Sholler: lead
• Beat NB Cancer Foundation: collaborator
• Team Parker for Life: collaborator

Study Sites (49)

University of Alabama, Children's of Alabama

Birmingham, Alabama, 35233, United States

RECRUITING

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

UCSF Benioff Children's Hospital Oakland-

Oakland, California, 94609, United States

RECRUITING

Rady Children's Hospital

San Diego, California, 92123, United States

RECRUITING

Rocky Mountain Pediatric Hematology

Denver, Colorado, 80205, United States

RECRUITING

Connecticut Children's Hospital

Hartford, Connecticut, 06106, United States

RECRUITING

University of Florida

Gainesville, Florida, 32611, United States

RECRUITING

Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States

RECRUITING

All Children's Hospital Johns Hopkins Medicine

St. Petersburg, Florida, 33701, United States

RECRUITING

St. Joseph's Children's Hospital

Tampa, Florida, 33614, United States

RECRUITING

Augusta University Health

Augusta, Georgia, 30912, United States

RECRUITING

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96813, United States

RECRUITING

St. Lukes

Boise, Idaho, 83712, United States

COMPLETED

Advocate Aurora Research Institute

Chicago, Illinois, 60453, United States

RECRUITING

University of Iowa

Iowa City, Iowa, 52242, United States

RECRUITING

Kentucky Children's Hospital

Lexington, Kentucky, 40536, United States

RECRUITING

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

Louisville, Kentucky, 40202, United States

RECRUITING

Tufts Medical Center

Boston, Massachusetts, 02111, United States

COMPLETED

University of Massachusetts Medical School Worcester

Worcester, Massachusetts, 01655, United States

WITHDRAWN

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

RECRUITING

Children's Hospital and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States

RECRUITING

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States

RECRUITING

Cardinal Glennon Children's Medical Center

St Louis, Missouri, 63104, United States

RECRUITING

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

RECRUITING

NYU Langone Health Hassenfeld Children's Hospital

New York, New York, 10016, United States

RECRUITING

The Children's Hospital at Montefiore

The Bronx, New York, 10467, United States

COMPLETED

Levine Children's Hospital

Charlotte, North Carolina, 28204, United States

RECRUITING

Duke University

Durham, North Carolina, 27708, United States

RECRUITING

Cleveland Clinic Children's

Cleveland, Ohio, 44106, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

ACTIVE NOT RECRUITING

Randall Children's Hospital

Portland, Oregon, 97227, United States

RECRUITING

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

Hershey, Pennsylvania, 17033, United States

RECRUITING

Hasbro Children's Hospital

Providence, Rhode Island, 02903, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

Monroe Carrell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, 37232, United States

RECRUITING

Dell Children's Blood and Cancer Center

Austin, Texas, 78723, United States

RECRUITING

Children's Medical Center

Dallas, Texas, 75235, United States

RECRUITING

Texas Children's Cancer and Hematology Centers

Houston, Texas, 77030, United States

ACTIVE NOT RECRUITING

Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

RECRUITING

Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23284, United States

RECRUITING

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

Alberta Children's Hospital

Calgary, Alberta, AB T3B 6A8, Canada

RECRUITING

CancerCare Manitoba

Winnipeg, Manitoba, MB R3E 0V9, Canada

RECRUITING

Janesway Children's Health and Rehabilitation Centre

St. John's, Newfoundland and Labrador, NL A1B 3V6, Canada

RECRUITING

UHC Sainte-Justine

Montreal, Quebec, QC H3S 2G4, Canada

RECRUITING

Montreal Children's Hospital

Montreal, Quebec, QC H4A 3H9, Canada

RECRUITING

CHUQ

Québec, Quebec, QC G1V 4W6, Canada

RECRUITING

CIUSSS de l'Estrie-CHUS

Sherbrooke, Quebec, QC J1H 5H3, Canada

RECRUITING

Related Publications (1)

• Duke ES, Bradford D, Sinha AK, Mishra-Kalyani PS, Lerro CC, Rivera D, Wearne E, Miller CP, Leighton J, Sabit H, Zhao H, Lane A, Scepura B, Pazdur R, Singh H, Kluetz PG, Donoghue M, Drezner N. US Food and Drug Administration Approval Summary: Eflornithine for High-Risk Neuroblastoma After Prior Multiagent, Multimodality Therapy. J Clin Oncol. 2024 Sep 1;42(25):3047-3057. doi: 10.1200/JCO.24.00546. Epub 2024 Jun 25.

  PMID: 38917371 DERIVED

Related Links

• Beat Childhood Cancer Consortium Website

MeSH Terms

Conditions

Neuroblastoma

Interventions

Eflornithine

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Ornithine; Amino Acids, Basic; Amino Acids; Amino Acids, Peptides, and Proteins; Amino Acids, Diamino

Study Officials

• Giselle Sholler, MD

  Beat Childhood Cancer at Atrium Health

  STUDY CHAIR

Central Study Contacts

BCC Enroll

CONTACT

7175310003; BCCEnroll@pennstatehealth.psu.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Beat Childhood Cancer Chair

Study Record Dates

• First Submitted: February 4, 2016
• First Posted: February 10, 2016
• Study Start: February 1, 2016
• Primary Completion (Estimated): February 1, 2028
• Study Completion (Estimated): February 1, 2033
• Last Updated: January 21, 2026

Record last verified: 2026-01

Locations

US (42); CA (7)