NCT01798004|CompletedPhase 1ResultsDMC

Busulfan, Melphalan, and Stem Cell Transplant After Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma

Pilot Study Using Myeloablative Busulfan/Melphalan (BuMel) Consolidation Following Induction Chemotherapy for Patients With Newly Diagnosed High-Risk Neuroblastoma

Children's Oncology Group ClinicalTrials.gov

Compare

5 other identifiers

ANBL12P1NCI-2012-02211COG-ANBL12P1U10CA180886U10CA098543

Study Type

interventional

Target

150

Locations

4 countries

Sites

123

Timeline

RegisteredFeb 2013

StartedApr 2013

CompletionJun 2024

Brief Summary

This pilot clinical trial studies busulfan, melphalan, and stem cell transplant after chemotherapy in treating patients with newly diagnosed neuroblastoma that is likely to come back or spread. Giving chemotherapy to the entire body before a stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood and stored. More chemotherapy or radiation therapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

150

participants targeted

Target at P75+ for phase_1

Timeline

Completed

Started Apr 2013

Longer than P75 for phase_1

Geographic Reach

4 countries

123 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

11.2 years study duration

First Submitted

Initial submission to the registry

January 6, 2013

Completed

2 months until next milestone

First Posted

Study publicly available on registry

February 25, 2013

Completed

1 month until next milestone

Study Start

First participant enrolled

April 9, 2013

Completed

2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 28, 2015

Completed

1.5 years until next milestone

Results Posted

Study results publicly available

February 9, 2017

Completed

7.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2024

Completed

Last Updated

July 15, 2024

Status Verified

July 1, 2024

Enrollment Period

2.3 years

First QC Date

January 6, 2013

Results QC Date

December 19, 2016

Last Update Submit

July 3, 2024

Conditions

Ganglioneuroblastoma Stage 1 Neuroblastoma Stage 2 Neuroblastoma Stage 2A Neuroblastoma Stage 2B Neuroblastoma Stage 3 Neuroblastoma Stage 4 Neuroblastoma Stage 4S Neuroblastoma

Outcome Measures

Primary Outcomes (1)

The Tolerability of BuMel Regimen
Number of patients who experience one or more unacceptable toxicities (severe sinusoidal obstruction syndrome \[SOS\] or Grade 4-5 pulmonary toxicity per Common Toxicity Criteria \[CTC\] v.4.0) during the Consolidation phase of therapy.
Up to 28 days post-consolidation therapy, up to 1 year

Other Outcomes (10)

Incidence of Non-hematologic Organ Toxicity (Grade 3 and Higher) and All Cause Mortality Graded According to CTC v4.0
Up to 180 days
Response Rate Determined Using the International Response Criteria
Up to 5 years
EFS
Up to 5 years
+7 more other outcomes

Study Arms (1)

Treatment (induction therapy, consolidation therapy, ASCT)

EXPERIMENTAL

INDUCTION THERAPY: COURSES 1-2: Patients receive cyclophosphamide IV over 15-30 minutes and topotecan hydrochloride IV over 30 minutes on days 1-5. Treatment repeats every 3 weeks for 2 courses. COURSES 3 AND 5: Patients receive cisplatin IV over 1 hour on days 1-4 and etoposide IV over 1-2 hours on days 1-3. Treatment repeats every 3 weeks for 2 courses. COURSE 4: Patients receive cyclophosphamide IV over 1-6 hours on days 1-2, vincristine sulfate IV over 1 minute on days 1-3, and doxorubicin hydrochloride IV over 24 hours on days 1-3. Treatment repeats every 3 weeks for 1 course. Treatment continues in the absence of disease progression or unacceptable toxicity. CONSOLIDATION THERAPY: Beginning 4-8 weeks following the 5th course of induction therapy, patients receive busulfan IV over 3 hours on days -6 to -3 and melphalan IV on day -1. Patients undergo ASCT on day 0. Some patients also undergo EBRT after induction and consolidation.

Procedure: Autologous Hematopoietic Stem Cell TransplantationDrug: BusulfanDrug: CisplatinDrug: CyclophosphamideDrug: Doxorubicin HydrochlorideDrug: EtoposideRadiation: External Beam Radiation TherapyBiological: FilgrastimOther: Laboratory Biomarker AnalysisDrug: MelphalanDrug: MesnaProcedure: Peripheral Blood Stem Cell TransplantationOther: Pharmacological StudyDrug: Topotecan HydrochlorideDrug: Vincristine Sulfate

Interventions

Autologous Hematopoietic Stem Cell TransplantationPROCEDURE

Undergo autologous peripheral blood stem cell transplant

Also known as: AHSCT, Autologous, Autologous Hematopoietic Cell Transplantation, Autologous Stem Cell Transplant, Autologous Stem Cell Transplantation, Stem Cell Transplantation, Autologous