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Alkylating agents

Chemotherapy + Stem Cell Transplant for Neuroblastoma

Phase 1

Waitlist Available

Led By Mary Meaghan Granger, MD

Research Sponsored by Children's Oncology Group

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites

Patients must have no known contraindications to peripheral blood stem cell collection

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Study Summary

This trial studies busulfan, melphalan, and stem cell transplant after chemotherapy in treating patients with newly diagnosed neuroblastoma.

Who is the study for?

This trial is for children and young adults with high-risk neuroblastoma, a type of cancer. Eligible participants include those with certain genetic features or stages of the disease, without prior extensive treatment. They must have proper kidney function, normal bilirubin levels, acceptable heart function, and no issues that would prevent stem cell collection.Check my eligibility

What is being tested?

The study tests a combination of chemotherapy drugs (Busulfan, Melphalan) followed by a stem cell transplant to treat newly diagnosed high-risk neuroblastoma. The goal is to stop tumor growth before transplanting the patient's own previously collected healthy stem cells to restore blood-forming cells destroyed by chemo.See study design

What are the potential side effects?

Potential side effects may include damage to organs from chemotherapy drugs like Busulfan and Melphalan; low blood counts; risk of infection; mouth sores; nausea and vomiting due to intense chemo; and complications related to stem cell transplantation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with neuroblastoma or ganglioneuroblastoma.

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I can safely undergo a procedure to collect stem cells from my blood.

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My kidney function tests are within the required range for my age and gender.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

The Tolerability of BuMel Regimen

Other outcome measures

EFS

First Dose Area Under the Curve (AUC) and Average Daily AUC for Busulfan

Incidence of Non-hematologic Organ Toxicity (Grade 3 and Higher) and All Cause Mortality Graded According to CTC v4.0

+7 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (induction therapy, consolidation therapy, ASCT)Experimental Treatment15 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cisplatin

2013

Completed Phase 3

~1940

Cyclophosphamide

1995

Completed Phase 3

~3780

Mesna

2003

Completed Phase 2

~1380

Peripheral Blood Stem Cell Transplantation

1997

Completed Phase 3

~1330

Filgrastim

2000

Completed Phase 3

~3670

Topotecan Hydrochloride

2013

Completed Phase 3

~6120

External Beam Radiation Therapy

2006

Completed Phase 3

~3070

Busulfan

2008

Completed Phase 3

~1120

Doxorubicin Hydrochloride

2019

Completed Phase 3

~17850

Melphalan

2008

Completed Phase 3

~1500

Etoposide

2010

Completed Phase 3

~2440

Autologous Hematopoietic Stem Cell Transplantation

2017

Completed Phase 3

~1730

Vincristine Sulfate

2005

Completed Phase 3

~10110

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Children's Oncology GroupLead Sponsor

453 Previous Clinical Trials

237,550 Total Patients Enrolled

5 Trials studying Ganglioneuroblastoma

11,478 Patients Enrolled for Ganglioneuroblastoma

National Cancer Institute (NCI)NIH

13,654 Previous Clinical Trials

40,933,003 Total Patients Enrolled

11 Trials studying Ganglioneuroblastoma

12,197 Patients Enrolled for Ganglioneuroblastoma

Mary Meaghan Granger, MDPrincipal InvestigatorChildren's Oncology Group

Media Library

Busulfan (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT01798004 — Phase 1

Ganglioneuroblastoma Clinical Trial 2023: Busulfan Highlights & Side Effects. Trial Name: NCT01798004 — Phase 1

Busulfan (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01798004 — Phase 1

Ganglioneuroblastoma Research Study Groups: Treatment (induction therapy, consolidation therapy, ASCT)

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

To what extent has this investigation been implemented in medical facilities?

"The trial is actively enrolling at Children's Specialty Center of Nevada II in Las Vegas, Providence Sacred Heart Medical Center and Children's Hospital in Spokane, Washington, The Toledo Hospital/Toledo Children's Hospital located in Toledo Ohio plus 97 other medical centers."

Answered by AI

Are there any other pharmaceutical experiments that have been conducted to investigate this issue?

"Initially conducted in 1997 at Spectrum Health Hospital - Butterworth Campus, this pharmacological study has since been the subject of 3762 completed clinical trials. Currently, 1859 medical researchers are actively recruiting patients for a vast number of studies primarily located in Las Vegas, Nevada."

Answered by AI

How many participants is this clinical trial recruiting?

"At the moment, this experiment is not taking on new candidates. It was initially posted on April 8th 2013 and last modified March 3rd 2022. If you are keen to explore other studies, 162 clinical trials for neuroblastoma and 1859 pharmacological experiments are currently seeking participants."

Answered by AI

Is there still capacity for enrolment in this clinical experiment?

"The clinical trial has ceased recruitment, as evidenced by the information posted on clinicaltrials.gov; which indicates that it was first listed April 8th 2013 and last edited March 3rd 2022. However, there are 2021 additional trials actively seeking to enrol participants."

Answered by AI

Is this trial the inaugural of its kind?

"Pharmacological study has been studied for over two decades, with the earliest research being sponsored by Alfacell in 1997. This drug was granted Phase 3 approval after trials on 300 patients, and currently there are 1859 live studies of this medication occurring across 81 nations."

Answered by AI

What symptoms are generally targeted by pharmacological research?

"Pharmacological studies are frequently utilized to combat merkel cell cancer, as well as other diseases such as leukemia, prostate cancer and lung cancers."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2013. "Busulfan, Melphalan, and Stem Cell Transplant After Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01798004.

All > Stem Cell Transplant > Neuroblastoma