Clinical trial · Interventional
Induction Chemotherapy Using Cyclophosphamide and Topotecan in Treating Patients Who Are Undergoing Autologous Peripheral Stem Cell Transplantation for Newly Diagnosed or Progressive Neuroblastoma
A Pilot Induction Regimen Incorporating Topotecan for Treatment of Newly Diagnosed High Risk Neuroblastoma
NCT00070200CI-TRIAL-00013754completedPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
RATIONALE: Drugs used in chemotherapy, such as topotecan and cyclophosphamide, use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. PURPOSE: This phase I trial is studying the side effects of induction chemotherapy using cyclophosphamide and topotecan in treating patients who are undergoing surgery and autologous stem cell transplantation followed by radiation therapy for newly diagnosed or progressive neuroblastoma.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (12)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| cisplatin | Drug | Cisplatin | ALIAS |
| conventional surgery | Procedure | — | UNRESOLVED |
| cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| doxorubicin hydrochloride | Drug | Doxorubicin | ALIAS |
| etoposide | Drug | Etoposide | ALIAS |
| filgrastim | Biological | Filgrastim | ALIAS |
| isotretinoin | Drug | — | UNRESOLVED |
| melphalan | Drug | Melphalan | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- All patients
- description
- Induction Cycles 1 and 2 (CT) (21 days each), Cyclophosphamide (Days 1 thru 5) weight based dosage (\> 12 kg 400 mg/m2/day, \< 12 kg 13.3 mg/kg/day, \< 2 years old N/A. Topotecan (Days 1 thru 5) weight based dosage (\> 12 kg 1.2 mg/m2/day, \< 12 kg 0.04 mg/kg/day, \< 2 years old 0.04 mg/kg/day). Filgrastim (Days 6 →) weight based dosage (\> 12 kg 5 micrograms/kg, \< 12 kg 5 micrograms /kg, \< 2 years old 5 micrograms /kg.
- interventionNames
- Biological: filgrastim
- Drug: cisplatin
- Drug: cyclophosphamide
- Drug: doxorubicin hydrochloride
- Drug: etoposide
- Drug: isotretinoin
- Drug: melphalan
- Drug: topotecan hydrochloride
- Drug: vincristine sulfate
- Procedure: conventional surgery
- Procedure: peripheral blood stem cell transplantation
- Radiation: radiation therapy
Primary outcomes (1)
- measure
- Proportion of patients who are classified as a "success"
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 30 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Histologically or cytologically confirmed neuroblastoma or ganglioneuroblastoma meeting 1 of the following staging criteria:
* Newly diagnosed disease, at least 1 year of age, and meets criteria for 1 of the following:
* International Neuroblastoma Staging System (INSS) stage 2a/2b with MYCN amplification (greater than 10) AND unfavorable pathology
* INSS stage 3 with MYCN amplification OR unfavorable pathology
* Newly diagnosed INSS stage 4 disease meeting criteria for 1 of the following:
* Over 18 months of age
* Age 12 to 18 months with any unfavorable biologic feature (MYCN amplification, unfavorable pathology, and/or DNA index=1) or any biologic feature that is indeterminant, unsatisfactory, or unknown
* No INSS stage 4 disease and age 12 to 18 months with all 3 favorable biologic features (i.e., nonamplified MYCN, favorable pathology, and DNA index greater than 1)
* Newly diagnosed INSS stage 3, 4, or 4S disease AND under 1 year of age with MYCN amplification
* At least 1 year of age and initially diagnosed with INSS stage 1, 2, or 4S disease that progressed to stage 4 without interval chemotherapy
* Must have been enrolled on COG-ANBL00B1 at initial diagnosis
PATIENT CHARACTERISTICS:
Age
* 30 and under at initial diagnosis
Performance status
* Not specified
Life expectancy
* Not specified
Hematopoietic
* Absolute neutrophil count at least 1,000/mm\^3\*
* Platelet count at least 100,000/mm\^3\* (transfusion independent)
* Hemoglobin at least 10.0 g/dL\* (red blood cell transfusions allowed) NOTE: \*Granulocytopenia, anemia, and/or thrombocytopenia allowed for patients with tumor metastatic to the bone marrow
Hepatic
* Bilirubin no greater than 1.5 mg/dL
* ALT less than 300 IU/L
Renal
* Creatinine no greater than 1.5 mg/dL
* Creatinine clearance or radioisotope glomerular filtration rate at least 60 mL/min
Cardiovascular
* ECG normal
* Shortening fraction at least 27% by echocardiogram OR
* Ejection fraction at least 50% by MUGA
Other
* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception
* HIV negative
PRIOR CONCURRENT THERAPY:
Biologic therapy
* Not specified
Chemotherapy
* See Disease Characteristics
* No more than 1 prior chemotherapy course on the low- or intermediate-risk neuroblastoma studies (COG-P9641, COG-A3961) prior to determination of MYCN amplification and Shimada histology
Endocrine therapy
* Not specified
Radiotherapy
* Prior localized emergency radiotherapy to sites of life-threatening or function-threatening disease allowed
Surgery
* Not specified
Other
* No other prior systemic therapyReferences
Publications (1)
- RESULTPark JR, Scott JR, Stewart CF, London WB, Naranjo A, Santana VM, Shaw PJ, Cohn SL, Matthay KK. Pilot induction regimen incorporating pharmacokinetically guided topotecan for treatment of newly diagnosed high-risk neuroblastoma: a Children's Oncology Group study. J Clin Oncol. 2011 Nov 20;29(33):4351-7. doi: 10.1200/JCO.2010.34.3293. Epub 2011 Oct 17. PMID 22010014