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Solid TumorPhase 1NCT00638898

Busulfan, Melphalan, Topotecan Hydrochloride, and a Stem Cell Transplant in Treating Patients With Newly Diagnosed or Relapsed Solid Tumor

A Phase 1 study testing busulfan, melphalan and topotecan hydrochloride in people with Solid Tumor and Adult Central Nervous System Germ Cell Tumor, run by City of Hope Medical Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

RATIONALE: Giving high-dose chemotherapy before an autologous stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as G-CSF, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Read more about this study

Who is it for?
Ages 6 months to 40 years
What is being tested?
busulfan, melphalan and topotecan hydrochloride
Could I receive a placebo?
No, everyone receives an active treatment
How many people
25 participants
Where
1 siteUnited States
Study dates
26 Feb 2007to 10 Dec 2024
Phase
Phase 1

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 months to 40 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Patients with relapsed neuroblastoma, rhabdomyosarcoma, Ewing's sarcoma, PNET, brain tumors, soft tissue sarcomas, Wilm's tumors, germ cell tumors or other solid tumors who achieved at least partial response (PR) to chemotherapy, surgery, or radiotherapy
  • Newly diagnosed patients for poor-risk pediatric solid tumors: metastatic Ewing's, metastatic PNET, rhabdomyosarcoma, soft tissue sarcomas, octeomesenchymoma, and others that are at a high risk of relapse and who have achieved at least partial response (PR) to chemotherapy, surgery, or radiotherapy
  • For any of the above categories, an attempt to achieve a complete response (CR) or PR should be made; pre-transplant modalities may include surgery, chemotherapy, or radiation therapy; radiation must not include lung fields; only patients in CR or PR at the primary site will be eligible
  • HIGH-DOSE CHEMOTHERAPY: Histologically confirmed diagnosis by Anatomic Pathology Department; if recurrent or metastatic disease, histologic confirmation should be obtained, with the exception of brain stem tumors; in neuroblastoma, demonstration of marrow metastases with elevated urinary catecholamines is adequate for diagnosis
  • HIGH-DOSE CHEMOTHERAPY: No contraindications to the stem cell collection by apheresis or by bone marrow harvesting
  • HIGH-DOSE CHEMOTHERAPY: All patients, or their legal guardians must have signed a voluntary informed consent in accordance with the institutional and federal guidelines
Show all 14Show fewer
  • HIGH-DOSE CHEMOTHERAPY: Adequate renal function as demonstrated by creatinine clearance (12 or 24 hour urine collection) or glomerular filtration rate (GFR) > 60 ml/min/1.73m^2
  • HIGH-DOSE CHEMOTHERAPY: Adequate cardiac function as demonstrated by ejection fraction > 55% by echocardiogram or MUGA
  • HIGH-DOSE CHEMOTHERAPY: Adequate hepatic function as demonstrated by bilirubin < 2 mg/dL, SGOT and SGPT < 5 x upper limits of normal
  • HIGH-DOSE CHEMOTHERAPY: Adequate bone marrow function as evidenced by platelet count > 50,000/ul and absolute granulocyte count >= 750 ul
  • HIGH-DOSE CHEMOTHERAPY: Adequate pulmonary function adults (older than 16 years): FEV1 > 2 liters, room air PaO2 > 70 mm Hg, room air PaCO2 < 42 mm Hg, and DLCO > 50% predicted; children (younger than 16 years): DLCO > 50% predicted
  • HIGH-DOSE CHEMOTHERAPY: Pretreatment tests and clinical and laboratory tests must have been performed within 4 weeks prior to initiation of high-dose chemotherapy
  • HIGH-DOSE CHEMOTHERAPY: No other medical and/or psychosocial problems which in the opinion of the primary physician or principal investigator would place the patient at unacceptable risk from this regimen
  • HIGH-DOSE CHEMOTHERAPY: Greater than 2-week period of recovery from prior modality used to control primary or recurrent site

Who cannot join

  • Histologically confirmed bone marrow metastases within 30 days prior to transplant; prior bone marrow metastases with clearing of bone marrow (< 5% contamination as measured by bilateral bone marrow biopsies) at the time for evaluation for this protocol is acceptable
  • Karnofsky performance status < 60% or Lansky performance status < 50% for patients younger than 16 years old
  • Females of reproductive age who are not using adequate birth control measures or who are pregnant
  • HIV disease
  • Patients with prior treatment with myeloablative therapy are excluded

Trial locations and contacts

1 site in 1 country.

United States1 site

  • City of Hope

    Duarte, California, 91010

Other trials in United States

About this study

Official title: Pilot Study of High-Dose Chemotherapy With Busulfan, Melphalan, and Topotecan Followed by Autologous Hematopoietic Stem Cell Transplant in Advanced Stage and Recurrent Tumors

RATIONALE: Giving high-dose chemotherapy before an autologous stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as G-CSF, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy is then 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.

PURPOSE: This clinical trial is studying how well giving busulfan, melphalan, and topotecan hydrochloride together with a stem cell transplant works in treating patients with newly diagnosed or relapsed solid tumor.

Read the detailed description

OBJECTIVES:

  1. To assess the feasibility of a novel combination conditioning therapy with busulfan/melphalan and topotecan followed by autologous hematopoietic stem cell transplantation (HSCT) in patients with relapsed, refractory and/or poor risk pediatric solid tumors.

II. To determine within the confines of this pilot study, myeloid and platelet engraftment, overall survival and disease-free survival in patients with relapsed, refractory pediatric solid tumors and in patients who have solid tumors with poor risk factors at the time of diagnosis.

III. To determine the pharmacokinetics of topotecan.

OUTLINE:

AUTOLOGOUS HEMATOPOIETIC STEM CELL OR AUTOLOGOUS BONE MARROW COLLECTION: Patients undergo stem cell mobilization per institutional guidelines with G-CSF IV or subcutaneously, continuing until the completion of leukapheresis. Patients undergo apheresis after mobilization and continue until a minimum of 2.0 x 10^6 CD34 cells/kg or more are collected. Cells are processed and cryopreserved following institutional guidelines. Patients who collect > 2.0 x 10^6 CD34+ cells/kg may proceed to high-dose chemotherapy.

HIGH-DOSE CHEMOTHERAPY: Patients receive topotecan hydrochloride IV continuously over 24 hours on days -8 to -4, busulfan IV every 6 hours on days -8 to -4, and melphalan IV over 30 minutes on days -3 and -2.

AUTOLOGOUS HEMATOPOIETIC STEM CELL OR AUTOLOGOUS BONE MARROW REINFUSION: Patients undergo autologous hematopoietic stem cell transplantation or autologous bone marrow transplantation on day 0. Patients also receive G-CSF IV daily beginning on day +5 and continuing until blood counts recover.

After completion of study treatment, patients are followed every 3 months for 1 year and then annually thereafter.

Being tested
busulfan, melphalan, topotecan hydrochloride, laboratory biomarker analysis, filgrastim
Medical subject headings
Rhabdomyosarcoma, Sarcoma, Ewing, Neuroectodermal Tumors, Primitive, Peripheral, Brain Neoplasms, Sarcoma, Astrocytoma, Familial ependymoma, Medulloblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • busulfanDrug

    Also called BSF, BU, Misulfan, Mitosan

    Given IV

  • melphalanDrug

    Also called Alkeran, CB-3025, L-PAM, L-phenylalanine mustard

    Given IV

  • topotecan hydrochlorideDrug

    Also called hycamptamine, Hycamtin, SKF S-104864-A, TOPO

    Given IV

  • laboratory biomarker analysisOther

    Correlative studies

  • filgrastimBiological

    Also called G-CSF, granulocyte colony-stimulating factor, Neupogen, r-metHuG-CSF

    Given IV or subcutaneously

  • autologous hematopoietic stem cell transplantationProcedure or surgery

    Undergo transplantation

  • pharmacological studyOther

    Also called pharmacological studies

    Correlative studies

  • autologous bone marrow transplantationProcedure or surgery

    Also called ABMT, bone marrow transplantation, autologous, transplantation, autologous bone marrow

    Undergo transplantation

Study groups in NCT00638898
Study groupRoleWhat they receive
Arm IExperimentalSee Detailed Description
Receives: Drug: busulfan; Drug: melphalan; Drug: topotecan hydrochloride; Other: laboratory biomarker analysis; Biological: filgrastim; Procedure: autologous hematopoietic stem cell transplantation; Other: pharmacological study; Procedure: autologous bone marrow transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Treatment feasibility in terms of investigational agent-related adverse events of a novel treatment combination followed by peripheral blood stem cell rescue

    Measured: Day 100 post stem cell rescue

Secondary outcomes (4)

  1. Overall survival

    Measured: 1 year post stem cell rescue

  2. Disease-free survival

    Measured: 1 year post stem cell rescue

  3. Incidence of myeloid and platelet engraftment

    Measured: Day 100 post stem cell rescue

  4. Pharmacokinetics and pharmacodynamics of topotecan hydrochloride and busulfan

    Measured: Baseline through day 4 of investigational agent treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Mar 2008 (estimated)
  2. Study start26 Feb 2007
  3. Primary completion19 Jan 2013
  4. Study completion10 Dec 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
25 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00638898
Sponsor's protocol ID
03112
Other IDs
NCI-2009-01600
Status last verified
Mar 2025
First submitted
18 Mar 2008
Last updated
20 Mar 2025
Results posted
No
Data monitoring committee
Yes

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