AzTrials
AzTrials

Brain and Central Nervous System TumorsPhase 1NCT00006246

Busulfan in Treating Children and Adolescents With Refractory CNS Cancer

A Phase 1 study testing busulfan in people with Brain and Central Nervous System Tumors and Childhood Germ Cell Tumor, run by Pediatric Brain Tumor Consortium at 8 sites in United States.

Completed

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

Official record
Search all trials

Trial summary

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the safety of delivering intrathecal busulfan in children and adolescents who have refractory CNS cancer and to estimate the maximum tolerated dose of this treatment… Read more about this study

Who is it for?
Ages 3 to 21 years
What is being tested?
busulfan
How many people
28 participants
Where
8 sitesUnited States
Study dates
Nov 2000to May 2003
Phase
Phase 1
Are there results?
None posted in the registry

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 3 to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

DISEASE CHARACTERISTICS:

  • Histologically confirmed CNS malignancy, including any of the following:
  • Primary malignant brain tumor refractory to standard therapy and metastatic to the cerebrospinal fluid (CSF) or leptomeningeal subarachnoid space
  • Recurrent or persistent leptomeningeal leukemia, lymphoma, or germ cell tumor refractory to conventional therapy
  • In second or greater relapse
  • CSF white blood count greater than 5 cells/mm3 with blasts on cytospin OR
  • Evidence of leptomeningeal tumor by MRI
  • No concurrent bone marrow disease
  • No obstruction or compartmentalization of CSF flow on CSF flow study

PATIENT CHARACTERISTICS:

Age:

  • 3 to 21

Performance status:

  • Lansky 50-100% (under 10 years)
  • Karnofsky 50-100% (10 to 21 years)

Life expectancy:

  • Greater than 8 weeks

Hematopoietic:

  • Absolute neutrophil count greater than 1,000/mm^3
  • Platelet count greater than 75,000/mm^3

Hepatic:

  • Bilirubin normal for age
  • ALT and AST less than 5 times upper limit of normal (ULN)
  • No hepatic disease

Renal:

  • Creatinine no greater than 1.5 times ULN OR
  • Glomerular filtration rate greater than 70 mL/min
  • No renal disease

Cardiovascular:

  • No cardiac disease

Pulmonary:

  • No pulmonary disease

Other:

  • No uncontrolled infection
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • Not specified

Chemotherapy:

  • At least 3 weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosoureas)
  • At least 1 week since prior intrathecal chemotherapy (2 weeks for cytarabine) and recovered
  • Evidence of subsequent disease progression
  • Concurrent systemic chemotherapy allowed for recurrent disease after first course of treatment except for the following:
  • Chemotherapy targeted at leptomeningeal disease
  • Other phase I agent
  • Any agent that significantly penetrates the CSF (e.g., high dose methotrexate greater than 1 g/m2, thiotepa, high dose cytarabine, fluorouracil, IV mercaptopurine, nitrosoureas, or topotecan)
  • Any agent that causes serious unpredictable CNS side effects

Endocrine therapy:

  • Prior dexamethasone allowed with decreasing or stable dose at least one week before study
  • Concurrent dexamethasone or prednisone with chemotherapy regimen allowed

Radiotherapy:

  • At least 1 week since prior focal irradiation to the brain or spine
  • At least 8 weeks since prior craniospinal irradiation
  • No concurrent cranial or craniospinal irradiation

Surgery:

  • Not specified

Other:

  • No other concurrent intrathecal or systemic therapy for leptomeningeal disease

Trial locations and contacts

8 sites in 1 country.

8 sites in 1 country

United States8 sites

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02115

  • Duke Comprehensive Cancer Center

    Durham, North Carolina, 27710

  • Baylor College of Medicine

    Houston, Texas, 77030

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104-4318

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15213

  • UCSF Cancer Center and Cancer Research Institute

    San Francisco, California, 94143-0128

  • Children's Hospital and Regional Medical Center - Seattle

    Seattle, Washington, 98105

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010-2970

Other trials in United States

About this study

Official title: Phase I Study of Intrathecal Spartaject-Busulfan in Children With Neoplastic Meningitis

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.

PURPOSE: Phase I trial to study the safety of delivering intrathecal busulfan in children and adolescents who have refractory CNS cancer and to estimate the maximum tolerated dose of this treatment regimen.

Read the detailed description

OBJECTIVES:

  • Determine the qualitative and quantitative toxicities of intrathecally administered busulfan in children and adolescents with refractory CNS malignancies.
  • Determine the maximum tolerated dose of this treatment regimen in these patients.
  • Determine the cerebrospinal fluid and serum pharmacokinetics of this treatment regimen in these patients.
  • Determine the efficacy of this treatment regimen in these patients.

OUTLINE: This is a dose-escalation study.

Patients receive intrathecal busulfan twice a week, at least 3 days apart, for 2 weeks. Patients with complete or partial response or stable disease may continue therapy once a week for 2 weeks, once a week every other week for 2 treatments, and then once a month thereafter in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of busulfan until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicities.

Patients are followed every 3 months for the first year, every 6 months for 4 years, and then annually for 5 years.

PROJECTED ACCRUAL: Approximately 18-24 patients will be accrued for this study over 18-38 months.

Being tested
busulfan
Keywords
recurrent childhood acute lymphoblastic leukemia, childhood infratentorial ependymoma, recurrent childhood rhabdomyosarcoma, recurrent childhood brain tumor, recurrent retinoblastoma, recurrent childhood lymphoblastic lymphoma, childhood central nervous system germ cell tumor, recurrent childhood acute myeloid leukemia, recurrent/refractory childhood Hodgkin lymphoma, leptomeningeal metastases, childhood high-grade cerebral astrocytoma, childhood oligodendroglioma, childhood choroid plexus tumor, childhood grade I meningioma, childhood grade II meningioma, childhood grade III meningioma, recurrent childhood large cell lymphoma, recurrent childhood brain stem glioma, recurrent childhood supratentorial primitive neuroectodermal tumor, recurrent childhood cerebellar astrocytoma
Medical subject headings
Central Nervous System Neoplasms, Leukemia, Lymphoma, Neoplasm Metastasis, Retinoblastoma, Sarcoma, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Recurrence

What participants receive

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

What is being measured

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

  1. Toxicities of IT administered busulfan in children and adolescents with refractory CNS malignancies

  2. Maximum tolerated dose of IT administered busulfan

  3. Serum and CSF pharmacokinetics of IT administered busulfan

Timeline

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

  1. First posted27 Jan 2003 (estimated)
  2. Study startNov 2000
  3. Primary completionMay 2003

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
Enrolment
28 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00006246
Sponsor's protocol ID
CDR0000068178
Other IDs
PBTC-004
Status last verified
Oct 2009
First submitted
11 Sep 2000
Last updated
7 Oct 2009
Results posted
No
Data monitoring committee
Yes

Publications

  • Gururangan S, Petros WP, Poussaint TY, Hancock ML, Phillips PC, Friedman HS, Bomgaars L, Blaney SM, Kun LE, Boyett JM. Phase I trial of intrathecal spartaject busulfan in children with neoplastic meningitis: a Pediatric Brain Tumor Consortium Study (PBTC-004). Clin Cancer Res. 2006 Mar 1;12(5):1540-6. doi: 10.1158/1078-0432.CCR-05-2094.

    PubMed 16533779

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing busulfan in people with Brain and Central Nervous System Tumors and Childhood Germ Cell Tumor, run by Pediatric Brain Tumor Consortium at 8 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 7 Oct 2009. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Toxicities of IT administered busulfan in children and adolescents with refractory CNS malignancies. The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00006246, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Oct 2009.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in