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Brain TumorPhase 2NCT00002594

Combination Chemotherapy Followed by Bone Marrow and/or Peripheral Stem Cell Transplantation in Treating Patients With Recurrent Medulloblastoma or CNS Germ Cell Tumors

A Phase 2 study testing Sargramostim, cyclophosphamide and melphalan in people with Brain Tumor and Central Nervous System Tumor, run by Children's Oncology Group at 118 sites in 6 countries.

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 that they stop growing or die. bone marrow transplantation and peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. Read more about this study

Who is it for?
Ages 2 to 25 years
Conditions the study is for
Brain Tumor, Central Nervous System Tumor
What is being tested?
Sargramostim, cyclophosphamide and melphalan
Could I receive a placebo?
No, everyone receives an active treatment
How many people
31 participants
Where
118 sitesin 6 countries
Study dates
Sep 1994to Mar 2007
Phase
Phase 2
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 2 to 25 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

DISEASE CHARACTERISTICS:

  • Diagnosis of recurrent medulloblastoma or CNS germ cell tumor
  • Histologic review of the primary intracranial or spinal cord tumor required
  • Biopsy and reduction of tumor bulk prior to study encouraged but not required
  • No more than 1 prior primary therapy (radiotherapy or chemoradiotherapy) and/or 1 prior salvage therapy
  • Patients with progression on salvage therapy ineligible
  • Minimal residual disease (tumor bulk no more than 1.5 cm) or in second clinical complete remission (CR)
  • Bone marrow infiltration with or without mass lesions or isolated abnormal CSF cytology as the only manifestation of recurrent disease allowed if a clinical CR is first achieved with conventional therapy

PATIENT CHARACTERISTICS:

Age:

  • 2 to 25

Performance status:

  • Karnofsky 80-100%

Life expectancy:

  • More than 8 weeks

Hematopoietic:

  • Absolute neutrophil count greater than 1,000/mm^3
  • Platelet count greater than 100,000/mm^3

Hepatic:

  • Bilirubin less than 1.5 mg/dL
  • SGPT less than 80 IU

Renal:

  • Creatinine less than 1.2 mg/dL

Cardiovascular:

  • LVEF normal

Other:

  • No infection
  • Able to tolerate vigorous hydration
  • Not pregnant
  • Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • Not specified

Chemotherapy:

  • See Disease Characteristics
  • Prior cyclophosphamide or ifosfamide allowed

Endocrine therapy:

  • No concurrent dexamethasone as an antiemetic
  • Other concurrent corticosteroids allowed

Radiotherapy:

  • See Disease Characteristics
  • Pretransplantation radiotherapy boost allowed

Surgery:

  • See Disease Characteristics
  • Pretransplantation surgery allowed

Other:

  • At least 4 weeks since prior therapy except corticosteroids

Trial locations and contacts

118 sites in 6 countries.

118 sites in 6 countries

United States103 sites

  • University of New Mexico Cancer Research and Treatment Center

    Albuquerque, New Mexico, 87131

  • Mission St. Joseph's Health System

    Asheville, North Carolina, 28801

  • Children's Healthcare of Atlanta - Scottish Rite

    Atlanta, Georgia, 30342

  • Marlene and Stewart Greenebaum Cancer Center, University of Maryland

    Baltimore, Maryland, 21201-1595

  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    Baltimore, Maryland, 21231-2410

  • Eastern Maine Medical Center

    Bangor, Maine, 04401

  • University of Alabama at Birmingham Comprehensive Cancer Center

    Birmingham, Alabama, 35233

  • Floating Hospital for Children

    Boston, Massachusetts, 02111

  • Massachusetts General Hospital Cancer Center

    Boston, Massachusetts, 02114

  • Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

    Boston, Massachusetts, 02115

  • Roswell Park Cancer Institute

    Buffalo, New York, 14263-0001

  • Vermont Cancer Center

    Burlington, Vermont, 05401-3498

  • Medical University of South Carolina

    Charleston, South Carolina, 29425-0721

  • West Virginia University - Charleston

    Charleston, West Virginia, 25302

  • Carolinas Medical Center

    Charlotte, North Carolina, 28232-2861

  • Presbyterian Healthcare

    Charlotte, North Carolina, 28233

  • Cancer Center at the University of Virginia

    Charlottesville, Virginia, 22908

  • Rush-Presbyterian-St. Luke's Medical Center

    Chicago, Illinois, 60612

  • Children's Memorial Hospital - Chicago

    Chicago, Illinois, 60614

  • University of Missouri-Columbia Hospital and Clinics

    Columbia, Missouri, 65212

  • Driscoll Children's Hospital

    Corpus Christi, Texas, 78466

  • Medical City Dallas Hospital

    Dallas, Texas, 75230

  • Simmons Cancer Center - Dallas

    Dallas, Texas, 75235-9154

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201

  • St. John Hospital and Medical Center

    Detroit, Michigan, 48236

  • Duke Comprehensive Cancer Center

    Durham, North Carolina, 27710

  • Inova Fairfax Hospital

    Falls Church, Virginia, 22042-3300

  • Hurley Medical Center

    Flint, Michigan, 48503

  • Broward General Medical Center

    Fort Lauderdale, Florida, 33316

  • Children's Hospital of Southwest Florida

    Fort Myers, Florida, 33908

  • Cook Children's Medical Center - Fort Worth

    Fort Worth, Texas, 76104

  • Shands Hospital and Clinics, University of Florida

    Gainesville, Florida, 32610-100277

  • University of Texas Medical Branch

    Galveston, Texas, 77555-0361

  • St. Vincent Hospital

    Green Bay, Wisconsin, 54307-9070

  • East Carolina University School of Medicine

    Greenville, North Carolina, 27858-4354

  • Children's Hospital of Greenville Hospital System

    Greenville, South Carolina, 29605

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Joe DiMaggio Children's Hospital at Memorial

    Hollywood, Florida, 33021

  • Cancer Research Center of Hawaii

    Honolulu, Hawaii, 96813

  • Tripler Army Medical Center

    Honolulu, Hawaii, 96859-5000

  • Texas Children's Cancer Center

    Houston, Texas, 77030-2399

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216-4505

  • Nemours Children's Clinic

    Jacksonville, Florida, 32207

  • East Tennessee State University Cancer Center at JCMC

    Johnson City, Tennessee, 37604

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160-7357

  • Keesler Medical Center - Keesler AFB

    Keesler Air Force Base, Mississippi, 39534-2511

  • Rebecca and John Moores UCSD Cancer Center

    La Jolla, California, 92093-0658

  • San Antonio Military Pediatric Cancer and Blood Disorders Center

    Lackland Air Force Base, Texas, 78236

  • Norris Cotton Cancer Center

    Lebanon, New Hampshire, 03756-0002

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105-2794

  • Sylvester Cancer Center, University of Miami

    Miami, Florida, 33136

  • Miami Children's Hospital

    Miami, Florida, 33155-4069

  • Baptist Hospital of Miami

    Miami, Florida, 33176-2197

  • Midwest Children's Cancer Center

    Milwaukee, Wisconsin, 53226

  • University of South Alabama Medical Center

    Mobile, Alabama, 36604

  • West Virginia University Hospitals

    Morgantown, West Virginia, 26506-9300

  • Yale Comprehensive Cancer Center

    New Haven, Connecticut, 06520-8028

  • Schneider Children's Hospital

    New Hyde Park, New York, 11042

  • MBCCOP - LSU Health Sciences Center

    New Orleans, Louisiana, 70112

  • Tulane University School of Medicine

    New Orleans, Louisiana, 70112

  • Children's Hospital of New Orleans

    New Orleans, Louisiana, 70118

  • Ochsner Clinic Foundation

    New Orleans, Louisiana, 70121

  • Mount Sinai School of Medicine

    New York, New York, 10029

  • Beth Israel Hospital North

    New York, New York, 10128

  • Advocate Hope Children's Hospital

    Oak Lawn, Illinois, 60453

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104

  • Florida Hospital Cancer Institute

    Orlando, Florida, 32804

  • Nemours Children's Clinic-Orlando

    Orlando, Florida, 32806

  • Sacred Heart Children's Hospital

    Pensacola, Florida, 32504

  • Saint Jude Midwest Affiliate

    Peoria, Illinois, 61637

  • St. Christopher's Hospital for Children

    Philadelphia, Pennsylvania, 19134-1095

  • Legacy Emanuel Hospital and Health Center

    Portland, Oregon, 97227

  • Naval Medical Center, Portsmouth

    Portsmouth, Virginia, 23708-5100

  • Rhode Island Hospital

    Providence, Rhode Island, 02818

  • Massey Cancer Center

    Richmond, Virginia, 23298-0037

  • Carilion Medical Center for Children

    Roanoke, Virginia, 24029

  • James P. Wilmot Cancer Center

    Rochester, New York, 14642

  • Sutter Cancer Center

    Sacramento, California, 95816

  • University of California Davis Cancer Center

    Sacramento, California, 95817

  • University of Texas Health Science Center at San Antonio

    San Antonio, Texas, 78207

  • Kaiser Permanente Medical Center/Kaiser Foundation Hospital - San Diego

    San Diego, California, 92120

  • Children's Hospital San Diego

    San Diego, California, 92123-4282

  • Kaiser Permanente Medical Center - Santa Clara

    Santa Clara, California, 95051-5386

  • Maine Children's Cancer Program

    Scarborough, Maine, 04074-9308

  • Cardinal Glennon Children's Hospital

    St Louis, Missouri, 63104

  • Washington University Medical Center

    St Louis, Missouri, 63105

  • All Children's Hospital

    St. Petersburg, Florida, 33701

  • Stanford University Medical Center

    Stanford, California, 94305-5208

  • State University of New York Health Sciences Center - Stony Brook

    Stony Brook, New York, 11790-7775

  • State University of New York - Upstate Medical University

    Syracuse, New York, 13210

  • Madigan Army Medical Center

    Tacoma, Washington, 98431-5000

  • Tampa Children's Hospital

    Tampa, Florida, 33677-4227

  • Scott and White Memorial Hospital

    Temple, Texas, 76508

  • Arizona Cancer Center

    Tucson, Arizona, 85724

  • Warren Clinic

    Tulsa, Oklahoma, 74136

  • Walter Reed Army Medical Center

    Washington D.C., District of Columbia, 20307-5000

  • St. Mary's Hospital

    West Palm Beach, Florida, 33407

  • CCOP - Wichita

    Wichita, Kansas, 67214-3882

  • Via Christi Regional Medical Center

    Wichita, Kansas, 67214

  • Wesley Medical Center

    Wichita, Kansas, 67214

  • Comprehensive Cancer Center at Wake Forest University

    Winston-Salem, North Carolina, 27157-1081

  • University of Massachusetts Memorial Medical Center - University Campus

    Worcester, Massachusetts, 01655

Other trials in United States

Canada8 sites

  • Alberta Children's Hospital

    Calgary, Alberta, T2T 5C7

  • Cross Cancer Institute

    Edmonton, Alberta, T6G 1Z2

  • Chedoke-McMaster Hospitals

    Hamilton, Ontario, L8N 3Z5

  • McGill University Health Center - Montreal Children's Hospital

    Montreal, Quebec, H3G 1A4

  • Hopital Sainte Justine

    Montreal, Quebec, H3T 1C5

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1

  • Centre de Recherche du Centre Hospitalier de l'Universite Laval

    Sainte-Foy, Quebec, GIV 4G2

  • Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

Other trials in Canada

Australia3 sites

  • Royal Children's Hospital

    Brisbane, Queensland, 4029

  • Royal Children's Hospital

    Parkville, Victoria, 3052

  • Children's Hospital at Westmead

    Westmead, New South Wales, 2145

Other trials in Australia

Switzerland2 sites

  • Swiss Pediatric Oncology Group Bern

    Bern, CH 3010

  • Swiss Pediatric Oncology Group Geneva

    Geneva, CH 1211

Other trials in Switzerland

Netherlands1 site

  • Academisch Ziekenhuis Groningen

    Groningen, 9700 RB

Other trials in Netherlands

Puerto Rico1 site

  • San Jorge Childrens Hospital

    Santurce, 00912

Other trials in Puerto Rico

About this study

Official title: Dose-Intensive Melphalan and Cyclophosphamide With Autologous Bone Marrow Rescue for Recurrent Medulloblastoma and Germ Cell Tumors

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so that they stop growing or die. bone marrow transplantation and peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells.

PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy followed by bone marrow transplantation and/or peripheral stem cell transplantation in treating patients who have recurrent medulloblastoma or CNS germ cell tumors.

Read the detailed description

OBJECTIVES:

  • Determine the response and progression-free survival in patients with recurrent medulloblastoma or CNS germ cell tumors treated with intensive melphalan and cyclophosphamide followed by autologous bone marrow and/or peripheral blood stem cell rescue.
  • Determine the acute and delayed toxic effects of this regimen in these patients.

OUTLINE: Autologous bone marrow and/or peripheral blood stem cells (PBSC) are harvested. Patients then receive intensive cyclophosphamide IV over 1 hour on days -8 to -5 and melphalan IV over 15 minutes on days -4 to -2. Bone marrow is reinfused on day 0. PBSC are reinfused on day 0 if used alone or on day 1 if used after autologous bone marrow transplantation (ABMT). Sargramostim (GM-CSF) is administered IV over 2 hours daily beginning 4 hours after ABMT and continuing until blood counts recover.

Patients are followed every 6 months through year 4 and then annually thereafter.

PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study within 2.5 years.

Being tested
Sargramostim, cyclophosphamide, melphalan, autologous bone marrow transplantation, bone marrow ablation with stem cell support
Keywords
recurrent childhood brain tumor, childhood central nervous system germ cell tumor, recurrent childhood medulloblastoma
Medical subject headings
Brain Neoplasms, Central Nervous System Neoplasms, Medulloblastoma

What participants receive

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

  • SargramostimBiological

    Also called M-CSF, GM-CSF, Granulocyte macrophage-colony stimulating factor, Prokine

    Given IV

  • cyclophosphamideDrug

    Also called Cytoxan, NSC #026271, IND #7089

    Given IV

  • melphalanDrug

    Also called ylalanine mustard, L-PAM, L-sarcolysin, Alkeran

    Given IV

  • autologous bone marrow transplantationProcedure or surgery

  • bone marrow ablation with stem cell supportProcedure or surgery

  • peripheral blood stem cell transplantationProcedure or surgery

Study groups in NCT00002594
Study groupRoleWhat they receive
Ablative chemo followed by autologous bone marrow (ABM) rescueExperimentalAutologous bone marrow and/or peripheral blood stem cells (PBSC) are harvested. Patients then receive intensive cyclophosphamide IV over 1 hour on days -8 to -5 and melphalan IV over 15 minutes on days -4 to -2. Bone marrow is reinfused on day 0. PBSC are reinfused on day 0 if used alone or on day 1 if used after autologous bone marrow transplantation (ABMT). Sargramostim (GM-CSF) is administered IV over 2 hours daily beginning 4 hours after ABMT and continuing until blood counts recover.
Receives: Biological: Sargramostim; Drug: cyclophosphamide; Drug: melphalan; Procedure: autologous bone marrow transplantation; Procedure: bone marrow ablation with stem cell support; Procedure: peripheral blood stem cell transplantation

What is being measured

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

  1. Progression-free survival

    Measured: 1-2 years

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 startSep 1994
  3. Primary completionSep 2003
  4. Study completionMar 2007

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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
31 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00002594
Sponsor's protocol ID
9430
Other IDs
COG-P9430, POG-9430, CDR0000063784
Status last verified
Jul 2014
First submitted
1 Nov 1999
Last updated
24 Jul 2014
Results posted
No
Data monitoring committee
Yes

Publications

  • Kadota RP, Mahoney DH, Doyle J, Duerst R, Friedman H, Holmes E, Kun L, Zhou T, Pollack IF. Dose intensive melphalan and cyclophosphamide with autologous hematopoietic stem cells for recurrent medulloblastoma or germinoma. Pediatr Blood Cancer. 2008 Nov;51(5):675-8. doi: 10.1002/pbc.21655.

    PubMed 18623206

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sargramostim, cyclophosphamide and melphalan in people with Brain Tumor and Central Nervous System Tumor, run by Children's Oncology Group at 118 sites in 6 countries.

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 24 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 118 sites in United States, Canada, Australia, Switzerland, Netherlands and 1 more country. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

Progression-free survival (time frame: 1-2 years). 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 NCT00002594, 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 24 Jul 2014; AzTrials last refreshed its copy on 2 Nov 2026.

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