Amifostine in Treating Young Patients With Newly Diagnosed De Novo Myelodysplastic Syndromes

February 11, 2014 updated by: Children's Oncology Group

A Phase II Study Of Amifostine In Children With Myelodysplastic Syndrome

RATIONALE: Drugs used in chemotherapy, such as amifostine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying how well amifostine works in treating young patients with newly diagnosed de novo myelodysplastic syndromes.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

OBJECTIVES:

Primary

  • Determine the hematologic effects of amifostine, in terms of, complete and partial response, in pediatric patients with newly diagnosed de novo myelodysplastic syndromes (MDS).
  • Determine the safety and efficacy of this drug in these patients.

Secondary

  • Determine the efficacy of this drug in preventing conversion of MDS to acute myeloid leukemia (AML) in terms of the proportion of patients who remain free of AML at the completion of study treatment.
  • Determine the duration of progression-free remission from MDS conversion to AML in patients treated with this drug.
  • Determine the effect of karyotypic abnormalities on survival and the duration from diagnosis of MDS until conversion to AML in patients treated with this drug.
  • Determine the effect of bone marrow blast count on survival and the duration from diagnosis of MDS until conversion to AML in patients treated with this drug.
  • Determine the effect of the number of cytopenias on survival in patients treated with this drug.
  • Correlate the duration of time from diagnosis of MDS until conversion to AML with survival in patients treated with this drug.

OUTLINE: This is a multicenter study.

Patients receive amifostine IV over 1-3 minutes on days 1, 3, 5, 8, 10, 12, 15, 17, and 19. Treatment repeats every 5 weeks for 2 courses in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease who are planning to undergo matched donor bone marrow or cord blood transplantation continue therapy until transplantation. Patients with stable or responding disease who are not undergoing transplantation may receive up to 4 additional courses of amifostine in the absence of disease progression or unacceptable toxicity.

Following completion of therapy with amifostine, patients are followed monthly for 1 year, every 2 months for 1 year, every 3 months for 1 year, every 6 months for 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 10-20 patients will be accrued for this study within 5-10 months.

Study Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Western Australia
      • Perth, Western Australia, Australia, 6001
        • Princess Margaret Hospital for Children
  • Canada
      • Quebec, Canada, G1V 4G2
        • Centre Hospitalier Universitaire de Quebec
    • Alberta
      • Edmonton, Alberta, Canada, T6G 1Z2
        • University of Alberta Hospital
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3V4
        • Children's & Women's Hospital of British Columbia
    • Manitoba
      • Winnipeg, Manitoba, Canada, R3E 0V9
        • CancerCare Manitoba
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3K 6R8
        • IWK Health Centre
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • McMaster Children's Hospital at Hamilton Health Sciences
      • Kingston, Ontario, Canada, K7L 3N6
        • Cancer Centre of Southeastern Ontario At Kingston General Hospital
    • Quebec
      • Montreal, Quebec, Canada, H3H 1P3
        • Montreal Children's Hospital at McGill University Health Center
      • Montreal, Quebec, Canada, H3T 1C5
        • Hopital Sainte Justine
  • Puerto Rico
      • Santurce, Puerto Rico, 00912
        • San Jorge Children's Hospital
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016-7710
        • Phoenix Children's Hospital
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • Arkansas Cancer Research Center at University of Arkansas for Medical Sciences
    • California
      • Long Beach, California, United States, 90801
        • Jonathan Jaques Children's Cancer Center at Miller Children's Hospital
      • Madera, California, United States, 93638-8762
        • Children's Hospital Central California
      • Sacramento, California, United States, 95825
        • Kaiser Permanente Medical Center - Oakland
    • Connecticut
      • Farmington, Connecticut, United States, 06360-2875
        • Carole and Ray Neag Comprehensive Cancer Center at the University of Connecticut Health Center
    • Florida
      • Fort Myers, Florida, United States, 33901
        • Lee Cancer Care of Lee Memorial Health System
      • Gainesville, Florida, United States, 32610-0232
        • University of Florida Shands Cancer Center
      • Hollywood, Florida, United States, 33021
        • Memorial Cancer Institute at Memorial Regional Hospital
      • Miami, Florida, United States, 33155
        • Miami Children's Hospital
      • Pensacola, Florida, United States, 32504
        • Sacred Heart Cancer Center at Sacred Heart Hospital
      • St. Petersburg, Florida, United States, 33701
        • All Children's Hospital
      • Tampa, Florida, United States, 33607
        • St. Joseph's Cancer Institute at St. Joseph's Hospital
      • West Palm Beach, Florida, United States, 33407
        • Kaplan Cancer Center at St. Mary's Medical Center
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Winship Cancer Institute of Emory University
    • Hawaii
      • Honolulu, Hawaii, United States, 95813
        • Cancer Research Center of Hawaii
    • Illinois
      • Oak Lawn, Illinois, United States, 60453
        • Advocate Christ Medical Center
    • Indiana
      • Indianapolis, Indiana, United States, 46202-5289
        • Indiana University Cancer Center
      • Indianapolis, Indiana, United States, 46260
        • St. Vincent Indianapolis Hospital
    • Kansas
      • Kansas City, Kansas, United States, 66160-7357
        • Kansas Masonic Cancer Research Institute at the University of Kansas Medical Center
    • Kentucky
      • Louisville, Kentucky, United States, 40232
        • Kosair Children's Hospital
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • Ochsner Cancer Institute at Ochsner Clinic Foundation
    • Maine
      • Bangor, Maine, United States, 04401
        • CancerCare of Maine at Eastern Maine Medial Center
    • Maryland
      • Baltimore, Maryland, United States, 21215
        • Alvin and Lois Lapidus Cancer Institute at Sinai Hospital
    • Michigan
      • Detroit, Michigan, United States, 48201-1379
        • Barbara Ann Karmanos Cancer Institute
      • Flint, Michigan, United States, 48503
        • Hurley Medical Center
      • Grand Rapids, Michigan, United States, 49503-2560
        • Spectrum Health Hospital - Butterworth Campus
      • Grosse Pointe Woods, Michigan, United States, 48236
        • Van Elslander Cancer Center at St. John Hospital and Medical Center
      • Lansing, Michigan, United States, 48910
        • Breslin Cancer Center at Ingham Regional Medical Center
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic Cancer Center
    • Mississippi
      • Jackson, Mississippi, United States, 39216-4505
        • University of Mississippi Medical Center
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Children's Mercy Hospital
      • St. Louis, Missouri, United States, 63110
        • Siteman Cancer Center at Barnes-Jewish Hospital
    • Nebraska
      • Omaha, Nebraska, United States, 68198-6805
        • UNMC Eppley Cancer Center at the University of Nebraska Medical Center
    • Nevada
      • Las Vegas, Nevada, United States, 89109-2306
        • Sunrise Hospital and Medical Center
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack University Medical Center Cancer Center
      • Morristown, New Jersey, United States, 07962
        • Overlook Hospital
      • New Brunswick, New Jersey, United States, 08901
        • Saint Peter's University Hospital
      • New Brunswick, New Jersey, United States, 08903
        • Cancer Institute of New Jersey at UMDNJ - Robert Wood Johnson Medical School
    • New Mexico
      • Albuquerque, New Mexico, United States, 87131-5636
        • University of New Mexico Cancer Research and Treatment Center
    • New York
      • Bronx, New York, United States, 10461
        • Albert Einstein Cancer Center at Albert Einstein College of Medicine
      • Brooklyn, New York, United States, 11219
        • Maimonides Cancer Center at Maimonides Medical Center
      • New York, New York, United States, 10032
        • Herbert Irving Comprehensive Cancer Center at Columbia University
      • Syracuse, New York, United States, 13210
        • SUNY Upstate Medical University Hospital
      • Valhalla, New York, United States, 10595
        • New York Medical College
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599-7295
        • Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill
      • Charlotte, North Carolina, United States, 28232-2861
        • Blumenthal Cancer Center at Carolinas Medical Center
    • Ohio
      • Akron, Ohio, United States, 44308-1062
        • Children's Hospital Medical Center of Akron
      • Cincinnati, Ohio, United States, 45229-3039
        • Cincinnati Children's Hospital Medical Center
      • Cleveland, Ohio, United States, 44106-5000
        • Rainbow Babies and Children's Hospital
      • Columbus, Ohio, United States, 43205-2696
        • Columbus Children's Hospital
      • Dayton, Ohio, United States, 45404-1815
        • Children's Medical Center - Dayton
      • Youngstown, Ohio, United States, 44501
        • Tod Children's Hospital - Forum Health
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Oklahoma University Medical Center
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033-0850
        • Penn State Cancer Institute at Milton S. Hershey Medical Center
      • Philadelphia, Pennsylvania, United States, 19134-1095
        • St. Christopher's Hospital for Children
      • Philadelphia, Pennsylvania, United States, 19104-9786
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 15213
        • Children's Hospital of Pittsburgh
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Hollings Cancer Center at Medical University of South Carolina
      • Greenville, South Carolina, United States, 29605
        • Greenville Hospital System Cancer Center
    • Tennessee
      • Knoxville, Tennessee, United States, 37916
        • East Tennessee Children's Hospital
    • Texas
      • Amarillo, Texas, United States, 79106
        • Texas Tech University Health Sciences Center School of Medicine - Amarillo
      • Austin, Texas, United States, 78701
        • Children's Hospital of Austin
      • Dallas, Texas, United States, 75230
        • Medical City Dallas Hospital
      • Fort Worth, Texas, United States, 76104-9958
        • Cook Children's Medical Center - Fort Worth
      • Houston, Texas, United States, 77030-2399
        • Baylor University Medical Center - Houston
      • San Antonio, Texas, United States, 78229-3993
        • Methodist Children's Hospital of South Texas
      • San Antonio, Texas, United States, 78207
        • University of Texas Health Science Center at San Antonio
    • Utah
      • Salt Lake City, Utah, United States, 84113-1100
        • Primary Children's Medical Center
    • Washington
      • Seattle, Washington, United States, 98105
        • Children's Hospital and Regional Medical Center - Seattle
    • West Virginia
      • Charleston, West Virginia, United States, 25302
        • West Virginia University - Robert C. Byrd Health Sciences Center - Charleston Division
      • Huntington, West Virginia, United States, 25701
        • Edwards Comprehensive Cancer Center at Cabell Huntington Hospital
    • Wisconsin
      • Marshfield, Wisconsin, United States, 54449
        • Marshfield Clinic - Marshfield Center
      • Milwaukee, Wisconsin, United States, 53226
        • Midwest Children's Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

DISEASE CHARACTERISTICS:

  • Histologically confirmed diagnosis of myelodysplastic syndromes (MDS)

    • One of the following subtypes:

      • Refractory anemia (RA)
      • RA with ringed sideroblasts
      • RA with excess blasts
      • Refractory cytopenia with multilineage dysplasia (RCMD)
      • RCMD and ringed sideroblasts
      • MDS, unclassified
      • MDS associated with isolated del 5(q)

  • De novo disease

    • No treatment-induced MDS
  • No juvenile myelomonocytic leukemia
  • No Down syndrome, Fanconi's anemia, or other inherited forms of hypoplastic bone marrow failure

PATIENT CHARACTERISTICS:

Age

  • 1 to 21 at original diagnosis

Performance status

  • Karnofsky 50-100% (patients > 16 years of age)
  • Lansky 50-100% (patients 1 to 16 years of age)

Life expectancy

  • At least 8 weeks

Hematopoietic

  • See Disease Characteristics

Hepatic

  • Bilirubin ≤ 1.5 times upper limit of normal (ULN)
  • ALT < 2.5 times ULN

Renal

  • Radioisotope glomerular filtration rate ≥ 60 mL/min OR
  • Creatinine clearance > 60 mL/min (based on Schwartz formula)
  • Calcium normal

Other

  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Serum electrolytes normal
  • Phosphorus normal
  • Magnesium normal
  • Glucose normal
  • No other concurrent malignancy

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • More than 8 weeks since prior growth factors
  • No concurrent growth factors
  • No concurrent hematopoietic stem cell transplantation
  • No concurrent immunomodulating agents

Chemotherapy

  • No prior amifostine
  • No other concurrent anticancer chemotherapy

Endocrine therapy

  • No concurrent daily steroid therapy

Radiotherapy

  • Not specified

Surgery

  • Not specified

Other

  • No prior therapy for MDS

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Safety and efficacy
Hematological effects (complete and partial response)

Secondary Outcome Measures

Outcome Measure
Efficacy
Duration of progression-free remission
Effect of karyotypic abnormalities on survival
Effect of the number of cytopenias on survival
Correlation of the duration of time from diagnosis of myelodysplastic syndromes until conversion to acute myeloid leukemia

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Oncology Group

Collaborators

National Cancer Institute (NCI)

Investigators

  • Study Chair: Prasad Mathew, MD, University of New Mexico Cancer Center
  • Robert J. Arceci, MD, PhD, Sidney kimmel comprehensive cancer center at johns hopkins

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2005

Primary Completion (Actual)

October 1, 2007

Study Completion (Actual)

October 1, 2009

Study Registration Dates

First Submitted

December 7, 2004

First Submitted That Met QC Criteria

December 7, 2004

First Posted (Estimate)

December 8, 2004

Study Record Updates

Last Update Posted (Estimate)

February 13, 2014

Last Update Submitted That Met QC Criteria

February 11, 2014

Last Verified

February 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AAML0121
  • CDR0000398140 (Other Identifier: Clinical Trials.gov)
  • COG-AAML0121 (Other Identifier: Children's Oncology Group)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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