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

NCT ID: NCT00098683

Last Updated: 2014-02-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-01-31

Study Completion Date

2009-10-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms

Study Design

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Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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amifostine trihydrate

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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
Minimum Eligible Age

1 Year

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Children's Oncology Group

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Prasad Mathew, MD

Role: STUDY_CHAIR

University of New Mexico Cancer Center

Robert J. Arceci, MD, PhD

Role:

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

Arkansas Cancer Research Center at University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Site Status

Jonathan Jaques Children's Cancer Center at Miller Children's Hospital

Long Beach, California, United States

Site Status

Children's Hospital Central California

Madera, California, United States

Site Status

Kaiser Permanente Medical Center - Oakland

Sacramento, California, United States

Site Status

Carole and Ray Neag Comprehensive Cancer Center at the University of Connecticut Health Center

Farmington, Connecticut, United States

Site Status

Lee Cancer Care of Lee Memorial Health System

Fort Myers, Florida, United States

Site Status

University of Florida Shands Cancer Center

Gainesville, Florida, United States

Site Status

Memorial Cancer Institute at Memorial Regional Hospital

Hollywood, Florida, United States

Site Status

Miami Children's Hospital

Miami, Florida, United States

Site Status

Sacred Heart Cancer Center at Sacred Heart Hospital

Pensacola, Florida, United States

Site Status

All Children's Hospital

St. Petersburg, Florida, United States

Site Status

St. Joseph's Cancer Institute at St. Joseph's Hospital

Tampa, Florida, United States

Site Status

Kaplan Cancer Center at St. Mary's Medical Center

West Palm Beach, Florida, United States

Site Status

Winship Cancer Institute of Emory University

Atlanta, Georgia, United States

Site Status

Cancer Research Center of Hawaii

Honolulu, Hawaii, United States

Site Status

Advocate Christ Medical Center

Oak Lawn, Illinois, United States

Site Status

Indiana University Cancer Center

Indianapolis, Indiana, United States

Site Status

St. Vincent Indianapolis Hospital

Indianapolis, Indiana, United States

Site Status

Kansas Masonic Cancer Research Institute at the University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Kosair Children's Hospital

Louisville, Kentucky, United States

Site Status

Ochsner Cancer Institute at Ochsner Clinic Foundation

New Orleans, Louisiana, United States

Site Status

CancerCare of Maine at Eastern Maine Medial Center

Bangor, Maine, United States

Site Status

Alvin and Lois Lapidus Cancer Institute at Sinai Hospital

Baltimore, Maryland, United States

Site Status

Barbara Ann Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Hurley Medical Center

Flint, Michigan, United States

Site Status

Spectrum Health Hospital - Butterworth Campus

Grand Rapids, Michigan, United States

Site Status

Van Elslander Cancer Center at St. John Hospital and Medical Center

Grosse Pointe Woods, Michigan, United States

Site Status

Breslin Cancer Center at Ingham Regional Medical Center

Lansing, Michigan, United States

Site Status

Mayo Clinic Cancer Center

Rochester, Minnesota, United States

Site Status

University of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status

Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Siteman Cancer Center at Barnes-Jewish Hospital

St Louis, Missouri, United States

Site Status

UNMC Eppley Cancer Center at the University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

Sunrise Hospital and Medical Center

Las Vegas, Nevada, United States

Site Status

Hackensack University Medical Center Cancer Center

Hackensack, New Jersey, United States

Site Status

Overlook Hospital

Morristown, New Jersey, United States

Site Status

Saint Peter's University Hospital

New Brunswick, New Jersey, United States

Site Status

Cancer Institute of New Jersey at UMDNJ - Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

Site Status

University of New Mexico Cancer Research and Treatment Center

Albuquerque, New Mexico, United States

Site Status

Maimonides Cancer Center at Maimonides Medical Center

Brooklyn, New York, United States

Site Status

Herbert Irving Comprehensive Cancer Center at Columbia University

New York, New York, United States

Site Status

SUNY Upstate Medical University Hospital

Syracuse, New York, United States

Site Status

Albert Einstein Cancer Center at Albert Einstein College of Medicine

The Bronx, New York, United States

Site Status

New York Medical College

Valhalla, New York, United States

Site Status

Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Blumenthal Cancer Center at Carolinas Medical Center

Charlotte, North Carolina, United States

Site Status

Children's Hospital Medical Center of Akron

Akron, Ohio, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Rainbow Babies and Children's Hospital

Cleveland, Ohio, United States

Site Status

Columbus Children's Hospital

Columbus, Ohio, United States

Site Status

Children's Medical Center - Dayton

Dayton, Ohio, United States

Site Status

Tod Children's Hospital - Forum Health

Youngstown, Ohio, United States

Site Status

Oklahoma University Medical Center

Oklahoma City, Oklahoma, United States

Site Status

Penn State Cancer Institute at Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Hollings Cancer Center at Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Greenville Hospital System Cancer Center

Greenville, South Carolina, United States

Site Status

East Tennessee Children's Hospital

Knoxville, Tennessee, United States

Site Status

Texas Tech University Health Sciences Center School of Medicine - Amarillo

Amarillo, Texas, United States

Site Status

Children's Hospital of Austin

Austin, Texas, United States

Site Status

Medical City Dallas Hospital

Dallas, Texas, United States

Site Status

Cook Children's Medical Center - Fort Worth

Fort Worth, Texas, United States

Site Status

Baylor University Medical Center - Houston

Houston, Texas, United States

Site Status

University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

Site Status

Methodist Children's Hospital of South Texas

San Antonio, Texas, United States

Site Status

Primary Children's Medical Center

Salt Lake City, Utah, United States

Site Status

Children's Hospital and Regional Medical Center - Seattle

Seattle, Washington, United States

Site Status

West Virginia University - Robert C. Byrd Health Sciences Center - Charleston Division

Charleston, West Virginia, United States

Site Status

Edwards Comprehensive Cancer Center at Cabell Huntington Hospital

Huntington, West Virginia, United States

Site Status

Marshfield Clinic - Marshfield Center

Marshfield, Wisconsin, United States

Site Status

Midwest Children's Cancer Center

Milwaukee, Wisconsin, United States

Site Status

Princess Margaret Hospital for Children

Perth, Western Australia, Australia

Site Status

University of Alberta Hospital

Edmonton, Alberta, Canada

Site Status

Children's & Women's Hospital of British Columbia

Vancouver, British Columbia, Canada

Site Status

CancerCare Manitoba

Winnipeg, Manitoba, Canada

Site Status

IWK Health Centre

Halifax, Nova Scotia, Canada

Site Status

McMaster Children's Hospital at Hamilton Health Sciences

Hamilton, Ontario, Canada

Site Status

Cancer Centre of Southeastern Ontario at Kingston General Hospital

Kingston, Ontario, Canada

Site Status

Montreal Children's Hospital at McGill University Health Center

Montreal, Quebec, Canada

Site Status

Hopital Sainte Justine

Montreal, Quebec, Canada

Site Status

Centre Hospitalier Universitaire de Quebec

Québec, , Canada

Site Status

San Jorge Children's Hospital

Santurce, , Puerto Rico

Site Status

Countries

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United States Australia Canada Puerto Rico

References

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Mathew P, Gerbing R, Alonzo TA, Wallas T, Gong JZ, Jasty R, Jorstad DT, Raimondi SC, Chavez CM, Eisenberg NL, Hirsch B, Gamis A, Smith FO, Arceci RJ. A phase II study of amifostine in children with myelodysplastic syndrome: a report from the Children's Oncology Group study (AAML0121). Pediatr Blood Cancer. 2011 Dec 15;57(7):1230-2. doi: 10.1002/pbc.23164. Epub 2011 Jun 16.

Reference Type RESULT
PMID: 21681928 (View on PubMed)

Other Identifiers

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CDR0000398140

Identifier Type: OTHER

Identifier Source: secondary_id

COG-AAML0121

Identifier Type: OTHER

Identifier Source: secondary_id

AAML0121

Identifier Type: -

Identifier Source: org_study_id

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