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

NCT00098683 | Completed Phase 2 DMC

• 3 other identifiers: AAML0121CDR0000398140COG-AAML0121
• Study Type: interventional
• Target: 10
• Locations: 4 countries
• Sites: 85

Brief Summary

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.

Conditions

Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms

Keywords

refractory anemia with excess blasts; refractory anemia; refractory anemia with ringed sideroblasts; de novo myelodysplastic syndromes; myelodysplastic/myeloproliferative neoplasm, unclassifiable; refractory cytopenia with multilineage dysplasia

Outcome Measures

Primary Outcomes (2)

• Hematological effects (complete and partial response)

• Safety and efficacy

Secondary Outcomes (5)

• 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

Interventions

amifostine trihydrate DRUG

Eligibility Criteria

• Age: 1 Year - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Children's Oncology Group: lead
• National Cancer Institute (NCI): collaborator

Study Sites (85)

Phoenix Children's Hospital

Phoenix, Arizona, 85016-7710, United States

Location

Arkansas Cancer Research Center at University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

Location

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

Long Beach, California, 90801, United States

Location

Children's Hospital Central California

Madera, California, 93638-8762, United States

Location

Kaiser Permanente Medical Center - Oakland

Sacramento, California, 95825, United States

Location

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

Farmington, Connecticut, 06360-2875, United States

Location

Lee Cancer Care of Lee Memorial Health System

Fort Myers, Florida, 33901, United States

Location

University of Florida Shands Cancer Center

Gainesville, Florida, 32610-0232, United States

Location

Memorial Cancer Institute at Memorial Regional Hospital

Hollywood, Florida, 33021, United States

Location

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

Sacred Heart Cancer Center at Sacred Heart Hospital

Pensacola, Florida, 32504, United States

Location

All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

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

Tampa, Florida, 33607, United States

Location

Kaplan Cancer Center at St. Mary's Medical Center

West Palm Beach, Florida, 33407, United States

Location

Winship Cancer Institute of Emory University

Atlanta, Georgia, 30322, United States

Location

Cancer Research Center of Hawaii

Honolulu, Hawaii, 95813, United States

Location

Advocate Christ Medical Center

Oak Lawn, Illinois, 60453, United States

Location

Indiana University Cancer Center

Indianapolis, Indiana, 46202-5289, United States

Location

St. Vincent Indianapolis Hospital

Indianapolis, Indiana, 46260, United States

Location

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

Kansas City, Kansas, 66160-7357, United States

Location

Kosair Children's Hospital

Louisville, Kentucky, 40232, United States

Location

Ochsner Cancer Institute at Ochsner Clinic Foundation

New Orleans, Louisiana, 70121, United States

Location

CancerCare of Maine at Eastern Maine Medial Center

Bangor, Maine, 04401, United States

Location

Alvin and Lois Lapidus Cancer Institute at Sinai Hospital

Baltimore, Maryland, 21215, United States

Location

Barbara Ann Karmanos Cancer Institute

Detroit, Michigan, 48201-1379, United States

Location

Hurley Medical Center

Flint, Michigan, 48503, United States

Location

Spectrum Health Hospital - Butterworth Campus

Grand Rapids, Michigan, 49503-2560, United States

Location

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

Grosse Pointe Woods, Michigan, 48236, United States

Location

Breslin Cancer Center at Ingham Regional Medical Center

Lansing, Michigan, 48910, United States

Location

Mayo Clinic Cancer Center

Rochester, Minnesota, 55905, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216-4505, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Siteman Cancer Center at Barnes-Jewish Hospital

St Louis, Missouri, 63110, United States

Location

UNMC Eppley Cancer Center at the University of Nebraska Medical Center

Omaha, Nebraska, 68198-6805, United States

Location

Sunrise Hospital and Medical Center

Las Vegas, Nevada, 89109-2306, United States

Location

Hackensack University Medical Center Cancer Center

Hackensack, New Jersey, 07601, United States

Location

Overlook Hospital

Morristown, New Jersey, 07962, United States

Location

Saint Peter's University Hospital

New Brunswick, New Jersey, 08901, United States

Location

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

New Brunswick, New Jersey, 08903, United States

Location

University of New Mexico Cancer Research and Treatment Center

Albuquerque, New Mexico, 87131-5636, United States

Location

Maimonides Cancer Center at Maimonides Medical Center

Brooklyn, New York, 11219, United States

Location

Herbert Irving Comprehensive Cancer Center at Columbia University

New York, New York, 10032, United States

Location

SUNY Upstate Medical University Hospital

Syracuse, New York, 13210, United States

Location

Albert Einstein Cancer Center at Albert Einstein College of Medicine

The Bronx, New York, 10461, United States

Location

New York Medical College

Valhalla, New York, 10595, United States

Location

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

Chapel Hill, North Carolina, 27599-7295, United States

Location

Blumenthal Cancer Center at Carolinas Medical Center

Charlotte, North Carolina, 28232-2861, United States

Location

Children's Hospital Medical Center of Akron

Akron, Ohio, 44308-1062, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

Location

Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106-5000, United States

Location

Columbus Children's Hospital

Columbus, Ohio, 43205-2696, United States

Location

Children's Medical Center - Dayton

Dayton, Ohio, 45404-1815, United States

Location

Tod Children's Hospital - Forum Health

Youngstown, Ohio, 44501, United States

Location

Oklahoma University Medical Center

Oklahoma City, Oklahoma, 73104, United States

Location

Penn State Cancer Institute at Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033-0850, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104-9786, United States

Location

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134-1095, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Hollings Cancer Center at Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Greenville Hospital System Cancer Center

Greenville, South Carolina, 29605, United States

Location

East Tennessee Children's Hospital

Knoxville, Tennessee, 37916, United States

Location

Texas Tech University Health Sciences Center School of Medicine - Amarillo

Amarillo, Texas, 79106, United States

Location

Children's Hospital of Austin

Austin, Texas, 78701, United States

Location

Medical City Dallas Hospital

Dallas, Texas, 75230, United States

Location

Cook Children's Medical Center - Fort Worth

Fort Worth, Texas, 76104-9958, United States

Location

Baylor University Medical Center - Houston

Houston, Texas, 77030-2399, United States

Location

University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78207, United States

Location

Methodist Children's Hospital of South Texas

San Antonio, Texas, 78229-3993, United States

Location

Primary Children's Medical Center

Salt Lake City, Utah, 84113-1100, United States

Location

Children's Hospital and Regional Medical Center - Seattle

Seattle, Washington, 98105, United States

Location

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

Charleston, West Virginia, 25302, United States

Location

Edwards Comprehensive Cancer Center at Cabell Huntington Hospital

Huntington, West Virginia, 25701, United States

Location

Marshfield Clinic - Marshfield Center

Marshfield, Wisconsin, 54449, United States

Location

Midwest Children's Cancer Center

Milwaukee, Wisconsin, 53226, United States

Location

Princess Margaret Hospital for Children

Perth, Western Australia, 6001, Australia

Location

University of Alberta Hospital

Edmonton, Alberta, T6G 1Z2, Canada

Location

Children's & Women's Hospital of British Columbia

Vancouver, British Columbia, V6H 3V4, Canada

Location

CancerCare Manitoba

Winnipeg, Manitoba, R3E 0V9, Canada

Location

IWK Health Centre

Halifax, Nova Scotia, B3K 6R8, Canada

Location

McMaster Children's Hospital at Hamilton Health Sciences

Hamilton, Ontario, L8N 3Z5, Canada

Location

Cancer Centre of Southeastern Ontario at Kingston General Hospital

Kingston, Ontario, K7L 3N6, Canada

Location

Montreal Children's Hospital at McGill University Health Center

Montreal, Quebec, H3H 1P3, Canada

Location

Hopital Sainte Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Centre Hospitalier Universitaire de Quebec

Québec, G1V 4G2, Canada

Location

San Jorge Children's Hospital

Santurce, 00912, Puerto Rico

Location

Related Publications (1)

• 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.

  PMID: 21681928 RESULT

MeSH Terms

Conditions

Myelodysplastic Syndromes; Myelodysplastic-Myeloproliferative Diseases; Anemia, Refractory, with Excess of Blasts; Anemia, Refractory; Myeloproliferative Disorders

Interventions

Amifostine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia

Intervention Hierarchy (Ancestors)

Organothiophosphates; Organophosphates; Organophosphorus Compounds; Organic Chemicals; Organothiophosphorus Compounds; Sulfur Compounds

Study Officials

• Prasad Mathew, MD

  University of New Mexico Cancer Center

  STUDY CHAIR

• Robert J. Arceci, MD, PhD

  Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Design

• Study Type: interventional
• Phase: phase 2
• Masking: NONE
• Purpose: TREATMENT
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 7, 2004
• First Posted: December 8, 2004
• Study Start: January 1, 2005
• Primary Completion: October 1, 2007
• Study Completion: October 1, 2009
• Last Updated: February 13, 2014

Record last verified: 2014-02

Locations

AU (1); US (74); PR (1); CA (9)