A Phase II Study Of Amifostine In Children With Myelodysplastic Syndrome
1 Year
21 Years
Both
Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Trial Information
A Phase II Study Of Amifostine In Children With Myelodysplastic Syndrome
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.
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
Type of Study:
Interventional
Study Design:
Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Hematological effects (complete and partial response)
Safety Issue:
No
Principal Investigator
Prasad Mathew, MD
Investigator Role:
Study Chair
Investigator Affiliation:
University of New Mexico Cancer Center
Authority:
United States: Federal Government
Study ID:
CDR0000398140
NCT ID:
NCT00098683
Start Date:
January 2005
Completion Date:
Related Keywords:
- Myelodysplastic Syndromes
- Myelodysplastic/Myeloproliferative Neoplasms
- 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
- Neoplasms
- Myelodysplastic Syndromes
- Preleukemia
- Myeloproliferative Disorders
- Myelodysplastic-Myeloproliferative Diseases
Name | Location |
|---|---|
| Children's Hospital of Philadelphia | Philadelphia, Pennsylvania 19104 |
| Mayo Clinic Cancer Center | Rochester, Minnesota 55905 |
| Indiana University Cancer Center | Indianapolis, Indiana 46202-5265 |
| Barbara Ann Karmanos Cancer Institute | Detroit, Michigan 48201 |
| University of Mississippi Medical Center | Jackson, Mississippi 39216-4505 |
| Hurley Medical Center | Flint, Michigan 48503 |
| Medical City Dallas Hospital | Dallas, Texas 75230 |
| University of Texas Health Science Center at San Antonio | San Antonio, Texas 78284-7811 |
| Midwest Children's Cancer Center | Milwaukee, Wisconsin 53226 |
| Carole and Ray Neag Comprehensive Cancer Center at the University of Connecticut Health Center | Farmington, Connecticut 06360-2875 |
| Van Elslander Cancer Center at St. John Hospital and Medical Center | Grosse Pointe Woods, Michigan 48236 |
| Penn State Cancer Institute at Milton S. Hershey Medical Center | Hershey, Pennsylvania 17033-0850 |
| Marshfield Clinic - Marshfield Center | Marshfield, Wisconsin 54449 |
| New York Medical College | Valhalla, New York 10595 |
| Siteman Cancer Center at Barnes-Jewish Hospital | Saint Louis, Missouri 63110 |
| Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill | Chapel Hill, North Carolina 27599-7570 |
| Cancer Research Center of Hawaii | Honolulu, Hawaii 96813 |
| Children's Mercy Hospital | Kansas City, Missouri 64108 |
| Children's Hospital of Pittsburgh | Pittsburgh, Pennsylvania 15213 |
| Children's Hospital and Regional Medical Center - Seattle | Seattle, Washington 98105 |
| Miami Children's Hospital | Miami, Florida 33155-4069 |
| All Children's Hospital | St. Petersburg, Florida 33701 |
| University of New Mexico Cancer Research and Treatment Center | Albuquerque, New Mexico 87131 |
| St. Christopher's Hospital for Children | Philadelphia, Pennsylvania 19134-1095 |
| Cook Children's Medical Center - Fort Worth | Fort Worth, Texas 76104 |
| Phoenix Children's Hospital | Phoenix, Arizona 85016-7710 |
| Children's Hospital Central California | Madera, California 93638-8762 |
| Kosair Children's Hospital | Louisville, Kentucky 40202-3830 |
| Sunrise Hospital and Medical Center | Las Vegas, Nevada 89109-2306 |
| Children's Hospital Medical Center of Akron | Akron, Ohio 44308 |
| Children's Medical Center - Dayton | Dayton, Ohio 45404 |
| East Tennessee Children's Hospital | Knoxville, Tennessee 37901 |
| Children's Hospital of Austin | Austin, Texas 78701 |
| Arkansas Cancer Research Center at University of Arkansas for Medical Sciences | Little Rock, Arkansas 72205 |
| Kansas Masonic Cancer Research Institute at the University of Kansas Medical Center | Kansas City, Kansas 66160-7353 |
| Herbert Irving Comprehensive Cancer Center at Columbia University | New York, New York 10032 |
| Blumenthal Cancer Center at Carolinas Medical Center | Charlotte, North Carolina 28232-2861 |
| Overlook Hospital | Summit, New Jersey 07902-0220 |
| Oklahoma University Medical Center | Oklahoma City, Oklahoma 73104 |
| Jonathan Jaques Children's Cancer Center at Miller Children's Hospital | Long Beach, California 90801 |
| Kaiser Permanente Medical Center - Oakland | Sacramento, California 95825 |
| Lee Cancer Care of Lee Memorial Health System | Fort Myers, Florida 33901 |
| University of Florida Shands Cancer Center | Gainesville, Florida 32610-0232 |
| Sacred Heart Cancer Center at Sacred Heart Hospital | Pensacola, Florida 32504 |
| St. Joseph's Cancer Institute at St. Joseph's Hospital | Tampa, Florida 33607 |
| Kaplan Cancer Center at St. Mary's Medical Center | West Palm Beach, Florida 33407 |
| Winship Cancer Institute of Emory University | Atlanta, Georgia 30322 |
| Advocate Christ Medical Center | Oak Lawn, Illinois 60453 |
| St. Vincent Indianapolis Hospital | Indianapolis, Indiana 46260 |
| Ochsner Cancer Institute at Ochsner Clinic Foundation | New Orleans, Louisiana 70121 |
| CancerCare of Maine at Eastern Maine Medial Center | Bangor, Maine 04401 |
| Alvin and Lois Lapidus Cancer Institute at Sinai Hospital | Baltimore, Maryland 21215 |
| Breslin Cancer Center at Ingham Regional Medical Center | Lansing, Michigan 48910 |
| Hackensack University Medical Center Cancer Center | Hackensack, New Jersey 07601 |
| Albert Einstein Cancer Center at Albert Einstein College of Medicine | Bronx, New York 10461 |
| SUNY Upstate Medical University Hospital | Syracuse, New York 13210 |
| Cincinnati Children's Hospital Medical Center | Cincinnati, Ohio 45229-3039 |
| Rainbow Babies and Children's Hospital | Cleveland, Ohio 44106-5000 |
| Tod Children's Hospital - Forum Health | Youngstown, Ohio 44501 |
| Hollings Cancer Center at Medical University of South Carolina | Charleston, South Carolina 29425 |
| Greenville Hospital System Cancer Center | Greenville, South Carolina 29605 |
| Texas Tech University Health Sciences Center School of Medicine - Amarillo | Amarillo, Texas 79106 |
| Baylor University Medical Center - Houston | Houston, Texas 77030-2399 |
| Methodist Children's Hospital of South Texas | San Antonio, Texas 78229-3993 |
| Primary Children's Medical Center | Salt Lake City, Utah 84113-1100 |
| West Virginia University - Robert C. Byrd Health Sciences Center - Charleston Division | Charleston, West Virginia 25302 |
| Edwards Comprehensive Cancer Center at Cabell Huntington Hospital | Huntington, West Virginia 25701 |
| Saint Peter's University Hospital | New Brunswick, New Jersey 08901-1780 |
| UNMC Eppley Cancer Center at the University of Nebraska Medical Center | Omaha, Nebraska 68198-7680 |
| Memorial Cancer Institute at Memorial Regional Hospital | Hollywood, Florida 33021 |
| Cancer Institute of New Jersey at UMDNJ - Robert Wood Johnson Medical School | New Brunswick, New Jersey 08903 |
| Maimonides Cancer Center at Maimonides Medical Center | Brooklyn, New York 11219 |
| Spectrum Health Hospital - Butterworth Campus | Grand Rapids, Michigan 49503 |
| Columbus Children's Hospital | Columbus, Ohio 43205-2696 |
