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A Phase II Study Of Amifostine In Children With Myelodysplastic Syndrome


Phase 2
1 Year
21 Years
Not Enrolling
Both
Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms

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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 PhiladelphiaPhiladelphia, Pennsylvania  19104
Mayo Clinic Cancer CenterRochester, Minnesota  55905
Indiana University Cancer CenterIndianapolis, Indiana  46202-5265
Barbara Ann Karmanos Cancer InstituteDetroit, Michigan  48201
University of Mississippi Medical CenterJackson, Mississippi  39216-4505
Hurley Medical CenterFlint, Michigan  48503
Medical City Dallas HospitalDallas, Texas  75230
University of Texas Health Science Center at San AntonioSan Antonio, Texas  78284-7811
Midwest Children's Cancer CenterMilwaukee, Wisconsin  53226
Carole and Ray Neag Comprehensive Cancer Center at the University of Connecticut Health CenterFarmington, Connecticut  06360-2875
Van Elslander Cancer Center at St. John Hospital and Medical CenterGrosse Pointe Woods, Michigan  48236
Penn State Cancer Institute at Milton S. Hershey Medical CenterHershey, Pennsylvania  17033-0850
Marshfield Clinic - Marshfield CenterMarshfield, Wisconsin  54449
New York Medical CollegeValhalla, New York  10595
Siteman Cancer Center at Barnes-Jewish HospitalSaint Louis, Missouri  63110
Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel HillChapel Hill, North Carolina  27599-7570
Cancer Research Center of HawaiiHonolulu, Hawaii  96813
Children's Mercy HospitalKansas City, Missouri  64108
Children's Hospital of PittsburghPittsburgh, Pennsylvania  15213
Children's Hospital and Regional Medical Center - SeattleSeattle, Washington  98105
Miami Children's HospitalMiami, Florida  33155-4069
All Children's HospitalSt. Petersburg, Florida  33701
University of New Mexico Cancer Research and Treatment CenterAlbuquerque, New Mexico  87131
St. Christopher's Hospital for ChildrenPhiladelphia, Pennsylvania  19134-1095
Cook Children's Medical Center - Fort WorthFort Worth, Texas  76104
Phoenix Children's HospitalPhoenix, Arizona  85016-7710
Children's Hospital Central CaliforniaMadera, California  93638-8762
Kosair Children's HospitalLouisville, Kentucky  40202-3830
Sunrise Hospital and Medical CenterLas Vegas, Nevada  89109-2306
Children's Hospital Medical Center of AkronAkron, Ohio  44308
Children's Medical Center - DaytonDayton, Ohio  45404
East Tennessee Children's HospitalKnoxville, Tennessee  37901
Children's Hospital of AustinAustin, Texas  78701
Arkansas Cancer Research Center at University of Arkansas for Medical SciencesLittle Rock, Arkansas  72205
Kansas Masonic Cancer Research Institute at the University of Kansas Medical CenterKansas City, Kansas  66160-7353
Herbert Irving Comprehensive Cancer Center at Columbia UniversityNew York, New York  10032
Blumenthal Cancer Center at Carolinas Medical CenterCharlotte, North Carolina  28232-2861
Overlook HospitalSummit, New Jersey  07902-0220
Oklahoma University Medical CenterOklahoma City, Oklahoma  73104
Jonathan Jaques Children's Cancer Center at Miller Children's HospitalLong Beach, California  90801
Kaiser Permanente Medical Center - OaklandSacramento, California  95825
Lee Cancer Care of Lee Memorial Health SystemFort Myers, Florida  33901
University of Florida Shands Cancer CenterGainesville, Florida  32610-0232
Sacred Heart Cancer Center at Sacred Heart HospitalPensacola, Florida  32504
St. Joseph's Cancer Institute at St. Joseph's HospitalTampa, Florida  33607
Kaplan Cancer Center at St. Mary's Medical CenterWest Palm Beach, Florida  33407
Winship Cancer Institute of Emory UniversityAtlanta, Georgia  30322
Advocate Christ Medical CenterOak Lawn, Illinois  60453
St. Vincent Indianapolis HospitalIndianapolis, Indiana  46260
Ochsner Cancer Institute at Ochsner Clinic FoundationNew Orleans, Louisiana  70121
CancerCare of Maine at Eastern Maine Medial CenterBangor, Maine  04401
Alvin and Lois Lapidus Cancer Institute at Sinai HospitalBaltimore, Maryland  21215
Breslin Cancer Center at Ingham Regional Medical CenterLansing, Michigan  48910
Hackensack University Medical Center Cancer CenterHackensack, New Jersey  07601
Albert Einstein Cancer Center at Albert Einstein College of MedicineBronx, New York  10461
SUNY Upstate Medical University HospitalSyracuse, New York  13210
Cincinnati Children's Hospital Medical CenterCincinnati, Ohio  45229-3039
Rainbow Babies and Children's HospitalCleveland, Ohio  44106-5000
Tod Children's Hospital - Forum HealthYoungstown, Ohio  44501
Hollings Cancer Center at Medical University of South CarolinaCharleston, South Carolina  29425
Greenville Hospital System Cancer CenterGreenville, South Carolina  29605
Texas Tech University Health Sciences Center School of Medicine - AmarilloAmarillo, Texas  79106
Baylor University Medical Center - HoustonHouston, Texas  77030-2399
Methodist Children's Hospital of South TexasSan Antonio, Texas  78229-3993
Primary Children's Medical CenterSalt Lake City, Utah  84113-1100
West Virginia University - Robert C. Byrd Health Sciences Center - Charleston DivisionCharleston, West Virginia  25302
Edwards Comprehensive Cancer Center at Cabell Huntington HospitalHuntington, West Virginia  25701
Saint Peter's University HospitalNew Brunswick, New Jersey  08901-1780
UNMC Eppley Cancer Center at the University of Nebraska Medical CenterOmaha, Nebraska  68198-7680
Memorial Cancer Institute at Memorial Regional HospitalHollywood, Florida  33021
Cancer Institute of New Jersey at UMDNJ - Robert Wood Johnson Medical SchoolNew Brunswick, New Jersey  08903
Maimonides Cancer Center at Maimonides Medical CenterBrooklyn, New York  11219
Spectrum Health Hospital - Butterworth CampusGrand Rapids, Michigan  49503
Columbus Children's HospitalColumbus, Ohio  43205-2696