Clinical trial NCT00003827: Combination Chemotherapy in Treating Patients With Myelodysplastic Syndrome
Official title: Treatment of Poor Risk Myelodysplasia With the Combination of Amifostine, Topotecan and ARA-C: A Phase II Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Leukemia
Myelodysplastic Syndromes
Keywords: refractory anemia with excess blasts
refractory anemia with excess blasts in transformation
de novo myelodysplastic syndromes
secondary myelodysplastic syndromes
childhood myelodysplastic syndromes
Lead sponsor: National Cancer Institute (NCI)
Sponsor class: NIH
First posted: 2003-09-25
Enrollment: 25
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

Brief summary: RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. Chemoprotective drugs such as amifostine may protect normal cells from the side effects of chemotherapy.

PURPOSE: Phase II trial to study the effectiveness of combining topotecan and cytarabine given with amifostine in treating patients who have myelodysplastic syndrome.

Detailed description: OBJECTIVES:

* Determine the toxic effects of amifostine, topotecan, and cytarabine in patients with poor risk myelodysplastic syndrome.
* Determine the hematologic response rate, cytogenetic response rate, and the rate of polyclonal hematopoiesis following this treatment regimen.
* Determine the duration of response and time to disease progression following this treatment regimen in these patients.

OUTLINE: Patients receive topotecan by continuous IV over 24 hours plus cytarabine IV over 2 hours, on days 1-5. Patients receive amifostine IV over 15 minutes every other day for a maximum of 60 days. Patients may receive a second course of the same regimen 8 weeks after the first.

Patients are followed at least monthly for 2 years, then every 3-6 months until death.

PROJECTED ACCRUAL: Approximately 25 patients will be accrued for this study within 1 to 1.5 years.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Histologically confirmed poor risk myelodysplastic syndrome, including at least one of the following:

  * Bilineage cytopenia
  * Unfavorable cytogenetic abnormalities
  * Refractory anemia with excess blasts and/or refractory anemia with excess blast in transformation (greater than 5% blast)
* At least 0.5 on the International Prognostic Score System
* No chronic myelomonocytic leukemia
* No hypocellular myelodysplastic syndrome (marrow cellularity less than 30%)

PATIENT CHARACTERISTICS:

Age:

* 16 and over

Performance status:

* ECOG 0-1

Life expectancy:

* Not specified

Hematopoietic:

* Absolute neutrophil count less than 1,500/mm3
* Platelet count less than 100,000/mm3
* Hemoglobin less than 10 g/dL

Hepatic:

* ALT less than 5 times upper limit of normal

Renal:

* Creatinine no greater than 1.4 mg/dL

Cardiovascular:

* No congestive heart failure

Other:

* Not pregnant or nursing
* Fertile patients must use effective contraception
* Must have right atrial catheter inserted

PRIOR CONCURRENT THERAPY:

Biologic therapy:

* No prior blood or bone marrow transplantations

Chemotherapy:

* No prior acute myeloid leukemia chemotherapy (except hydroxyurea or low dose cytarabine)
* No prior topotecan
* No prior amifostine

Endocrine therapy:

* Not specified

Radiotherapy:

* Not specified

Surgery:

* Not specified

Other:

* At least 24 hours since prior antihypertensive medication prior to amifostine

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Duarte
country: United States
facility: Cancer Center and Beckman Research Institute, City of Hope
state: California
Study officials:
affiliation: City of Hope Comprehensive Cancer Center
name: Henry C. Fung, MD, FRCPE
role: STUDY_CHAIR
References: