Blood Cancer, Myelodysplastic Syndromes, Myelodysplastic Syndromes (MDS)
Conditions
Brief summary
The primary objectives of the trial are to assess erythroid response to darbepoetin alfa, as determined by changes in hemoglobin and/or red blood cell (RBC) transfusion-dependence and to describe the safety profile of darbepoetin alfa in patients with MDS. The secondary objective is to assess bone marrow progenitor BFU-E growth before and after treatment with darbepoetin alfa.
Interventions
DRUGDarbepoetin alfa
During the induction phase, the investigational agent DARBEPOETIN ALFA will be initiated at a dose of 4.5 ug/kg/week subcutaneously.If patients do not achieve a major erythroid response by 6 weeks, the dose of DARBEPOETIN ALFA will be doubled to 9.0 ug/kg/week.
Sponsors
Amgen
Peter L Greenberg
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
- Diagnosis: * Bone marrow aspirate/biopsy-proven MDS for \> 2 months prior to enrollment. * MDS French-American-British (FAB) subtypes refractory anemia (RA), RA with ringed sideroblasts (RARS), RA with excess blasts (RAEB), and non-proliferative chronic myelomonocytic leukemia (CMML) \[WBC \< 12,000/ml\]. * Patients must have an untransfused hemoglobin \< 10.0 g/dL and/or patients must be red cell transfusion-dependent for a period of at least 2 months prior to study entry. \- Laboratory: * Bilirubin \< or = to 2 mg/dL * ALT/SGPT \< or = to 2.5 x the upper limit of normal (ULN) * Normal renal function (Stanford: serum creatinine \< 1.2 mg/dL \[male\], \< 1.0 mg/dL \[female\]; Vanderbilt: \< 1.5 mg/dL). * Age: \> or = to 18 * Other: * ECOG performance status 0-2. * Patients may receive standard supportive care, including transfusions and antibiotics as required. * Patients must be r-HuEPO naive or must not have received prior treatment with r-HuEPO \> or = to 40,000 U/week for more than 4 weeks.
Exclusion criteria
- Patients with secondary MDS or prior allogeneic bone marrow transplant.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| hemoglobin and/or red blood cell (RBC) transfusion-dependence. | — |
| To assess erythroid responses to DARBEPOETIN ALFA, as determined by changes in | — |
Secondary
| Measure | Time frame |
|---|---|
| To assess bone marrow progenitor BFU-E growth before and after treatment | — |
| DARBEPOETIN ALFA | — |
Countries
United States
Outcome results
None listed