Prospective, Multicenter, Open Label and Single-arm Study of Darbepoetin Alfa for Anemia in Myelodisplastic Syndrome Patients.
18 Years
N/A
Both
Myelodysplastic Syndrome
Trial Information
Prospective, Multicenter, Open Label and Single-arm Study of Darbepoetin Alfa for Anemia in Myelodisplastic Syndrome Patients.
This is an open-label, single-arm, multicentre, prospective study of darbepoetin alfa to
treat anaemia in patients with low and intermediate-1 IPSS risk MDS. The study will consist
of a 14-day screening period followed by a maximum 24-week treatment period and a final
visit. Darbepoetin alfa will be initiated at a dose of 300 mcg QW SC over a period of 8
weeks. After 8 weeks, erythroid response will be evaluated, and treatment algorithm adapted
to it.
The study treatment period will last for a maximum of 24 weeks. The treatment will end at
the start of week 24. If the scheduled 24-week treatment period is not completed, it will
end during the week of the last administration of the study drug.
The follow-up period will last for a minimum of 4 weeks and a maximum of 8 weeks after the
last dose of darbepoetin alfa.
Subjects will be stratified at enrolment according to IPSS (low risk versus intermediate-1
risk).
Inclusion Criteria:
- Age ³ 18 years
- Low or intermediate-1 risk MDS according to IPSS, and FAB classification of RA, RARS,
or RAEB with blasts £ 10%
- Predictive variables of good response (serum erythropoietin levels < 500 IU/l and
transfusion requirements < 2 packed RBC/month over the preceding 2 months)
- Anaemia (Hb £ 10 g/dL), confirmed in the 14 days before day 1 of the study
- Life expectancy of at least 6 months
- ECOG Performance status score of 0, 1, or 2
- Subject must sign and date the Informed Consent (approved by a Clinical Research
Ethics Committee - CREC), before any study-specific procedure is performed
Exclusion Criteria:
- Known history of convulsive disorders
- Poorly controlled hypertension (diastolic blood pressure > 100 mmHg) at screening
- Inadequate liver function (total bilirubin > two times the upper limit of the normal
range (ULN), and liver enzymes (ALT, AST) > two times ULN)
- Inadequate renal function (serum creatinine concentration > 2 mg/dL)
- Ferritin < 100 ng/ml or transferrin saturation index (TSI) < 16%; Vitamin B12
deficiency (< 200 pg/ml) or folate deficiency (< 2 ng/ml)
- Clinically-relevant haemorrhages
- Haemolytic anaemia
- Cardiac condition: uncontrolled angina, congestive heart failure, or uncontrolled
cardiac arrhythmia
- Clinically significant systemic infection or chronic inflammatory disease present at
time of screening
- Any concomitant therapy used to treat MDS (including other growth factors than those
described as part of this protocol, chemotherapy, antibody-based cancer treatment,
hormonal therapy, interferon, and interleukins)
- Treatment with rHuEPO or darbepoetin alfa over the 4 weeks prior to Day 1 of the
study
- More than 2 RBC transfusions over the 28 days prior to Day 1 of the study
- Pregnant or breast feeding women
- Subjects of childbearing-potential who do not take adequate contraceptive measures,
in the opinion of the investigator
- Known hypersensitivity to any mammal-derived recombinant product
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Proportion of patients achieving an erythroid response during the 24-week treatment period.
Outcome Time Frame:
weeks 8; 12; 16 and 24
Safety Issue:
No
Principal Investigator
Ana M Villegas, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Fundacion para el Estudio de la Hematologia y Hemoterapia en Aragon
Authority:
Spain: Spanish Agency of Medicines
Study ID:
GEE200401
NCT ID:
NCT01039350
Start Date:
February 2006
Completion Date:
July 2009
Related Keywords:
- Myelodysplastic Syndrome
- Myelodysplastic syndrome
- Myelodysplastic Syndromes
- Preleukemia
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