Prospective, Multicenter, Open Label and Single-arm Study of Darbepoetin Alfa for Anemia in Myelodisplastic Syndrome Patients.

Fundacion para el Estudio de la Hematologia y Hemoterapia en Aragon

Status and phase

Terminated

Phase 2

Conditions

Myelodysplastic Syndrome

Treatments

Drug: Darbepoetin alfa

Study type

Interventional

Funder types

Other

Identifiers

NCT01039350

2005-002414-38 (EudraCT Number)

GEE200401

Details and patient eligibility

About

Full description

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).

Enrollment

80 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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