Prospective, Multicenter, Open Label and Single-arm Study of Darbepoetin Alfa for Anemia in Myelodisplastic Syndrome Patients.
- Conditions
- Myelodysplastic Syndrome
- Registration Number
- NCT01039350
- Lead Sponsor
- Fundacion para el Estudio de la Hematologia y Hemoterapia en Aragon
- Brief Summary
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.
- Detailed 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).
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 80
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Proportion of patients achieving an erythroid response during the 24-week treatment period. weeks 8; 12; 16 and 24
- Secondary Outcome Measures
Name Time Method Time to erythroid response and time it is maintained. week 24 Proportion of non-responders to darbepoetin alfa who obtain an erythroid response after the addition of Filgrastim weeks 8, 12, 16 and 24 Proportion of patients receiving RBC transfusions (more than 1 unit) from week 5 to 24, inclusive weeks 8; 12; 16 and 24 Score changes in the FACT-Fatigue quality-of-life scale between the baseline visit, and weeks 8, 16, 24, and the end of the study. weeks 8; 16 and 24 Number of morphological and cytogenetic disorders at baseline and end of treatment week 24 Incidence of adverse events and serious adverse events weeks 8; 12; 16 and 24 Proportion of patients with haemoglobin values over 12 g/dL at any time during the study weeks 8; 12; 16 and 24
Trial Locations
- Locations (16)
Hospital de Cruces
🇪🇸Barakaldo, Bilbao, Spain
Hospital Virgen del Puerto
🇪🇸Plasencia, Caceres, Spain
Hospital General Universitario de Alicante
🇪🇸Alicante, Spain
Hospital Central de Asturias
🇪🇸Oviedo, Asturias, Spain
Hospital Duran i Reynals
🇪🇸Barcelona, Spain
Hospital Ramón y Cajal
🇪🇸Madrid, Spain
Hospital General Yagüe
🇪🇸Burgos, Spain
Hospital de la Santa Creu i Sant Pau
🇪🇸Barcelona, Spain
Hospital Vall D´Hebron
🇪🇸Barcelona, Spain
Hospital Clinic i Provincial de Barcelona
🇪🇸Barcelona, Spain
Hospital Universitario Doce de Octubre
🇪🇸Madrid, Spain
Hospital Universitario Puerta del Mar
🇪🇸Cádiz, Spain
Complejo Hospitalario Universitario Juan Canalejo
🇪🇸La Coruña, Spain
Hospital Universitario La Fé
🇪🇸Valencia, Spain
Hospital Universitario de Salamanca
🇪🇸Salamanca, Spain
Hospital Arnau de Vilanova
🇪🇸Valencia, Spain