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

This study has been terminated.
(It was stopped due to a lack of recruitment after 48 patients included)
Sponsor:
Information provided by:
Fundacion para el Estudio de la Hematologia y Hemoterapia en Aragon
ClinicalTrials.gov Identifier:
NCT01039350
First received: December 23, 2009
Last updated: January 12, 2010
Last verified: January 2010
  Purpose

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.


Condition Intervention Phase
Myelodysplastic Syndrome
Drug: Darbepoetin alfa
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Prospective, Multicenter, Open Label and Single-arm Study of Darbepoetin Alfa for Anemia in Myelodisplastic Syndrome Patients.

Resource links provided by NLM:


Further study details as provided by Fundacion para el Estudio de la Hematologia y Hemoterapia en Aragon:

Primary Outcome Measures:
  • Proportion of patients achieving an erythroid response during the 24-week treatment period. [ Time Frame: weeks 8; 12; 16 and 24 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Time to erythroid response and time it is maintained. [ Time Frame: week 24 ] [ Designated as safety issue: No ]
  • Proportion of non-responders to darbepoetin alfa who obtain an erythroid response after the addition of Filgrastim [ Time Frame: weeks 8, 12, 16 and 24 ] [ Designated as safety issue: No ]
  • Proportion of patients receiving RBC transfusions (more than 1 unit) from week 5 to 24, inclusive [ Time Frame: weeks 8; 12; 16 and 24 ] [ Designated as safety issue: No ]
  • 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. [ Time Frame: weeks 8; 16 and 24 ] [ Designated as safety issue: No ]
  • Number of morphological and cytogenetic disorders at baseline and end of treatment [ Time Frame: week 24 ] [ Designated as safety issue: No ]
  • Incidence of adverse events and serious adverse events [ Time Frame: weeks 8; 12; 16 and 24 ] [ Designated as safety issue: Yes ]
  • Proportion of patients with haemoglobin values over 12 g/dL at any time during the study [ Time Frame: weeks 8; 12; 16 and 24 ] [ Designated as safety issue: Yes ]

Enrollment: 80
Study Start Date: February 2006
Estimated Study Completion Date: July 2009
Estimated Primary Completion Date: July 2008 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Darbepoetin alfa
    Darbepoetin alfa will be initiated at a weekly (QW) subcutaneous dose of 300 mcg over 8 weeks.
    Other Name: Darbepoetin alfa
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).

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01039350

Locations
Spain
Hospital Central de Asturias
Oviedo, Asturias, Spain, 33006
Hospital de Cruces
Barakaldo, Bilbao, Spain, 48903
Hospital Virgen del Puerto
Plasencia, Caceres, Spain, 10600
Hospital General Universitario de Alicante
Alicante, Spain, 03010
Hospital Clinic i Provincial de Barcelona
Barcelona, Spain, 08036
Hospital Duran i Reynals
Barcelona, Spain, 08907
Hospital Vall D´Hebron
Barcelona, Spain, 08035
Hospital de la Santa Creu i Sant Pau
Barcelona, Spain, 08025
Hospital General Yagüe
Burgos, Spain, 09005
Hospital Universitario Puerta del Mar
Cádiz, Spain, 11009
Complejo Hospitalario Universitario Juan Canalejo
La Coruña, Spain, 15006
Hospital Universitario Doce de Octubre
Madrid, Spain, 28041
Hospital Ramón y Cajal
Madrid, Spain, 28034
Hospital Universitario de Salamanca
Salamanca, Spain, 37007
Hospital Arnau de Vilanova
Valencia, Spain, 46015
Hospital Universitario La Fé
Valencia, Spain, 46009
Sponsors and Collaborators
Fundacion para el Estudio de la Hematologia y Hemoterapia en Aragon
Investigators
Principal Investigator: Ana M Villegas, MD Fundacion para el Estudio de la Hematologia y Hemoterapia en Aragon
  More Information

Publications:
Hellstrom-Lindberg E. Growth factor treatment for the anemia of MDS: mechanisms and efficacy. Educational Programme at the 8th Congress of the European Haematology Association (EHA), June 2003
Verhoef GEG, Boogaerts MA. RHuEPO in the treatment of the myelodysplastic syndromes. In: Smyth JF, Boogaerts MA, Ehmer BR-M (eds): rHuEPO in cancer supportive treatment. Marcel Dekker, New York 1996;pp.13-34.
Glaspy J, Appelbaum S, Henry D et al. Effects of darbepoetin alfa (Aranesp®) timing with chemotherapy administration: a randomized study. SIOG 2003. Poster.

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Ana M Villegas, FEHH
ClinicalTrials.gov Identifier: NCT01039350     History of Changes
Other Study ID Numbers: GEE200401, 2005-002414-38
Study First Received: December 23, 2009
Last Updated: January 12, 2010
Health Authority: Spain: Spanish Agency of Medicines

Keywords provided by Fundacion para el Estudio de la Hematologia y Hemoterapia en Aragon:
Myelodysplastic syndrome

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Darbepoetin alfa
Hematinics
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 22, 2014