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A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study Evaluating Epoetin Alfa Versus Placebo in Anemic Patients With IPSS Low- or Intermediate-1-Risk Myelodysplastic Syndromes

Phase 3
18 Years
Open (Enrolling)
Myelodysplastic Syndromes

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Trial Information

A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study Evaluating Epoetin Alfa Versus Placebo in Anemic Patients With IPSS Low- or Intermediate-1-Risk Myelodysplastic Syndromes

This is a randomized (the treatment you receive will be assigned by chance), double-blind
(neither physician nor patient knows the name of the assigned drug), placebo-controlled
(comparison with patients that receive treatment without active ingredient), multicenter
study of epoetin alfa in anemic patients who are diagnosed with myelodysplastic syndromes
(MDS) according to protocol-specified criteria. This study includes a 2-week screening
phase, a 24-week treatment phase and a 24-week treatment extension phase. All patients
enrolled in the study will complete an end-of-study visit 4 weeks after the last dose of
study drug (Week 28 or Week 52), or 4 weeks after early withdrawal (unless the reason for
early withdrawal is withdrawal of consent). Between 125 and 159 patients will be enrolled in
the treatment phase of the study. During the screening phase, which will take place within 2
weeks before starting study drug, the study doctor will do tests to see if the patient is
suitable for this study. Patients meeting entry criteria for the study will then be randomly
assigned to one of the 2 treatment groups. This means that each patient who is allowed to
join the study is put into a group by chance, like flipping a coin. Group 1 patients will
receive epoetin alfa 450 or increased up to 1050 International Units (IU) per kg body weight
administered by subcutaneous injection (injection beneath the skin) using pre-filled
syringes. Injections will be done once every week at a weight-based dose regimen (the total
weekly dose received will depend on your weight) with a possible total maximum dose of
40,000 IU once every week for the first 8 weeks of the treatment phase and 80,000 IU once
every week at any other time during the study. Group 2 patients will receive a matching
volume of placebo administered once every week by subcutaneous injection. The chance that
the patient will get epoetin alfa is 2 to 1. Doses of study drug will be withheld,
decreased, or increased on the basis of erythroid response, weekly hemoglobin concentrations
monitored in patients and predefined dose adjustment guidelines. Patients will see the study
doctor every 4 weeks for a period of 24 weeks. At each visit the patient will undergo a full
hematologic evaluation, serum chemistry evaluation, measurement of blood pressure and pulse
rate, recording of blood product transfusions and transfusion complications, adverse events,
concomitant therapies and an evaluation for disease progression. The patient's Erythroid
response will be assessed at Week 8 and every 4 weeks thereafter, until Week 24. Blinded
study treatment will be administered to all patients at Week 24. However, at the end of the
treatment phase (after the Week 24 response assessment), only responders will enter the
double-blind treatment extension phase to measure the duration of response. Patients will
continue to receive the same treatment, in the same blinded fashion, and at the same dose as
received at Week 24, and will return to the study center every 4 weeks, until Week 48, for
assessment of the Erythroid response and the evaluations as described above. For all
non-responders at Week 24 the treatment code will be broken after Week 28 assessments. For
responders at Week 48, the treatment code will be broken after the Week 48 visit, following
completion of the response assessment. The treatment code will not be broken for subjects
who discontinue study treatment before Week 24, irrespective of whether they are responders
or nonresponders. For these subjects, the blind will not be broken until all subjects have
completed the study and the database is final. Once the patient stops receiving doses of
study drug, he/she will be asked to see the study doctor for the safety follow-up visit,
which is scheduled 4 weeks after the last dose of study drug. Safety will be monitored
throughout the study at predetermined intervals and as clinically indicated by physical
examination, laboratory tests and evaluation of adverse events. An Independent Data
Monitoring Committee (IDMC) will periodically review study data and for the assessment of
disease progression. The total duration of study participation will be for about 30 or 54

Inclusion Criteria:

- Diagnosis of MDS according to World Health Organization or French-American-British
pathologic classification (confirmed via bone marrow aspirate/biopsy) within 12 weeks
prior to screening

- Documentation of an International Prognostic Scoring System score indicating Low- or
Intermediate-1-risk disease within 12 weeks prior to screening

- Hemoglobin concentration at screening and baseline (before the first dose of study
drug) of 10.0 g/dL or less

- Screening serum erythropoietin concentration of less than 500 mU/mL

- Red Blood Cell transfusion requirement of less than or equal to 4 red blood cell
units over the last 8 weeks before randomization

Exclusion Criteria:

- Anemia attributed to factors other than MDS (including hemolysis, chronic renal
failure, hepatitis, gastrointestinal bleeding)

- Secondary MDS (ie, MDS arising after chemotherapy, immunotherapy or radiation

- History of malignancy, except in situ skin basal cell carcinoma or carcinoma in situ
of the cervix or breast curatively treated

- Prior therapy with any erythropoiesis-stimulating agent (ESA) (including innovative
ESAs and biosimilar ESAs for approved indications or for investigational use) in the
last 8 weeks before randomization

- Prior use of approved or experimental agents for the treatment of MDS

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Outcome Measure:

Erythroid response

Outcome Time Frame:

at week 24

Safety Issue:


Principal Investigator

Janssen-Cilag International NV Clinical Trial

Investigator Role:

Study Director

Investigator Affiliation:

Janssen-Cilag International NV


Germany: Ethics Commission

Study ID:




Start Date:

October 2011

Completion Date:

October 2015

Related Keywords:

  • Myelodysplastic Syndromes
  • Myelodysplastic Syndromes
  • Myeloproliferative Disorders
  • Bone Marrow disease
  • Anemia
  • Epoetin Alfa
  • Eprex
  • Erypo
  • Erythropoiesis-stimulating agent
  • Myelodysplastic Syndromes
  • Preleukemia