Clinical trial NCT02145026: A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)
Official title: A Prospective Open-Label Study of the Effectiveness of Epoetin Beta for Treating Anemic Patients With Low/Intermediate-1-Risk Myelodysplastic Syndrome (MDS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Myelodysplastic Syndromes
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2014-05-22
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a Phase IV, prospective, multi-center, open-label study to assess the effectiveness and safety profile of epoetin beta (Recormon®) for treatment of symptomatic anemia in adult participants associated with low/intermediate-1-risk MDS. After screening, eligible participants will be treated with epoetin beta as recommended in the approved label and international guidelines for the use of epoetin in MDS participants and the dosage will be adjusted on the basis of erythroid response.


Eligibility criteria: Inclusion Criteria:

* Adult participants with low or intermediate-1 risk MDS
* No previous treatment with hematopoietic growth factors within 3 months prior to screening
* Symptomatic anemia (hemoglobin \<10 g/dL) as determined by investigator
* Serum erythropoietin \<500 milliunits/milliliter (mU/mL) within 14 days prior to the first dose of study treatment
* Require no red blood cell transfusion or dependent on \<4 units within 8 weeks prior to screening
* Clinically stable for at least 1 month prior to entry into the study
* For female participants of childbearing potential and male participants with partners of childbearing potential, agreement (by participants and/or partner) to use highly effective form(s) of contraception

Exclusion Criteria:

* Contraindications and/or known hypersensitivity to the active substance and/or any of the excipients of epoetin beta treatment
* Poorly controlled hypertension as assessed by the investigator
* History of Acute Myeloid Leukemia (AML) or high risk for AML
* Administration of another investigational drug within 1 month before screening or planned during the study period
* Previously documented evidence of Pure Red Cell Aplasia (PRCA)

Primary outcomes:
measure: Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria
timeFrame: Week 12
Secondary outcomes:
measure: Percentage of Participants With Platelet Response (in Participants With Pre-Treatment Platelets <100*10^9 Per Liter) at Week 12 as Assessed by IWG 2006 Response Criteria
timeFrame: Week 12
measure: Percentage of Participants With Neutrophil Response (in Participants With Pre-Treatment Neutrophil <1.0*10^9 Per Liter) at Week 12 as Assessed by IWG 2006 Response Criteria
timeFrame: Week 12
measure: Percentage of Participants With Adverse Events
timeFrame: From signing of informed consent up to 4 weeks after last dose (up to 18 weeks)
Other outcomes:
Locations:
city: Bangkok
country: Thailand
facility: King Chulalongkorn Memorial Hospital; Division of Hematology, Department of Medicine
city: Bangkok
country: Thailand
facility: Rajavithi Hospital; Medicine
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital; Division of Hematology, Department of Medicine
city: Bangkok
country: Thailand
facility: Siriraj Hospital; Division of Hematology, Department of Medicine
city: Chiang Mai
country: Thailand
facility: Chiang Mai Uni Hospital; Division of Hematology,Dept of Medicine,Faculty of Medicine
city: Khon Kaen
country: Thailand
facility: Srinagarind Hospital, Khon Kaen Uni ; Dept of Medicine
city: Khon Kean
country: Thailand
facility: Khonkean Regional Hospital; Medicine
city: Pathumthani
country: Thailand
facility: Thammasart Chalermprakiert Hospital, Thammasart Uni; Hematology
city: Phitsanulok
country: Thailand
facility: Naresaun University hospital
city: Ubon Ratchathani
country: Thailand
facility: Sapprasitthiprasong Hospital
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: