A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)

NCT02145026 | Completed Phase 4 Results

• 1 other identifier: ML29005
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 10

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.

Conditions

Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria

  Erythroid response at Week 12 according to IWG 2006 criteria was defined as a hemoglobin (Hb) increase of \>/= 1.5 grams/deciliter (g/dL), and a reduction of units of red blood cell (RBC) transfusions by at least 4 transfusions/8 weeks compared with the pre-treatment transfusion number in the previous 8 weeks. Only RBC transfusions given for an Hb of \</= 9.0 g/dL pre-treatment were counted in the RBC transfusion response evaluation.

  Week 12

Secondary Outcomes (3)

• 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

  Week 12

• 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

  Week 12

• Percentage of Participants With Adverse Events

  From signing of informed consent up to 4 weeks after last dose (up to 18 weeks)

Study Arms (1)

Epoetin Beta

EXPERIMENTAL

Participants will receive epoetin beta at an initial dose of 30,000 International Units (IU) per week administered subcutaneously (SC). Response will be firstly evaluated at Week 4 and the subsequent dose will be based on the response: if hemoglobin level reaches greater than or equal to (\>/=)12 grams per deciliter (g/dL) at any time, epoetin beta will be discontinued until hemoglobin levels are less than or equal to (\</=) 10 g/dL; if the hemoglobin level increases less than (\<) 1 g/dL from screening level and hemoglobin level ˂12 g/dL, a 60,000 IU per week epoetin beta will be administered SC until Week 12; if the hemoglobin level increases \>/=1 g/dL from screening level and hemoglobin level ˂12 g/dL, a 30,000 IU per week epoetin beta will be continued until Week 12.

Drug: Epoetin beta

Interventions

Epoetin beta DRUG

Epoetin beta 30,000 or 60,000 IU per week SC injection

Also known as: Recormon

Epoetin Beta

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (10)

King Chulalongkorn Memorial Hospital; Division of Hematology, Department of Medicine

Bangkok, 10330, Thailand

Location

Rajavithi Hospital; Medicine

Bangkok, 10400, Thailand

Location

Ramathibodi Hospital; Division of Hematology, Department of Medicine

Bangkok, 10400, Thailand

Location

Siriraj Hospital; Division of Hematology, Department of Medicine

Bangkok, 10700, Thailand

Location

Chiang Mai Uni Hospital; Division of Hematology,Dept of Medicine,Faculty of Medicine

Chiang Mai, 50200, Thailand

Location

Khonkean Regional Hospital; Medicine

Khon Kaen, 40000, Thailand

Location

Srinagarind Hospital, Khon Kaen Uni ; Dept of Medicine

Khon Kaen, 40002, Thailand

Location

Thammasart Chalermprakiert Hospital, Thammasart Uni; Hematology

Pathum Thani, 12120, Thailand

Location

Naresaun University hospital

Phitsanulok, 65000, Thailand

Location

Sapprasitthiprasong Hospital

Ubon Ratchathani, 34000, Thailand

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

epoetin beta

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Limitations and Caveats

Data for the secondary efficacy endpoints (% of participants with platelet response and % of participants with neutrophil response) is not conclusive given the small number of participants meeting the evaluation criteria for each endpoint.

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

1-800-821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 20, 2014
• First Posted: May 22, 2014
• Study Start: August 6, 2014
• Primary Completion: April 9, 2019
• Study Completion: April 9, 2019
• Last Updated: April 24, 2020
• Results First Posted: April 24, 2020

Record last verified: 2020-04

Locations

TH (10)