Myelodysplastic SyndromesPhase 4NCT02145026
A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)
A Phase 4 study testing Epoetin beta in people with Myelodysplastic Syndromes, run by Hoffmann-La Roche at 10 sites in Thailand.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Epoetin beta
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 10 sitesThailand
- Study dates
- 6 Aug 2014to 9 Apr 2019
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
Thailand10 sites
King Chulalongkorn Memorial Hospital; Division of Hematology, Department of Medicine
Bangkok, 10330
Rajavithi Hospital; Medicine
Bangkok, 10400
Ramathibodi Hospital; Division of Hematology, Department of Medicine
Bangkok, 10400
Siriraj Hospital; Division of Hematology, Department of Medicine
Bangkok, 10700
Chiang Mai Uni Hospital; Division of Hematology,Dept of Medicine,Faculty of Medicine
Chiang Mai, 50200
Khonkean Regional Hospital; Medicine
Khon Kaen, 40000
Srinagarind Hospital, Khon Kaen Uni ; Dept of Medicine
Khon Kaen, 40002
Thammasart Chalermprakiert Hospital, Thammasart Uni; Hematology
Pathum Thani, 12120
Naresaun University hospital
Phitsanulok, 65000
Sapprasitthiprasong Hospital
Ubon Ratchathani, 34000
About this study
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)
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
- Being tested
- Epoetin beta
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Epoetin betaDrug
Also called Recormon
Epoetin beta 30,000 or 60,000 IU per week SC injection
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Epoetin beta |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Platelet response according to IWG 2006 criteria was defined as an absolute increase of >/= 30x10^9/L for participants starting with >20x10^9/L platelets.
Measured: 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
Neutrophil response according to IWG 2006 criteria was defined as at least 100% increase and an absolute increase of >0.5x10^9/L.
Measured: Week 12
Percentage of Participants With Adverse Events
Measured: From signing of informed consent up to 4 weeks after last dose (up to 18 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2014 (estimated)
- Study start6 Aug 2014
- Primary completion9 Apr 2019
- Study completion9 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02145026
- Sponsor's protocol ID
- ML29005
- Status last verified
- Apr 2020
- First submitted
- 20 May 2014
- Last updated
- 24 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02145026
- Acronym / sponsor ID
- ML29005
- Conditions
- Myelodysplastic Syndromes
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 6 Aug 2014 (actual)
- Primary completion
- 9 Apr 2019 (actual)
- Study completion
- 9 Apr 2019 (actual)
- First posted
- 22 May 2014 (estimated)
- Results
- Posted 24 Apr 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Epoetin Beta | Experimental | Drug: Epoetin betaParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (5 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Epoetin betaAlso: RecormonEpoetin beta 30,000 or 60,000 IU per week SC injection | Drug | Epoetin Beta |
Primary outcome
Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria
Time frame: Week 12
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.
3 secondary outcomes
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
Time frame: 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
Time frame: Week 12
Percentage of Participants With Adverse Events
Time frame: From signing of informed consent up to 4 weeks after last dose (up to 18 weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Myelodysplastic Syndromes
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Epoetin beta in people with Myelodysplastic Syndromes, run by Hoffmann-La Roche at 10 sites in Thailand.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Thailand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Apr 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria (time frame: Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2020.