Myelodysplastic SyndromesPhase 3NCT00695396
A Study Evaluating Epoetin Alfa 40,000 IU (International Units) Every Week or 80,000 IU Every Week Compared to Placebo in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes at Risk for Transfusion
A Phase 3 study testing Epoetin alfa in people with Myelodysplastic Syndromes and Anemia, run by Johnson & Johnson Pharmaceutical Research & Development, L.L.C..
Trial summary
The purpose of this study is to demonstrate that Epoetin alfa treatment reduces red blood cell transfusions in anemic patients with myelodysplastic syndromes (MDS). Myelodysplastic syndromes are a group of disorders characterized by progressive bone marrow failure and an increased risk of development of leukemia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Anemia
- What is being tested?
- Epoetin alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- No sites listed yet
- Study dates
- Jun 2008to Jan 2010
- Phase
- Phase 3
- 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
- Diagnosis of MDS according to protocol-specified criteria via bone marrow studies performed within 12 weeks before randomization
Who cannot join
- No prior or concurrent treatment with epoetin alfa or any other approved or experimental erythropoietin stimulating agents (ESAs) within the previous 12 months before randomization
- No prior use of approved or experimental agents for the treatment of MDS or recent treatment with granulocyte colony stimulating factor (G-CSF) or granulocyte macrophage colony stimulating factor (GM-CSF) for the treatment of neutropenia
- Patients must not have secondary MDS or anemia caused by factors other than MDS (including iron deficiency, vitamin B12 or folate deficiencies, hemolysis, chronic renal failure, or gastrointestinal bleeding)
- No history (within 12 months) of deep venous thrombosis
- or history (within 6 months) of stroke, acute coronary syndrome or other arterial thrombosis
- Not currently receiving therapeutic anticoagulants or have uncontrolled hypertension
- No uncontrolled disease or dysfunction deemed clinically significant by the Investigator not attributable to MDS
About this study
Official title: A Randomized, Double Blind, Placebo Controlled, Multicenter Study Evaluating Epoetin Alfa Initiated at 40,000 IU Every Week or 80,000 IU Every Week Versus Placebo in Subjects With IPSS Low- or Intermediate-1 Risk Myelodysplastic Syndromes at Risk For Transfusion
The purpose of this study is to demonstrate that Epoetin alfa treatment reduces red blood cell transfusions in anemic patients with myelodysplastic syndromes (MDS). Myelodysplastic syndromes are a group of disorders characterized by progressive bone marrow failure and an increased risk of development of leukemia.
Read the detailed description
This is a randomized (patients are assigned by chance to a treatment group), double-blind (neither the patient or the physician know which treatment is being received by the patient), placebo-controlled, multicenter study of epoetin alfa in anemic patients who are diagnosed with myelodysplastic syndromes (MDS) according to protocol-specified criteria. Patients meeting entry criteria for the study will be randomly assigned to receive epoetin alfa 40,000 IU or 80,000 IU or a matching volume of placebo administered by subcutaneous (under the skin) injection once every week. Doses of study drug will be withheld, decreased, or increased on the basis of weekly hemoglobin concentrations monitored in patients and predefined dose adjustment guidelines. An Independent Data Monitoring Committee (IDMC) will periodically review study data and for the assessment of disease progression, an independent central reviewer will review bone marrow specimens and peripheral blood counts. Safety will be monitored throughout the study at predetermined intervals and as clinically indicated by physical examination, laboratory tests and evaluation of adverse events. Patients in the Treatment Phase will be randomly assigned to receive once weekly epoetin alfa subcutaneously (SC) at a dose of 40,000 IU (1 mL) or 80,000 IU (2ML) or matching volume of placebo (1 mL or 2 mL) once every week for 48 weeks. Patients may continue to receive double-blinded treatment after 48-weeks.
- Conditions
- Myelodysplastic Syndromes, Anemia
- Being tested
- Placebo, Epoetin alfa
- Keywords
- MDS, Myelodysplastic syndromes, Anemia, Epoetin alfa, EPO
- Medical subject headings
- Myelodysplastic Syndromes, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Matching volume 2 mLfor 48 weeks
Epoetin alfaDrug
40,000 IU subcutaneously once every week (1 mL dose) for 48 weeks
PlaceboDrug
Matching volume 1 mL for 48 weeks
Epoetin alfaDrug
80,000 IU subcutaneously once every week (2 mL dose) for 48 weeks
| Study group | Role | What they receive |
|---|---|---|
| 001 | Experimental | Epoetin alfa 40 000 IU subcutaneously once every week (1 mL dose) for 48 weeks Receives: Drug: Epoetin alfa |
| 002 | Experimental | Epoetin alfa 80 000 IU subcutaneously once every week (2 mL dose) for 48 weeks Receives: Drug: Epoetin alfa |
| 003 | Placebo comparator | Placebo Matching volume 1 mL for 48 weeks Receives: Drug: Placebo |
| 004 | Placebo comparator | Placebo Matching volume 2 mLfor 48 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Red Blood Cell (RBC) Transfusion
Incidence of participants who received at least 1 Red Blood Cell (RBC) transfusion during the study (from randomization through the end of study)
Measured: Approximately 48 weeks
Secondary outcomes (2)
RBC Transfusion From Day 29 Through the End of Study
incidence of participants who received at least 1 RBC transfusion from Day 29 through the end of study (approximately 48 weeks).
Measured: Day 29 through the end of study (approximately 48 weeks)
Transfusion Dependent
Participants who were transfusion-dependent were those who received 4 or more RBC units during a consecutive 8-week period.
Measured: Approximately 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2008 (estimated)
- Study startJun 2008
- Primary completionJan 2010
- Study completionJan 2010
Why it stopped: The study was stopped due to low subject enrollment. No safety issue or other concern factored into this decision.
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.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Centocor Ortho Biotech Services, L.L.C.
- Investigators
- Johnson & Johnson Pharmaceutical Research & Development, L.L. C. Clinical Trial, Study director, Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00695396
- Sponsor's protocol ID
- CR013651
- Other IDs
- EPOANE3018
- Status last verified
- Oct 2012
- First submitted
- 5 Jun 2008
- Last updated
- 5 Oct 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double Blind, Placebo Controlled, Multicenter Study Evaluating Epoetin Alfa Initiated at 40,000 IU Every Week or 80,000 IU Every Week Versus Placebo in Subjects With IPSS Low- or Intermediate-1 Risk Myelodysplastic Syndromes at Risk For Transfusion
- ClinicalTrials.gov ID
- NCT00695396
- Acronym / sponsor ID
- CR013651
- Conditions
- Myelodysplastic Syndromes, Anemia
- Keywords
- MDS, Myelodysplastic syndromes, Anemia, Epoetin alfa, EPO
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was stopped due to low subject enrollment. No safety issue or other concern factored into this decision.
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.Industry (pharma, biotech, devices)
- Collaborators
- Centocor Ortho Biotech Services, L.L.C.
- Study start
- Jun 2008
- Primary completion
- Jan 2010 (actual)
- Study completion
- Jan 2010 (actual)
- First posted
- 11 Jun 2008 (estimated)
- Results
- Posted 17 Mar 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to demonstrate that Epoetin alfa treatment reduces red blood cell transfusions in anemic patients with myelodysplastic syndromes (MDS). Myelodysplastic syndromes are a group of disorders characterized by progressive bone marrow failure and an increased risk of development of leukemia.
Detailed description
This is a randomized (patients are assigned by chance to a treatment group), double-blind (neither the patient or the physician know which treatment is being received by the patient), placebo-controlled, multicenter study of epoetin alfa in anemic patients who are diagnosed with myelodysplastic syndromes (MDS) according to protocol-specified criteria. Patients meeting entry criteria for the study will be randomly assigned to receive epoetin alfa 40,000 IU or 80,000 IU or a matching volume of placebo administered by subcutaneous (under the skin) injection once every week. Doses of study drug will be withheld, decreased, or increased on the basis of weekly hemoglobin concentrations monitored in patients and predefined dose adjustment guidelines. An Independent Data Monitoring Committee (IDMC) will periodically review study data and for the assessment of disease progression, an independent central reviewer will review bone marrow specimens and peripheral blood counts. Safety will be monitored throughout the study at predetermined intervals and as clinically indicated by physical examination, laboratory tests and evaluation of adverse events. Patients in the Treatment Phase will be randomly assigned to receive once weekly epoetin alfa subcutaneously (SC) at a dose of 40,000 IU (1 mL) or 80,000 IU (2ML) or matching volume of placebo (1 mL or 2 mL) once every week for 48 weeks. Patients may continue to receive double-blinded treatment after 48-weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 001 | Experimental | Drug: Epoetin alfaEpoetin alfa 40 000 IU subcutaneously once every week (1 mL dose) for 48 weeks |
| 002 | Experimental | Drug: Epoetin alfaEpoetin alfa 80 000 IU subcutaneously once every week (2 mL dose) for 48 weeks |
| 003 | Placebo comparator | Drug: PlaceboPlacebo Matching volume 1 mL for 48 weeks |
| 004 | Placebo comparator | Drug: PlaceboPlacebo Matching volume 2 mLfor 48 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Diagnosis of MDS according to protocol-specified criteria via bone marrow studies performed within 12 weeks before randomization
Key exclusion criteria (7 listed)
- No prior or concurrent treatment with epoetin alfa or any other approved or experimental erythropoietin stimulating agents (ESAs) within the previous 12 months before randomization
- No prior use of approved or experimental agents for the treatment of MDS or recent treatment with granulocyte colony stimulating factor (G-CSF) or granulocyte macrophage colony stimulating factor (GM-CSF) for the treatment of neutropenia
- Patients must not have secondary MDS or anemia caused by factors other than MDS (including iron deficiency, vitamin B12 or folate deficiencies, hemolysis, chronic renal failure, or gastrointestinal bleeding)
- No history (within 12 months) of deep venous thrombosis
- or history (within 6 months) of stroke, acute coronary syndrome or other arterial thrombosis
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 |
|---|---|---|
| PlaceboMatching volume 2 mLfor 48 weeks | Drug | 004 |
| Epoetin alfa40,000 IU subcutaneously once every week (1 mL dose) for 48 weeks | Drug | 001 |
| PlaceboMatching volume 1 mL for 48 weeks | Drug | 003 |
| Epoetin alfa80,000 IU subcutaneously once every week (2 mL dose) for 48 weeks | Drug | 002 |
Primary outcome
Red Blood Cell (RBC) Transfusion
Time frame: Approximately 48 weeks
Incidence of participants who received at least 1 Red Blood Cell (RBC) transfusion during the study (from randomization through the end of study)
2 secondary outcomes
RBC Transfusion From Day 29 Through the End of Study
Time frame: Day 29 through the end of study (approximately 48 weeks)
Transfusion Dependent
Time frame: Approximately 48 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
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ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
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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.
Johnson & Johnson Pharmaceutical Research & Development, L.L. C. Clinical Trial study director
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Epoetin alfa in people with Myelodysplastic Syndromes and Anemia, run by Johnson & Johnson Pharmaceutical Research & Development, L.L.C..
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 1 inclusion and 7 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: stopped early, as of the sponsor's last update on 5 Oct 2012. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 17 Mar 2011). 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?
Red Blood Cell (RBC) Transfusion (time frame: Approximately 48 weeks). 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 5 Oct 2012.