AnemiaPhase 3NCT00003138
Erythropoietin (EPO)+/- Filgrastim (G-CSF) vs. Supportive Therapy Alone for Patients With Myelodysplastic Syndromes
A Phase 3 study testing Erythropoietin, Filgrastim and Transfusion in people with Anemia and Myelodysplastic Syndromes, run by Eastern Cooperative Oncology Group at 28 sites in United States.
Trial summary
RATIONALE: Erythropoietin and colony-stimulating factors such as filgrastim stimulate the production of blood cells. It is not yet known whether erythropoietin with or without filgrastim is more effective than standard blood transfusions in reducing the need for transfusions in patients who have anemia associated with… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anemia, Myelodysplastic Syndromes
- What is being tested?
- Erythropoietin, Filgrastim and Transfusion
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 118 participants
- Where
- 28 sitesUnited States
- Study dates
- 4 Mar 1998to May 2014
- 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
- At least 18 years of age
- Diagnosis of a myelodysplastic syndrome
- Refractory anemia (RA)
- RA with ringed sideroblasts
- RA with excess blasts (RAEB). RAEB patients must have a bone marrow blast count of less than 20% and less than 5% blast forms on peripheral blood
- Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 3
Show all 15Show fewer
- Platelet count greater than 30,000/mm^3 (without platelet transfusions)
- Hematocrit less than 30% (pretransfusion)
- Bilirubin less than 3 mg/dL
- Blood urea nitrogen (BUN) less than 40 mg/dL or Creatinine less than 2.0 mg/dL
- Prior epoetin alfa allowed provided dosage was less than 30,000 units per week for less than 1 month duration
- At least 1 month since prior erythropoietin
- At least 2 months since prior recombinant growth factor
- At least 2 months since prior chemotherapy for other malignancy or autoimmune disease
- At least 2 weeks since prior androgen or steroids for treatment of myelodysplastic syndromes
Who cannot join
- RAEB in transformation
- Chronic myelomonocytic leukemia
- Splenomegaly greater than 6 cm below the left costal margin or greater than 3 times normal size
- Uncontrolled hypertension
- Sensitivity to E. coli-derived proteins
- Sensitivity to epoetin alfa or any of its components (e.g., human albumin)
Show all 10Show fewer
- Documented iron deficiency. If marrow iron stain is not available, the transferrin saturation must be greater than 20% or ferritin greater than 100 ng/dL
- Active infection or bleeding
- Other uncontrolled malignancy
- Pregnant or nursing. Fertile patients must use effective contraception.
Trial locations and contacts
28 sites in 1 country.
28 sites in 1 country
United States28 sites
CCOP - Michigan Cancer Research Consortium
Ann Arbor, Michigan, 48106
Tufts - New England Medical Center
Boston, Massachusetts, 02111
CCOP - Cedar Rapids Oncology Project
Cedar Rapids, Iowa, 52403-1206
Veterans Affairs Medical Center - Lakeside Chicago
Chicago, Illinois, 60611-4494
Robert H. Lurie Comprehensive Cancer Center at Northwestern University
Chicago, Illinois, 60611
MetroHealth's Cancer Care Center at MetroHealth Medical Center
Cleveland, Ohio, 44109
CCOP - Columbus
Columbus, Ohio, 43206
CCOP - Geisinger Clinic and Medical Center
Danville, Pennsylvania, 17822-2001
CCOP - Colorado Cancer Research Program, Incorporated
Denver, Colorado, 80224
CCOP - Iowa Oncology Research Association
Des Moines, Iowa, 50309-1016
Veterans Affairs Medical Center - East Orange
East Orange, New Jersey, 07019
CCOP - Merit Care Hospital
Fargo, North Dakota, 58122
CCOP - St. Vincent Hospital Cancer Center, Green Bay
Green Bay, Wisconsin, 54307-3453
CCOP - Northern New Jersey
Hackensack, New Jersey, 07601
CCOP - Kalamazoo
Kalamazoo, Michigan, 49007-3731
West Michigan Cancer Center
Kalamazoo, Michigan, 49007
CCOP - Southern Nevada Cancer Research Foundation
Las Vegas, Nevada, 89106
CCOP - Marshfield Clinic Research Foundation
Marshfield, Wisconsin, 54449
Medical College of Wisconsin Cancer Center
Milwaukee, Wisconsin, 53226-3596
Cancer Institute of New Jersey
New Brunswick, New Jersey, 08903
MBCCOP - LSU Health Sciences Center
New Orleans, Louisiana, 70112
NYU School of Medicine's Kaplan Comprehensive Cancer Center
New York, New York, 10016
CCOP - Missouri Valley Cancer Consortium
Omaha, Nebraska, 68106
CCOP - Illinois Oncology Research Association
Peoria, Illinois, 61602
CCOP - Metro-Minnesota
Saint Louis Park, Minnesota, 55416
CCOP - Sioux Community Cancer Consortium
Sioux Falls, South Dakota, 57104
CCOP - Scott and White Hospital
Temple, Texas, 76508
CCOP - Carle Cancer Center
Urbana, Illinois, 61801
About this study
Official title: Phase III Evaluation of EPO With or Without G-CSF Versus Supportive Therapy Alone in the Treatment of Myelodysplastic Syndromes
RATIONALE: Erythropoietin and colony-stimulating factors such as filgrastim stimulate the production of blood cells. It is not yet known whether erythropoietin with or without filgrastim is more effective than standard blood transfusions in reducing the need for transfusions in patients who have anemia associated with myelodysplastic syndrome.
PURPOSE: Randomized phase III trial to compare the effectiveness of erythropoietin with or without filgrastim with that of standard blood transfusions in reducing the need for transfusions in patients who have anemia associated with myelodysplastic syndrome.
Read the detailed description
OBJECTIVES:
- Compare the benefit of erythropoietin vs standard transfusion support in reducing transfusion requirements in patients with myelodysplastic syndromes.
- Compare the clinical response, disease progression, and survival in patients treated with these regimens.
- Compare the toxicity of these regimens in these patients.
- Evaluate whether adding filgrastim (G-CSF) or increasing the erythropoietin dose will reduce the transfusion requirement in patients who do not respond to erythropoietin alone.
- To compare the benefit of erythropoietin versus supportive care alone on quality of life (QOL) in persons with myelodysplastic syndromes.
OUTLINE: This is a randomized, controlled, multicenter, cross-over study. Patients are stratified according to morphologic subtype (refractory anemia [RA] vs RA with ringed sideroblasts vs RA with excess blasts), transfusion requirement (yes vs no), prior erythropoietin treatment (yes vs no), and erythropoietin level (at least 200 mU/mL vs less than 200 mU/mL). Patients are randomized to one of two treatment arms.
- Arm I (standard transfusion support): Patients receive red cell and platelet transfusions for symptoms or to maintain hematocrit level of 25% or above. Patients undergo bone marrow aspirate and biopsy at 4 months and then every year until development of acute leukemia or completion of study. Patients with progressive disease may cross over to arm II after at least 4 months on study and up to 1 year from the time of randomization. Patients who cross over receive erythropoietin alone.
- Arm II (Erythropoietin): Patients receive erythropoietin subcutaneously (SC) or intravenously (IV) daily. Patients undergo bone marrow aspirate and biopsy as in arm I. Treatment continues daily for a maximum of 1 year.
Patients with stable or progressive disease at day 120 receive filgrastim (G-CSF) SC daily or 3 days a week and erythropoietin SC daily for up to 6 months. Patients with no response to G-CSF and lower-dose erythropoietin may proceed to a higher dose of erythropoietin.
Quality of life is assessed at baseline, every 4 months during study, and at study completion.
Patients are followed every 4 months for 2 years, every 6 months for 3 years, and then annually for 5 years.
ACTUAL ACCRUAL: A total of 118 patients were accrued for this study.
- Conditions
- Anemia, Myelodysplastic Syndromes
- Being tested
- Erythropoietin, Filgrastim, Transfusion
- Keywords
- anemia, refractory anemia, myelodysplastic syndromes, erythropoietin, filgrastim
- Medical subject headings
- Anemia, Myelodysplastic Syndromes, Anemia, Refractory
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ErythropoietinBiological
Also called r-HuEPO, EPO
Administered at 150 units/kg subcutaneously every day. Rotating sites should be used. The dose should be rounded off to the nearest 1000 U. The dose should be adjusted based on hematocrit.
FilgrastimBiological
Also called G-CSF, Neupogen, Recombinant-methionyl human granulocyte colony-stimulating factor, Granulocyte colony-stimulating factor
G-CSF should start at a dose of 1 mcg/kg per day or 2.5 mcg/kg three times a week subcutaneously. Rotating sites should be used The dose should be rounded off to the nearest 10 mcg.
TransfusionProcedure or surgery
Red cell and platelet transfusions
| Study group | Role | What they receive |
|---|---|---|
| Supportive Care | Active comparator | Patients received red cell and platelet transfusions for symptoms or to maintain hematocrit at or above 25% by volume. Patients who required transfusion support for symptomatic anemia prior to entering the study and who developed an increase in their transfusion requirement of >= 50% shall cross over to the Erythropoietin treatment arm, after at least four months on the supportive therapy arm. Receives: Procedure: Transfusion |
| Erythropoietin | Experimental | Erythropoietin was administered at 150 units/kg subcutaneously every day. If patients stopped responding, they were subsequently treated with Erythropoietin (150 units/kg) and filgrastim and then Erythropoietin (300 units/kg) and filgrastim. Receives: Biological: Erythropoietin; Biological: Filgrastim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Patients Free of Transfusion at 4 Months
Whether a patient required transfusion or not at 4 months was recorded.
Measured: Assessed at 4 months
Secondary outcomes (2)
Overall Survival
Time from randomization to death from any cause. Patients alive at the time of analysis were censored at the date of last contact.
Measured: Assessed every 3 months for 2 years, every 6 months for 3 subsequent years, and annually thereafter
Quality of Life- Total Functional Assessment of Cancer Therapy - General (FACT-G) Score at 4 Months
The FACT-G scale has 4 dimensions, including physical well-being, social/family well-being, emotional well-being, and functional well-being. The score for each subscale was added together to obtain the total FACT-G score that was evaluated on this study. The total FACT-G score ranges from 0 to 108 with higher scores reflecting better quality of life. It was administered at the time of study entry, every 4 months for the first year, and at the time patient went off treatment. Due to limited data after 4 months on treatment, the analysis was restricted to the four-month time point.
Measured: Assessed at 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2003 (estimated)
- Study start4 Mar 1998
- Primary completionAug 2008
- Study completionMay 2014
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.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for AnemiaStudies 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
- None (open label)
- Enrolment
- 118 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eastern Cooperative Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Kenneth B. Miller, MD, Study chair, Beth Israel Deaconess Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003138
- Sponsor's protocol ID
- CDR0000065907
- Other IDs
- E1996, U10CA021115
- Status last verified
- Jun 2023
- First submitted
- 1 Nov 1999
- Last updated
- 29 Jun 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Cherry AM, Brockman SR, Paternoster SF, Hicks GA, Neuberg D, Higgins RR, Bennett JM, Greenberg PL, Miller K, Tallman MS, Rowe J, Dewald GW. Comparison of interphase FISH and metaphase cytogenetics to study myelodysplastic syndrome: an Eastern Cooperative Oncology Group (ECOG) study. Leuk Res. 2003 Dec;27(12):1085-90. doi: 10.1016/s0145-2126(03)00104-8.
PubMed 12921944Greenberg PL, Sun Z, Miller KB, Bennett JM, Tallman MS, Dewald G, Paietta E, van der Jagt R, Houston J, Thomas ML, Cella D, Rowe JM. Treatment of myelodysplastic syndrome patients with erythropoietin with or without granulocyte colony-stimulating factor: results of a prospective randomized phase 3 trial by the Eastern Cooperative Oncology Group (E1996). Blood. 2009 Sep 17;114(12):2393-400. doi: 10.1182/blood-2009-03-211797. Epub 2009 Jun 29.
PubMed 19564636Miller KB, Kim HT, Greenberg P, et al.: Phase III prospective randomized trial of EPO with or without G-CSF versus supportive therapy alone in the treatment of myelodysplastic syndromes (MDS): results of the ECOG- CLSG trial (E1996). [Abstract] Blood 104 (11): A-70, 2004.
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Evaluation of EPO With or Without G-CSF Versus Supportive Therapy Alone in the Treatment of Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT00003138
- Acronym / sponsor ID
- CDR0000065907
- Conditions
- Anemia, Myelodysplastic Syndromes
- Keywords
- anemia, refractory anemia, myelodysplastic syndromes, erythropoietin, filgrastim
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 118 participants (actual)
- Lead sponsor
- Eastern Cooperative Oncology GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 4 Mar 1998 (actual)
- Primary completion
- Aug 2008 (actual)
- Study completion
- May 2014 (actual)
- First posted
- 27 Jan 2003 (estimated)
- Results
- Posted 6 Dec 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Erythropoietin and colony-stimulating factors such as filgrastim stimulate the production of blood cells. It is not yet known whether erythropoietin with or without filgrastim is more effective than standard blood transfusions in reducing the need for transfusions in patients who have anemia associated with myelodysplastic syndrome.
PURPOSE: Randomized phase III trial to compare the effectiveness of erythropoietin with or without filgrastim with that of standard blood transfusions in reducing the need for transfusions in patients who have anemia associated with myelodysplastic syndrome.
Detailed description
OBJECTIVES:
- Compare the benefit of erythropoietin vs standard transfusion support in reducing transfusion requirements in patients with myelodysplastic syndromes.
- Compare the clinical response, disease progression, and survival in patients treated with these regimens.
- Compare the toxicity of these regimens in these patients.
- Evaluate whether adding filgrastim (G-CSF) or increasing the erythropoietin dose will reduce the transfusion requirement in patients who do not respond to erythropoietin alone.
- To compare the benefit of erythropoietin versus supportive care alone on quality of life (QOL) in persons with myelodysplastic syndromes.
OUTLINE: This is a randomized, controlled, multicenter, cross-over study. Patients are stratified according to morphologic subtype (refractory anemia [RA] vs RA with ringed sideroblasts vs RA with excess blasts), transfusion requirement (yes vs no), prior erythropoietin treatment (yes vs no), and erythropoietin level (at least 200 mU/mL vs less than 200 mU/mL). Patients are randomized to one of two treatment arms.
- Arm I (standard transfusion support): Patients receive red cell and platelet transfusions for symptoms or to maintain hematocrit level of 25% or above. Patients undergo bone marrow aspirate and biopsy at 4 months and then every year until development of acute leukemia or completion of study. Patients with progressive disease may cross over to arm II after at least 4 months on study and up to 1 year from the time of randomization. Patients who cross over receive erythropoietin alone.
- Arm II (Erythropoietin): Patients receive erythropoietin subcutaneously (SC) or intravenously (IV) daily. Patients undergo bone marrow aspirate and biopsy as in arm I. Treatment continues daily for a maximum of 1 year.
Patients with stable or progressive disease at day 120 receive filgrastim (G-CSF) SC daily or 3 days a week and erythropoietin SC daily for up to 6 months. Patients with no response to G-CSF and lower-dose erythropoietin may proceed to a higher dose of erythropoietin.
Quality of life is assessed at baseline, every 4 months during study, and at study completion.
Patients are followed every 4 months for 2 years, every 6 months for 3 years, and then annually for 5 years.
ACTUAL ACCRUAL: A total of 118 patients were accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Supportive Care | Active comparator | Procedure: TransfusionPatients received red cell and platelet transfusions for symptoms or to maintain hematocrit at or above 25% by volume. Patients who required transfusion support for symptomatic anemia prior to entering the study and who developed an increase in their transfusion requirement of >= 50% shall cross over to the Erythropoietin treatment arm, after at least four months on the supportive therapy arm. |
| Erythropoietin | Experimental | Biological: Erythropoietin, Biological: FilgrastimErythropoietin was administered at 150 units/kg subcutaneously every day. If patients stopped responding, they were subsequently treated with Erythropoietin (150 units/kg) and filgrastim and then Erythropoietin (300 units/kg) and filgrastim. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- At least 18 years of age
- Diagnosis of a myelodysplastic syndrome
- Refractory anemia (RA)
- RA with ringed sideroblasts
- RA with excess blasts (RAEB). RAEB patients must have a bone marrow blast count of less than 20% and less than 5% blast forms on peripheral blood
Key exclusion criteria (10 listed)
- RAEB in transformation
- Chronic myelomonocytic leukemia
- Splenomegaly greater than 6 cm below the left costal margin or greater than 3 times normal size
- Uncontrolled hypertension
- Sensitivity to E. coli-derived proteins
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 |
|---|---|---|
| ErythropoietinAlso: r-HuEPO, EPOAdministered at 150 units/kg subcutaneously every day. Rotating sites should be used. The dose should be rounded off to the nearest 1000 U. The dose should be adjusted based on hematocrit. | Biological | Erythropoietin |
| FilgrastimAlso: G-CSF, Neupogen, Recombinant-methionyl human granulocyte colony-stimulating factor, Granulocyte colony-stimulating factorG-CSF should start at a dose of 1 mcg/kg per day or 2.5 mcg/kg three times a week subcutaneously. Rotating sites should be used The dose should be rounded off to the nearest 10 mcg. | Biological | Erythropoietin |
| TransfusionRed cell and platelet transfusions | Procedure or surgery | Supportive Care |
Primary outcome
Proportion of Patients Free of Transfusion at 4 Months
Time frame: Assessed at 4 months
Whether a patient required transfusion or not at 4 months was recorded.
2 secondary outcomes
Overall Survival
Time frame: Assessed every 3 months for 2 years, every 6 months for 3 subsequent years, and annually thereafter
Quality of Life- Total Functional Assessment of Cancer Therapy - General (FACT-G) Score at 4 Months
Time frame: Assessed at 4 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 12921944Cherry AM, Brockman SR, Paternoster SF, Hicks GA, Neuberg D, Higgins RR, Bennett JM, Greenberg PL, Miller K, Tallman MS, Rowe J, Dewald GW. Comparison of interphase FISH and metaphase cytogenetics to study myelodysplastic syndrome: an Eastern Cooperative Oncology Group (ECOG) study. Leuk Res. 2003 Dec;27(12):1085-90. doi: 10.1016/s0145-2126(03)00104-8.
- Results publication: PubMed 19564636Greenberg PL, Sun Z, Miller KB, Bennett JM, Tallman MS, Dewald G, Paietta E, van der Jagt R, Houston J, Thomas ML, Cella D, Rowe JM. Treatment of myelodysplastic syndrome patients with erythropoietin with or without granulocyte colony-stimulating factor: results of a prospective randomized phase 3 trial by the Eastern Cooperative Oncology Group (E1996). Blood. 2009 Sep 17;114(12):2393-400. doi: 10.1182/blood-2009-03-211797. Epub 2009 Jun 29.
- Results publicationMiller KB, Kim HT, Greenberg P, et al.: Phase III prospective randomized trial of EPO with or without G-CSF versus supportive therapy alone in the treatment of myelodysplastic syndromes (MDS): results of the ECOG- CLSG trial (E1996). [Abstract] Blood 104 (11): A-70, 2004.
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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.
Kenneth B. Miller, MD study chair
Beth Israel Deaconess Medical Center
28 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Erythropoietin, Filgrastim and Transfusion in people with Anemia and Myelodysplastic Syndromes, run by Eastern Cooperative Oncology Group at 28 sites in United States.
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 15 inclusion and 10 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 29 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States. 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 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 6 Dec 2013). 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 Patients Free of Transfusion at 4 Months (time frame: Assessed at 4 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00003138, which also lists 3 linked publications.
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 29 Jun 2023.