Myelodysplastic SyndromePhase 2NCT00520468
Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS
A Phase 2 study testing Erythropoietin, Cyclosporin A and G-CSF in people with Myelodysplastic Syndrome, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
Objectives: Primary: To evaluate the response rate of total cytokine-immunotherapy for low-risk myelodysplastic syndromes (MDS). Secondary: To evaluate response duration, survival and side effects of the treatment. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Myelodysplastic Syndrome
- What is being tested?
- Erythropoietin, Cyclosporin A and G-CSF
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2004to Jun 2009
- Phase
- Phase 2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with MDS and </= 10% blasts or International Prognostic Scoring System (IPSS) low or intermediate 1. No prior intensive chemotherapy or high-dose ara-C (>/= 1g/m2). Prior cytokines, biologic therapies, targeted therapies, or single agent chemotherapy allowed. Procrit, G-CSF are allowed before therapy. Patients with blasts < 5% must have an indication for therapy, such as transfusion needs, symptomatic anemia or Hb < 11g/dl, platelets < 100 x 10 9/L, or granulocytes < 10 9/L.
- Performance 0-2 (Eastern Cooperative Oncology Group (ECOG)). Adequate liver function (bilirubin of < 2mg/dl) and renal function (creatinine < 2mg/dl). Adequate cardiac functions (New York Heart Association (NYHA) cardiac III-IV excluded)
- Signed informed consent
Who cannot join
- Nursing and pregnant females are excluded. Women of childbearing potential should practice effective methods of contraception. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
- Patients with active and uncontrolled infections.
Trial locations and contacts
1 site in 1 country.
United States1 site
U.T.M.D. Anderson Cancer Center
Houston, Texas, 77030
About this study
Objectives:
Primary: To evaluate the response rate of total cytokine-immunotherapy for low-risk myelodysplastic syndromes (MDS).
Secondary: To evaluate response duration, survival and side effects of the treatment.
Read the detailed description
MDS is a disease that produces low blood counts and may cause anemia, infections, and/or bleeding. Erythropoietin and Granulocyte colony-stimulating factor (G-CSF or GCSF) are drugs that stimulate the production of red cells and white cells. Prednisone and cyclosporin are drugs that work against MDS by affecting your immune system.
Before you can start treatment on this study, you will have what are called "screening tests". These tests will help the doctor decide if you are eligible to take part in the study. You will have a complete medical history and physical exam. Routine blood tests (between 4-6 tablespoons) will be performed. You will have a bone marrow aspiration. To collect a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. Women who are able to have children must have a negative blood or urine pregnancy test.
If you are found to be eligible to take part in this study, you will receive erythropoietin as an injection under the skin once a week and G-CSF as an injection under the skin 1-2 times a week for as long as you respond well to treatment. You will take prednisone by mouth every day for a month and cyclosporin tablets by mouth every day for 6 months.
During this study, you will need to visit your doctor for a physical exam and measurement of your vital signs. The frequency of doctor visits will vary depending on your physical condition, but will be required at least once every 3 months.
Blood tests (about 2 teaspoons) will be done about every 1-2 weeks during the first 12 weeks of treatment, then every 2 to 4 weeks for the remainder of the study. The blood samples will be collected for routine lab tests. Periodic (every 3 to 6 months) bone marrow samples will also be taken to check cells related to the disease before, during, and after completion of this study.
You will be taken off study if the disease gets worse or intolerable side effects occur.
This is an investigational study. All drugs are FDA approved and commercially available. Up to 60 patients will take part in this study. All will be enrolled at M. D. Anderson.
- Conditions
- Myelodysplastic Syndrome
- Being tested
- Erythropoietin, Cyclosporin A, G-CSF, Prednisone
- Keywords
- Myelodysplastic Syndrome, MDS, Erythropoietin, Darbepoetin alfa, Aranesp, Erythropoiesis stimulating protein, G-CSF, Filgrastim, Neupogen, Prednisone, Cyclosporin A, Sandimmune, CYA, Cyclosporine
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ErythropoietinDrug
Also called Darbepoetin alfa, Aranesp, Erythropoiesis stimulating protein
40,000 units injected under the skin (SQ) weekly
Cyclosporin ADrug
Also called CYA, Cyclosporine, Sandimmune
300 mg (tablets) by mouth daily for 6 months
G-CSFDrug
Also called Filgrastim, Neupogen
300 mcg injected under the skin (SQ) two times per week
PrednisoneDrug
60 mg per day for 7 days, taper over 1 month
| Study group | Role | What they receive |
|---|---|---|
| Cytokine-Immunotherapy | Experimental | Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice a week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily Receives: Drug: Erythropoietin; Drug: Cyclosporin A; Drug: G-CSF; Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Response
Periodic bone marrow samples (every 3-6 months) to check cells related to disease before/during/after study. Response classifications categorized by the International Working Group Response Criteria for Myelodysplastic Syndrome (MDS) as: Complete Remission, Partial Response, Hematologic Improvement or No Response.
Measured: Response evaluation within first 3 months from start of therapy, then every 3 to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2007 (estimated)
- Study startJun 2004
- Primary completionJun 2009
- Study completionJun 2009
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 SyndromeStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gautam Borthakur, MBBS, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00520468
- Sponsor's protocol ID
- 2004-0253
- Status last verified
- Aug 2012
- First submitted
- 23 Aug 2007
- Last updated
- 7 Aug 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS
- ClinicalTrials.gov ID
- NCT00520468
- Acronym / sponsor ID
- 2004-0253
- Conditions
- Myelodysplastic Syndrome
- Keywords
- Myelodysplastic Syndrome, MDS, Erythropoietin, Darbepoetin alfa, Aranesp, Erythropoiesis stimulating protein, G-CSF, Filgrastim
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- Jun 2004
- Primary completion
- Jun 2009 (actual)
- Study completion
- Jun 2009 (actual)
- First posted
- 24 Aug 2007 (estimated)
- Results
- Posted 14 Apr 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Objectives:
Primary: To evaluate the response rate of total cytokine-immunotherapy for low-risk myelodysplastic syndromes (MDS).
Secondary: To evaluate response duration, survival and side effects of the treatment.
Detailed description
MDS is a disease that produces low blood counts and may cause anemia, infections, and/or bleeding. Erythropoietin and Granulocyte colony-stimulating factor (G-CSF or GCSF) are drugs that stimulate the production of red cells and white cells. Prednisone and cyclosporin are drugs that work against MDS by affecting your immune system.
Before you can start treatment on this study, you will have what are called "screening tests". These tests will help the doctor decide if you are eligible to take part in the study. You will have a complete medical history and physical exam. Routine blood tests (between 4-6 tablespoons) will be performed. You will have a bone marrow aspiration. To collect a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. Women who are able to have children must have a negative blood or urine pregnancy test.
If you are found to be eligible to take part in this study, you will receive erythropoietin as an injection under the skin once a week and G-CSF as an injection under the skin 1-2 times a week for as long as you respond well to treatment. You will take prednisone by mouth every day for a month and cyclosporin tablets by mouth every day for 6 months.
During this study, you will need to visit your doctor for a physical exam and measurement of your vital signs. The frequency of doctor visits will vary depending on your physical condition, but will be required at least once every 3 months.
Blood tests (about 2 teaspoons) will be done about every 1-2 weeks during the first 12 weeks of treatment, then every 2 to 4 weeks for the remainder of the study. The blood samples will be collected for routine lab tests. Periodic (every 3 to 6 months) bone marrow samples will also be taken to check cells related to the disease before, during, and after completion of this study.
You will be taken off study if the disease gets worse or intolerable side effects occur.
This is an investigational study. All drugs are FDA approved and commercially available. Up to 60 patients will take part in this study. All will be enrolled at M. D. Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cytokine-Immunotherapy | Experimental | Drug: Erythropoietin, Drug: Cyclosporin A, Drug: G-CSF, Drug: PrednisoneErythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice a week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients with MDS and </= 10% blasts or International Prognostic Scoring System (IPSS) low or intermediate 1. No prior intensive chemotherapy or high-dose ara-C (>/= 1g/m2). Prior cytokines, biologic therapies, targeted therapies, or single agent chemotherapy allowed. Procrit, G-CSF are allowed before therapy. Patients with blasts < 5% must have an indication for therapy, such as transfusion needs, symptomatic anemia or Hb < 11g/dl, platelets < 100 x 10 9/L, or granulocytes < 10 9/L.
- Performance 0-2 (Eastern Cooperative Oncology Group (ECOG)). Adequate liver function (bilirubin of < 2mg/dl) and renal function (creatinine < 2mg/dl). Adequate cardiac functions (New York Heart Association (NYHA) cardiac III-IV excluded)
- Signed informed consent
Key exclusion criteria (2 listed)
- Nursing and pregnant females are excluded. Women of childbearing potential should practice effective methods of contraception. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
- Patients with active and uncontrolled infections.
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: Darbepoetin alfa, Aranesp, Erythropoiesis stimulating protein40,000 units injected under the skin (SQ) weekly | Drug | Cytokine-Immunotherapy |
| Cyclosporin AAlso: CYA, Cyclosporine, Sandimmune300 mg (tablets) by mouth daily for 6 months | Drug | Cytokine-Immunotherapy |
| G-CSFAlso: Filgrastim, Neupogen300 mcg injected under the skin (SQ) two times per week | Drug | Cytokine-Immunotherapy |
| Prednisone60 mg per day for 7 days, taper over 1 month | Drug | Cytokine-Immunotherapy |
Primary outcome
Number of Participants With Response
Time frame: Response evaluation within first 3 months from start of therapy, then every 3 to 6 months
Periodic bone marrow samples (every 3-6 months) to check cells related to disease before/during/after study. Response classifications categorized by the International Working Group Response Criteria for Myelodysplastic Syndrome (MDS) as: Complete Remission, Partial Response, Hematologic Improvement or No Response.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- M.D. Anderson Cancer Center's WebsiteLink provided by the sponsor.
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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.
Gautam Borthakur, MBBS principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Erythropoietin, Cyclosporin A and G-CSF in people with Myelodysplastic Syndrome, run by M.D. Anderson Cancer Center at 1 site 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 3 inclusion and 2 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 7 Aug 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 14 Apr 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?
Number of Participants With Response (time frame: Response evaluation within first 3 months from start of therapy, then every 3 to 6 months). 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 7 Aug 2012.