Myelodysplastic SyndromePhase 2NCT00806598
Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
A Phase 2 study testing Thymoglobulin, Cyclosporine and Methylprednisolone in people with Myelodysplastic Syndrome and Aplastic Anemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to learn if combining the drugs thymoglobulin, methylprednisolone, cyclosporine, and G-CSF (NeupogenTM or NeulastaTM ) can help to control severe aplastic anemia (AA) or hypoplastic myelodysplastic syndrome (MDS). The safety of this combination therapy will also be studied. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, Aplastic Anemia
- What is being tested?
- Thymoglobulin, Cyclosporine and Methylprednisolone
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 53 participants
- Where
- 1 siteUnited States
- Study dates
- May 2005to Jun 2012
- 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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of severe aplastic anemia (bone marrow cellularity < 30%, with two of three peripheral counts at the time of initial presentation or currently low with absolute neutrophil count (ANC) < 500/mL, pre-transfusion platelet (PLT) < 20,000/mL, or pre-transfusion hemoglobin < 8 g/dL and presence of no other underlying disorder.
- Diagnosis of MDS (World Health Organization) with bone marrow cellularity < 30%, with two of three peripheral counts at the time of initial presentation or currently low with ANC < 500/mL, pre-transfusion PLT < 20,000/mL, or pre-transfusion hemoglobin < 8 g/dL.
- Patients with MDS who have received prior biological therapy (not chemotherapy) are eligible. Hypomethylating agents and histone deacetylase inhibitors are considered as biological therapy.
- Age 15 or greater
- Adequate renal function (creatinine less than or equal to 2.0 mg/dL) unless related to the disease
- Adequate hepatic function (bilirubin less than or equal to 3.5 mg/dL) unless related to the disease
Show all 10Show fewer
- No other investigational therapy in the past 14 days
- Able to sign consent form
- Able to comply with the need for contraception (abstinence, condom, birth control pill, or other acceptable form of contraception) during the entire study period
- Diagnosis of MDS (WHO) with bone marrow cellularity greater than 30%, with low or intermediate-1 risk by the International Prognostic Scoring System (IPSS) score, and requiring treatment (i.e. transfusion-dependent)
Who cannot join
- Active and uncontrolled infection
- HIV positive test
- Pregnant or breast feeding
- Active and uncontrolled medical illness (pulmonary, cardiac, neurological, or other) that in the opinion of treating physician would likely interfere with study treatment
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Study of Combination of Thymoglobulin, Cyclosporine, Methylprednisone, and Granulocyte Colony-stimulating Factor (GCSF) in Patients With Newly Diagnosed Aplastic Anemia or With Hypoplastic or Low/Intermediate-1 Risk Myelodysplastic Syndrome
The goal of this clinical research study is to learn if combining the drugs thymoglobulin, methylprednisolone, cyclosporine, and G-CSF (NeupogenTM or NeulastaTM ) can help to control severe aplastic anemia (AA) or hypoplastic myelodysplastic syndrome (MDS). The safety of this combination therapy will also be studied.
Read the detailed description
Aplastic anemia is a condition that involves a low level of red blood cells (anemia), white blood cells, and platelets without evidence of another bone marrow disease. Anemia leads to fatigue, shortness of breath, and heart problems. Low platelet counts can lead to bruising and bleeding, and low white blood cell counts may cause an increased risk of infection, including pneumonia. Some of the treatment for AA includes transfusion, antibiotics and a combination of anti-thymocyte globulin (ATG) and cyclosporine with or without steroids, and growth factors such as G-CSF. For those who are eligible and have a donor, stem cell/bone marrow transplantation may be used.
MDS is a bone marrow disorder that usually affects older adults. Treatment of the bone marrow failure that accompanies MDS is usually with supportive care with red blood cell and platelet transfusions, antibiotics, and combinations of hematopoietic growth factors, which may partially improve blood cell counts.
It is often difficult to distinguish the hypoplastic variety of MDS from severe AA because both can result in bone marrow tests with very low cell count numbers. Earlier studies have shown that in some patients with the hypoplastic MDS, low blood counts respond to immunosuppressive treatment with ATG and cyclosporine. ATG is made from horse plasma. Thymoglobulin is a type of ATG made from rabbit plasma. Thymoglobulin has been successfully used to treat patients with AA who were previously treated with horse ATG but whose disease has returned. G-CSF is a growth factor that helps raise the white cell count after receiving chemotherapy. Methylprednisolone is a steroid that is commonly used in treating a number of medical conditions associated with people's abnormal immune response against themselves.
If you are found to be eligible to take part in this study, you will receive a combination of thymoglobulin, cyclosporine, G-CSF, and methylprednisolone. Treatment will be with thymoglobulin, which will be dosed depending on your age and weight. It will be given by vein over several hours once a day for a total of 5 days. You will receive the steroid methylprednisolone by vein before each dose of thymoglobulin to decrease the risk of developing allergic reactions to thymoglobulin. After 5 days of receiving methylprednisolone by vein, you will start taking it by mouth once a day at a decreasing dose over about 3 weeks.
The first 5 days of treatment will be given at M. D. Anderson but you will treated outside the hospital for the rest of the time unless complications develop.
You will be started on cyclosporine as well as G-CSF after completion of thymoglobulin. You will take cyclosporine by mouth twice a day for 6 months. Your physician may continue cyclosporine longer at his discretion. You will receive G-CSF as an injection under the skin for 3 months (or longer) once a day at the discretion of the treating physician starting at the same time as cyclosporine is started.
You will also receive antibiotic pills to help decrease the risk of infection. You will take levofloxacin, valacyclovir, fluconazole, or a similar antibiotic by mouth every day for the length of the study or until your treating physician finds appropriate.
If you have a history of heart disease and you take aspirin for this, your treating physician may consider stopping the aspirin because of your low platelet count as a result of your disease. This may increase your risk of heart attacks.
You will have blood tests (about 2 tablespoons each) once or twice a week for the first month and then once every 2-4 weeks until the end of the study to check if your blood counts are improving. The level of cyclosporine in your blood will also be checked at these times. It will require about 1 tablespoon for each of these tests. Monitoring of cyclosporine levels is a routine test done on all patients who receive this drug in order to avoid toxic blood levels and side effects.
At about 3 months, you will have a repeat bone marrow biopsy and aspiration as well as blood tests (about 3 tablespoons) to evaluate your response to the treatment.
You will continue cyclosporine for about 6 months (or longer if your doctor feels that it is in your interest) and will receive G-CSF for up to about 3 months (or longer if in your interest). If you develop serious side effects or if the disease gets worse at any time you will be taken off the study.
At the time when your doctor feels you have had the best possible response to the treatment and you are coming off study, blood tests (about 3 tablespoons) will be repeated.
Your doctor will continue to remain in touch with you to ensure that your disease remains under control. This may be done by arranging follow-up visits or through phone or other means of communication.
This is an investigational study. All the drugs used in this study are FDA approved and commercially available. Their use together in this study is experimental. A total of 60 patients will take part in this study. All will be enrolled at M. D. Anderson.
- Conditions
- Myelodysplastic Syndrome, Aplastic Anemia
- Being tested
- Thymoglobulin, Cyclosporine, Methylprednisolone, G-CSF
- Keywords
- Myelodysplastic Syndrome, Aplastic Anemia, Thymoglobulin, Cyclosporine, Methylprednisolone, G-CSF
- Medical subject headings
- Myelodysplastic Syndromes, Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThymoglobulinDrug
Also called ATG, Antithymocyte globulin
3.5 or 2.5 mg/kg/day IV for 5 days
- Aplastic anemia patients receive 3.5 mg/kg/day for 5 days
- MDS patients <55 years receive 3.5 mg/kg/day for 5 days
- MDS patients >55 years receive 2.5 mg/kg/day for 5 days
CyclosporineDrug
Also called CYA, Sandimmune, Cyclosporine A
5 mg/kg orally for 6 months; start after completing thymoglobulin.
Also called Duralone®, Medralone®, Medrol®, M-Prednisol®
1 mg/kg/day IV for 5 days, given before each dose of thymoglobulin.
G-CSFDrug
Also called Neupogen®, Granulocyte - Colony Stimulating Factor
5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin.
| Study group | Role | What they receive |
|---|---|---|
| Thymoglobulin + Cyclosporin | Experimental | Combination of Thymoglobulin 3.5 or 2.5 mg/kg/day intravenous (IV) for 5 days + Methylprednisone 1 mg/kg/day IV for 5 days, before each dose Thymoglobulin + Cyclosporin 5 mg/kg orally for 6 months following Thymoglobulin + Granulocyte - Colony Stimulating Factor (G-CSF) 5 microgram/kg subcutaneously daily up to 3 months Receives: Drug: Thymoglobulin; Drug: Cyclosporine; Drug: Methylprednisolone; Drug: G-CSF |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response
Complete response (CR) was defined as normalization of peripheral blood and bone marrow with <5% blasts, a peripheral absolute neutrophil count (ANC) >/= 1 * 10^9/l, hemoglobin >/= 100g/l, and a platelet count >/= * 10^9/l, Partial Response (PR) was defined as transfusion independence with a peripheral blood ANC >=/ 0.05 * 10^9/l, a platelet count >/= 20 * 10^9/l, and a hemoglobin >/= 40 g/l. Hematologic improvement was defined as a clinically relevant increase in hemoglobin, platelets or absolute neutrophil count.
Measured: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first. Assessed first at 3 months on study, continuing monthly up to 3 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2008 (estimated)
- Study startMay 2005
- Primary completionJun 2012
- Study completionJun 2012
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 53 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Responsible party
- Sponsor
- Investigators
- Tapan M. Kadia, M.D., Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00806598
- Sponsor's protocol ID
- 2005-0115
- Status last verified
- Mar 2013
- First submitted
- 9 Dec 2008
- Last updated
- 12 Mar 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Combination of Thymoglobulin, Cyclosporine, Methylprednisone, and Granulocyte Colony-stimulating Factor (GCSF) in Patients With Newly Diagnosed Aplastic Anemia or With Hypoplastic or Low/Intermediate-1 Risk Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT00806598
- Acronym / sponsor ID
- 2005-0115
- Conditions
- Myelodysplastic Syndrome, Aplastic Anemia
- Keywords
- Myelodysplastic Syndrome, Aplastic Anemia, Thymoglobulin, Cyclosporine, Methylprednisolone, G-CSF
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 53 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Study start
- May 2005
- Primary completion
- Jun 2012 (actual)
- Study completion
- Jun 2012 (actual)
- First posted
- 11 Dec 2008 (estimated)
- Results
- Posted 6 Mar 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical research study is to learn if combining the drugs thymoglobulin, methylprednisolone, cyclosporine, and G-CSF (NeupogenTM or NeulastaTM ) can help to control severe aplastic anemia (AA) or hypoplastic myelodysplastic syndrome (MDS). The safety of this combination therapy will also be studied.
Detailed description
Aplastic anemia is a condition that involves a low level of red blood cells (anemia), white blood cells, and platelets without evidence of another bone marrow disease. Anemia leads to fatigue, shortness of breath, and heart problems. Low platelet counts can lead to bruising and bleeding, and low white blood cell counts may cause an increased risk of infection, including pneumonia. Some of the treatment for AA includes transfusion, antibiotics and a combination of anti-thymocyte globulin (ATG) and cyclosporine with or without steroids, and growth factors such as G-CSF. For those who are eligible and have a donor, stem cell/bone marrow transplantation may be used.
MDS is a bone marrow disorder that usually affects older adults. Treatment of the bone marrow failure that accompanies MDS is usually with supportive care with red blood cell and platelet transfusions, antibiotics, and combinations of hematopoietic growth factors, which may partially improve blood cell counts.
It is often difficult to distinguish the hypoplastic variety of MDS from severe AA because both can result in bone marrow tests with very low cell count numbers. Earlier studies have shown that in some patients with the hypoplastic MDS, low blood counts respond to immunosuppressive treatment with ATG and cyclosporine. ATG is made from horse plasma. Thymoglobulin is a type of ATG made from rabbit plasma. Thymoglobulin has been successfully used to treat patients with AA who were previously treated with horse ATG but whose disease has returned. G-CSF is a growth factor that helps raise the white cell count after receiving chemotherapy. Methylprednisolone is a steroid that is commonly used in treating a number of medical conditions associated with people's abnormal immune response against themselves.
If you are found to be eligible to take part in this study, you will receive a combination of thymoglobulin, cyclosporine, G-CSF, and methylprednisolone. Treatment will be with thymoglobulin, which will be dosed depending on your age and weight. It will be given by vein over several hours once a day for a total of 5 days. You will receive the steroid methylprednisolone by vein before each dose of thymoglobulin to decrease the risk of developing allergic reactions to thymoglobulin. After 5 days of receiving methylprednisolone by vein, you will start taking it by mouth once a day at a decreasing dose over about 3 weeks.
The first 5 days of treatment will be given at M. D. Anderson but you will treated outside the hospital for the rest of the time unless complications develop.
You will be started on cyclosporine as well as G-CSF after completion of thymoglobulin. You will take cyclosporine by mouth twice a day for 6 months. Your physician may continue cyclosporine longer at his discretion. You will receive G-CSF as an injection under the skin for 3 months (or longer) once a day at the discretion of the treating physician starting at the same time as cyclosporine is started.
You will also receive antibiotic pills to help decrease the risk of infection. You will take levofloxacin, valacyclovir, fluconazole, or a similar antibiotic by mouth every day for the length of the study or until your treating physician finds appropriate.
If you have a history of heart disease and you take aspirin for this, your treating physician may consider stopping the aspirin because of your low platelet count as a result of your disease. This may increase your risk of heart attacks.
You will have blood tests (about 2 tablespoons each) once or twice a week for the first month and then once every 2-4 weeks until the end of the study to check if your blood counts are improving. The level of cyclosporine in your blood will also be checked at these times. It will require about 1 tablespoon for each of these tests. Monitoring of cyclosporine levels is a routine test done on all patients who receive this drug in order to avoid toxic blood levels and side effects.
At about 3 months, you will have a repeat bone marrow biopsy and aspiration as well as blood tests (about 3 tablespoons) to evaluate your response to the treatment.
You will continue cyclosporine for about 6 months (or longer if your doctor feels that it is in your interest) and will receive G-CSF for up to about 3 months (or longer if in your interest). If you develop serious side effects or if the disease gets worse at any time you will be taken off the study.
At the time when your doctor feels you have had the best possible response to the treatment and you are coming off study, blood tests (about 3 tablespoons) will be repeated.
Your doctor will continue to remain in touch with you to ensure that your disease remains under control. This may be done by arranging follow-up visits or through phone or other means of communication.
This is an investigational study. All the drugs used in this study are FDA approved and commercially available. Their use together in this study is experimental. A total of 60 patients will take part in this study. All will be enrolled at M. D. Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thymoglobulin + Cyclosporin | Experimental | Drug: Thymoglobulin, Drug: Cyclosporine, Drug: Methylprednisolone, Drug: G-CSFCombination of Thymoglobulin 3.5 or 2.5 mg/kg/day intravenous (IV) for 5 days + Methylprednisone 1 mg/kg/day IV for 5 days, before each dose Thymoglobulin + Cyclosporin 5 mg/kg orally for 6 months following Thymoglobulin + Granulocyte - Colony Stimulating Factor (G-CSF) 5 microgram/kg subcutaneously daily up to 3 months |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Diagnosis of severe aplastic anemia (bone marrow cellularity < 30%, with two of three peripheral counts at the time of initial presentation or currently low with absolute neutrophil count (ANC) < 500/mL, pre-transfusion platelet (PLT) < 20,000/mL, or pre-transfusion hemoglobin < 8 g/dL and presence of no other underlying disorder.
- Diagnosis of MDS (World Health Organization) with bone marrow cellularity < 30%, with two of three peripheral counts at the time of initial presentation or currently low with ANC < 500/mL, pre-transfusion PLT < 20,000/mL, or pre-transfusion hemoglobin < 8 g/dL.
- Patients with MDS who have received prior biological therapy (not chemotherapy) are eligible. Hypomethylating agents and histone deacetylase inhibitors are considered as biological therapy.
- Age 15 or greater
- Adequate renal function (creatinine less than or equal to 2.0 mg/dL) unless related to the disease
Key exclusion criteria (4 listed)
- Active and uncontrolled infection
- HIV positive test
- Pregnant or breast feeding
- Active and uncontrolled medical illness (pulmonary, cardiac, neurological, or other) that in the opinion of treating physician would likely interfere with study treatment
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 |
|---|---|---|
| ThymoglobulinAlso: ATG, Antithymocyte globulin3.5 or 2.5 mg/kg/day IV for 5 days * Aplastic anemia patients receive 3.5 mg/kg/day for 5 days * MDS patients <55 years receive 3.5 mg/kg/day for 5 days * MDS patients >55 years receive 2.5 mg/kg/day for 5 days | Drug | Thymoglobulin + Cyclosporin |
| CyclosporineAlso: CYA, Sandimmune, Cyclosporine A5 mg/kg orally for 6 months; start after completing thymoglobulin. | Drug | Thymoglobulin + Cyclosporin |
| MethylprednisoloneAlso: Duralone®, Medralone®, Medrol®, M-Prednisol®1 mg/kg/day IV for 5 days, given before each dose of thymoglobulin. | Drug | Thymoglobulin + Cyclosporin |
| G-CSFAlso: Neupogen®, Granulocyte - Colony Stimulating Factor5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin. | Drug | Thymoglobulin + Cyclosporin |
Primary outcome
Overall Response
Time frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first. Assessed first at 3 months on study, continuing monthly up to 3 years.
Complete response (CR) was defined as normalization of peripheral blood and bone marrow with <5% blasts, a peripheral absolute neutrophil count (ANC) >/= 1 * 10^9/l, hemoglobin >/= 100g/l, and a platelet count >/= * 10^9/l, Partial Response (PR) was defined as transfusion independence with a peripheral blood ANC >=/ 0.05 * 10^9/l, a platelet count >/= 20 * 10^9/l, and a hemoglobin >/= 40 g/l. Hematologic improvement was defined as a clinically relevant increase in hemoglobin, platelets or absolute neutrophil count.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- University of Texas MD Anderson Cancer Center Official websiteLink provided by the sponsor.
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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.
Tapan M. Kadia, M.D. 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 Thymoglobulin, Cyclosporine and Methylprednisolone in people with Myelodysplastic Syndrome and Aplastic Anemia, 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 10 inclusion and 4 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 12 Mar 2013. 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 6 Mar 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?
Overall Response (time frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first. Assessed first at 3 months on study, continuing monthly up to 3 years.). 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 12 Mar 2013.