Chronic Myeloproliferative DisordersPhase 2NCT00005852
Bone Marrow Transplantation in Treating Patients With Severe Aplastic Anemia or Rejection of Previous Bone Marrow Transplant
A Phase 2 study testing anti-thymocyte globulin, filgrastim and muromonab-CD3 in people with Chronic Myeloproliferative Disorders and Graft Versus Host Disease, run by H. Lee Moffitt Cancer Center and Research Institute.
Trial summary
RATIONALE: Bone marrow from donors may be able to treat patients with severe aplastic anemia and patients whose bodies have rejected previous bone marrow transplantation. PURPOSE: Phase II trial to study the effectiveness of bone marrow transplantation in treating patients who have severe aplastic anemia or whose… Read more about this study
- Who is it for?
- Ages 15 to 55 years
- Conditions the study is for
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia
- What is being tested?
- anti-thymocyte globulin, filgrastim and muromonab-CD3
- Type of treatment
- Surgery or procedure
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Jun 1996to Jul 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS: Histologically confirmed aplastic anemia Must have related donor with a genotypic 6 out of 6 HLA A, B, and DR match OR Bone marrow transplant failure Must have related donor with at least a 5 out of 6 HLA match OR Must have an unrelated donor with at least a 5 out of 6 HLA match
PATIENT CHARACTERISTICS: Age: 15 to 55 Performance status: Aplastic anemia patients: Karnofsky 80-100% Graft failure patients: Karnofsky 20-100% Life expectancy: Not specified Hematopoietic: See Disease Characteristics Hepatic: Aplastic anemia patients: Bilirubin no greater than 2.0 mg/dL SGOT/SGPT no greater than 3 times normal PT/PTT normal (except when aplastic anemia secondary to viral hepatitis) Graft failure patients: Mild to severe venoocclusive disease allowed Renal: Aplastic anemia patients: Creatinine no greater than 2.0 mg/dL Creatinine clearance at least 60 mL/min Cardiovascular: Aplastic anemia patients: Left ventricular ejection fraction at least 45% by MUGA or echocardiography No myocardial infarction within the past 6 months No uncontrolled arrhythmias Pulmonary: Aplastic anemia patients: FEV1 and DLCO at least 50% predicted Other: Aplastic anemia patients: No uncontrolled diabetes mellitus or thyroid disease No active serious infections HIV negative Not pregnant or nursing Negative pregnancy test No psychosocial problem that would preclude study compliance
PRIOR CONCURRENT THERAPY: Biologic therapy: See Disease Characteristics Chemotherapy: Not specified Endocrine therapy: Not specified Radiotherapy: Not specified Surgery: Not specified
About this study
Official title: Allogeneic Bone Marrow Transplantation for Marrow Failure States
RATIONALE: Bone marrow from donors may be able to treat patients with severe aplastic anemia and patients whose bodies have rejected previous bone marrow transplantation.
PURPOSE: Phase II trial to study the effectiveness of bone marrow transplantation in treating patients who have severe aplastic anemia or whose bodies have rejected previous bone marrow transplant.
Read the detailed description
OBJECTIVES: I. Determine the graft failure rate and overall survival of patients with severe aplastic anemia after receiving an allogeneic bone marrow transplant. II. Determine the efficacy of high dose cyclophosphamide and antithymocyte globulin as conditioning regimen for a second bone marrow transplant in patients who reject their first graft. III. Determine the efficacy of methylprednisolone and anti-CD3 monoclonal antibody as a conditioning regimen for a second bone marrow transplant in patients who reject their first graft and are poor candidates for cyclophosphamide and antithymocyte globulin.
OUTLINE: Patients with graft failure with the following organ function and performance status are assigned to arm I: Performance status: Karnofsky 60-100% Pulmonary: FEV1 and DLCO at least 50% Cardiac: Left ventricular ejection fraction at least 45% Hepatic: None to mild venoocclusive disease Patients with graft failure with the following organ function and performance status are assigned to arm II: Performance status: Karnofsky 20-50% Pulmonary: FEV1 or DLCO less than 50% Cardiac: Left ventricular ejection fraction less than 45% Hepatic: Moderate to severe venoocclusive disease Patients with aplastic anemia are assigned to arm I. Arm I: Patients receive cyclophosphamide IV over 1 hour on days -5 to -2, antithymocyte globulin IV over 8 hours on days -5 to -3, and bone marrow transplant on day 0. Arm II: Patients receive methylprednisolone IV twice a day on days -5 to 62; monoclonal antibody OKT3 IV on days -1 to 24; bone marrow transplant on day 0; oral cyclosporine twice a day starting day 20; and filgrastim (G-CSF) subcutaneously once a day starting day -5 until blood counts recover. Patients are followed once a week for 3 months and then monthly for one year.
PROJECTED ACCRUAL: A total of 40 patients (20 with aplastic anemia and 20 with graft failure) will be accrued for this study.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Being tested
- anti-thymocyte globulin, filgrastim, muromonab-CD3, cyclophosphamide, cyclosporine
- Keywords
- recurrent childhood acute lymphoblastic leukemia, recurrent adult Hodgkin lymphoma, recurrent cutaneous T-cell non-Hodgkin lymphoma, refractory multiple myeloma, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, recurrent childhood lymphoblastic lymphoma, recurrent childhood acute myeloid leukemia, recurrent adult acute myeloid leukemia, recurrent adult acute lymphoblastic leukemia, relapsing chronic myelogenous leukemia, refractory chronic lymphocytic leukemia, primary myelofibrosis, childhood acute promyelocytic leukemia (M3), refractory hairy cell leukemia, recurrent/refractory childhood Hodgkin lymphoma, recurrent grade 1 follicular lymphoma, recurrent grade 2 follicular lymphoma, recurrent grade 3 follicular lymphoma
- Medical subject headings
- Myeloproliferative Disorders, Graft vs Host Disease, Leukemia, Lymphoma, Multiple Myeloma, Neoplasms, Plasma Cell, Myelodysplastic Syndromes, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
filgrastimBiological
muromonab-CD3Biological
cyclophosphamideDrug
cyclosporineDrug
allogeneic bone marrow transplantationProcedure or surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2004 (estimated)
- Study startJun 1996
- Primary completionJul 2007
- Study completionJul 2007
Why it stopped: low accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myeloproliferative DisordersStudies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe 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
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Steven C. Goldstein, MD, Study chair, H. Lee Moffitt Cancer Center and Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00005852
- Sponsor's protocol ID
- MCC-11309
- Other IDs
- MCC-11309, MCC-IRB-4251, NCI-G00-1791
- Status last verified
- Dec 2012
- First submitted
- 2 Jun 2000
- Last updated
- 11 Dec 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Bone Marrow Transplantation for Marrow Failure States
- ClinicalTrials.gov ID
- NCT00005852
- Acronym / sponsor ID
- MCC-11309
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Keywords
- recurrent childhood acute lymphoblastic leukemia, recurrent adult Hodgkin lymphoma, recurrent cutaneous T-cell non-Hodgkin lymphoma, refractory multiple myeloma, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, recurrent childhood lymphoblastic lymphoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: low accrual
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jun 1996
- Primary completion
- Jul 2007 (actual)
- Study completion
- Jul 2007 (actual)
- First posted
- 26 Jul 2004 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Bone marrow from donors may be able to treat patients with severe aplastic anemia and patients whose bodies have rejected previous bone marrow transplantation.
PURPOSE: Phase II trial to study the effectiveness of bone marrow transplantation in treating patients who have severe aplastic anemia or whose bodies have rejected previous bone marrow transplant.
Detailed description
OBJECTIVES: I. Determine the graft failure rate and overall survival of patients with severe aplastic anemia after receiving an allogeneic bone marrow transplant. II. Determine the efficacy of high dose cyclophosphamide and antithymocyte globulin as conditioning regimen for a second bone marrow transplant in patients who reject their first graft. III. Determine the efficacy of methylprednisolone and anti-CD3 monoclonal antibody as a conditioning regimen for a second bone marrow transplant in patients who reject their first graft and are poor candidates for cyclophosphamide and antithymocyte globulin.
OUTLINE: Patients with graft failure with the following organ function and performance status are assigned to arm I: Performance status: Karnofsky 60-100% Pulmonary: FEV1 and DLCO at least 50% Cardiac: Left ventricular ejection fraction at least 45% Hepatic: None to mild venoocclusive disease Patients with graft failure with the following organ function and performance status are assigned to arm II: Performance status: Karnofsky 20-50% Pulmonary: FEV1 or DLCO less than 50% Cardiac: Left ventricular ejection fraction less than 45% Hepatic: Moderate to severe venoocclusive disease Patients with aplastic anemia are assigned to arm I. Arm I: Patients receive cyclophosphamide IV over 1 hour on days -5 to -2, antithymocyte globulin IV over 8 hours on days -5 to -3, and bone marrow transplant on day 0. Arm II: Patients receive methylprednisolone IV twice a day on days -5 to 62; monoclonal antibody OKT3 IV on days -1 to 24; bone marrow transplant on day 0; oral cyclosporine twice a day starting day 20; and filgrastim (G-CSF) subcutaneously once a day starting day -5 until blood counts recover. Patients are followed once a week for 3 months and then monthly for one year.
PROJECTED ACCRUAL: A total of 40 patients (20 with aplastic anemia and 20 with graft failure) will be accrued for this study.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 15 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
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 |
|---|---|---|
| anti-thymocyte globulin | Biological | – |
| filgrastim | Biological | – |
| muromonab-CD3 | Biological | – |
| cyclophosphamide | Drug | – |
| cyclosporine | Drug | – |
| methylprednisolone | Drug | – |
| allogeneic bone marrow transplantation | Procedure or surgery | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early (reason given: low accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2012.
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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.
Steven C. Goldstein, MD study chair
H. Lee Moffitt Cancer Center and Research Institute
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing anti-thymocyte globulin, filgrastim and muromonab-CD3 in people with Chronic Myeloproliferative Disorders and Graft Versus Host Disease, run by H. Lee Moffitt Cancer Center and Research Institute.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 11 Dec 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. 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?
The study was stopped early (reason given: low accrual). Results are not available in the source record.
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.
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 11 Dec 2012.