Chronic Myeloproliferative DisordersPhase 2MPDMDSBMTNCT00069992
Total-Body Irradiation, Fludarabine, and Alemtuzumab Followed By Stem Cell Transplant in Treating Patients With Myeloproliferative Disorder, MS, AML, or CML
A Phase 2 study testing Total Body Irradiation, Fludarabine and Campath 1H in people with Chronic Myeloproliferative Disorders and Leukemia, run by Baylor College of Medicine at 2 sites in United States.
Trial summary
Patients are being asked to participate in this study because they have a malignant blood disease such as Myelodysplastic Syndrome (MDS), Myeloproliferative Disorder (MPD), Acute Myelogenous Leukemia (AML) or Chronic Myelogenous Leukemia (CML). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Chronic Myeloproliferative Disorders, Leukemia, Myelodysplastic Syndromes
- What is being tested?
- Total Body Irradiation, Fludarabine and Campath 1H
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 2 sitesUnited States
- Study dates
- Dec 2001to Apr 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Myelodysplastic syndrome with IPSS score > 0.(Appendix B) Or
- Myeloproliferative disorders
- Primary Myelofibrosis with Lile score of 1 or 2 (Appendix C)
- Polycythemia Vera or Essential Thrombocythemia transformed to AML or Myelofibrosis and PV "spent phase" or
- Acute myelogenous leukemia or
- Chronic myelogenous leukemia
- Available Healthy Donor without any contraindications for donation. 5/6 or 6/6 related donor or 5/6 or 6/6 unrelated donor (molecular typing for DRB1)
- Able to give informed consent
Who cannot join
- Patient is pregnant or lactating or unwilling to use contraceptives.
- HIV positive patient
- Uncontrolled intercurrent infection
- Unstable angina and uncompensated congestive heart failure (Zubrod of 3 or greater)
- Severe chronic pulmonary disease requiring oxygen (Zubrod of 3 or greater)
- Hemodialysis dependent.
Show all 10Show fewer
- Active hepatitis or cirrhosis with total bilirubin, SGOT, and SGPT greater than 3 x normal.
- Concurrent solid organ malignancy not in remission, except for Stage 0 or A prostate cancer.
- Unstable cerebral vascular disease or recent hemorrhagic stroke (less than 6 months)
- Active CNS disease from hematological disorder.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Texas Children's Hospital
Houston, Texas, 77030
The Methodist Hospital
Houston, Texas, 77030
About this study
Official title: Safety And Efficacy of Sub-Myeloablative Allogeneic Stem Cell Transplantation For Patients With Myeloproliferative Disorder (MPD), Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML) or Chronic Myelogenous Leukemia
Patients are being asked to participate in this study because they have a malignant blood disease such as Myelodysplastic Syndrome (MDS), Myeloproliferative Disorder (MPD), Acute Myelogenous Leukemia (AML) or Chronic Myelogenous Leukemia (CML). We feel that patients could benefit from an allogeneic (meaning the cells come from a donor other than themself) stem cell transplant. The donor would be a family member or an unrelated person that is felt to be a good match for the patient. Stem cells are cells that are made in the bone marrow (spongy material that fills the middle of the bones). As the stem cells grow, they change into different types of blood cells that they need. This includes red blood cells that carry oxygen around the body, white blood cells that help to fight infections, and platelets that help to prevent and stop bleeding. Usually, patients are given high doses of chemotherapy before a stem cell transplant. High doses of chemo destroy the bone marrow. Healthy stem cells from a donor are then given to replace the patient's unhealthy cells. However, because of complications with the patient's disease, they have a high risk of having life-threatening side effects. These include serious damage to organs such as the lung, liver, kidney and heart. There is also an increased risk of bacterial, fungal, and viral infections. The other major problem is when a donor's stem cells (also called the graft) find that the patient's cells ( the host cells) are not the same. The donor cells may try to destroy the host's cells. The cells at high risk are those of the skin, liver and intestines. This is called graft versus host disease (GVHD) and it can be fatal.
Recently, doctors have been able to use less toxic chemotherapy treatments before patients receive their transplants. This less toxic treatment helps reduce some of the treatment related problems mentioned above. Patient's are being asked to be involved in a research study that uses this approach. One major risk of this low dose treatment is that the patient's body may reject the donor cells. This is called graft rejection. This study is designed to see if this low dose treatment is safe and effective.
Read more
This treatment plan adds CAMPATH 1H (a special protein called an antibody) to a low dose chemotherapy regimen. After chemo, the patient will receive an allogeneic (cells come from a donor) stem cell transplant. Adding CAMPATH 1H to the transplant medicines may help in treating the disease. CAMPATH 1H may reduce life-threatening and treatment related side effects like GVHD. CAMPATH 1H stays active in the body for a long time which means it may work longer to prevent GVHD. CAMPATH 1H destroys lymphocytes, a type of white cells that help fight infection, and this helps prevent graft rejection.
We want to see if the addition of CAMPATH 1H to the patient's pre-transplant low dose chemotherapy will decrease the side effects from an allogeneic stem cell transplant, while providing a curative treatment for patients with blood disorders.
Read the detailed description
We expect that the patient's participation in this study will last approximately 18 months to 2 years.
Before treatment begins, they will be evaluated to confirm they meet the requirements of this study. The evaluation includes HIV testing, HIV (Human Immunodeficiency Virus) is the virus that causes Acquired Immune Deficiency Syndrome (AIDS). If the patient is HIV positive, they will not be able to be treated on this protocol.
The patient will need to have a central line. This is a thin plastic catheter or tube that is placed during surgery into one of the large veins in the chest or neck. Central lines are used to give medications IV (intravenous, by vein) or to take blood samples without having to endure frequent needle sticks.
After admission to the hospital the subject will receive:
Day -6: a single dose of total body irradiation
Day -5 to Day -2 Chemotherapy: Fludarabine plus Campath 1H through a catheter inserted into a vein (IV)
Day -2: FK506 given IV over a 24 hour period until the patient can take medication by mouth. When they can take oral medication they will take this medication by mouth every 12 hours.
Day -1 : a day of rest
Day 0: the stem cell transplant (infusion) will be given
Day +7: G-CSF will be given by subcutaneous injection until your white blood cells (granulocytes) are greater than 1000/ul.
After transplantation, they will be evaluated as follows. Routine history, physical examination, blood tests and radiology studies will be done as needed for clinical care. Bone marrow aspirate and biopsy will be done on or about day 30, 60 and 100, 180 and then yearly and as needed.
- Being tested
- Total Body Irradiation, Fludarabine, Campath 1H
- Keywords
- polycythemia vera, essential thrombocythemia, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes, primary myelofibrosis, chronic myelogenous leukemia, adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with inv(16)(p13;q22), adult acute myeloid leukemia with t(15;17)(q22;q12), adult acute myeloid leukemia with t(16;16)(p13;q22), adult acute myeloid leukemia with t(8;21)(q22;q22), childhood myelodysplastic syndromes
- Medical subject headings
- Myeloproliferative Disorders, Leukemia, Myelodysplastic Syndromes, Polycythemia Vera, Thrombocythemia, Essential, Primary Myelofibrosis, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Congenital Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Total Body IrradiationRadiation
Also called TBI
Total body irradiation of 450cGy as a single dose, day -6
FludarabineDrug
Also called Fludara
Fludarabine 30mg/m2 Day -5 to -2
Campath 1HDrug
Also called Alemtuzumab
Campath 1H dosing as per institutional SOPs Day -5 to -2
| Study group | Role | What they receive |
|---|---|---|
| Submyeloablative Allogeneic Stem Cell Transplant | Experimental | Total Body Irradiation Fludarabine Campath 1H Receives: Radiation: Total Body Irradiation; Drug: Fludarabine; Drug: Campath 1H |
What is being measured
The primary outcome is the main question the study is designed to answer.
Day 100 Non-relapse mortality,
Safety and feasibility of submyeloablative conditioning as a preparative regimen for blood stem cell transplantation
Measured: 100 days
Day 100 graft rejection
Safety and feasibility of submyeloablative conditioning as a preparative regimen for blood stem cell transplantation
Measured: 100 days
Secondary outcomes (2)
1 year disease free survival
Measured: 1 year
Complete Remission at 100 days
Measured: 100 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2003 (estimated)
- Study startDec 2001
- Primary completionSep 2006
- Study completionApr 2007
Why it stopped: Closed due to competing protocols
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 Baylor College of MedicineThe 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
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
- Responsible party
- Principal investigator, George Carrum, Baylor College of Medicine
- Investigators
- George Carrum, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00069992
- Sponsor's protocol ID
- H-10857-MPDMDSBMT
- Acronym
- MPDMDSBMT
- Status last verified
- Oct 2012
- First submitted
- 3 Oct 2003
- Last updated
- 10 Oct 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety And Efficacy of Sub-Myeloablative Allogeneic Stem Cell Transplantation For Patients With Myeloproliferative Disorder (MPD), Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML) or Chronic Myelogenous Leukemia
- ClinicalTrials.gov ID
- NCT00069992
- Acronym / sponsor ID
- MPDMDSBMT / H-10857-MPDMDSBMT
- Keywords
- polycythemia vera, essential thrombocythemia, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes, primary myelofibrosis, chronic myelogenous leukemia, adult acute myeloid leukemia with 11q23 (MLL) abnormalities
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Closed due to competing protocols
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
- Study start
- Dec 2001
- Primary completion
- Sep 2006 (actual)
- Study completion
- Apr 2007 (actual)
- First posted
- 7 Oct 2003 (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.
Patients are being asked to participate in this study because they have a malignant blood disease such as Myelodysplastic Syndrome (MDS), Myeloproliferative Disorder (MPD), Acute Myelogenous Leukemia (AML) or Chronic Myelogenous Leukemia (CML). We feel that patients could benefit from an allogeneic (meaning the cells come from a donor other than themself) stem cell transplant. The donor would be a family member or an unrelated person that is felt to be a good match for the patient. Stem cells are cells that are made in the bone marrow (spongy material that fills the middle of the bones). As the stem cells grow, they change into different types of blood cells that they need. This includes red blood cells that carry oxygen around the body, white blood cells that help to fight infections, and platelets that help to prevent and stop bleeding. Usually, patients are given high doses of chemotherapy before a stem cell transplant. High doses of chemo destroy the bone marrow. Healthy stem cells from a donor are then given to replace the patient's unhealthy cells. However, because of complications with the patient's disease, they have a high risk of having life-threatening side effects. These include serious damage to organs such as the lung, liver, kidney and heart. There is also an increased risk of bacterial, fungal, and viral infections. The other major problem is when a donor's stem cells (also called the graft) find that the patient's cells ( the host cells) are not the same. The donor cells may try to destroy the host's cells. The cells at high risk are those of the skin, liver and intestines. This is called graft versus host disease (GVHD) and it can be fatal.
Recently, doctors have been able to use less toxic chemotherapy treatments before patients receive their transplants. This less toxic treatment helps reduce some of the treatment related problems mentioned above. Patient's are being asked to be involved in a research study that uses this approach. One major risk of this low dose treatment is that the patient's body may reject the donor cells. This is called graft rejection. This study is designed to see if this low dose treatment is safe and effective.
This treatment plan adds CAMPATH 1H (a special protein called an antibody) to a low dose chemotherapy regimen. After chemo, the patient will receive an allogeneic (cells come from a donor) stem cell transplant. Adding CAMPATH 1H to the transplant medicines may help in treating the disease. CAMPATH 1H may reduce life-threatening and treatment related side effects like GVHD. CAMPATH 1H stays active in the body for a long time which means it may work longer to prevent GVHD. CAMPATH 1H destroys lymphocytes, a type of white cells that help fight infection, and this helps prevent graft rejection.
We want to see if the addition of CAMPATH 1H to the patient's pre-transplant low dose chemotherapy will decrease the side effects from an allogeneic stem cell transplant, while providing a curative treatment for patients with blood disorders.
Detailed description
We expect that the patient's participation in this study will last approximately 18 months to 2 years.
Before treatment begins, they will be evaluated to confirm they meet the requirements of this study. The evaluation includes HIV testing, HIV (Human Immunodeficiency Virus) is the virus that causes Acquired Immune Deficiency Syndrome (AIDS). If the patient is HIV positive, they will not be able to be treated on this protocol.
The patient will need to have a central line. This is a thin plastic catheter or tube that is placed during surgery into one of the large veins in the chest or neck. Central lines are used to give medications IV (intravenous, by vein) or to take blood samples without having to endure frequent needle sticks.
After admission to the hospital the subject will receive:
Day -6: a single dose of total body irradiation
Day -5 to Day -2 Chemotherapy: Fludarabine plus Campath 1H through a catheter inserted into a vein (IV)
Day -2: FK506 given IV over a 24 hour period until the patient can take medication by mouth. When they can take oral medication they will take this medication by mouth every 12 hours.
Day -1 : a day of rest
Day 0: the stem cell transplant (infusion) will be given
Day +7: G-CSF will be given by subcutaneous injection until your white blood cells (granulocytes) are greater than 1000/ul.
After transplantation, they will be evaluated as follows. Routine history, physical examination, blood tests and radiology studies will be done as needed for clinical care. Bone marrow aspirate and biopsy will be done on or about day 30, 60 and 100, 180 and then yearly and as needed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Submyeloablative Allogeneic Stem Cell Transplant | Experimental | Radiation: Total Body Irradiation, Drug: Fludarabine, Drug: Campath 1HTotal Body Irradiation Fludarabine Campath 1H |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Myelodysplastic syndrome with IPSS score > 0.(Appendix B) Or
- Myeloproliferative disorders
- Acute myelogenous leukemia or
- Chronic myelogenous leukemia
- Available Healthy Donor without any contraindications for donation. 5/6 or 6/6 related donor or 5/6 or 6/6 unrelated donor (molecular typing for DRB1)
Key exclusion criteria (10 listed)
- Patient is pregnant or lactating or unwilling to use contraceptives.
- HIV positive patient
- Uncontrolled intercurrent infection
- Unstable angina and uncompensated congestive heart failure (Zubrod of 3 or greater)
- Severe chronic pulmonary disease requiring oxygen (Zubrod of 3 or greater)
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 |
|---|---|---|
| Total Body IrradiationAlso: TBITotal body irradiation of 450cGy as a single dose, day -6 | Radiation | Submyeloablative Allogeneic Stem Cell Transplant |
| FludarabineAlso: FludaraFludarabine 30mg/m2 Day -5 to -2 | Drug | Submyeloablative Allogeneic Stem Cell Transplant |
| Campath 1HAlso: AlemtuzumabCampath 1H dosing as per institutional SOPs Day -5 to -2 | Drug | Submyeloablative Allogeneic Stem Cell Transplant |
Primary outcome
Day 100 Non-relapse mortality,
Time frame: 100 days
Safety and feasibility of submyeloablative conditioning as a preparative regimen for blood stem cell transplantation
Day 100 graft rejection
Time frame: 100 days
Safety and feasibility of submyeloablative conditioning as a preparative regimen for blood stem cell transplantation
2 secondary outcomes
1 year disease free survival
Time frame: 1 year
Complete Remission at 100 days
Time frame: 100 days
Results and safety
The study was stopped early (reason given: Closed due to competing protocols). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 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.
George Carrum, MD principal investigator
Baylor College of Medicine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Total Body Irradiation, Fludarabine and Campath 1H in people with Chronic Myeloproliferative Disorders and Leukemia, run by Baylor College of Medicine at 2 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 8 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: stopped early, as of the sponsor's last update on 10 Oct 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 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: Closed due to competing protocols). 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.
What does the primary outcome measure?
Day 100 Non-relapse mortality, (time frame: 100 days). The study lists 2 primary outcomes in all. 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 10 Oct 2012.