Multiple MyelomaPhase 2NCT00133367
Study of Unrelated Cord Blood Transplantation Using Tacrolimus and Sirolimus
A Phase 2 study testing Tacrolimus, Sirolimus and G-CSF in people with Multiple Myeloma and Non-Hodgkin's Lymphoma, run by Massachusetts General Hospital at 2 sites in United States.
Trial summary
The purpose of this study is to measure the effectiveness of 2 drugs, tacrolimus and sirolimus, in preventing graft versus host disease (GVHD) after treatment with chemotherapy followed by donor cord blood transplantation. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Multiple Myeloma, Non-Hodgkin's Lymphoma, Hodgkin's Disease
- What is being tested?
- Tacrolimus, Sirolimus and G-CSF
- Type of treatment
- Injection or infusion
- How many people
- 32 participants
- Where
- 2 sitesUnited States
- Study dates
- Aug 2005to Nov 2011
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate
- Non-Hodgkin's lymphoma, or Hodgkin's lymphoma: in Complete Remission >2 (second complete remission, third complete remission, etc) or in partial remission
- Multiple myeloma: relapsed
- Chronic lymphocytic leukemia, Rai stage III or IV, or lymphocyte doubling time of 6 months, or stage I-II, having progressed after > 2 chemotherapy regimens, in partial remission.
- Acute myelogenous or lymphoblastic leukemia in second or subsequent remission or in first remission with adverse cytogenetic or antecedent hematologic disorder
- Chronic myelogenous leukemia in accelerated or second stable phase, or imatinib resistant and not eligible for an ablative transplant
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- Myelodysplasia, previously treated or not eligible for ablative transplant
- Age 18-65 years.
- ECOG performance status of 0, 1, or 2.
- Lack of 6/6 or 5/6 HLA-matched related, 10/10 matched unrelated donor, or unrelated donor not available within the time frame necessary to perform a potentially curative stem cell transplant.
Who cannot join
- Cardiac disease:
- symptomatic congestive heart failure or
- radionuclide ventriculogram (RVG) or echocardiogram determined left ventricular ejection fraction of < 40%,
- active angina pectoris, or
- uncontrolled hypertension.
- Pulmonary disease:
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- severe chronic obstructive lung disease, or
- symptomatic restrictive lung disease, or
- corrected DLCO of < 50% of predicted.
- Renal disease:
- serum creatinine > 2.0 mg/dl.
- Hepatic disease:
- serum bilirubin > 2.0 mg/dl (except in the case of Gilbert's syndrome or hemolytic anemia in which the bilirubin can be elevated greater than 2.0mg/dl),
- SGOT or SGPT > 3 x normal.
- Neurologic disease:
- symptomatic leukoencephalopathy,
- active central nervous system (CNS) malignancy or other neuropsychiatric abnormalities believed to preclude transplantation (previous CNS malignancy, presently in complete remission [CR] is not exclusion).
- HIV antibody.
- Uncontrolled infection.
- Pregnancy or breast feeding mother.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
About this study
Official title: Phase II Study of Sequential Unrelated Cord Blood Transplantation Using Tacrolimus and Sirolimus as Graft Versus Host Disease Prophylaxis
The purpose of this study is to measure the effectiveness of 2 drugs, tacrolimus and sirolimus, in preventing graft versus host disease (GVHD) after treatment with chemotherapy followed by donor cord blood transplantation.
Read the detailed description
- The chemotherapy portion of the study involves the intravenous administration of fludarabine, for six days (Days 8, 7, 6, 5,4, and 3) before transplant, melphalan, for one day (Day 2) before transplant. Antithymocyte globulin, or thymoglobulin, will be given IV daily for 4 days (days 7, 5, 3, and 1 before transplant). This drug also helps to suppress the immune system, allowing the cord blood cells to grow and reproduce.
- Immunosuppression therapy consists of the drugs tacrolimus and sirolimus. The patient will receive these 3 days before the transplant and every day for 3-6 months after transplant. After the first 100 days post transplant, the doses of tacrolimus and sirolimus will begin to be reduced with the goal of having the patient off both drugs by 6-9 months after transplant.
- After completion of conditioning therapy described above, the patient will receive 2 cord blood units 1-6 hours apart. To help with engraftment, the patient will also receive G-CSF starting on day five after transplant, until the patients white blood cells recover.
- Follow-up visits will continue every 6 months after the last treatment dose and will last up to 2 years.
- Blood tests will be drawn frequently to test whether the donor's immune cells have engrafted as well as to test the levels of Tacrolimus and Sirolimus.
- Conditions
- Multiple Myeloma, Non-Hodgkin's Lymphoma, Hodgkin's Disease, Myelogenous Leukemia, Lymphoblastic Leukemia
- Being tested
- Tacrolimus, Sirolimus, G-CSF, Antithymocyte globulin, Thymoglobulin
- Keywords
- Hematologic malignancy, Cord blood transfusion, Graft versus host disease, tacrolimus, sirolimus
- Medical subject headings
- Multiple Myeloma, Lymphoma, Non-Hodgkin, Hodgkin Disease, Leukemia, Myeloid, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Hematologic Neoplasms, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TacrolimusDrug
Given three days before transplant and every day for 3-6 months after transplant. After first 100 days post-transplant, the dose will be reduced.
SirolimusDrug
Given three days before transplant and every day for 3-6 months after transplant. After first 100 days post-transplant, the dose will be reduced.
G-CSFDrug
Given starting on day 5 after transplant until the subjects white blood cell count recovers.
Antithymocyte globulinDrug
Given intravenously for 4 days before transplant (days 7, 5, 3, 1).
ThymoglobulinDrug
Given intravenously for 4 days before transplant (days 7, 5, 3, 1).
FludarabineDrug
Given intravenously for six days prior to transplant (days 8,7,6,5,4,3).
MelphalanDrug
Given intravenously on day 2 before transplant.
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the effectiveness of tacrolimus and sirolimus in preventing graft versus host disease
Measured: 2 years
Secondary outcomes (2)
To evaluate the days to neutrophil engraftment and platelet engraftment
Measured: 2 years
To evaluate the relapse rate and overall disease free survival
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2005 (estimated)
- Study startAug 2005
- Primary completionNov 2007
- Study completionNov 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe 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
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Dana-Farber Cancer Institute
- Responsible party
- Principal investigator, Karen Ballen, Massachusetts General Hospital
- Investigators
- Karen K Ballen, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00133367
- Sponsor's protocol ID
- 05-154
- Status last verified
- Jul 2016
- First submitted
- 19 Aug 2005
- Last updated
- 25 Jul 2016
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Brown JA, Stevenson K, Kim HT, Cutler C, Ballen K, McDonough S, Reynolds C, Herrera M, Liney D, Ho V, Kao G, Armand P, Koreth J, Alyea E, McAfee S, Attar E, Dey B, Spitzer T, Soiffer R, Ritz J, Antin JH, Boussiotis VA. Clearance of CMV viremia and survival after double umbilical cord blood transplantation in adults depends on reconstitution of thymopoiesis. Blood. 2010 May 20;115(20):4111-9. doi: 10.1182/blood-2009-09-244145. Epub 2010 Jan 27.
PubMed 20107229
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Sequential Unrelated Cord Blood Transplantation Using Tacrolimus and Sirolimus as Graft Versus Host Disease Prophylaxis
- ClinicalTrials.gov ID
- NCT00133367
- Acronym / sponsor ID
- 05-154
- Conditions
- Multiple Myeloma, Non-Hodgkin's Lymphoma, Hodgkin's Disease, Myelogenous Leukemia, Lymphoblastic Leukemia
- Keywords
- Hematologic malignancy, Cord blood transfusion, Graft versus host disease, tacrolimus, sirolimus
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Dana-Farber Cancer Institute
- Study start
- Aug 2005
- Primary completion
- Nov 2007 (actual)
- Study completion
- Nov 2011 (actual)
- First posted
- 23 Aug 2005 (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.
The purpose of this study is to measure the effectiveness of 2 drugs, tacrolimus and sirolimus, in preventing graft versus host disease (GVHD) after treatment with chemotherapy followed by donor cord blood transplantation.
Detailed description
- The chemotherapy portion of the study involves the intravenous administration of fludarabine, for six days (Days 8, 7, 6, 5,4, and 3) before transplant, melphalan, for one day (Day 2) before transplant. Antithymocyte globulin, or thymoglobulin, will be given IV daily for 4 days (days 7, 5, 3, and 1 before transplant). This drug also helps to suppress the immune system, allowing the cord blood cells to grow and reproduce.
- Immunosuppression therapy consists of the drugs tacrolimus and sirolimus. The patient will receive these 3 days before the transplant and every day for 3-6 months after transplant. After the first 100 days post transplant, the doses of tacrolimus and sirolimus will begin to be reduced with the goal of having the patient off both drugs by 6-9 months after transplant.
- After completion of conditioning therapy described above, the patient will receive 2 cord blood units 1-6 hours apart. To help with engraftment, the patient will also receive G-CSF starting on day five after transplant, until the patients white blood cells recover.
- Follow-up visits will continue every 6 months after the last treatment dose and will last up to 2 years.
- Blood tests will be drawn frequently to test whether the donor's immune cells have engrafted as well as to test the levels of Tacrolimus and Sirolimus.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate
- Non-Hodgkin's lymphoma, or Hodgkin's lymphoma: in Complete Remission >2 (second complete remission, third complete remission, etc) or in partial remission
- Multiple myeloma: relapsed
- Chronic lymphocytic leukemia, Rai stage III or IV, or lymphocyte doubling time of 6 months, or stage I-II, having progressed after > 2 chemotherapy regimens, in partial remission.
- Acute myelogenous or lymphoblastic leukemia in second or subsequent remission or in first remission with adverse cytogenetic or antecedent hematologic disorder
Key exclusion criteria (20 listed)
- Cardiac disease:
- Pulmonary disease:
- Renal disease:
- Hepatic disease:
- Neurologic disease:
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 |
|---|---|---|
| TacrolimusGiven three days before transplant and every day for 3-6 months after transplant. After first 100 days post-transplant, the dose will be reduced. | Drug | – |
| SirolimusGiven three days before transplant and every day for 3-6 months after transplant. After first 100 days post-transplant, the dose will be reduced. | Drug | – |
| G-CSFGiven starting on day 5 after transplant until the subjects white blood cell count recovers. | Drug | – |
| Antithymocyte globulinGiven intravenously for 4 days before transplant (days 7, 5, 3, 1). | Drug | – |
| ThymoglobulinGiven intravenously for 4 days before transplant (days 7, 5, 3, 1). | Drug | – |
| FludarabineGiven intravenously for six days prior to transplant (days 8,7,6,5,4,3). | Drug | – |
| MelphalanGiven intravenously on day 2 before transplant. | Drug | – |
Primary outcome
To determine the effectiveness of tacrolimus and sirolimus in preventing graft versus host disease
Time frame: 2 years
2 secondary outcomes
To evaluate the days to neutrophil engraftment and platelet engraftment
Time frame: 2 years
To evaluate the relapse rate and overall disease free survival
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2016.
- Derived from this study: PubMed 20107229Brown JA, Stevenson K, Kim HT, Cutler C, Ballen K, McDonough S, Reynolds C, Herrera M, Liney D, Ho V, Kao G, Armand P, Koreth J, Alyea E, McAfee S, Attar E, Dey B, Spitzer T, Soiffer R, Ritz J, Antin JH, Boussiotis VA. Clearance of CMV viremia and survival after double umbilical cord blood transplantation in adults depends on reconstitution of thymopoiesis. Blood. 2010 May 20;115(20):4111-9. doi: 10.1182/blood-2009-09-244145. Epub 2010 Jan 27.
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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.
Karen K Ballen, MD principal investigator
Massachusetts General Hospital
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 Tacrolimus, Sirolimus and G-CSF in people with Multiple Myeloma and Non-Hodgkin's Lymphoma, run by Massachusetts General Hospital 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 10 inclusion and 20 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 25 Jul 2016. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To determine the effectiveness of tacrolimus and sirolimus in preventing graft versus host disease (time frame: 2 years). 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 NCT00133367, which also lists 1 linked publication.
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 25 Jul 2016.