LymphomaPhase 2NCT01135329
Reduced-intensity, Related-donor Bone Marrow Transplantation Followed by High-dose Cyclophosphamide for Hematologic Cancers
A Phase 2 study testing Fludarabine, Busulfan and Cyclophosphamide in people with Lymphoma and Leukemia, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.
Trial summary
This research is being done to learn more about reduced-intensity bone marrow transplantation (BMT), also known as a "mini" transplant for patients with blood cancers, using bone marrow from a relative. The main goal of the study is to determine how quickly the donor's bone marrow "takes" in your body. Read more about this study
- Who is it for?
- Ages 6 months to 75 years
- Conditions the study is for
- Lymphoma, Leukemia, Myelodysplastic Syndrome
- What is being tested?
- Fludarabine, Busulfan and Cyclophosphamide
- Type of treatment
- 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
- Aug 2010to May 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 6 months to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- First-degree related donor who is at minimum HLA haploidentical
- Eligible diagnoses:
- Low-grade non-Hodgkin's lymphoma or plasma cell neoplasm that has progressed during multiagent therapy, failed at least two prior therapies (excluding single agent rituximab and single agent steroids), or in the case of lymphoma undergone histological conversion:
- Follicular grade 1 or 2 lymphoma
- Follicular lymphoma not otherwise specified
- Marginal zone (or MALT) lymphoma
Show all 53Show fewer
- Lymphoplasmacytic lymphoma / Waldenstrom's macroglobulinemia
- Hairy cell leukemia
- Small lymphocytic lymphoma (SLL) or chronic lymphocytic leukemia (CLL)
- Prolymphocytic leukemia
- Low grade B-cell lymphoma, unspecified
- Multiple myeloma
- Plasma cell leukemia
- Poor-risk SLL or CLL, defined by an 11q or 17p deletion, histological conversion, or disease progression < 6 months after a purine analog-containing regimen
- Aggressive lymphoma that has failed at least one prior regimen of multiagent chemotherapy, and patient is either ineligible for autologous BMT or autologous BMT is not recommended:
- Hodgkin lymphoma
- Follicular grade 3 lymphoma
- Mantle cell lymphoma or leukemia
- Diffuse large B-cell lymphoma (excluding primary CNS lymphoma). Eligible subtypes include primary mediastinal large B-cell lymphoma, T-cell rich large B-cell lymphoma, and large B-cell lymphoma not otherwise specified.
- Burkitt's lymphoma/leukemia
- Atypical Burkitt's lymphoma/leukemia (high grade B-cell lymphoma, unclassified, including that with features intermediate between Burkitt's and diffuse large B-cell lymphoma)
- Anaplastic large cell lymphoma
- Plasmablastic lymphoma
- Peripheral T-cell lymphoma
- Relapsed or refractory acute leukemia in second or subsequent remission
- Poor-risk acute leukemia in first remission
- AML with at least one of the following:
- AML arising from MDS or a myeloproliferative disorder, or secondary AML
- Presence of Flt3 internal tandem duplications
- Poor-risk cytogenetics
- Primary refractory disease
- ALL (leukemia and/or lymphoma) with at least one of the following:
- Adverse cytogenetics
- Clear evidence of hypodiploidy
- Primary refractory disease
- Biphenotypic leukemia
- MDS with at least one of the following features:
- Poor-risk cytogenetics
- IPSS score of INT-2 or greater
- Treatment-related MDS
- MDS diagnosed before age 21 years
- Progression on or lack of response to standard DNA-methyltransferase inhibitor therapy
- Life-threatening cytopenias, including those generally requiring greater than weekly transfusions
- Interferon- or imatinib-refractory CML in first chronic phase, or non-blast crisis CML beyond first chronic phase
- Philadelphia chromosome negative myeloproliferative disease (including myelofibrosis)
- Chronic myelomonocytic leukemia
- Juvenile myelomonocytic leukemia
- For patients with SLL, CLL, or prolymphocytic leukemia, < 20% of bone marrow cellularity involved by this process
- Adequate end-organ function:
- Left ventricular ejection fraction greater than or equal to 35%
- Bilirubin ≤ 3.0 mg/dL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST < 5 x ULN
- FEV1 and FVC > 40% of predicted; or in pediatric patients, if unable to perform pulmonary function tests due to young age, oxygen saturation >92% on room air
- ECOG performance status < 2 or Karnofsky or Lansky score > 60
Who cannot join
- Pregnant or breast-feeding
- Uncontrolled infection Note: Infection is permitted if there is evidence of response to medication. Eligibility of HIV infected patients will be determined on a case-by-case basis.
- Any previous BMT within 3 months prior to start of conditioning
- Active extra-medullary leukemia or known active Central Nervous System (CNS) involvement by malignancy. Such disease treated into remission is permitted.
Trial locations and contacts
1 site in 1 country.
United States1 site
The Sydney Kimmel Comprehensive Cancer Center
Baltimore, Maryland, 21231
About this study
Official title: Reduced-intensity, Related-donor Allogeneic BMT With Fludarabine, Busulfan, and High-dose Posttransplantation Cyclophosphamide for Hematologic Malignancies
This research is being done to learn more about reduced-intensity bone marrow transplantation (BMT), also known as a "mini" transplant for patients with blood cancers, using bone marrow from a relative.
The main goal of the study is to determine how quickly the donor's bone marrow "takes" in your body. Other goals include describing how many people accept the bone marrow and how quickly the blood counts come up; describing Graft-versus-host disease (GVHD) and other complications; and describing how many people survive without progressive cancer and survive overall
Read the detailed description
At the present time there are few or no cures for people with cancer of the blood or lymph glands outside of a bone marrow transplant (BMT). BMT has developed over several decades of research as an effective treatment of various malignant and nonmalignant hematologic diseases.
This research is being done to learn more about reduced-intensity bone marrow transplantation (BMT), also known as a "mini" transplant for patients with blood cancers, using bone marrow from a relative. The bone marrow for this transplant comes from a relative who is a half-match or "haplo" match to you. Possible donors include parents, siblings, and children.
"Mini" transplants have been given to many people with various cancers but are considered experimental. Over 200 people at Johns Hopkins have received mini transplants with high doses of cyclophosphamide after the transplant. However, the chemotherapy combination and other treatment given before those transplants were different from what is in this study. Although all of the chemotherapy and immune-lowering drugs used in this study are approved by the Food and Drug Administration (FDA), the combination of medications used in this study are not FDA approved and are experimental.
- Conditions
- Lymphoma, Leukemia, Myelodysplastic Syndrome
- Being tested
- Fludarabine, Busulfan, Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus
- Keywords
- Lymphoma, Hodgkin's lymphoma, Non hodgkin's lymphoma, Leukemia, Acute myeloid leukemia (AML), Acute lymphoblastic leukemia(ALL), Chronic myeloid leukemia (CML), Chronic Myelomonocytic (CMML), Myelodysplastic syndrome (MDS), High-risk acute leukemia in first remission, Relapsed leukemia in remission, Cyclophosphamide, High-dose cyclophosphamide, Fludarabine, Busulfan, Allogeneic, Nonmyeloablative, Reduced intensity, Haploidentical, Bone marrow transplant (BMT)
- Medical subject headings
- Lymphoma, Leukemia, Myelodysplastic Syndromes, Hodgkin Disease, Lymphoma, Non-Hodgkin, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
Also called Fludara, Flu
30 mg/m^2 IV daily on Day -6 through Day -2.
BusulfanDrug
Also called Busulfex, Myleran
1 mg/kg PO OR 0.8 mg/kg IV four times daily on Day -6 through Day -3.
CyclophosphamideDrug
Also called CTX, Cytoxan, Cy
50 mg/kg IV daily on Day +3 and Day +4.
Also called MMF, CellCept
15 mg/kg PO three times daily (max daily dose of 3g) starting on Day +5.
TacrolimusDrug
Also called FK506, FK-506, Prograf
Dosed based on drug levels; begin on Day +5 at 1 mg IV daily.
| Study group | Role | What they receive |
|---|---|---|
| BMT Allogenic Transplantation | Experimental | Reduced-intensity transplant with a fludarabine- and busulfan-based preparative regimen. GVHD prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil. Receives: Drug: Fludarabine; Drug: Busulfan; Drug: Cyclophosphamide; Drug: Mycophenolate Mofetil; Drug: Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Chimerism in Unsorted Peripheral Blood
Percentage of participants achieving full-donor chimerism in unsorted peripheral blood.
Measured: Day 60
Chimerism in CD3+ Sorted Peripheral Blood
Percentage of participants achieving full-donor chimerism in CD3+ sorted peripheral blood
Measured: Day 60
Secondary outcomes (6)
Overall Survival
Percentage of participants alive
Measured: 1 year
Progression-free Survival
Percentage of participants alive without disease relapse or progression.
Measured: 1 year
Incidence of Relapse
Percentage of participants experiencing disease relapse or progression
Measured: 1 year
Non-relapse Mortality
Percentage of participants who died due to BMT-related reasons
Measured: 1 year
Incidence of Graft-versus-host-disease (GVHD)
Percentage of participants experiencing acute and chronic GVHD. Acute GVHD is graded by Przepiorka criteria. Chronic GVHD is graded by NIH consensus criteria and Seattle criteria.
Measured: 1 year
Participants Who Failed to Engraft
Number of participants who failed to engraft
Measured: Day 60
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2010 (estimated)
- Study startAug 2010
- Primary completionMay 2012
- Study completionMay 2012
Why it stopped: The stopping rule was met and hence the study was closed
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 LymphomaStudies of the same condition, open and under way.
- More trials run by Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsThe 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
- 15 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Yvette Kasamon, M.D., Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01135329
- Sponsor's protocol ID
- J1046
- Other IDs
- NA_00039363
- Status last verified
- May 2026
- First submitted
- 1 Jun 2010
- Last updated
- 27 May 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reduced-intensity, Related-donor Allogeneic BMT With Fludarabine, Busulfan, and High-dose Posttransplantation Cyclophosphamide for Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT01135329
- Acronym / sponsor ID
- J1046
- Conditions
- Lymphoma, Leukemia, Myelodysplastic Syndrome
- Keywords
- Lymphoma, Hodgkin's lymphoma, Non hodgkin's lymphoma, Leukemia, Acute myeloid leukemia (AML), Acute lymphoblastic leukemia(ALL), Chronic myeloid leukemia (CML), Chronic Myelomonocytic (CMML)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The stopping rule was met and hence the study was closed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Study start
- Aug 2010
- Primary completion
- May 2012 (actual)
- Study completion
- May 2012 (actual)
- First posted
- 2 Jun 2010 (estimated)
- Results
- Posted 3 Jul 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research is being done to learn more about reduced-intensity bone marrow transplantation (BMT), also known as a "mini" transplant for patients with blood cancers, using bone marrow from a relative.
The main goal of the study is to determine how quickly the donor's bone marrow "takes" in your body. Other goals include describing how many people accept the bone marrow and how quickly the blood counts come up; describing Graft-versus-host disease (GVHD) and other complications; and describing how many people survive without progressive cancer and survive overall
Detailed description
At the present time there are few or no cures for people with cancer of the blood or lymph glands outside of a bone marrow transplant (BMT). BMT has developed over several decades of research as an effective treatment of various malignant and nonmalignant hematologic diseases.
This research is being done to learn more about reduced-intensity bone marrow transplantation (BMT), also known as a "mini" transplant for patients with blood cancers, using bone marrow from a relative. The bone marrow for this transplant comes from a relative who is a half-match or "haplo" match to you. Possible donors include parents, siblings, and children.
"Mini" transplants have been given to many people with various cancers but are considered experimental. Over 200 people at Johns Hopkins have received mini transplants with high doses of cyclophosphamide after the transplant. However, the chemotherapy combination and other treatment given before those transplants were different from what is in this study. Although all of the chemotherapy and immune-lowering drugs used in this study are approved by the Food and Drug Administration (FDA), the combination of medications used in this study are not FDA approved and are experimental.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BMT Allogenic Transplantation | Experimental | Drug: Fludarabine, Drug: Busulfan, Drug: Cyclophosphamide, Drug: Mycophenolate Mofetil, Drug: TacrolimusReduced-intensity transplant with a fludarabine- and busulfan-based preparative regimen. GVHD prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil. |
Who may qualify
- Ages
- 6 months to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (53 listed)
- First-degree related donor who is at minimum HLA haploidentical
- Eligible diagnoses:
- Left ventricular ejection fraction greater than or equal to 35%
- Bilirubin ≤ 3.0 mg/dL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST < 5 x ULN
- FEV1 and FVC > 40% of predicted; or in pediatric patients, if unable to perform pulmonary function tests due to young age, oxygen saturation >92% on room air
Key exclusion criteria (4 listed)
- Pregnant or breast-feeding
- Uncontrolled infection Note: Infection is permitted if there is evidence of response to medication. Eligibility of HIV infected patients will be determined on a case-by-case basis.
- Any previous BMT within 3 months prior to start of conditioning
- Active extra-medullary leukemia or known active Central Nervous System (CNS) involvement by malignancy. Such disease treated into remission is permitted.
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 |
|---|---|---|
| FludarabineAlso: Fludara, Flu30 mg/m^2 IV daily on Day -6 through Day -2. | Drug | BMT Allogenic Transplantation |
| BusulfanAlso: Busulfex, Myleran1 mg/kg PO OR 0.8 mg/kg IV four times daily on Day -6 through Day -3. | Drug | BMT Allogenic Transplantation |
| CyclophosphamideAlso: CTX, Cytoxan, Cy50 mg/kg IV daily on Day +3 and Day +4. | Drug | BMT Allogenic Transplantation |
| Mycophenolate MofetilAlso: MMF, CellCept15 mg/kg PO three times daily (max daily dose of 3g) starting on Day +5. | Drug | BMT Allogenic Transplantation |
| TacrolimusAlso: FK506, FK-506, PrografDosed based on drug levels; begin on Day +5 at 1 mg IV daily. | Drug | BMT Allogenic Transplantation |
Primary outcome
Chimerism in Unsorted Peripheral Blood
Time frame: Day 60
Percentage of participants achieving full-donor chimerism in unsorted peripheral blood.
Chimerism in CD3+ Sorted Peripheral Blood
Time frame: Day 60
Percentage of participants achieving full-donor chimerism in CD3+ sorted peripheral blood
6 secondary outcomes
Overall Survival
Time frame: 1 year
Progression-free Survival
Time frame: 1 year
Incidence of Relapse
Time frame: 1 year
Non-relapse Mortality
Time frame: 1 year
Incidence of Graft-versus-host-disease (GVHD)
Time frame: 1 year
Participants Who Failed to Engraft
Time frame: Day 60
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Yvette Kasamon, M.D. principal investigator
Johns Hopkins University
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 Fludarabine, Busulfan and Cyclophosphamide in people with Lymphoma and Leukemia, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 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 53 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: stopped early, as of the sponsor's last update on 27 May 2026. 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 3 Jul 2018). 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?
Chimerism in Unsorted Peripheral Blood (time frame: Day 60). 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 27 May 2026.