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Hematologic MalignanciesPhase 1/2NCT01203722

Reduced Intensity, Partially HLA Mismatched BMT to Treat Hematologic Malignancies

A Phase 1/2 study testing Fludarabine, Cytoxan and Total Body Irradiation in people with Hematologic Malignancies, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

If transplantation using mismatched unrelated donors or non-first-degree relatives could be performed with an acceptable toxicity profile, an important unmet need would be served. Read more about this study

Who is it for?
Ages 6 months to 75 yearsHealthy volunteers can take part
Conditions the study is for
Hematologic Malignancies
What is being tested?
Fludarabine, Cytoxan and Total Body Irradiation
Could I receive a placebo?
No, everyone receives an active treatment
How many people
87 participants
Where
1 siteUnited States
Study dates
Sep 2010to 28 May 2024
Phase
Phase 1/2
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
  • Healthy volunteers accepted
  • Child, Adult, Older adult

Patient Inclusion Criteria:

  1. Patient age 0.5-75 years
  2. Absence of a suitable related or unrelated bone marrow donor who is molecularly matched at HLA-A, B, Cw, DRB1, and DQB1.
  3. Absence of a suitable partially HLA-mismatched (haploidentical), first-degree related donor. Donors who are homozygous for the CCR5delta32 polymorphism are given preference.
  4. Eligible diagnoses:
  5. Relapsed or refractory acute leukemia in second or subsequent remission, with remission defined as <5% bone marrow blasts morphologically
  6. Poor-risk acute leukemia in first remission, with remission defined as <5% bone marrow blasts morphologically:
  • 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: Complex karyotype [> 3 abnormalities], inv(3), t(3;3), t(6;9), MLL rearrangement with the exception of t(9;11), or abnormalities of chromosome 5 or 7
  • Primary refractory disease
  • ALL (leukemia and/or lymphoma) with at least one of the following:
  • Adverse cytogenetics such as t(9;22), t(1;19), t(4;11), or MLL rearrangement
  • Clear evidence of hypodiploidy
  • Primary refractory disease
  • Biphenotypic leukemia
  1. MDS with at least one of the following poor-risk features:
  • Poor-risk cytogenetics (7/7q minus or complex 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
  1. Interferon- or imatinib-refractory CML in first chronic phase, or non-blast crisis CML beyond first chronic phase.
  2. Philadelphia chromosome negative myeloproliferative disease.
  3. Chronic myelomonocytic leukemia.
  4. Juvenile myelomonocytic leukemia.
  5. Low-grade non-Hodgkin lymphoma (including SLL and CLL) or plasma cell neoplasm that has:
  • progressed after at least two prior therapies (excluding single agent rituximab and single agent steroids), or
  • in the case of lymphoma undergone histologic conversion;
  • patients with transformed lymphomas must have stable disease or better.
  1. Poor-risk CLL or SLL as follows:
  • 11q deletion disease that has progressed after a combination chemotherapy regimen,
  • 17p deletion disease,
  • or histologic conversion;
  • patients with transformed lymphomas must have stable disease or better.
  1. Aggressive non-Hodgkin lymphoma as follows, provided there is stable disease or better to last therapy:
  • NK or NK-T cell lymphoma, hepatosplenic T-cell lymphoma, or subcutaneous panniculitic T-cell lymphoma, blastic/ blastoid variant of mantle cell lymphoma
  • Hodgkin or aggressive non Hodgkin lymphoma that has failed at least one multiagent regimen, and the patient is either ineligible for autologous BMT or autologous BMT is not recommended.
  • Eligible subtypes of aggressive non-Hodgkin lymphoma include:
  • mantle cell lymphoma
  • follicular grade 3 lymphoma
  • diffuse large B-cell lymphoma or its subtypes, excluding primary CNS lymphoma
  • primary mediastinal large B-cell lymphoma
  • large B-cell lymphoma, unspecified
  • anaplastic large cell lymphoma, excluding skin-only disease
  • Burkitt's lymphoma or atypical Burkitt's lymphoma (high-grade B-cell lymphoma, unclassifiable, with features intermediate between diffuse large B-cell lymphoma and Burkitt's), in complete remission
  1. Patients with CLL, SLL, or prolymphocytic leukemia must have < 20% bone marrow involvement by malignancy (to lower risk of graft rejection).
  2. One of the following, in order to lower risk of graft rejection:
  • Cytotoxic chemotherapy, an adequate course of 5-azacitidine or decitabine, or alemtuzumab within 3 months prior to start of conditioning; or
  • Previous BMT within 6 months prior to start of conditioning. NOTE: Patients who have received treatment outside of these windows may be eligible if it is deemed sufficient to reduce graft rejection risk; this will be decided on a case-by-case basis by the PI or co-PI.
  1. Any previous BMT must have occurred at least 3 months prior to start of conditioning.
  2. Adequate end-organ function as measured by:
  3. Left ventricular ejection fraction greater than or equal to 35%, or shortening fraction > 25%, unless cleared by a cardiologist
  4. Bilirubin ≤ 3.0 mg/dL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST < 5 x ULN
  5. 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
  6. ECOG performance status < 2 or Karnofsky or Lansky score > 60

Patient Exclusion Criteria:

  • Not pregnant or breast-feeding.
  • No uncontrolled bacterial, viral, or fungal infection.
  • Note: HIV-infected patients are potentially eligible. Eligibility of HIV-infected patients will be determined on a case-by-case basis.
  • No previous allogeneic BMT (syngeneic BMT permissible).
  • Active extramedullary leukemia or known active CNS involvement by malignancy. Such disease treated into remission is permitted.

Donor Inclusion Criteria:

  1. Potential donors consist of:
  • Unrelated donors
  • Second-degree relatives
  • First cousins
  1. The donor and recipient must be identical at at least 5 HLA alleles based on high resolution typing of HLA-A, -B, -Cw, -DRB1, and -DQB1, with at least one allele matched for a HLA class I gene (HLA-A, -B, or -Cw) and at least one allele matched for a class II gene (HLA-DRB1 or -DQB1).
  2. Meets institutional selection criteria and medically fit to donate. 4 . Lack of recipient anti-donor HLA antibody. Note: In some instances, low level, non-cytotoxic HLA specific antibodies may be permissible if they are found to be at a level well below that detectable by flow cytometry. This will be decided on a case-by-case basis by the PI and one of the immunogenetics directors. Pheresis to reduce anti-HLA antibodies is permissible; however eligibility to proceed with the transplant regimen would be contingent upon the result.

Donor Exclusion Criteria:

  • Donor must not be HLA identical to the recipient.
  • Has not donated blood products to recipient.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    Baltimore, Maryland, 21231-2410

Other trials in United States

About this study

Official title: Reduced Intensity, Partially HLA Mismatched Allogeneic BMT for Hematologic Malignancies Using Donors Other Than First-degree Relatives

If transplantation using mismatched unrelated donors or non-first-degree relatives could be performed with an acceptable toxicity profile, an important unmet need would be served. Towards this goal, the current study extends our platform of nonmyeloablative, partially HLA-mismatched bone marrow transplant (BMT) and Peripheral Blood Stem Cell Transplant (PBSCT) to the use of such donors, investigating up to several postgrafting immunosuppression regimens that incorporate high-dose Cy. Of central interest is the incorporation of sirolimus into this postgrafting immunosuppression regimen.

The primary goal for phase 1 is to identify a transplant regimen associated with acceptable rates of severe acute GVHD and NRM by Day 100 and for phase 2 estimate the 6-month probability of survival without having had acute grade III- IV GVHD or graft failure.

Being tested
Fludarabine, Cytoxan, Total Body Irradiation, Allogeneic Blood or Marrow Transplant, Peripheral Blood Stem Cell Transplant
Keywords
Reduced intensity, partially HLA mismatched allogeneic BMT, Acute leukemias, Chronic leukemias, Myelodysplasia, Lymphomas, Unrelated or non-first-degree related donors, Peripheral blood stem cell transplant
Medical subject headings
Hematologic Neoplasms, Anemia, Refractory, with Excess of Blasts, Lymphoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FludarabineDrug

    Fludarabine 30 mg/m2/day

  • CytoxanDrug

    Also called High-dose Cytoxan

    Pre-BMT: Cytoxan 14.5 mg/kg/day administered IV; Post-Transplantation: High-dose Cytoxan 50mg/kg/day

  • Total Body IrradiationRadiation

    Also called TBI

    400 cGy TBI administered in a single fraction

  • Allogeneic Blood or Marrow TransplantProcedure or surgery

    Also called BMT

  • Peripheral Blood Stem Cell TransplantProcedure or surgery

    Also called PBSCT

  • Mycophenolate MofetilDrug

    Also called MMF

    15mg/kg by mouth three times daily

  • SirolimusDrug

    Loading Dose: Sirolimus 6mg by mouth once; Maintenance dose: Sirolimus 2mg by mouth daily

  • TacrolimusDrug

    Tacrolimus 1mg intravenously, daily

Study groups in NCT01203722
Study groupRoleWhat they receive
REGIMEN BActive comparatorPre-BMT : * Day -6 through -2: Fludarabine 30 mg/m2/day (adjusted for renal function; maximum cumulative dose, 150 mg/m2) administered IV * Day -6 and -5: Cytoxan 14.5 mg/kg/day administered IV * Day -1: 400 cGy total body irradiation (TBI) administered in a single fraction Day 0: Allogeneic blood or marrow transplantation (BMT) Post-Transplantation Immunosuppression Consisting of: * Day 3 and 4: High-dose Cytoxan 50mg/kg/day (adjusted according to IBW) administered IV * Day 5: Sirolimus loading dose 6 mg PO once * Day 5 thru Day 35: Mycophenolate Mofetil (MMF) 15 mg/kg PO TID (maximum daily dose 3 g/day) * Day 6 thru Day 180: Sirolimus maintenance dose 2 mg PO QD with dose adjustments to maintain trough of 3 - 12 ng/mL
Receives: Drug: Fludarabine; Drug: Cytoxan; Radiation: Total Body Irradiation; Procedure: Allogeneic Blood or Marrow Transplant; Drug: Mycophenolate Mofetil; Drug: Sirolimus
REGIMEN CActive comparatorPre-BMT: * Day -6 through -2: Fludarabine 30 mg/m2/day (adjusted for renal function; maximum cumulative dose, 150 mg/m2) administered IV * Day -6 and -5: Cytoxan 14.5 mg/kg/day administered IV * Day -1: 400 cGy TBI administered in a single fraction Day 0: BMT Post-Transplantation Immunosuppression Consisting of: * Day 3 and 4: High-dose Cytoxan 50mg/kg/day (adjusted according to IBW) administered IV * Day 5 thru Day 35: MMF 15 mg/kg PO TID (maximum daily dose 3 g/day) * Day 5 thru Day 180: Tacrolimus 1 mg administered IV QD
Receives: Drug: Fludarabine; Drug: Cytoxan; Radiation: Total Body Irradiation; Procedure: Allogeneic Blood or Marrow Transplant; Drug: Mycophenolate Mofetil; Drug: Tacrolimus
REGIMEN B2Active comparatorPre-PBSCT: * Day -6 through -2: Fludarabine 30 mg/m2/day (adjusted for renal function; maximum cumulative dose, 150 mg/m2) administered IV * Day -6 and -5: Cytoxan 14.5 mg/kg/day administered IV * Day -1: 400 cGy TBI administered in a single fraction Day 0: Peripheral Blood Stem Cell Transplant (PBSCT) Post-Transplantation Immunosuppression Consisting of: * Day 3 and 4: High-dose Cytoxan 50mg/kg/day (adjusted according to IBW) administered IV * Day 5: Sirolimus loading dose 6 mg PO once * Day 5 thru Day 35: MMF 15 mg/kg PO TID (maximum daily dose 3 g/day) * Day 6 thru Day 180: Sirolimus maintenance dose 2 mg PO QD with dose adjustments to maintain trough of 3 - 12 ng/mL
Receives: Drug: Fludarabine; Drug: Cytoxan; Radiation: Total Body Irradiation; Procedure: Peripheral Blood Stem Cell Transplant; Drug: Mycophenolate Mofetil; Drug: Sirolimus
REGIMEN B3: HIV patients with CCRd32 homozygous donorsActive comparatorPre-PBSCT: * Day -6 through -2: Fludarabine 30 mg/m2/day (adjusted for renal function; maximum cumulative dose, 150 mg/m2) administered IV * Day -6 and -5: Cytoxan 14.5 mg/kg/day administered IV * Day -1: 400 cGy TBI administered in a single fraction Day 0: Peripheral Blood Stem Cell Transplant (PBSCT) Post-Transplantation Immunosuppression Consisting of: * Day 3 and 4: High-dose Cytoxan 50mg/kg/day (adjusted according to IBW) administered IV * Day 5: Sirolimus loading dose 6 mg PO once * Day 5 thru Day 35: MMF 15 mg/kg PO TID (maximum daily dose 3 g/day) * Day 6 thru Day 180: Sirolimus maintenance dose 2 mg PO QD with dose adjustments to maintain trough of 3 - 12 ng/mL
Receives: Drug: Fludarabine; Drug: Cytoxan; Radiation: Total Body Irradiation; Procedure: Peripheral Blood Stem Cell Transplant; Drug: Mycophenolate Mofetil; Drug: Sirolimus

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants Who Have Severe Acute Graft-versus-host-disease (GVHD)

    Two immunosuppressive regimens with Fludarabine-Cytoxan-TBI conditioning will be studied in reduced-intensity, partially HLA mismatched allogeneic BMT from unrelated or non-first-degree related donors. Transplant regimen will be determined by acceptable rates of severe acute GVHD (< 25%).

    Measured: Study Day 100

  2. Number of Participants Who Have Transplant-related Nonrelapse Mortality (NRM)

    Two immunosuppressive regimens with Fludarabine-Cytoxan-TBI conditioning will be studied in reduced-intensity, partially HLA mismatched allogeneic BMT from unrelated or non-first-degree related donors. Transplant regimen will be determined by acceptable number of participants with transplant-related NRM.

    Measured: Study Day 100

  3. 6-month Probability of Survival as Assessed by Absence of Grade III-IV GVHD or Evidence of Graft Failure.

    Number of participants who do not have grade II-IV GVHD or evidence of graft failure will be assessed.

    Measured: 6 months

Secondary outcomes (13)
  1. Progression-free Survival

    All patients will be tracked from Day 0 to date of first objective disease progression, death from any cause, or last patient evaluation. Patients who have not progressed or died will be censored at the last date they were assessed and deemed free of relapse or progression.

    Measured: 2 years

  2. Event-free Survival

    All patients will be tracked from Day 0 to date of first observed disease progression, or death from any cause, or last patient evaluation. Patients will be followed on study to identify instances of death, progression or disease recurrence.

    Measured: 7 years

  3. Overall Survival

    All patients will be tracked from Day 0 to date of first objective disease progression, death from any cause, or last patient evaluation. Patients who have not progressed or died will be censored at the last date they were assessed and deemed free of relapse or progression.

    Measured: 7 years

  4. Cumulative Incidence of Progression or Relapse

    All patients will be tracked from Day 0 to date of first objective disease progression, death from any cause, or last patient evaluation. Patients who have not progressed or died will be censored at the last date they were assessed and deemed free of relapse or progression.

    Measured: 7 years

  5. Cumulative Incidence of Non-relapse Mortality (NRM).

    All patients will be tracked from Day 0 to date of first observed disease relapse or progression, or death from disease, or last patient evaluation. Patients who have not progressed or died by their last patient contact visit will be censored at the last date they were assessed and deemed free of relapse or progression.

    Measured: 7 years

  6. Cumulative Incidence of Acute Grade II-IV GVHD.

    All suspected cases of acute GVHD must be confirmed histologically by biopsy of an affected organ (skin, liver, or gastrointestinal tract). Date of symptom onset, date of biopsy confirmation of GVHD, maximum clinical grade, and dates and types of treatment will be recorded. Dates of symptom onset of grade II or higher GVHD and grade III-IV GVHD will be recorded.

    Measured: 1 year

  7. Cumulative Incidence of Acute Grade III-IV GVHD

    All suspected cases of acute GVHD must be confirmed histologically by biopsy of an affected organ (skin, liver, or gastrointestinal tract). Date of symptom onset, date of biopsy confirmation of GVHD, maximum clinical grade, and dates and types of treatment will be recorded. Dates of symptom onset of grade II or higher GVHD and grade III-IV GVHD will be recorded.

    Measured: 1 year

  8. Cumulative Incidence of Chronic GVHD

    All suspected cases of acute GVHD must be confirmed histologically by biopsy of an affected organ (skin, liver, or gastrointestinal tract). Date of symptom onset, date of biopsy confirmation of GVHD, maximum clinical grade, and dates and types of treatment will be recorded. Dates of symptom onset of grade II or higher GVHD and grade III-IV GVHD will be recorded.

    Measured: 1 year

  9. Cumulative Incidence of Graft Failure

    Number of cases with less than 5% donor chimerism in blood and/or bone marrow on ~Day 30 or after and on all subsequent measurements, in the absence of documented bone marrow involvement by malignancy.

    Measured: 1 year

  10. Cumulative Incidence of Neutrophil Recovery

    Number of cases who achieve a post nadir ANC greater or equal to 500/mm3 for three consecutive measurements on different days.

    Measured: 1 year

  11. Cumulative Incidence of Platelet Recovery

    Number of cases who achieve a platelet count greater than 20,000/mm3 or greater than 50,000/mm3 with no platelet transfusions in the preceding seven days, as measured as maintained on at least three consecutive measurements on different days.

    Measured: 1 year

  12. Cumulative Incidence of Donor Engraftment

    Number of cases who achieve either mixed donor chimerism is defined as greater than 5%, but less than 95%, donor or full donor chimerism is defined as > 95% donor.

    Measured: 1 year

  13. Cumulative Incidence of Failure Free Survival

    Number of cases who reach the end of the trial without severe acute (grade III-IV) GVHD, graft failure, or non-relapse mortality

    Measured: 7 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Sep 2010 (estimated)
  2. Study startSep 2010
  3. Primary completion28 May 2024
  4. Study completion28 May 2024

Resources

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 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
87 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01203722
Sponsor's protocol ID
J1055
Other IDs
NA_00039823, P01CA015396
Status last verified
Feb 2026
First submitted
15 Sep 2010
Last updated
19 Feb 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Rappazzo KC, Zahurak M, Bettinotti M, Ali SA, Ambinder AJ, Bolanos-Meade J, Borrello I, Dezern AE, Gladstone D, Gocke C, Fuchs E, Huff CA, Imus PH, Jain T, Luznik L, Rahmat L, Swinnen LJ, Wagner-Johnston N, Jones RJ, Ambinder RF. Nonmyeloablative, HLA-Mismatched Unrelated Peripheral Blood Transplantation with High-Dose Post-Transplantation Cyclophosphamide. Transplant Cell Ther. 2021 Nov;27(11):909.e1-909.e6. doi: 10.1016/j.jtct.2021.08.013. Epub 2021 Aug 20.

    PubMed 34425261
  • Kasamon YL, Ambinder RF, Fuchs EJ, Zahurak M, Rosner GL, Bolanos-Meade J, Levis MJ, Gladstone DE, Huff CA, Swinnen LJ, Matsui WH, Borrello I, Brodsky RA, Jones RJ, Luznik L. Prospective study of nonmyeloablative, HLA-mismatched unrelated BMT with high-dose posttransplantation cyclophosphamide. Blood Adv. 2017;1(4):288-292. doi: 10.1182/bloodadvances.2016002766. Epub 2017 Jan 6.

    PubMed 29242852

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Fludarabine, Cytoxan and Total Body Irradiation in people with Hematologic Malignancies, 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?

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 19 Feb 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 18 Feb 2026). 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?

Number of Participants Who Have Severe Acute Graft-versus-host-disease (GVHD) (time frame: Study Day 100). The study lists 3 primary outcomes in all. 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 NCT01203722, which also lists 2 linked publications.

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 19 Feb 2026.

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