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Bone marrow transplant breakthrough: more donors now possible for blood cancer patients?

NCT ID NCT01203722

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study tested a gentler bone marrow transplant for people with blood cancers who don't have a perfectly matched family donor. Instead, it used donors who are less closely matched, like unrelated volunteers or distant relatives. The goal was to see if this approach could be safe and effective, reducing severe side effects like graft-versus-host disease while helping patients survive longer.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Fludarabine, Cytoxan, Sirolimus, Mycophenolate Mofetil, total body irradiation, and allogeneic blood or marrow transplant
What this could lead to
If successful, this approach could expand donor options for blood cancer patients who lack a matched family donor, potentially improving access to life-saving transplants.
What could go wrong
This is an early-phase trial (Phase 1/2) with only 87 participants, so results may not apply to all patients. Risks include severe graft-versus-host disease, infection, and transplant failure.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

87 people

The number who actually took part.

Started

Sep 2010

Finished

May 2024

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 months to 75 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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: 1. Relapsed or refractory acute leukemia in second or subsequent remission, with remission defined as \<5% bone marrow blasts morphologically 2. 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 3. 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 4. Interferon- or imatinib-refractory CML in first chronic phase, or non-blast crisis CML beyond first chronic phase. 5. Philadelphia chromosome negative myeloproliferative disease. 6. Chronic myelomonocytic leukemia. 7. Juvenile myelomonocytic leukemia. 8. 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. 9. 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. 10. 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 5. Patients with CLL, SLL, or prolymphocytic leukemia must have \< 20% bone marrow involvement by malignancy (to lower risk of graft rejection). 6. 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. 7. Any previous BMT must have occurred at least 3 months prior to start of conditioning. 8. Adequate end-organ function as measured by: 1. Left ventricular ejection fraction greater than or equal to 35%, or shortening fraction \> 25%, unless cleared by a cardiologist 2. Bilirubin ≤ 3.0 mg/dL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST \< 5 x ULN 3. 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 9. 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 2. 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). 3. 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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    Baltimore, Maryland, 21231-2410, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.