Bone Marrow Transplant for Leukemia
Recruiting in Palo Alto (17 mi)
+3 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: Ossium Health, Inc.
Must not be taking: Investigational drugs
Disqualifiers: Living donor, Prior HCT, Pregnancy, Infections, others
No Placebo Group
Approved in 4 Jurisdictions
Trial Summary
What is the purpose of this trial?This trial is testing the safety of using bone marrow from a deceased donor to treat patients with severe leukemia. The goal is to see if this new bone marrow can help produce healthy blood cells. Patients will be monitored closely for any side effects and overall effectiveness over several months.
Do I need to stop my current medications for the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you are on an investigational drug for your condition, you must stop it at least 5 half-lives before starting the trial.
What data supports the effectiveness of the treatment Bone Marrow Transplant for Leukemia?
Research shows that allogeneic bone marrow transplantation is an effective treatment for leukemia, with increased long-term survival and potential cure rates, especially when an HLA-identical donor is available. Additionally, donor leukocyte transfusions after transplantation have induced lasting remissions in many patients with chronic myelogenous leukemia and other types of leukemia.
12345Is bone marrow transplant generally safe for humans?
Bone marrow transplants can be effective for treating leukemia and other blood diseases, but they come with risks such as graft failure, graft-versus-host disease (where the donor cells attack the recipient's body), and infections. These risks can be higher when the donor is not a perfect match, but improvements in treatment are helping to reduce complications.
35678How is the bone marrow transplant treatment for leukemia different from other treatments?
Bone marrow transplant for leukemia is unique because it involves infusing healthy bone marrow from a donor to replace the patient's diseased marrow, offering a potential cure. This treatment requires a matching donor, often a sibling, and involves high-dose chemotherapy and sometimes radiation to prepare the patient, which is different from standard chemotherapy or radiation treatments alone.
59101112Eligibility Criteria
This trial is for adults aged 18-55 (or up to 70 for a specific regimen) with certain types of acute leukemia in remission, who need a bone marrow transplant and match the donor's tissue type at least half-way. They must be able to consent, have decent heart function and overall health status, and commit to study procedures for one year.Inclusion Criteria
I am mostly able to care for myself but may need help.
My heart's pumping ability is within the required range.
I am willing and able to follow all study rules and attend all appointments.
+7 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Pre-transplant Conditioning
Patients receive myeloablative or reduced intensity conditioning regimen prior to the transplant
1-2 weeks
Transplantation
Bone marrow transplant with Ossium HPC, Marrow
1 day
Post-Transplant Treatment
Post-transplant treatment with Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, and Filgrastim
56 days
Follow-up
Participants are monitored for safety and effectiveness after treatment
1 year
Participant Groups
The study tests the safety of bone marrow transplants from deceased donors in patients with blood cancers. It compares two pre-transplant conditioning regimens: myeloablative (stronger) or reduced intensity (milder), followed by close monitoring for nearly two months post-transplant.
2Treatment groups
Experimental Treatment
Group I: Cohort 2Experimental Treatment4 Interventions
\*Open to enrollment after DSMB review of Cohort 1 safety events through Day 56\*
Bone Marrow Transplant with Ossium HPC, Marrow
Pre-transplant conditioning treatment with:Regimen A(MAC): Busulfan and Fludarabine \[OR\] Regimen B(MAC): Fludarabine and Total Body Irradiation \[OR\] Regimen C(RIC): Fludarabine, Cyclophosphamide, and Total Body Irradiation
Post-Transplant treatment with Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, and Filgrastim
Group II: Cohort 1Experimental Treatment3 Interventions
Bone Marrow Transplant with Ossium HPC, Marrow
Pre-transplant myeloablative conditioning treatment with: Regimen A(MAC): Busulfan and Fludarabine \[OR\] Regimen B(MAC): Fludarabine and Total Body Irradiation
Post-Transplant treatment with Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, and Filgrastim
Bone Marrow Transplant is already approved in European Union, United States, Canada, Japan for the following indications:
🇪🇺 Approved in European Union as Allogeneic Bone Marrow Transplant for:
- Acute Leukemias
- Chronic Leukemias
- Lymphomas
- Multiple Myeloma
- Myelodysplastic Syndromes
- Aplastic Anemia
🇺🇸 Approved in United States as Allogeneic Bone Marrow Transplant for:
- Acute Leukemias
- Chronic Leukemias
- Lymphomas
- Multiple Myeloma
- Myelodysplastic Syndromes
- Aplastic Anemia
🇨🇦 Approved in Canada as Allogeneic Bone Marrow Transplant for:
- Acute Leukemias
- Chronic Leukemias
- Lymphomas
- Multiple Myeloma
- Myelodysplastic Syndromes
- Aplastic Anemia
🇯🇵 Approved in Japan as Allogeneic Bone Marrow Transplant for:
- Acute Leukemias
- Chronic Leukemias
- Lymphomas
- Multiple Myeloma
- Myelodysplastic Syndromes
- Aplastic Anemia
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Columbia University - Herbert Irving Comprehensive Cancer CenterNew York, NY
Moffitt Cancer CenterTampa, FL
TriStar Bone Marrow TransplantNashville, TN
St. David's South Austin Medical CenterAustin, TX
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Who Is Running the Clinical Trial?
Ossium Health, Inc.Lead Sponsor
Center for International Blood and Marrow Transplant ResearchCollaborator
References
Busulfan and melphalan as conditioning regimen for allogeneic hematopoietic stem cell transplantation in acute myeloid leukemia in first complete remission. [2021]Allogeneic hematopoietic stem cell transplantation with HLA-identical donors has been established for the treatment of acute myeloid leukemia patients for over 30 years with a cure rate of 50% to 60%.
Donor leukocyte transfusions for treatment of leukemic relapse after bone marrow transplantation. EBMT Immunology and Chronic Leukemia Working Parties. [2019]Allogeneic bone marrow transplantation is an effective treatment of leukemia. Intensive chemo- and radiotherapy used for conditioning and T-cells of the graft contribute to the control of leukemia. Animal experiments indicate that transfusion of lymphocytes from the marrow donor convert into complete chimerism without producing graft-versus-host disease, if delayed for two months or more. Transfusion of donor leukocytes (DLT) after marrow transplantation has induced lasting remissions in the majority of patients with chronic myelogenous leukemia (CML) in hematological or cytogenetic relapse, some patients with acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), transformed phase CML and multiple myeloma (MMY). The mechanism of the graft-versus-leukemia reaction is discussed.
Mismatched bone marrow transplantation. [2019]Most patients seeking allogeneic bone marrow transplantation lack an HLA genotypically identical sibling and require an alternative donor. Alternate donor options include the patient (autologous bone marrow transplantation), an HLA-haploidentical partially mismatched related donor, an HLA-phenotypically matched or partially mismatched unrelated donor, and an HLA-similar cord blood stem cell donor. When an allogeneic approach is preferred the risk of graft failure, graft-versus-host disease, fatal infection, and delayed immunoreconstitution is significantly greater than expected with a matched sibling donor. This has not always translated into inferior disease-free survival, possibly due to lower relapse rates seen in some studies from a graft-versus-leukemia effect potentially enhanced by major histocompatibility complex disparity. The degree and type of mismatch differs between alternative donors with broad variation in donor availability. Improvements are emerging in the prevention and treatment of graft rejection, acute and chronic graft-versus-host disease, and infectious complications that should result in improved survival, particularly when transplantation is applied to patients earlier in the disease course. No clear preference among alternate donor options has been established. In centers with experience, the use of an alternative donor option is not experimental in nature. The risk and outcome must be weighed on an individual patient basis. This approach to treatment is indicated in selective cases when conventional therapy is expected to offer little or no hope for cure.
[Preliminary study of HLA haplotype matched and T-cell undepleted allogeneic bone marrow transplantation for treatment of leukemia]. [2006]To explore the feasibility of allogeneic bone marrow transplantation (Allo-BMT) with graft from HLA haplotype matched related donor without T-cell depleted for the treatment of leukemia.
Allogeneic bone marrow transplantation in acute myeloid and lymphocytic leukemia. [2019]Allogeneic bone marrow transplantation is an accepted therapy for acute leukemia, with an increased rate of long-term survival and possible cure. Unfortunately, it can be given only when an HLA-identical donor is found. For this reason, large international registries containing normal HLA-typed volunteer marrow donors have been established. Although preliminary, results are encouraging. Approaches using stem cell purification and expansion, fetal and neonatal cells, and new immunosuppressive agents are currently under investigation.
Stem cell source and outcome after hematopoietic stem cell transplantation (HSCT) in children and adolescents with acute leukemia. [2010]Allogeneic hematopoietic stem cell transplantation from siblings, unrelated donors or HLA mismatched family members has become an important procedure to offer a chance of cure to children and adolescents with acute leukemia at high risk of relapse and those with certain genetic diseases. Bone marrow (BM) was the only stem cell source for many years. During the past 15 years, peripheral blood stem cells from granulocyte colony-stimulating factor (G-CSF) mobilized healthy donors, or umbilical cord blood from related or unrelated donors, have become available. Each stem cell source has different risks/benefits for patients and donors, the choice depending not only on availability, but also on HLA compatibility and urgency of the HSCT. This review will analyze the advantages and limitations of each of these options, and the main criteria which can be applied when choosing the appropriate stem cell source for pediatric transplant recipients with acute leukemia.
Current status of allogeneic bone marrow transplantation. [2019]Use of allogeneic bone marrow transplants continues to increase. During the 36-year period between 1955 and 1990, more than 33,000 patients received allogeneic bone marrow transplants; more than 45% of these were performed during the 3 years 1988-1990. Transplants are effective therapy for leukemia and other hematologic diseases. It is widely considered that transplants are the treatment of choice for aplastic anemia and chronic myelogenous leukemia, those who fail conventional therapy for acute leukemia and a variety of genetic, metabolic and immune deficiency disorders. Successful application of bone marrow transplantation is limited by complications such as graft failure, graft versus host disease GVHD and interstitial pneumonia and, until recently, the requirement for an HLA-identical sibling donor. In the past few years, an increasing number of transplants were performed using unrelated or HLA-partially matched related donors with some success. Development of post-transplant complications can often be predicted by risk factor assessment. In this report, current data from the IBMTR are summarized and several risk factors affecting outcome identified.
[Bone marrow transplantation (author's transl)]. [2006]Bone marrow transplantation is an experimental therapy, which has been used with success in patients with aplastic anemia, severe combined immunodeficiency and leukemia. We report here on the procedure of transplantation and the possible difficulties which can be encountered.
[Allogenic bone marrow grafts (author's transl)]. [2021]Bone marrow transplantation is a major therapeutic approach for treatment of patients with severe aplastic anemia or acute leukemia refractory to chemotherapy. It is possible only if an HLA matched sibing is found. ABO compatibility is not necessary. The conditionning regimen includes high doses of cyclophosphamide associated or not with a 1 000 rads total body irradiation. In acute leukemia, the two year percentage survival is 17%, in aplastic anemia it is 40%. Complications are immunologic : rejection, graft versus host disease and persistent severe immune deficiency.
[The probability of finding HLA identical or partially matched unrelated donors in the population of Vojvodina]. [2019]Allogeneic bone marrow transplantation and hematopoietic stem cell transplantation from unrelated donors are treatments of choice for patients lacking HLA identical siblings or family matched donors.
Allogeneic marrow transplantation: the Seattle experience. [2021]Allogeneic marrow transplantation from a normal donor provides potential life-saving therapy for a variety of inherited and acquired diseases of the hematopoietic system. Donor selection is a major issue, and most marrow transplants have been done only when an HLA-identical sibling donor has been available. Successful transplants can be done from partially HLA-matched relatives, but graft failure, graft rejection, and GvHD become increasingly serious complications as the number of HLA incompatibilities increase. More recently it has been shown that marrow transplants from HLA-identical unrelated donors can be successful. The recent development of a National Marrow Donor Program to recruit large numbers of HLA-typed volunteers and to facilitate the unrelated donor search process promises to make marrow transplants available to a growing number of patients.
Allogeneic bone marrow transplantation in the treatment of hematologic diseases. [2021]The current use of allogeneic bone marrow transplantation in various hematologic diseases is reviewed. Bone marrow transplantation (BMT) involves infusion of bone marrow from a suitable donor into a properly conditioned recipient. Most BMT is allogeneic, in which the donor is genetically dissimilar but shares some common tissue antigens with the recipient. Almost all patients undergoing allogeneic BMT must be "prepared" with high-dose cyclophosphamide to prevent graft rejection. Most patients with hematologic malignancy also receive total body irradiation to eradicate malignant cells located in areas inaccessible to the systemic circulation. Bone marrow transplantation is the treatment of choice for severe aplastic anemia. In acute myelogenous leukemia, the best results are observed in young patients undergoing BMT in first remission. In acute lymphoblastic leukemia, BMT is usually reserved for patients in second or subsequent remission. Early results are promising in patients with chronic myelogenous leukemia who receive BMT before the accelerated phase or blast crisis of this disease. Allogeneic BMT offers an opportunity for cure in some patients with relapses of Hodgkin's disease or those with certain subtypes of non-Hodgkin's lymphoma. Other diseases for which BMT has been used include severe combined immune deficiency disease, Fanconi's anemia, and multiple myeloma. Complications of BMT include graft failure or rejection, acute and chronic graft-versus-host disease, and infectious complications; late complications, such as restrictive and obstructive pulmonary disease, cataracts, sterility, and secondary malignancies, may also occur. Bone marrow transplantation has become an important treatment for many hematologic diseases, but it will probably remain a treatment reserved for only a few highly specialized centers. If morbidity and mortality caused by transplant-related complications can be reduced, BMT may be offered to older patients and those without HLA-identical sibling donors.