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Condition Guide

New Treatments & Clinical Trials for Bone Marrow Transplantation

Last updated September 2026Data from ClinicalTrials.gov310 active trials
← Browse all Bone Marrow Transplantation trials

Bone marrow transplantation, also called hematopoietic stem cell transplantation, replaces damaged or diseased bone marrow with healthy stem cells. It's used to treat blood cancers like leukemia and lymphoma, inherited blood disorders like sickle cell disease, and immune system conditions. The procedure has become safer over the past two decades, with better donor matching, infection control, and management of graft-versus-host disease.

What's actually going on in research

Trials are testing ways to make transplant safer and more accessible — expanding donor options through half-matched family members, reducing transplant-related complications, and improving outcomes for older patients. Studies are examining CAR T-cell therapy as an alternative to transplant in some blood cancers, post-transplant maintenance drugs to prevent relapse, and new approaches to graft-versus-host disease that preserve the transplant's anti-cancer effect while reducing tissue damage.

Haploidentical transplant

Using half-matched family donors makes transplant possible for patients who lack a fully matched donor. New techniques using post-transplant cyclophosphamide have made these transplants nearly as successful as matched transplants.

GVHD prevention

Trials are testing drugs like abatacept and ruxolitinib given early after transplant to prevent graft-versus-host disease. The goal is to reduce GVHD without eliminating the transplant's ability to fight cancer.

Reduced-intensity conditioning

Lower doses of chemotherapy before transplant allow older patients and those with other health conditions to undergo the procedure. Studies are refining which patients benefit most from this approach.

What to know before you search

Eligibility depends on the underlying disease, disease status at time of transplant, prior treatments, organ function, and availability of a suitable donor.

What types of trials are currently open

  • Conditioning regimen trialsTesting different chemotherapy or radiation doses given before transplant to prepare the body for donor cells while minimizing toxicity.
  • GVHD prevention trialsTesting drugs or cell manipulation techniques to prevent graft-versus-host disease while preserving the transplant's anti-cancer effect.
  • Donor source trialsComparing outcomes using different donor types — matched siblings, unrelated donors, half-matched family members, or cord blood.
  • Post-transplant maintenanceTesting drugs given after transplant to prevent cancer relapse, particularly in high-risk leukemia and myeloma.
  • Supportive care trialsTesting treatments for transplant complications like infections, organ damage, and late effects on quality of life.

Recently added Bone Marrow Transplantation trials

RecruitingObservational study

Receive antifungal medication to prevent serious infections during cancer treatment

This is a single-center, single-arm, observational clinical study evaluating the efficacy and safety of low-dose liposomal amphotericin B (50 mg/day, intravenous, once daily) for the prevention of invasive fungal infections in adult patients (aged 18-75 years) with hematological malignancies who develop prolonged neutropenia (absolute neutrophil count ≤0.5×10\^9/L, expected to last \>7 days) and are at high risk for invasive fungal disease. Participants are those who, per the treating physician's routine clinical decision, have been initiated on liposomal amphotericin B prophylaxis at 50 mg/day due to intolerance or toxicity to other antifungal agents. The primary outcome is the incidence of proven or probable invasive fungal disease. Secondary outcomes include incidence of pneumonia, persistent unexplained fever \>4 days, use of additional systemic antifungal therapy, and adverse events. A total of 30 participants will be enrolled. Data will be collected at baseline, during treatment, and within 7 days after treatment completion.

Hainan, Hainan, China
RecruitingInterventional study

Complete a specialized breathing test to monitor lung function after stem cell transplant

Bronchiolitis obliterans syndrome (BOS) is a severe pulmonary complication following allogeneic hematopoietic stem cell transplantation (HSCT). This prospective study evaluates the usefulness of the Forced Oscillation Technique (FOT) for the diagnosis, early detection, monitoring, and assessment of treatment response in patients undergoing allogeneic HSCT. FOT results will be compared with standard pulmonary function tests (spirometry, lung volumes, and DLCO).

Jerusalem, Israel
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