Treatment
Busulfan clinical trials
We’re tracking 53 recruiting studies that include Busulfan, each written for real people, not researchers, below.
Recruiting studies
- NCT06959771RecruitingPhase 1/Phase 2
Gene therapy for CD40L deficiency (Hyper-IgM syndrome) - single patient study
This trial tests a new gene therapy that uses a person's own blood stem cells and T cells, which are corrected with a gene-editing tool called base editing. It is designed for people with a specific CD40L gene mutation who also have liver problems and portal hypertension. The goal is to see if this treatment can improve their immune system and liver health.
Bethesda, MarylandAges 37–120 - NCT04221035RecruitingPhase 3
Trial for children with high-risk neuroblastoma testing next steps
This Phase 3 study tests treatment strategies for children with high-risk neuroblastoma, aiming to improve how well the cancer responds and how long patients can stay in control. It compares different intensities and add-ons at key treatment steps depending on your child’s response and health.
Sydney, RandwickAges Up to 21 years - NCT06872333RecruitingPhase 2
Stem cell transplant from a matched sibling for blood disorders
This trial tests whether a stem cell transplant from a fully matched sibling donor can help people with serious blood disorders like sickle cell disease or thalassemia. It aims to replace the faulty blood system with a healthy one from the donor.
Minneapolis, MinnesotaAges Up to 55 years - NCT06851767Enrolling by invitationPhase 1/Phase 2
Gene therapy for X-linked severe combined immunodeficiency
This study tests a gene therapy to treat X-linked severe combined immunodeficiency (X-SCID) without a stem cell transplant from a matched sibling. It aims to improve immune function and reduce infections using your own stem cells modified to carry the correct gene.
Bethesda, MarylandAges 3–99 - NCT06809712RecruitingPhase 1
Stem cell transplant from a partially matched unrelated donor
This trial tests a transplant using stem cells from an unrelated donor who is not a perfect match for your tissue type. It may be an option for people with blood cancers who can't find a fully matched related donor and need a transplant quickly.
HangzhouAges 18–60 - NCT06802315RecruitingPhase 2
Radiation therapy before stem cell transplant for high-risk blood cancers
This trial tests a precise type of radiation called intensity modulated total marrow irradiation before a stem cell transplant. It aims to destroy cancer cells while reducing side effects for patients with high-risk leukemia or myelodysplastic syndrome.
Chicago, IllinoisAges 18–65 - NCT06506461RecruitingPhase 1
Gene editing for sickle cell disease in young adults
This trial uses gene editing to fix your own blood stem cells, so you can stop having severe sickle cell crises. It is for young adults with frequent pain episodes or who need regular blood transfusions.
Memphis, TennesseeAges 18–24 - NCT06345495RecruitingPhase 2
High-dose ruxolitinib and stem cell transplant for enlarged spleen in myelofibrosis
This trial tests a higher dose of ruxolitinib before a stem cell transplant for people with myelofibrosis who have an enlarged spleen. It aims to see if this approach helps the transplant work better.
Houston, TexasAges 18–75 - NCT06325709RecruitingPhase 1/Phase 2
Gene editing trial for X-linked chronic granulomatous disease
This trial tests a new gene-editing treatment for men with a specific genetic mutation that causes X-linked chronic granulomatous disease (CGD). The goal is to fix the faulty gene in your own blood stem cells to help your immune system fight infections better.
Bethesda, MarylandAges 18–75 - NCT06252870RecruitingPhase 2
Comparing two conditioning regimens for stem cell transplant
This trial tests two different chemotherapy and radiation plans (conditioning regimens) given before a stem cell transplant from a matched donor. Both are followed by the same drugs to prevent graft-versus-host disease. The goal is to find which regimen works better for people with blood cancers.
AngersAges 18–70 - NCT06207799RecruitingPhase 2
Drug combo before and after transplant for high-risk myeloma
This trial tests a two-step approach for people with newly diagnosed, high-risk multiple myeloma who are eligible for a stem cell transplant. You'll get a drug called elranatamab before transplant to 'purge' the cancer, and then after transplant, you'll get maintenance therapy guided by how well your cancer is controlled. The goal is to see if this reduces the chance of the cancer coming back.
Houston, TexasAges 18–75 - NCT06158828RecruitingPhase 1/Phase 2
Natural Killer Cells After Half-Matched Stem Cell Transplant for High-Risk AML
This study tests a new way to prevent leukemia from returning after a half-matched stem cell transplant. It uses specially activated natural killer cells to attack remaining cancer cells. This may help people with high-risk or relapsed AML who have few other options.
St Louis, MissouriAges 18 years+ - NCT03016806Recruiting
Umbilical cord blood transplant from unrelated donors
This trial studies transplanting healthy blood-forming cells from an umbilical cord blood donor who is not related to you. It may help people with certain blood cancers, immune disorders, or other serious diseases when a standard transplant plan is needed.
Rochester, New YorkAges 2 months–75 years - NCT02143830RecruitingPhase 2
Stem cell transplant chemotherapy for Fanconi anemia
This Phase 2 study tests a stem cell transplant approach using adjusted chemotherapy doses for people with Fanconi anemia. It may help replace damaged bone marrow and treat blood problems like severe aplastic anemia, MDS, or leukemia.
New York, New YorkAges 3 months+ - NCT05917405RecruitingPhase 2
Clofarabine vs fludarabine for AML before stem cell transplant
This Phase 2 study compares two chemotherapy medicines—clofarabine and fludarabine—given at a reduced intensity before an allogeneic stem cell transplant. It aims to see which regimen works better and is safer for people with AML who are eligible for this transplant approach.
Nantes, Loire AtlantiqueAges 18–99 - NCT05735717RecruitingPhase 2
T-cell transplant using depleted receptors for blood cancers
This Phase 2 study tests a specialized stem cell transplant that removes certain immune “T-cell” receptors to help treat blood cancers like acute leukemia. It may help your body rebuild a healthier immune system after high-dose treatment.
Minneapolis, MinnesotaAges Up to 60 years - NCT05674539Enrolling by invitationPhase 3
Less intense transplant regimen for AML or MDS patients
This trial tests whether a less intense chemotherapy and transplant plan can work for adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who are getting a stem cell transplant. It may help reduce transplant-related harm, especially in people considered higher risk due to age or other health factors.
Wuhan, HubeiAges 18 years+ - NCT05564390RecruitingPhase 2
MyeloMATCH screening to match you to AML or MDS treatment
This study screens people with newly suspected acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) to see which treatment approach fits best—either a specific study treatment or standard care. It may help by directing you to the right option and by sharing your blood/bone marrow samples for research.
Birmingham, AlabamaAges 18 years+ - NCT03311503RecruitingPhase 1/Phase 2
Gene therapy for X-linked SCID in young children
This trial tests a gene therapy using a lentivirus to give missing immune system instructions in children with X-linked SCID (SCID-X1). It also uses a low dose of targeted chemotherapy (busulfan) to help the new genes work, and it requires long-term follow-up for safety.
Los Angeles, CaliforniaAges birth–5 years - NCT05327023RecruitingPhase 1/Phase 2
Donor immune cells after stem cell transplant for high-risk blood cancer
This trial uses donor immune cells (lymphocytes) after a stem cell transplant to help fight any remaining cancer in people with high-risk blood cancers. It aims to lower the chance of the cancer coming back.
Bethesda, MarylandAges 12–120 - NCT04644016RecruitingPhase 2
Cord blood transplant for children and young adults with blood cancers
This Phase 2 trial studies using a cord blood transplant after finding it is risky enough that standard treatment may not be sufficient. It may help certain children and young adults with specific leukemia, lymphoma, and some non-cancer blood/immune or inherited disorders who do not have a well-matched donor available in time.
New York, New YorkAges Up to 21 years - NCT03970096RecruitingPhase 2
Reduce graft-versus-host disease after stem cell transplant
This Phase 2 study tests ways to lower the risk of graft-versus-host disease (GVHD), a serious complication after an allogeneic stem cell transplant from a matched donor. It is for people with acute leukemia or MDS who are going to receive intensive conditioning therapy, aiming to improve safety while keeping the transplant effective.
Tampa, FloridaAges 1–60 - NCT03579875RecruitingPhase 2
Testing an inherited bone marrow transplant approach for rare blood disorders
This Phase 2 study tests a special “T-cell depleted” transplant plan for people with inherited bone marrow failure disorders (Fanconi anemia or T-Beta-thalassemia—or “TBD”). It may help restore healthy blood production while lowering the chance of certain transplant complications.
Minneapolis, MinnesotaAges Up to 65 years - NCT03538899RecruitingPhase 1/Phase 2
Gene therapy for children with an Artemis SCID immune problem
This early-phase study tests an “autologous” gene therapy, meaning they use the child’s own cells, adjusted with a working gene, to help restore immune function. It is for babies and children with Artemis-deficient SCID, aiming to improve T-cell immunity and reduce the need for lifelong infection risk management.
San Francisco, CaliforniaAges 2 months+
Common questions
- Are there clinical trials studying Busulfan?
- Yes. Clin2 currently lists 53 recruiting studies that include Busulfan, drawn from the U.S. registry and rewritten for real people so you can see what each is testing and who it's for.
- How do I know if I qualify for a Busulfan trial?
- Each study lists its own eligibility criteria — rules about condition, age, and prior treatments. On every Clin2 trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Is Busulfan an approved treatment?
- A drug being studied in a trial may be investigational (still being tested) or already approved and being studied for a new use. Each study page explains what phase it's in. Clin2 does not provide medical advice — talk with your doctor about whether Busulfan or a specific trial is right for you.
Related treatments
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. A drug appearing here is being studied in a trial and may be investigational. Talk with your doctor about whether a specific treatment or trial is right for you.