Treatment
Busulfan clinical trials
We’re tracking 53 recruiting studies that include Busulfan, each written for real people, not researchers, below.
Recruiting studies
- NCT05711732Recruiting
Collecting blood samples during IV busulfan treatment
This study looks at how to collect and handle blood samples from people receiving IV busulfan, a chemotherapy medicine. It may help improve the process of monitoring treatment by ensuring samples are collected correctly.
Chicago, IllinoisAges Any age - NCT07014943RecruitingPhase 2
High-dose chemo and stem cell transplant for brain lymphoma
This trial tests a combination of high-dose chemotherapy drugs (gemcitabine, busulfan, and thiotepa) followed by a stem cell transplant for people with primary central nervous system lymphoma. It aims to see if this treatment can improve outcomes for patients whose disease has responded to initial therapy.
Chengdu, SichuanAges 16–70 - NCT04339777RecruitingPhase 2
Bone marrow transplant for inherited immune system disorders
This trial studies how well a donor bone marrow (stem cell) transplant works for people with inherited immune system problems (called immune deficiencies). It’s for patients who haven’t improved with usual treatment or don’t have another good option.
Bethesda, MarylandAges 4–69 - NCT04708054RecruitingPhase 2/Phase 3
Venetoclax with busulfan chemo for high-risk AML or MDS
This study tests whether adding venetoclax to a specific busulfan-based chemotherapy plan improves outcomes for people with high-risk AML or MDS. It mainly targets patients whose leukemia/marrow disease still has high-risk features or measurable disease after initial treatment.
Houston, TexasAges 18–70 - NCT05463133RecruitingPhase 1/Phase 2
Stem cell transplant for CGD using targeted medications and steroids-free support
This trial tests an intensive stem cell transplant approach for people with chronic granulomatous disease (CGD). The study aims to improve outcomes by preparing the body for a donor stem cell graft and using immune “brakes” to reduce immune problems.
Bethesda, MarylandAges 4–65 - NCT05477589RecruitingPhase 3
Conditioning plan for kids with AML before stem cell transplant
This Phase 3 trial studies the best “conditioning” chemotherapy plan before an allogeneic stem cell transplant for children with AML. It may help improve outcomes by testing an approach for kids who are ready for transplant after relapse or specific high-risk features.
BrusselsAges Up to 18 years - NCT05991908RecruitingPhase 3
Comparing chemotherapy regimens before stem cell transplant for blood cancers
This trial is testing two different chemotherapy regimens (with one or two alkylating agents) before a stem cell transplant for people with acute myeloid leukemia or myelodysplastic syndrome. The goal is to find which conditioning treatment is safer and more effective.
Fuzhou, FujianAges 16–55 - NCT05027945RecruitingPhase 2
Stem cell transplant for people with VEXAS syndrome
This Phase 2 trial tests whether an allogeneic stem cell transplant (from a donor) can treat VEXAS syndrome. It may help calm the body’s overactive inflammation and improve symptoms in eligible adults.
Bethesda, MarylandAges 18–75 - NCT06869265RecruitingPhase 2
Thiotepa added to standard chemo before a stem cell transplant for older adults with high-risk AML
This trial tests whether adding a drug called thiotepa to the usual chemotherapy (busulfan and fludarabine) before a half-matched stem cell transplant can safely and effectively treat older adults (55–70) with high-risk AML.
Beijing, Beijing MunicipalityAges 55–70 - NCT01861106RecruitingPhase 2
Bone marrow transplant for people with GATA2 gene changes
This trial tests whether an allogeneic bone marrow (stem cell) transplant can improve outcomes for people with GATA2 gene changes or “MonoMAC” syndrome. It aims to rebuild a healthier immune system in patients who have serious infections and immune cell deficiencies.
Bethesda, MarylandAges 6–70 - 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 - NCT03126916RecruitingPhase 3
Testing added therapy for high-risk neuroblastoma in children
This Phase 3 study tests whether adding either a targeted drug (lorlatinib) or a radioactive treatment (131I-MIBG) to intensive chemotherapy improves outcomes for children with high-risk neuroblastoma. It may help by aiming at tumor cells more directly alongside standard treatment.
Birmingham, AlabamaAges 12 months–30 years - 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+ - NCT07565220RecruitingPhase 1
Thiotepa-based stem cell transplant for blood cancers
This trial tests a new chemotherapy preparation (using thiotepa) before a stem cell transplant from a matched donor to treat blood cancers like leukemia and myelodysplastic syndrome. The goal is to improve survival while reducing severe side effects.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT07113743Enrolling by invitationPhase 1/Phase 2
Gene therapy for x-linked chronic granulomatous disease
This trial tests a new gene therapy for X-linked chronic granulomatous disease (CGD), a condition where the immune system cannot fight certain infections. The treatment uses your own blood stem cells, which are modified in the lab to help your body produce working immune cells.
Bethesda, MarylandAges 3–60 - 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+ - NCT07074470Recruiting
New drug combo for relapsed brain lymphoma
This trial tests a new combination of drugs (teniposide, a PD-1 checkpoint inhibitor, and selinexor) for people with relapsed or refractory primary central nervous system lymphoma. It aims to see if this three-drug approach can shrink tumors or slow the disease when previous treatments haven't worked.
Suzhou, JiangsuAges 18–75 - 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 - 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 - NCT03779854RecruitingPhase 2
Preventing graft-versus-host disease in kids after stem cell transplant
This trial tests whether removing certain immune cells (T cells) from the donor transplant can lower the risk of chronic graft-versus-host disease (long-term immune attack after transplant) in children and young adults with blood cancers. If you qualify, it may help improve transplant outcomes while tracking safety and side effects.
Los Angeles, CaliforniaAges 6 months–26 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 - 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 - 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+ - 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
What Busulfan is being studied for
Conditions with recruiting Busulfan studies.
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.