Busulfan clinical trials
Busulfan is named as an intervention in 23 current clinical trials in our China, France, Canada, Australia, Israel, Switzerland, Germany, Spain, Italy, Netherlands, Belgium, Sweden, Mexico, Poland, Turkey, South Korea, Denmark, Austria, Czechia, Norway, Ireland, UK, USA, Portugal, Japan and Taiwan registers. 13 are recruiting patients now. The most common phase is Phase 3. The sponsor with the most trials is Ruijin Hospital (2). The most-studied condition is Acute Myeloid Leukemia. The EU clinical trials register (CTIS) separately lists 1 authorised or ongoing trial.
Where Busulfan is approved
All regulators →Of the three regulators tracked here, the first to approve Busulfan was the US, on 26 Jun 1954. It is approved in 3 of the 3 regulators tracked here.
- 1 EU orphan designation, most recently for conditioning treatment prior to haematopoietic-progenitor-cell transplantation (30 Dec 2000, expired)
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
What health systems spend on Busulfan
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Busulfan works
All drug targets →- Ewing sarcomaPhase 3
- Acute leukemiaPhase 3
- Acute lymphoblastic leukemiaPhase 3
- Acute myeloid leukemiaPhase 3
- Acute myeloid leukemia by FAB classificationPhase 3
- Breast cancerPhase 3
- Diffuse large B-cell lymphomaPhase 3
- Hemophagocytic syndromePhase 3
- Myelodysplastic syndromePhase 3
- NeuroblastomaPhase 3
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL820, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 7136, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
Patient leaflets for Busulfan
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 2 products on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: Busulfan Fresenius Kabi (generic)EMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Reference identifiers for Busulfan
- ATC code
- L01AB01
- ChEMBL
- CHEMBL820
- DrugBank
- DB01008
- PubChem CID
- 2478
- CAS number
- 55-98-1
- FDA UNII
- G1LN9045DK
- Wikidata
- Q348922
- Wikipedia
- Busulfan
Codes that identify this medicine in other databases, from Wikidata (CC0), matched by its ChEMBL ID. The ATC code is the WHO classification of what the medicine is used for.
Trials by status
Trials by phase
Clinical trials of Busulfan
23 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Busulfan in the EU trials register (CTIS)
1 trialAuthorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Busulfan. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
Framework for Optimizing, Refining, and Unifying Management of HSCT in Pediatric ALL.
2025-522052-13-00 · CTIS ↗ · Hemic and Lymphatic Diseases
|
Phase 3 | Ongoing | Ospedale Pediatrico Bambino Gesu | Germany, Denmark, Finland + 14 more |
Busulfan across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
2 single-substance products containing Busulfan on the HPRA medicines list.
- Busulfan Fresenius Kabi 6 mg/ml concentrate for solution for infusionFresenius Kabi Deutschland GmbH · EU/1/14/951/001
- Myleran 2 mg film-coated tabletsAspen Pharma Trading Limited · PA1691/008/001 · Not marketed
Supply shortages
- Busulfan-ratiopharm 6 mg/ml Konzentrat zur Herstellung einer InfusionslösungGermany (BfArM) · Production problem · 1 Jan 2026 to 21 Oct 2026
Conditions studied with Busulfan
36 conditionsAll 36 conditions
| Condition (as registered) | Trials |
|---|---|
| Acute Myeloid Leukemia | 4 |
| MDS | 2 |
| Neuroblastoma | 2 |
| Acute Myeloid Leukaemia | 1 |
| Essential Thrombocythemia | 1 |
| AML, Adult | 1 |
| Leukemia | 1 |
| Hematologic Diseases | 1 |
| Acute Myeloid Leukemia (AML) | 1 |
| High Risk Acute Myeloid Leukemia(AML) | 1 |
| Relapsed/Refractory Acute Myeloid Leukemia (AML) | 1 |
| Leukemia, Myeloid, Acute(AML) | 1 |
| Hematopoietic Stem Cell Transplantation (HSCT) | 1 |
| Cancer | 1 |
| Hematologic Disease | 1 |
| Chronic Myelomonocytic Leukemia (CMML) | 1 |
| High-Risk Acute Myeloid Leukemia | 1 |
| Myelodysplastic Syndromes | 1 |
| High-Risk Myelodysplastic Syndromes | 1 |
| Acute Lymphoblastic Leukaemia | 1 |
| High-Risk Neuroblastoma | 1 |
| Patient With Insufficient Response Chemoimmunotherapy | 1 |
| Acute Myeloid Leukemia in Remission | 1 |
| Graft Versus Host Disease | 1 |
| Hematologic Malignancy | 1 |
| Ganglioneuroblastoma | 1 |
| Ganglioneuroblastoma, Nodular | 1 |
| Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm | 1 |
| Acute Myeloid Leukemia Post Cytotoxic Therapy | 1 |
| Acute Myeloid Leukemia, Myelodysplasia-Related | 1 |
| SCID | 1 |
| Acute Lymphoblastic Leukemia | 1 |
| Mixed Phenotype Acute Leukemia | 1 |
| Myelodysplastic Syndrome | 1 |
| T-cell Non-Hodgkin Lymphoma | 1 |
| Lymphoma, Extranodal NK-T-Cell | 1 |
Sponsors
lead sponsor per trial+ 2 more sponsors in the trial list above.
By country register
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
Related compounds
Questions about Busulfan trials
How many clinical trials are testing Busulfan?
23 current trials in our China, France, Canada, Australia, Israel, Switzerland, Germany, Spain, Italy, Netherlands, Belgium, Sweden, Mexico, Poland, Turkey, South Korea, Denmark, Austria, Czechia, Norway, Ireland, UK, USA, Portugal, Japan and Taiwan registers name Busulfan (also registered as L01AB01) as a drug or biological intervention, as of the registry sync on 9 November 2026. Completed and withdrawn studies are not counted.
Are Busulfan trials recruiting patients?
Yes. 13 of the 23 trials are recruiting: 6 with sites in China, 4 with sites in France, 3 with sites in Canada, 2 with sites in Australia, 4 with sites in Israel, 2 with sites in Switzerland, 2 with sites in Germany, 2 with sites in Spain, 2 with sites in Italy, 2 with sites in Netherlands, 2 with sites in Belgium, 2 with sites in Sweden, 3 with sites in Mexico, 1 with sites in Poland, 1 with sites in Turkey, 1 with sites in South Korea, 2 with sites in Denmark, 2 with sites in Austria, 2 with sites in Czechia and 2 with sites in Norway. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Busulfan clinical trials?
Ruijin Hospital (2), First Affiliated Hospital of Zhejiang University (2), He Huang (2) and Peking University People's Hospital (2), among 17 lead sponsors in total. The number in brackets is how many trials each organisation leads.
Are there Busulfan clinical trials in Ireland?
Yes. 1 trial in our Ireland register (ClinicalTrials.gov). Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.
Is Busulfan licensed in Ireland?
Yes. The HPRA medicines list includes 2 single-substance products containing Busulfan, for example Busulfan Fresenius Kabi 6 mg/ml concentrate for solution for infusion (Fresenius Kabi Deutschland GmbH). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Busulfan shortage?
The registers we track list 1 entry for Busulfan: 1 German notice. Shortages are product- and country-specific; check with a pharmacist.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Busulfan works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Busulfan works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Busulfan (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 9 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.