Clinical Trials

Multiple clinical trials evaluate treosulfan as a conditioning regimen prior to allogeneic or autologous hematopoietic stem cell transplantation for hematological malignancies, including acute myeloid leukemia, myelodysplastic syndrome, multiple myeloma, acute lymphoblastic leukemia, and juvenile myelomonocytic leukemia. Spanning Phase 1, Phase 2, and combined Phase 1/2 designs, these active and completed studies focus on pharmacokinetic parameters and comparative regimen efficacy. Research is sponsored by pharmaceutical entities such as medac GmbH alongside academic medical centers, hospital trusts, and blood and marrow transplant research groups.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05534620 Recruiting
Acute Myeloid Leukaemia (AML)|Myelodysplastic Syndrome (MDS)|Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
medac GmbH|Synteract Inc.
 2024-02-01 Phase 1
NCT05636787 Recruiting
Multiple Myeloma
Insel Gruppe AG University Hospital Bern
 2023-06-06 Phase 2
NCT02333058 Completed
Acute Lymphoblastic Leukaemias (ALL)|Acute Myeloid Leukaemias (AML)|Myelodysplastic Syndromes (MDS)|Juvenile Myelomonocytic Leukaemias (JMML)
medac GmbH|Celerion|Syneos Health
 2014-11-21 Phase 2
NCT02048800 Completed
Allogeneic Haematopoietic Stem Cell Transplantation
Great Ormond Street Hospital for Children NHS Foundation Trust|Newcastle-upon-Tyne Hospitals NHS Trust
 2014-03 --
NCT01772953 Completed
Acute Myeloid Leukemia (AML)|Myelodysplastic Syndrome (MDS)
Center for International Blood and Marrow Transplant Research|National Marrow Donor Program|Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI BMT)|Pediatric Blood and Marrow Transplant Consortium|St. Baldrick''s Foundation|medac GmbH
 2013-09 Phase 2
NCT01063647 Completed
Hematological Malignancies
medac GmbH
 2001-11 Phase 1|Phase 2

(data from https://clinicaltrials.gov, updated on 2024-05-22)

Check the Treosulfan product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

As a bifunctional alkylating prodrug, treosulfan undergoes spontaneous non-enzymatic conversion into reactive epoxide intermediates that covalently bind to nucleophilic sites on genomic DNA, inducing interstrand DNA cross-links. This blockade of DNA replication and RNA transcription triggers apoptotic cell death and profound myelosuppression, establishing the cytotoxic foundation for myeloablative conditioning therapy in patients undergoing hematopoietic stem cell transplantation for acute myeloid leukemia and myelodysplastic syndrome.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.