A phase II trial of targeted busulfan and cyclophosphamide conditioning for allogeneic hematopoietic stem cell transplantation for hematological malignancies

A phase II trial of targeted busulfan and cyclophosphamide conditioning for allogeneic hematopoietic stem cell transplantation for hematological malignancies - Targeted busulfan and cyclophosphamide for allogeneic hematopoietic stem cell transplantation (C-SHOT 0301)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-C000000156

Enrollment

Registered

2005-09-13

Start date

2003-10-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Chronic Myelogenous Leukemia, Acute Myeloid Leukemia, Myelodysplastic Syndrome

Interventions

Check plasma busulfan concentrations over time after the first administration, and calculate the optimal dose of busulfan to achieve optimal AUC from 7th to 16th administrations.

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: (1) Diagnosis of either MDS, AML or CML (except for blastic crisis) (2) Age between 16 and 65 years old (3) Available HLA-identical family donor, or HLA-identical or DRB1-mismatched unrelated donor (4) First hematopoietic stem cell transplantation including auto transplant (5) Performance status ranging from 0 to 2 (6)Written informed consent of participation

Exclusion criteria

Exclusion criteria: (1) Uncontrollable and active infection (2) Positivity for HIV antibody and/or HBs antigen and/or HCV antibody (3) Graft manipulation such as T cell depletion (4) Impaired organ function (kidney; serum creatinine >2.0 mg/dl, Liver; aspartate aminotransferase more than 5 times high than upper limit or serum bilirubin >2.0mg/dl, heart; left ventricular ejection fraction less than 50%, lung; arterial oxygen saturation < 93% without oxygen inhalation.) (5) History of drug allergy used in the present conditioning or GVHD prophylaxis.

Design outcomes

Primary

Measure	Time frame
Overall survival at one year after transplantation	—

Secondary

Measure	Time frame
Disease-free survival, overall survival, pharmacokinetics of busulfan, incidence and severity of acute GVHD, incidence and severity of chronic GVHD, incidence and severity of hepatic veno-occlusive disease, mortality rate at 100 days after transplantaion, time to hematopoietic recovery, overall survival and disease-free survival in each disease group	—

Countries

Japan

Contacts

Public ContactAkio Kohno

JA Aichi Showa Hospital Department of Hematology and Oncology

akohno@wd5.so-net.ne.jp0587-56-4155

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026