Pediatric hematological malignancies and non-malignant diseases
Conditions
Interventions
Treosulfan: For patients with a body surface area (BSA) >=0.4 m2 and <0.9 m2, administer 12 g/m2 once daily as a 2-hour intravenous infusion for 3 consecutive days (Day 2 to Day 4). If a patient with
Sponsors
Umeda Katsutsugu
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1)Those who have hematological malignancy(ALL, AML, MDS, ML, CML, and JMML) and non-malignant disease(primary immunodeficiency syndrome, inborn error of metabolism, and inherited bone marrow failure syndrome) for which allogeneic HSCT(including second transplants) is indicated. 2)Patients who are under 18 years of age at the time of obtaining consent. 3)Those who have obtained a matched related or unrelated donor. 4)Patient and/or the legally acceptable representative who provides a written consent or assent for participation in this study. 5)ECOG performance status: 0-2 6)Those who meet all the following criteria for the results of organ function test. a)No uncontrolled cardiac failure with 50% or more of left ventricular ejection fraction. b)FEV1 >= 50% and %VC >= 50% on lung function tests.(however, in case of pulmonary function test is not available, SpO2>=95% without requiring oxygen administration) c)AST/ALT less than 5 times of the upper limit of normal on examination by age. d)T.Bil less than 3 times of the upper limit of normal on examination by age. e)No uncontrolled renal failure with serum creatinine less than 2 times of the upper limit of normal on examination by age. 7)Are willing to consent to using a highly effective method of birth control such as condoms, implants, injectables, combined oral contraceptives, intrauterine devices, sexual abstinence or vasectomised partner while on treatment and for at least 6 months thereafter if females of childbearing potential(defined according to the Clinical Trials Facilitation and Coordination Group guidelines as a fertile woman, following menarche and until becoming postmenopausal unless permanently sterile) and males capable of reproduction. 8)Have a negative pregnancy test if females have childbearing potential.
Exclusion criteria
Exclusion criteria: 1)Those who have uncontrollable infections at the time of enrollment. 2)Those who are ineligible for allogeneic HSCT due to severe comorbidities at the time of enrollment. 3)Those with Fanconi anemia and other DNA breakage repair disorders, or these disease-related secondary leukemia. 4)Those who have congenital disease or mental disease interfering with study treatment. 5)Those who have double cancer. 6)Those with extreme obesity(BMI > 30 kg/m2). 7)A history of hypersensitivity to Treo or Flu. 8)Currently pregnant, breastfeeding, or interrupting breastfeeding. 9)Those who exhibit non cooperative behavior or non compliance. 10)Those who have psychiatric diseases or conditions that might compromise the ability to give informed consent. 11)Those who have received cytotoxic drugs(excluding administration for GVHD prophylaxis), alemtuzumab, or radiation therapy within 10 days(Day -10) prior to the start of the conditioning regimen(Day 1). 12)Those who are within six months from the completion of conditioning for a re-transplant. 13)Participation in another interventional study at the time of informed consent. 14)When the treating physician considers interfering with performing treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| DLT from Day 1 to Day 35 after HSCT | — |
Secondary
| Measure | Time frame |
|---|---|
| Time to engraftment after HSCT (up to 100 days) Occurrence of complete chimerism after HSCT Overall survival after HSCT (up to 100 days) (Serious) Adverse events Occurrence of hepatic SOS Occurrence of grade II-IV and III-IV acute GVHD Area under the concentration-time curve (AUC) Cmax Clearance (CL) | — |
Contacts
Public ContactKatsutsugu Umeda
University of Fukui
Outcome results
None listed