Clinical study to examine the safety and efficacy of Treosulfan used as part of a conditioning therapy prior to a stem cell transplantation in children with blood cancer.
- Conditions
- Male and female children with haematologic malignant diseases as acute lymphoblastic leukaemias (ALL), acute myeloid leukaemias (AML), myelodysplastic syndromes (MDS) and juvenile myelomonocytic leukaemias (JMML), requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT)MedDRA version: 21.1 Level: LLT Classification code 10054439 Term: Juvenile chronic myelomonocytic leukemia System Organ Class: 100000004864MedDRA version: 21.0 Level: LLT Classification code 10060355 Term: Acute myeloid leukaemia in remission System Organ Class: 100000004864MedDRA version: 21.0 Level: LLT Classification code 10028534 Term: Myelodysplastic syndrome NOS System Organ Class: 100000004864MedDRA version: 21.0 Level: LLT Classification code 10000844 Term: Acute lymphoblastic leukaemia System Organ Class: 100000004864Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2013-003604-39-AT
- Lead Sponsor
- medac GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 70
1. Haematologic malignant disease i.e. ALL, AML, MDS or JMML, indicated for allo-HSCT.
2.Indication for first allo-HSCT or second allo-HSCT due to disease relapse, graft failure or secondary malignancy after previous HSCT.
3. Available matched sibling donor (MSD), matched family donor (MFD) or matched unrelated donor (MUD). For bone marrow (BM) and peripheral blood (PB) match is defined as 9/10 or 10/10 allele match after four digit typing in human leucocyte antigen (HLA)-A, B, C, DRB1 and DQB1 antigens.
4. Patients with ALL or AML in complete morphologic remission (blast counts <5% in BM) and patients with MDS or JMML with blast counts < 20% in BM at study entry.
5. Age at time of registration from 28 days to less than 18 years of age.
6. Lansky (patients aged <16 years) or Karnofsky (patients aged = 16 years) performance score of at least 70%.
7. Written informed consent of the parents/ legal guardians and patient assent/consent according to national regulations.
8. Females of child-bearing potential or male patients’ partners with child-bearing potential must use a highly effective method of contraception (pearl index < 1%) such as complete sexual abstinence, combined oral contraceptive, hormone intrauterine contraceptive device (IUCD), vaginal hormone ring, transdermal contraceptive patch, contraceptive implant or depot contraceptive injection in combination with a second method of contraception like a condom or a cervical cap / diaphragm with spermicide or surgical sterilisation (vasectomy) in male patients or male partners during the study and at least 6 months thereafter.
9. Negative pregnancy test for females of child-bearing potential.
Are the trial subjects under 18? yes
Number of subjects for this age range: 70
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Third or later allo-HSCT.
2. HSCT from haploidentical or umbilical cord blood donor.
3. Concomitant involvement of central nervous system (CNS) e.g.
presence of the leukaemic blasts in the cerebrospinal fluid (CSF) at study entry.
4. Treatment with cytotoxic drugs within 10 days prior to day 6.
5. Obese paediatric patients with body mass index: weight (kg)/[height (m)]² > 30 kg/m².
6. Concomitant solid tumours (e.g. neuroblastoma, peripheral neuroectodermal tumour [PNET], Ewing sarcoma).
7. Fanconi anaemia and other deoxyribonucleic acid (DNA) breakage repair disorders; secondary leukaemia
developed from the Fanconi anaemia or other DNA breakage repair disorders.
8. Impaired liver function indicated by Bilirubin > three times the upper limit of normal (ULN) or aspartate aminotransferase/alanine aminotransferase (AST/ALT) > ten times ULN or clinical significant coagulopathy, or active infectious hepatitis with clinical evidence.
9. Impaired renal function indicated by estimated glomerular filtration rate ([GFR], according to the Schwartz formula) < 60 mL/min/1,73m2.
10. Impaired cardiac function: severe cardiac insufficiency indicated by left ventricle ejection fraction (LVEF) 35%.
11. Requirement for supplementary continuous oxygen.
12. Severe active infection requiring deferral of conditioning.
13. Human immunodeficiency virus (HIV) positivity.
14. Known pregnancy, breast feeding.
15. Known hypersensitivity to Treosulfan and/or Fludarabine.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method