EUCTR2013-005508-33-PL进行中（未招募）1 期
Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- medac GmbH
- 入组人数
- 100
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •1. Non-malignant disease indicated for first myeloablative allogeneic
- •HSCT, including inborn errors of metabolism, primary
- •immunodeficiencies, haemoglobinopathies and bone marrow failure
- •2. First allogeneic 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 at least 9/10 allele matches
- •after four digit typing in human leucocyte antigen (HLA)-A, -B, -C, –
- •DRB1 and DQB1 antigens. For umbilical cord blood (UCB) match is
- •defined as at least 5/6 matches after two digit typing in HLA-A and -B
- •and four digit typing in DRB1 antigens.
- •4. Age at time of registration from 28 days to less than 18 years of age.
- •5. Lansky (patients <16 years of age) or Karnofsky (patients = 16 years
- •of age) performance score of at least 70%.
- •6. Written informed consent of the parents/legal guardians and patient's
- •assent/consent according to national regulations.
- •7. Female patients of child-bearing potential or partner of male patients
- •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 six months
- •thereafter. For female patients on the study, the vasectomized male partner should be the sole partner for this patient.
- •8. Negative pregnancy test for females of child-bearing potential.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 100
- •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
排除标准
- •1. Second or later HSCT.
- •2. HSCT from mismatched donor (less than 9/10 BM/peripheral blood
- •stem cells (PBSC) or less than 5/6 matched cord donor).
- •3. Preterm newborn infants (<37 weeks gestational age) and term
- •newborn infants aged 0 – 27 days at time of registration.
- •4. Obese paediatric patients with body mass index weight (kg)/[height
- •(m)]² > 30 kg/m².
- •5. Diagnosis of Fanconi anaemia and other chromosomal breakage
- •disorders, radiosensitivity disorders (deoxyribonucleic acid (DNA) Ligase
- •4, Cernunnos- X-ray repair cross-complementing protein 4 (XRCC4) like
- •factor (XLF), Nijmegen Breakage Syndrome (NBS)) and Dyskeratosis
- •6. Treatment with cytotoxic drugs within 10 days prior to day 7.
- •7. Impaired liver function indicated by Bilirubin > three times the upper
- •limit of normal (ULN) or aspartate aminotransferase/alanine
- •aminotransferase (AST/GOT, ALT/GPT) > ten times ULN or clinically significant coagulopathy, or active
- •infectious hepatitis with clinical evidence.
- •8. Impaired renal function indicated by estimated glomerular filtration
- •rate ([GFR], according to the Schwartz formula) < 60 mL/min/1.73m2.
- •9. Impaired cardiac function: severe cardiac insufficiency indicated by
- •left ventricular ejection fraction (LVEF) ? 35%.
- •10. Requirement for supplementary continuous oxygen.
- •11. Severe active infection requiring deferral of conditioning.
- •12. Human immunodeficiency virus (HIV) positivity.
- •13. Severe concomitant illness, comorbidity or condition that would
- •severely limit life expectancy.
- •14. Known pregnancy, breast feeding.
- •15. Known hypersensitivity to Treosulfan, Busulfan, Fludarabine and/or
- •16. Participation in another interventional clinical study with an
- •experimental drug, within four weeks prior to patient inclusion.
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