drugset / Trial / NCT02349906
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
Phase 2
Completed
106 enrolled
medac GmbH
Celerion · collabSyneos Health · collabVenn Life Sciences · collab
RandomizedParallel-groupOpen-labelTreatment
Summary
The aim of the trial is to describe the safety and efficacy of intravenous (i.v.) Treosulfan compared to the conventional (myeloablative) dose of i.v. Busulfan, each administered as part of a standardised Fludarabine-containing conditioning regimen and to contribute to a PK model which permits - in conjunction with data comparing Treosulfan and Busulfan in adults with malignant diseases - to extend the use of Treosulfan in the paediatric population by extrapolating efficacy.
Timeline
- Start
- 2015-04
- Primary completion
- 2020-05-07
- Completion
- 2023-02-13
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Busulfan | Small molecule | 3.2 mg/kg | Intravenous |
| Comparator | Busulfan | Small molecule | 4.8 mg/kg | Intravenous |
| Subject | Treosulfan | Small molecule | 10 g | Intravenous |
| Subject | Treosulfan | Small molecule | 12 g | Intravenous |
| Subject | Treosulfan | Small molecule | 14 g | Intravenous |