Overview

Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases

Status:
Active, not recruiting

Trial end date:
2023-01-01

Target enrollment:

Participant gender:

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.

Phase:

Phase 2

Details

Lead Sponsor:

medac GmbH

Collaborators:

Celerion
Syneos Health
Therametrics
Venn Life Sciences

Treatments:

Busulfan
Treosulfan