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Treosulfan-based Conditioning in Paediatric Patients With Haematological Malignancies

NCT02333058 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 70

Last updated 2020-05-04

No results posted yet for this study

Summary

The primary goal of this study is to evaluate an alternative myeloablative, but reduced toxicity conditioning regimen in children, to describe the safety and efficacy of intravenous (i.v.) Treosulfan administered as part of a standardised Fludarabine-containing conditioning and to contribute to the current pharmacokinetic model to be able to finally give age (or body surface area) dependent dose recommendations. The treatment regimens given in the protocol MC-FludT.17/M are based on sufficient clinical safety and efficacy data. Considering the vital indication for allogeneic haematopoietic stem cell transplantation of the selected patient population, the risk-benefit assessment is therefore reasonably in favour of the study conduct.

Conditions

  • Acute Lymphoblastic Leukaemias (ALL)
  • Acute Myeloid Leukaemias (AML)
  • Myelodysplastic Syndromes (MDS)
  • Juvenile Myelomonocytic Leukaemias (JMML)

Interventions

DRUG

Treosulfan

Treosulfan dose per day is to be calculated by using BSA: One dose of Treosulfan per day on three consecutive days (day -6, day -5 and day -4) as intravenous (i.v.) infusion, given over 2 hours. Two background conditioning regimens with Treosulfan are allowed: One regimen consists of a standardised Fludarabine-containing regimen (regimen A) and the other consists of an intensified regimen with Fludarabine and ThioTEPA (regimen B). The investigator decides for each individual patient whether to treat the patient with regimen A or with regimen B. Treosulfan: i.v., BSA adapted: 10, 12 or 14 g/m²/day within 120 min to be administered prior to Fludarabine; Fludarabine: i.v., 30 mg/m2/day on days from -7 to -3 prior to HSCT; ThioTEPA (Regimen B): i.v., 2 x 5mg/kg/day on day -2.

Sponsors & Collaborators

  • Celerion

    collaborator INDUSTRY

  • Syneos Health

    collaborator OTHER

  • medac GmbH

    lead INDUSTRY

Principal Investigators

  • Ajay Vora, MD, Prof. · Great Ormond Street Hospital NHS Trust

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
28 Days
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-11-21
Primary Completion
2016-12-24
Completion
2019-09-30

Countries

  • Austria
  • Czechia
  • Germany
  • Italy
  • Poland
  • United Kingdom

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02333058 on ClinicalTrials.gov