TREOSULFAN LEVELS IN CHILDREN UNDERGOING STEM CELL TRANSPLANTATION

• Register: EUCTR
• Last refreshed on: 28 February 2019
• Main ID: EUCTR2013-003257-20-GB
• Date of registration: 12/03/2014
• Prospective Registration: Yes
• Primary sponsor: GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST
• Public title: TREOSULFAN LEVELS IN CHILDREN UNDERGOING STEM CELL TRANSPLANTATION
• Scientific title: Evaluation of Treosulfan pharmacokinetics (PK) in children undergoing allogeneic haematopoietic stem cell transplantation (HSCT) - Treosulfan PK in children
• Date of first enrolment: 02/05/2014
• Target sample size: 90
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-003257-20
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
Patients affected by malignant or non-malignant diseases with an indication to allogeneic HSCT.
Patients must meet all of the following criteria for admission in the study:

1) age = 28 days and = 18 years old;
2) Karnofsky Performance Status = 50 or Lansky Performance Status = 30;
3) provide signed, written informed consent from parent or guardian;
4) be able to comply with study procedures and follow-up examinations;
5) have adequate organ function (as indicated by Table 1, page 27), within 14 days prior enrollment;
6) negative pregnancy test in post-pubertal female patients.

Are the trial subjects under 18? yes
Number of subjects for this age range: 90
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
Patients who meet any of the following criteria will be excluded from study admission:

1) Patients aged < 28 days and > 18 years old;
2) patients with compromised organ function*;
3) patients with any other severe concurrent disease, which, in the judgment of the Investigator, would make the patient inappropriate for entry into this study;
4) Known hypersensitivity to Treosulfan or Fludarabine;
5) Pregnancy/lactation.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Any paediatric disease with an indication to an allogeneic stem cell transplantation (inclusing leukaemia, primary immunodeficiencies, metabolic disorders and autoimmune or genetic inflammatory bowel disorders).

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Intervention(s)

Trade Name: Treosulfan
Product Name: TREOSULFAN
Pharmaceutical Form: Injection
INN or Proposed INN: Treosulfan
CAS Number: 299-75-2

Primary Outcome(s)

Primary end point(s): Analyze Cmax, t1/2 and AUC0-8 of Treosulfan with serial blood tests;

Main Objective: This study aims to describe Treosulfan PK in children and infants undergoing allogeneic stem cell transplantation and to determine if this is a significant variable in determining transplant outcome.

The primary objective of the study is:

1) Assess PK parameters such as Cmax, t1/2 and AUC0-8 after Treosulfan infusion in children prior to allogeneic haematopoietic stem cell transplantation.

Secondary Objective: These are the secondary objectives of this study:

1) Assess interindividual and intraindividual variability of PK parameters in children of different age and weight;
2) Assess the relationship between PK parameters and patient characteristics;
3) Assess the relationship between Treosulfan PK and regimen related toxicity (using the NCI toxicity criteria scoring system) and survival;
4) Assess the relationship between Treosulfan PK and efficacy parameters, such as rate of engraftment and donor chimerism

Timepoint(s) of evaluation of this end point: Day -7 and -5

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: first 100 days after transplant,Assess donor engraftment in different cell lineages (myeloid and lymphoid) at 30, 60, 90, 180 and 360 days after transplant,follow up at 1 year

Secondary end point(s): 1) Assess the incidence of grade III-IV adverse events (NCI common toxicity criteria, version 4.0) within the first 100 days after transplant;
2) Assess transplant related mortality at 100 days after transplant;
3) Assess cumulative incidence and severity of acute GvHD
4) Assess the timing of neutrophil and platelet recovery;
5) Assess donor engraftment in different cell lineages (myeloid and lymphoid) at 30, 60, 90, 180 and 360 days after transplant;
6) Assess overall survival and disease-free survival at 1 year post transplant

Secondary ID(s)

NCT02048800

10MI28

Ethics review

Status: Approved
Approval date:
Contact: