Prospective Analysis of an individualized dosing Regimen of ATG (Thymoglobulin) in Children Undergoing HCT: redUcing Toxicity and improving Efficacy – a single arm phase II study

Completed

Conditions: Bone Marrow TransplantationStem cell transplantationThymoglobulinATGThymoglobulineBeenmergtransplantatieStamceltransplantatieImmuunreconstitutieImmune Reconstitution

Registration Number: NL-OMON26849

Lead Sponsor: MC Utrecht

Brief Summary: Admiraal et al, Clinical Pharmacokinetics 2014<br> Admiraal et al, Lancet Haematology 2015.

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 53

Inclusion Criteria

All patients eligible for a non-haplo-identical non-T-cell depleted HCT with Thymoglobulin as part of the conditioning regimen treated in the pediatric ward of the participating centers

- Any hematopoietic stem cell source

Exclusion Criteria

- Ex-vivo T-cell depleted grafts

- Other serotherapy in conditioning (e.g. Campath, or Campath in the bag)

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Incidence of CD4+ T-cell immune reconstitution, defined as a CD4+ T- cell count > 50 x 106/L in 2 consecutive measurements within 100 days.

Secondary Outcome Measures

Name	Time	Method
• Survival (overall survival, event free survival, non-relapse mortality, relapse mortality) at 1 year follow-up<br /><br>• Relapse incidence at 1 year follow-up<br /><br>• Incidence of viral reactivations (CMV, Adenovirus, EBV, HHV6, BK-virus) at 1 year<br>follow-up<br /><br>• Acute graft versus host disease (according to Glucksberg criteria3)<br /><br>• Chronic graft versus host disease (according to NIH criteria4) at 1 year follow-up<br /><br>• Engraftment defined as a neutrophil count > 0·5 x 109/L with use of granulocyte-<br>colony stimulating factor (G-CSF) within 40 days<br /><br>• Rejection defined as >95% recipient chimerism, or reinfusion of donor cells after<br>successful engraftment at 1 year follow-up<br /><br>• Prospective validation of the pharmacokinetic model<br /><br>• Lymphocyte subset reconstitution monitored throughout the treatment (including<br>some rare populations) for future studies<br />