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

Phase 2

Completed

Conditions: Individualised ATG dosing in standard of care in stem cell transplantion
10024324
10021429
10021460

Registration Number: NL-OMON47267

Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary: Not available

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 UMCU Utrecht or the LUMC Leiden
* Any stem cell source
* First transplantation
* Age at time of transplantation < 18 years
* Signed written informed consent according to local law and regulations
* Lansky/Karnofsky * 80%

Exclusion Criteria

* Withdrawal of or no informed consent
* No Thymoglobuline in conditioning regimen
* Lansky / Karnofsky <80%
* Ex-vivo T-cell depleted grafts
* Other serotherapy in conditioning (e.g. campath, or campath in the bag)
* Received serotherapy within 3 months before this transplantation
* Pregnancy
* Sensibility to rabbit proteins or previous treatment with Thymoglobulin
* Acute or chronic infections, in which each form of immune suppression is contra-indicated
* Patients not receiving the full intentioned dose of Thymoglobulin due to any reason
* Cardiac ejection fraction < 30%
* No complete remission (CR-status) in case of malignancy
* History of serious immune-mediated reactions or hypersensitivity to any biological product
* Participation in other trial in which the dose of Thymoglobulin is fixed to amounts other than the individualized dose.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Incidence of CD4+ T-cell immune reconstitution, defined as a CD4+ T-cell count<br /><br>> 50 x 10e6/L in 2 consecutive measurements within 100 days.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>* Survival (overall survival, event free survival, non-relapse mortality,<br /><br>relapse mortality)<br /><br>* Relapse incidence<br /><br>* Incidence of viral reactivations (CMV, Adenovirus, EBV, HHV6, BK-virus)<br /><br>* Acute graft versus host disease (according to Glucksberg criteria)<br /><br>* Chronic graft versus host disease (according to Shulman criteria)<br /><br>* Engraftment defined as a neutrophil count > 0.5 x 109/L with use of<br /><br>granulocyte-colony stimulating factor (G-CSF) within 40 days<br /><br>* Rejection defined as >95% recipient chimerism, or reinfusion of donor cells<br /><br>after successful engraftment<br /><br>* Prospective validation of the pharmacokinetic model<br /><br>* Lymphocyte subset reconstitution monitored throughout the treatment<br /><br>(including some rare populations) for future studies</p><br>