Pharmacokinetics of Thymoglobulin in Paediatric Haematopoietic Stem-cell Transplants

Study Overview
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Brief Summary
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This study will describe the pharmacokinetic disposition of biologically active rabbit anti-thymocyte globulin (rATG) after a consistent dose of 7.5 mg/kg/course given as part of the conditioning regimen in children undergoing hematopoeitic stem cell transplantation (HSCT).

Detailed Description
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Allogeneic hematopoeitic stem cell transplantation (HSCT) is a therapeutic option for patients with malignancies as well as metabolic and genetic diseases. Conditioning regimens given prior to donor cell infusion aim to ablate the recipient bone-marrow, to allow engraftment of the stem-cells infused, and to prevent acute versus host disease (aGVHD). Anti-thymocyte globulin (ATG) is one of the immunosuppressive drugs given as a preparative regimen for HSCT. Subjects will be given an ATG infusion daily for 3 days prior to HSCT and serum levels will be collected, as per schedule, with the last sample taken +100 days post-HSCT.

Official Title
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Pharmacokinetics and Pharmacodynamics of Thymoglobulin in Paediatric Haematopoietic Stem-cell Transplant Recipients

Conditions
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Malignancy, Metabolic Disease, Genetic Disorder

Intervention / Treatment
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* Biological: Thymoglobulin (rATG)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: All patients who are scheduled to receive ATG 2.5mg/kg/day for 3 days as part of the preparative regimen for HSCT, as determined by the responsible HSCT physician. Written, informed consent Exclusion Criteria: Hypersensitivity to rabbit proteins or to any product excipients Active acute or chronic infections, which would contraindicate any additional immunosuppression Known pregnancy or breastfeeding

Ages Eligible for Study
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Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Thymoglobulin<br>Thymoglobulin 7.5 mg/kg/course prior to HSCT | Biological: Thymoglobulin (rATG)<br>* Thymoglobulin 2.5 mg/kg of body weight IV administered daily for 3 days prior to HSCT. Thymoglobulin infused over a minimum of 6 hours for the first infusion and over at least 4 to 6 hours on subsequent days of therapy.<br>* Other names: Anti-thymocyte Globulin (Rabbit);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetic Disposition of ATG After a 7.5 mg/kg/Course | ATG pharmacokinetic parameters were estimated using a noncompartmental model. Maximum Observed Concentration (Cmax) of ATG After a 7.5 mg/kg/Course was measured | 100 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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allogeneic hematopoietic stem cell transplantation, pharmacokinetics, pediatrics