Impact of Alemtuzumab exposure on immune reconstitution, autoimmunity, risk of infection, chimerism and Graft-versus-Host Disease in children with non-malignant diseases undergoing allogeneic stem cell transplantation. ARTIC International Multicenter Observational Study.
- Conditions
- Group of inherited blood diseases that affect red blood cells (e.g. sickle cell diseasethalassemia) and/or white blood cells (e.g. severe combined immunodeficiency characterized by a profound reduction or absence of T lymphocyte function) requiring an allogeneic stem cell transplantation.100189021002142910021460
- Registration Number
- NL-OMON55607
- Lead Sponsor
- eids Universitair Medisch Centrum
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 7
• diagnosis of severe congenital immune deficiency or of congenital
hematological disorder with an indication for HSCT, e.g. (severe) combined
immunodeficiency ((S)CID), hemophagocytic lymphohistiocytosis (HLH), chronic
granulomatous disease (CGD), thalassemia major, sickle cell disease (SCD);
• age at diagnosis and at the time of HSCT <= 18 years;
• Alemtuzumab treatment intravenously (iv) is given as part of a treosulfan- or
a busulfan-based reduced intensity conditioning regimen prior to alloHSCT;
• all donor types and hematopoietic stem cell sources will be considered;
• allogeneic hematopoietic stem cell transplantation is performed in a study
participating center; written consent of the parents (legal guardian) and of
the patient herself or himself if >=14 years old (>=12 years old in the
Netherlands);
• in case of multiple alloHSCT per patient, further transplantations will only
be considered if a minimal serotherapy-free interval of 3 months is preceding
the second transplant
• patients who do not fulfill the inclusion criteria;
• patients with known hypersensitivity to Alemtuzumab;
• patients treated with other serotherapy drugs (e.g. anti-thymocyte globulin -
ATG) within the same conditioning regimen prior to HSCT;
• patients who received any other serotherapy in the last 3 months before
starting this observational study;
• known HIV-positivity;
• severe uncontrolled infections before alloHSCT;
• active malignancies;
• pregnancy/lactation.
Study & Design
- Study Type
- Observational non invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Campath® (Alemtuzumab) levels measured 15-30 minutes before (please note: no<br /><br>sample will be taken before the first Alemtuzumab dose) and after each Campath®<br /><br>(Alemtuzumab) administration as well as at day 0 (= day HSCT), +1, +7, +14, +21<br /><br>and at week +4 and +6 after alloHSCT.</p><br>
- Secondary Outcome Measures
Name Time Method