T&B depletion non malignant
- Conditions
- graft-versus-host diseaseMedDRA version: 14.1Level: SOCClassification code 10021428Term: Immune system disordersSystem Organ Class: 10021428 - Immune system disordersTherapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2011-004730-34-IT
- Lead Sponsor
- OSPEDALE PEDIATRICO BAMBINO GESU' DI ROMA
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- Not specified
?Availability of a matched related donor (MRD) or Matched Unrelated Donor (MUD)
?Lansky or Karnofsky Index = 60
?Inherited metabolic disorders: DQ =70 (+ MRI Loes score = 9 for adrenoleukodystrophy)
?Adequate cardiac, renal, hepatic and pulmonary functions as evidenced by:
?Serum creatinine =1.5 × upper limit of normal (ULN)
?Heart shortening fraction (left-ventricle) > 28 % or LVEF > 55%
?Serum bilirubin =1.5 × ULN (except for Wolman disease),
?AST and ALT = 2.5 × ULN (except for thalassemic syndromes and Wolman disease)
?Pulmonary function: if cooperative: FEV1 and FVC on pulmonary function testing > 60 %; if non cooperative: pulse oximetry > 95 % in room air
?Availability of autologous back up marrow (> 2 x 108 TNC+ cells/kg or > 2 x 106 CD34+ cells/kg) for MUD
?Adequate contraception in female patients of child-bearing potential
?Signed informed consent
Are the trial subjects under 18? yes
Number of subjects for this age range: 90
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
?Any malignancy
?Liver cirrhosis evidenced on liver histology (performed in suspicious cases or in case of Wolman disease)
?HIV- positivity
?Clinically significant pleural effusion or ascites
?Pregnancy or lactation
?Known hypersensitivity to trial drugs
?Participation in another experimental drug trial in the 2 months preceding enrolment
?Non-cooperative behaviour or non-compliance
?Previous HSCT
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Assess in a randomized fashion the benefit on standard graft-versus-host disease (GVHD) prophylaxis of the addition of ATG-Fresenius S in transplants from matched related donors (MRD) and of anti-CD20 rituximab in transplants from matched unrelated donors (MUD);Secondary Objective: For patients transplanted from a MUD<br><br>Reduced incidence and severity of cGVHD after transplantation using rituximab;Primary end point(s): For patients transplanted from a MRD<br><br>The primary end-point is the cumulative incidence of a combined end-point defined as the time from randomization to:<br>- primary and secondary graft failure, <br>- aGVHD II-IV, <br>- cGVHD, <br>- death, <br>whichever occurs first.<br>For patients transplanted from a MUD<br><br>The primary end-point is the cumulative incidence of a combined end-point defined as the time from randomization to:<br>- aGVHD II-IV, <br>- EBV viremia, <br>whichever occurs first.;Timepoint(s) of evaluation of this end point: NA
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. the cumulative incidence and severity of cGVHD after randomization<br>2. the incidence of TRM after randomization <br>3. the overall survival after randomization;Timepoint(s) of evaluation of this end point: NA