World Health Organization site
Skip Navigation Links

Main
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 19 March 2012
Main ID:  EUCTR2008-003368-21-IT
Date of registration: 06/02/2009
Prospective Registration: No
Primary sponsor: ISTITUTO PER L`INFANZIA BURLO GAROFOLO
Public title: Pilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndrome
Scientific title: Pilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndrome
Date of first enrolment: 04/06/2008
Target sample size:
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-003368-21
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other:  
Phase: 
Countries of recruitment
Italy
Contacts
Name:    
Address: 
Telephone:
Email:
Affiliation: 
Name:    
Address: 
Telephone:
Email:
Affiliation: 
Key inclusion & exclusion criteria
Inclusion criteria:
Confirmed diagnosis of IPEX. Unfavourable cost/benefit ratio for Hematopoietic Stem Cell Transplantation. In particular: 1)patients who already developed autoimmune disorders and/or organ failure because of the disease 2)disease not fully controlled by an intensive treatment at doses of conventional immunosuppressants producing severe side effects (infections, adrenal-hypotalamic suppression).
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Positivity for mycobacterius tuberculosis infection.


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes
Health Condition(s) or Problem(s) studied
For patients with a stabilized clinical condition, not recovered in care units for acute problems.
MedDRA version: 9.1 Level: HLGT Classification code 10003816
MedDRA version: 9.1 Level: HLT Classification code 10027657
Intervention(s)

Trade Name: ORENCIA
Pharmaceutical Form: Powder for infusion*
INN or Proposed INN: ABATACEPT
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-

Primary Outcome(s)
Main Objective: To improve the ratio between benefits and costs for the treatment with drugs in subjects not eligible for hematopoietic stem cell transplantation. In particular, to obtain a better control of the disease with less of the immunosuppressants-related side effects (infections and suppression of the hypothalamic axis).
Primary end point(s): Survival without the occurrence of new autoimmune phenomena; number and severity of autoimmune flares (cutanous, intestinal, haematological, respiratory). Residual growth.
Secondary Objective: To widen the trial and to propose new protocols for treating IPEX.
Secondary Outcome(s)
Secondary ID(s)
RC 03/08
Source(s) of Monetary Support
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.
Copyright - World Health Organization - Version 3.6 - Version history