|
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: |
|
|
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)
|
|
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.
|
|
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).
|
|
Source(s) of Monetary Support
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|