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
Conditions
Interventions
Trade Name: ORENCIA
Pharmaceutical Form: Powder for infusion*
INN or Proposed INN: ABATACEPT
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Sponsors
ISTITUTO PER L`INFANZIA BURLO GAROFOLO
Eligibility
Sex/Gender
Male
Inclusion 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
Exclusion criteria: Positivity for mycobacterius tuberculosis infection.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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).;Secondary Objective: To widen the trial and to propose new protocols for treating IPEX.;Primary end point(s): Survival without the occurrence of new autoimmune phenomena; number and severity of autoimmune flares (cutanous, intestinal, haematological, respiratory). Residual growth. | — |
Countries
Italy
Outcome results
None listed