Dettagli per singolo studio

DEC-NET Serial number FR378

Published online	31/03/2005 15.16.00
Last updated	27/11/2006 11.02.51

Other protocol ID number
Current trial status	Open (actively recruiting new participants)
Major Disease (ICD9 class)	COMPLICATIONS OF TRANSPLANTED BONE MARROW
Experimental drug
Hematopoietic stem cell Treatment regimen (dosage and duration) 15 days after the injection of the hematopoietic stem cell partially compatible, administration of the donor lymphocytes deprived of antirecipient reactivity. Only one dose will be injected to the patient but five doses will be studied (from 3 x 10+5 to 4.8 x 10+6 T/kg
Gender	Both
Age (range)	1 -1 5 years
Eligibility criteria
Inclusion criteria
1/Age: 0 to 15 years (limiting age included). 2/Absence of visceral anomalies contra-indicating the planned conditioning prior hematopoietic stem cell transplantation. 3/Written consent of the parents and /or legal guardian 4/Presence one of hereditary disease: -T cell immunodeficiency -Severe Combined Immunodeficiency (SCID) with serious infection -Wiskott-Aldrich syndrome -adhesion leucocytic proteins defect -Chediak-Higashi syndrome -Family lymphohistiocytosis -Other hereditary hemophagocytic syndrome(Purtilo and Griscelli disease) -Lysosomal storage diseases -Osteopetrosis
Exclusion criteria
1/Acute visceral diseases 2/Life expectancy of less than 3 weeks. 3/Severe infection or white lung disease. 4/Family or childr refusal. 5/Risk of study interruption
Trial design/methodology
Phase	2
Kind of study	Efficacy Safety Pharmacokinetics
Design	Phase I/II
Purpose of study
1/Research of the maximum number of donor T lymphocytes deprived of antirecipient reactivity that can be reinjected and that satisfies both similar conditions of tolerance (prevention of acute GvHD with grade II or higher) and efficacy (reconstitution of lymphocytes T-CD4+ number in 60 days). 2/Kinetics of the immune reconstitution according to the periodic measurements of CD3+,CD4+ lymphocytes counts
Primary outcomes
-Appearance of GvHD grade II or more -Kinetics of the immune reconstitution
Secondary outcomes
-Hematologic reconstitution. -Early and late infectious complications. -Immune reconstitution and immune anti-infectious response. -Post-graft chimerism
Summary of study design, objectives, and ongoing research findings
Patients with hereditary diseases (immune deficits, metabolic diseases) are treated by peripheral hematopoietic stem cell (HSC) partially HLA compatible transplantation, depleted in T lymphocytes by enrichment in CD3,4+ cells. After day 15 of HSC, a fixed number of T lymphocytes of the allodepleted donor will be reinjected. The aim of this phase I/II trial is to determine an amount of T lymphocytes both non toxic in term of GvH and effective in term of T immune reconstitution. The evaluation of the results is based on conventional follow-up criteria of hematopoietic stem cell transplantation.