SCID pediatric patients (n=12 for analysis) requiring an HLA partially compatible allogeneic HSCT. MedDRA version: 20.0 Level: PT Classification code 10010099 Term: Combined immunodeficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: HTLP
Product Code: HTLP
Pharmaceutical Form: Suspension for injection
Sponsors
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Pediatric patients affected by any type of SCID confirmed by clinical, immunological and/or molecular diagnosis and eligible for an allogeneic HSCT • Absence of a matched sibling donor or a matched unrelated donor (MUD) 10/10 • Clinical conditions incompatible with the search of a MUD • Written, informed consent of parents/ legal representative (child) • Age = 2 years at the time of screening • No prior therapy with allogeneic stem cell transplantation • No treatment with another investigational drug within one month before inclusion • Injection occurs 7 days prior to HSCT with CD34+ selected graft if all inclusion criteria are met • Patient affiliated to social security Are the trial subjects under 18? yes Number of subjects for this age range: 12 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: " Presence of an HLA genoidentical donor " Absence of written parental consent " Treatment with another investigational drug within one month before inclusion " Positive for HIV infection by genome PCR " Contra-indication to allogeneic transplantation or conditioning therapy (except SCID patients with DNA repair deficiency)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main objective is to assess the procedure's efficacy and safety: 1.(= 300/µL total CD3+ TCRaß+ T cells at 3 months) 2.Dose-limiting toxicity (DLT), including graft versus host disease (GVHD) grade II-IV and grade III or higher common terminology criteria adverse events (CTCAEs). ;Secondary Objective: "Time course of reconstitution of the different T cell subpopulations (numbers, functions and repertoires), thymic output and diversity of the T cell receptor repertoire by immunophenotyping, proliferation assays in the presence of mitogens and antigens, TCR repertoire analysis and TRECs values. A complete immunological assessment will be performed at 3, 6, 12 and 24 months and as soon as the number of CD3+ TCR aß cells in the blood reaches > 300/µl with particular attention to the time necessary to reach a normal number of naïve CD4 and CD8 T cells for the patient according to age "Cumulative incidence of opportunistic infections at 3, 6 and 12 months "Cumulative incidence of acute and chronic episodes of GVHD and their grade (at 3, 6, 12 and 24 months post-transplantation) according to Glucksgber GVHD staging "Overall survival at 2 years and EFS ;Primary end point(s): na | — |
Countries
France, Germany, Italy
Contacts
Public ContactDRCI Hôpital St Louis
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
Outcome results
None listed