HTLP

Phase 1

• Conditions: SCID pediatric patients (n=12 for analysis) requiring an HLA partially compatible allogeneic HSCT.; Therapeutic area: Diseases [C] - Immune System Diseases [C20]; MedDRA version: 20.0Level: PTClassification code 10010099Term: Combined immunodeficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders

• Registration Number: EUCTR2018-001029-14-FR
• Lead Sponsor: ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2018-07-02
• Last Update Posted: 6 November 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 12

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

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)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified