Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients

• Status: Recruiting
• Trial end date: 2025-06-03
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

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

• Patient affiliated to social security

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)