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

Assistance Publique - Hôpitaux de Paris

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT); Pediatric Patients; Any Type of Severe Combined Immunodeficiency (SCID)

Treatments: Drug: Human T Lymphoid Progenitor (HTLP) injection

Study type: Interventional

Funder types: Other

Identifiers

NCT03879876

2018-001029-14 (EudraCT Number)

P150949J

Details and patient eligibility

About

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.

Full description

Not provided

Enrollment: 12 estimated patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

Human T Lymphoid Progenitor (HTLP) injection

Experimental group

Description:

Human T Lymphoid Progenitor cells (HTLPs) obtained after a brief period of ex vivo culture in the presence of the fusion protein DL-4, Retronectin® and a combination of cytokines

Treatment:

Drug: Human T Lymphoid Progenitor (HTLP) injection