Trial intelligence / Profile preview

HTLP Injection After HSCT in SCID Patients

NCT03879876Phase 2

Key facts

Status: Completed
Start: May 2020
Primary completion: Sep 2023
Countries: 1
Sponsor: Assistance publique - Hôpitaux de Paris
Modality: Gene Editing, Gene Silencing, Stem Cell Therapies

Drugs: human T lymphoid progenitor
Conditions: severe combined immunodeficiency (SCID)

Overview

Phase 2, open-label, single-arm trial of human T lymphoid progenitor in severe combined immunodeficiency (SCID), sponsored by Assistance publique - Hôpitaux de Paris. Primary endpoints: Dose Limiting Toxicity at 3 months and Reconstitution of the CD3+ TCRαβ+ cell compartment at Month 3. Started May 2020; status: completed.

Design

1 arm

Study design: Non-randomizedOpen-labelSingle group

Human T Lymphoid Progenitor (HTLP) injection
Human T Lymphoid Progenitor cells (HTLPs) obtained after ex vivo culture injected at Day 8-12 post-HSCT

Who it enrols

Pediatric patients ≤ 2 years with SCID eligible for allogeneic HSCT, lacking a matched donor, with no prior allogeneic stem cell transplantation.

Endpoints

Primary

Dose Limiting Toxicity3 months
Reconstitution of the CD3+ TCRαβ+ cell compartmentMonth 3

Secondary

Time course of reconstitution of the different T cell subpopulationsMonth 12
Presence of recent thymic emigrantsMonth 12
T-cell receptor excision circles number in peripheral bloodMonth 12
TCR rearrangementsMonth 12
B-cell reconstitutionMonth 12
Immunoglobulin levelsMonth 12
NK cell numbersMonth 12
Cumulative incidence of infections12 months

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