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Completed

NCT Number: NCT03879876

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

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.

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Key information

Conditions

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

Age range

2 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Unité d'Immunologie Hématologie Rhumatologie Pédiatrique (UIHR),

Paris, Île-de-France Region, 75015, France

About this study

Not provided

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Human T Lymphoid Progenitor (HTLP) injection

Drug

Injection of progenitors derived from HTLP culture at Day 8-Day 12 after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients (Day7 of culture)

Primary outcomes

  1. Dose limiting toxicity (DLT)

    Time frame: 3 months post-transplant

    to evaluate the procedure safety

  2. reconstitution of the CD3+ TCRαβ+ cell compartment

    Time frame: Month 3

    determined by the presence of ≥ 300/µL total, circulating CD3+ TCRαβ+ T cells to evaluate the efficacy

Secondary outcomes

  1. Time course of reconstitution of the different T cell subpopulations

    Time frame: Month 3, month 6, month 12

    time necessary to reach a normal number of naïve CD4+ and CD8+ T cells

  2. presence of recent thymic emigrants

    Time frame: Month 3, month 6, month 12

    To evaluate the active thymopoiesis

  3. T-cell receptor excision circles (TREC ) number in peripheral blood

    Time frame: Month 3, month 6, month 12

    To evaluate the active thymopoiesis

  4. TCR rearrangements

    Time frame: Month 3, month 6, month 12

    By NGS analysis

  5. B-cell reconstitution

    Time frame: Month 6, month 12

    number and phenotype for naïve IgD+CD27-, marginal zone IgD+CD27+, switched memory IgD-CD27+, and IgD-CD27- cells

  6. Immunoglobulin (Ig) levels

    Time frame: Month 6, month 12

  7. NK cell numbers

    Time frame: Month 6, month 12

  8. Cumulative incidence of infections

    Time frame: 12 months post-transplant

  9. Cumulative incidence of acute and chronic episodes of graft versus host disease (GVHD)

    Time frame: 24 months post-transplant

  10. Overall survival

    Time frame: 2 years post-transplant

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Collaborators

  • URC-CIC Paris Descartes Necker Cochin

Registry information

Official study title

A Phase I/II Study Evaluating 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

Acronym: HTLP Necker

Important dates

Study start
2020
Primary completion
2023
Study completion
2024
First posted
Mar 19, 2019
Registry last updated
May 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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