NCT03879876

Brief 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started May 2020

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 15, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 19, 2019

Completed
1.2 years until next milestone

Study Start

First participant enrolled

May 13, 2020

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 3, 2023

Completed
1.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 3, 2025

Completed
Last Updated

September 27, 2022

Status Verified

September 1, 2022

Enrollment Period

3.3 years

First QC Date

March 15, 2019

Last Update Submit

September 26, 2022

Conditions

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

Keywords

SCIDhematopoietic stem cell transplantationHuman T Lymphoid Progenitor

Outcome Measures

Primary Outcomes (2)

  • Dose limiting toxicity (DLT)

    to evaluate the procedure safety

    3 months post-transplant

  • reconstitution of the CD3+ TCRαβ+ cell compartment

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

    Month 3

Secondary Outcomes (10)

  • Time course of reconstitution of the different T cell subpopulations

    Month 3, month 6, month 12

  • presence of recent thymic emigrants

    Month 3, month 6, month 12

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

    Month 3, month 6, month 12

  • TCR rearrangements

    Month 3, month 6, month 12

  • B-cell reconstitution

    Month 6, month 12

  • +5 more secondary outcomes

Study Arms (1)

Human T Lymphoid Progenitor (HTLP) injection

EXPERIMENTAL

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

Drug: Human T Lymphoid Progenitor (HTLP) injection

Interventions

Human T Lymphoid Progenitor (HTLP) injectionDRUG

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)

Human T Lymphoid Progenitor (HTLP) injection

Eligibility Criteria

Age2 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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
  • Patient affiliated to social security

You may not qualify if:

  • Presence of an HLA genoidentical donor
  • Absence of written parental consent
  • Positive for HIV infection by genome PCR
  • Contra-indication to allogeneic transplantation or conditioning therapy (except SCID patients with DNA repair deficiency)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Unité d'Immunologie Hématologie Rhumatologie Pédiatrique (UIHR),

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

RECRUITING

MeSH Terms

Interventions

Injections

Intervention Hierarchy (Ancestors)

Drug Administration RoutesDrug TherapyTherapeutics

Study Officials

  • Isabelle ANDRE, PhD

    Institut National de la Santé Et de la Recherche Médicale, France

    STUDY DIRECTOR

  • Despina MOSHOUS, MD, PhD

    Assistance Publique - Hôpitaux de Paris and Université Paris Descartes

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Marina CAVAZZANA, MD & PhD

CONTACT

+33 1 44 49 50 68m.cavazzana@aphp.fr

Jinmi BAEK

CONTACT

+33 1 42 19 28 49jinmi.beak@aphp.fr

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 15, 2019

First Posted

March 19, 2019

Study Start

May 13, 2020

Primary Completion

September 3, 2023

Study Completion

June 3, 2025

Last Updated

September 27, 2022

Record last verified: 2022-09

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)