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

NCT ID: NCT03879876

Last Updated: 2022-09-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-05-13

Study Completion Date

2025-06-03

Brief Summary

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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.

Detailed Description

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Not provided

Conditions

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Human T Lymphoid Progenitor (HTLP) injection

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

Group Type EXPERIMENTAL

Human T Lymphoid Progenitor (HTLP) injection

Intervention Type 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)

Interventions

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Human T Lymphoid Progenitor (HTLP) injection

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)

Intervention Type DRUG

Eligibility Criteria

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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)
Minimum Eligible Age

2 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Isabelle ANDRE, PhD

Role: STUDY_DIRECTOR

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

Despina MOSHOUS, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Assistance Publique - Hôpitaux de Paris and Université Paris Descartes

Locations

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Unité d'Immunologie Hématologie Rhumatologie Pédiatrique (UIHR),

Paris, Île-de-France Region, France

Site Status RECRUITING

Countries

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France

Central Contacts

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Marina CAVAZZANA, MD & PhD

Role: CONTACT

+33 1 44 49 50 68

Jinmi BAEK

Role: CONTACT

+33 1 42 19 28 49

Facility Contacts

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Marina CAVAZZANA, MD, PhD

Role: primary

33 1 44 49 50 68

Other Identifiers

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2018-001029-14

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

P150949J

Identifier Type: -

Identifier Source: org_study_id

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