Clinical trial NCT03879876: Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients
Official 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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Pediatric Patients
Any Type of Severe Combined Immunodeficiency (SCID)
Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Keywords: SCID
hematopoietic stem cell transplantation
Human T Lymphoid Progenitor
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2019-03-19
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Detailed description: Not provided

Eligibility criteria: 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)

Primary outcomes:
measure: Dose limiting toxicity (DLT)
timeFrame: 3 months post-transplant
measure: reconstitution of the CD3+ TCRαβ+ cell compartment
timeFrame: Month 3
Secondary outcomes:
measure: Time course of reconstitution of the different T cell subpopulations
timeFrame: Month 3, month 6, month 12
measure: presence of recent thymic emigrants
timeFrame: Month 3, month 6, month 12
measure: T-cell receptor excision circles (TREC ) number in peripheral blood
timeFrame: Month 3, month 6, month 12
measure: TCR rearrangements
timeFrame: Month 3, month 6, month 12
measure: B-cell reconstitution
timeFrame: Month 6, month 12
measure: Immunoglobulin (Ig) levels
timeFrame: Month 6, month 12
measure: NK cell numbers
timeFrame: Month 6, month 12
measure: Cumulative incidence of infections
timeFrame: 12 months post-transplant
measure: Cumulative incidence of acute and chronic episodes of graft versus host disease (GVHD)
timeFrame: 24 months post-transplant
measure: Overall survival
timeFrame: 2 years post-transplant
Other outcomes:
Locations:
city: Paris
country: France
facility: Unité d'Immunologie Hématologie Rhumatologie Pédiatrique (UIHR),
state: Ile De France
Study officials:
affiliation: Institut National de la Santé Et de la Recherche Médicale, France
name: Isabelle ANDRE, PhD
role: STUDY_DIRECTOR
affiliation: Assistance Publique - Hôpitaux de Paris and Université Paris Descartes
name: Despina MOSHOUS, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: