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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-001029-14 | EudraCT Number |
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| Name | Class |
|---|---|
| URC-CIC Paris Descartes Necker Cochin | OTHER |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Time course of reconstitution of the different T cell subpopulations | time necessary to reach a normal number of naïve CD4+ and CD8+ T cells | Month 3, month 6, month 12 |
| presence of recent thymic emigrants |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| 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 |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Unité d'Immunologie Hématologie Rhumatologie Pédiatrique (UIHR), | Paris | Île-de-France Region | 75015 | France |
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To evaluate the active thymopoiesis
| Month 3, month 6, month 12 |
| T-cell receptor excision circles (TREC ) number in peripheral blood | To evaluate the active thymopoiesis | Month 3, month 6, month 12 |
| TCR rearrangements | By NGS analysis | Month 3, month 6, month 12 |
| B-cell reconstitution | number and phenotype for naïve IgD+CD27-, marginal zone IgD+CD27+, switched memory IgD-CD27+, and IgD-CD27- cells | Month 6, month 12 |
| Immunoglobulin (Ig) levels | Month 6, month 12 |
| NK cell numbers | Month 6, month 12 |
| Cumulative incidence of infections | 12 months post-transplant |
| Cumulative incidence of acute and chronic episodes of graft versus host disease (GVHD) | 24 months post-transplant |
| Overall survival | 2 years post-transplant |
| ID | Term |
|---|---|
| D007267 | Injections |
| ID | Term |
|---|---|
| D004333 | Drug Administration Routes |
| D004358 | Drug Therapy |
| D013812 | Therapeutics |
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