HTLP injection to improve immune recovery in SCID patients after stem cell transplant
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
This study is testing if HTLP injections can help young children with Severe Combined Immunodeficiency recover their immune system faster after a stem cell transplant.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 2 Years to 18 Years |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Locations | 1 site (Paris, Ile De France) |
| Trial ID | NCT03879876 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and efficacy of Human T Lymphoid Progenitor (HTLP) injections aimed at enhancing immune recovery in pediatric patients with Severe Combined Immunodeficiency (SCID) following partially HLA compatible allogeneic hematopoietic stem cell transplantation. The trial focuses on children under the age of two who lack a matched sibling or unrelated donor and are eligible for this type of transplant. By administering HTLP injections, the study seeks to accelerate the reconstitution of the immune system in these vulnerable patients. The trial is conducted in a controlled environment to ensure rigorous assessment of outcomes.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients under 2 years old with any type of SCID who are eligible for allogeneic HSCT and lack a matched donor.
Not a fit: Patients with an HLA genoidentical donor or those who have previously undergone allogeneic stem cell transplantation may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could significantly improve immune recovery and overall health outcomes for SCID patients post-transplant.
How similar studies have performed: While this approach is novel, similar studies targeting immune reconstitution in SCID patients have shown promise, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion 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)
Where this trial is running
Paris, Ile De France
- Unité d'Immunologie Hématologie Rhumatologie Pédiatrique (UIHR), — Paris, Ile De France, France (Recruiting)
Study contacts
- Principal investigator: Despina MOSHOUS, MD, PhD — Assistance Publique - Hôpitaux de Paris and Université Paris Descartes
- Study coordinator: Marina CAVAZZANA, MD & PhD
- Email: m.cavazzana@aphp.fr
- Phone: +33 1 44 49 50 68
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.