Effect of memory T cells after mismatched donor transplant in children with immunodeficiency

Not Applicable

• Conditions: on-severe combined immunodeficiency inborn errors of immunity; Haematological Disorders

• Registration Number: ISRCTN11859866
• Lead Sponsor: ewcastle upon Tyne Hospitals NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-10-25
• Last Update Posted: 11 March 2024
• Study Completion: 2027-09-01

Recruitment & Eligibility

• Status: Ongoing
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

1. Age at least 1 month and up to 18 years at the time of scheduled transplant
2. Patients deemed clinically eligible for allogeneic HSCT for non-SCID IEI
3. No suitable conventional matched family donor#.
4. Patient has an eligible donor identified by the clinical transplant team:
4.1. Planned mismatched family or mismatched unrelated donor for TCRaß-HaploSCT (Intervention and control group 1)*
OR
4.2. 10/10 HLA-matched unrelated donor for MUD HSCT (control group 2)
5. Capacity for patient or the patient’s parent or guardian to provide written informed consent

1. # Donor choice is independent of this study and will be decided by the clinical team according to best clinical practice. Generally the hierarchy of preferred donor is 10/10 matched family donor > 10/10 matched unrelated donor > mismatched family donor > mismatched unrelated donor. However, depending on the specific disorder, a family member might not be a suitable donor if they are a carrier of the genetic disease, or the underlying molecular defect is unknown.

Exclusion Criteria

1. Lansky/Karnofsky performance score <30%
2. Ongoing active acute GvHD or chronic extensive GvHD due to previous allograft at the time of screening
3. Patient receiving an immunosuppressive treatment for GvHD due to previous allograft at time of screening
4. Presence of a medical condition indicating that survival will be dismal such as the requirement for a high setting of mechanical ventilation and severe failure of a major organ system
5. Pregnancy or breastfeeding in female patients

Additional exclusion criteria for intervention group and control group 1 only (TCRaß-HaploSCT)
1. Patients with donor-specific antibodies (DSA) against the potential stem cell donor using the standard test according to the institutional guideline

Study & Design

• Study Type: Interventional
• Study Design: Not specified