Clinical Evidence

Evidence: First-in-human phase I/II clinical trial design

Randomised, exploratory and prospective phase I/II clinical trial to evaluate the safety and efficacy of the transfusion of autologous Treg cells obtained from thymic tissue in the prevention of rejection in heart transplant children (NCT04924491).

Graphic summary of the heart transplant clinical trial (NCT04924491)

Capacity to restore the Treg pool

  • In a first-in-human pediatric heart-transplant case, autologous thyTreg® infusion increased peripheral Treg frequency above pre-transplant baseline and maintained elevated levels over 24 months.
  • Expression of lineage/function markers (e.g., FOXP3, HELIOS, CTLA-4) was preserved or increased during follow-up.

JexpMed: Bernaldo-de-Quirós E, et al. First-in-human therapy with Treg produced from thymic tissue (thyTreg) in a heart transplant infant. J Exp Med. 2023;220(12):e20231045.

Capacity to restore the Treg pool

  • No infusion-related complications or product-attributable adverse events were observed over 24 months.
  • No clinical signs of graft rejection were reported during follow-up, supporting that autologous thyTregs® may be safe and well tolerated.

Clinical studies

  • Pediatric heart transplantation phase I: Nine heart transplant infants yet treated with autologous thyTreg®. Safety confirmed and first evidences of efficacy preventing acute rejection.
  • Pediatric heart transplantation phase II: Re-dosing thyTreg® in heart transplant children at 24 months seeking to reduce the doses of immunosuppressants maintaining immune tolerance. Starting in 2026.
  • Immune hyperactivation in acute respiratory distress syndrome (ARDS): Authorised phase I/IIa clinical trial for the allogeneic use of thyTreg® therapy in adults. Recruiting patients.
  • Pediatric VEO-BD: Allogeneic use of thyTreg® therapy in children with very early onset inflammatory bowel disease (VEO-IBD).
  • Crohn’s disease: Phase I/IIa clinical trial for the allogeneic administration of thyTregs® in the control of refractory Crohn’s disease in adult patients. Starting in 2026.