Idefirix 11 mg powder for concentrate for solution for infusion
Sponsors
Hansa Biopharma AB, Genethon, Sarepta Therapeutics Inc., Charite Universitaetsmedizin Berlin KöR, Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Conditions
Anti-GBM antibody disease (Goodpasture disease)Anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis with severe diffuse alveolar hemorrhageCrigler-Najjar syndromeDuchenne muscular dystrophy (DMD)End stage chronic kidney disease (CKD) patients who are highly sensitised and on the kidney transplant list awaiting a kidney transplantHighly sensitised paediatric patients with a positive crossmatch against a living or deceased donor kidney.Highly sensitised patients who have undergone kidney transplantation after Idefirix (imlifidase) administration and less sensitized patients transplanted with compatible kidney transplantdevic disease
Phase 1
Phase 2
A single-arm, multi-centre trial to evaluate efficacy and safety of imlifidase in highly sensitised children (1-17 years) receiving a kidney transplant with positive crossmatch against a living or deceased donor converted to negative after imlifidase treatment
RecruitingCTIS2022-500230-28-00
Start: 2023-06-02Target: 8Updated: 2025-12-22
An open-label, phase 2 trial to evaluate the efficacy and safety of a single intravenous administration of GNT0003 (an adeno-associated viral vector expressing the UGT1A1 transgene) following imlifidase pre-treatment in participants aged 16 years and older with severe Crigler-Najjar syndrome requiring daily phototherapy and presenting pre-existing anti-AAV8 antibodies.
RecruitingCTIS2023-510405-18-00
Start: 2024-11-08Target: 3Updated: 2025-11-19
lmlifidase treatment tor acute inflammation in AQP4-lgG associated neuromyelitis optica spectrum disorder
(DEFEAT NMOSD)
Not yet recruitingCTIS2024-517176-38-00
Target: 5Updated: 2024-09-10
Imlifidase in ANCA-associated Vasculitis
RecruitingCTIS2024-516727-13-00
Start: 2023-05-17Target: 10Updated: 2025-11-19
Phase 3
A Phase 3 open-label, controlled, randomised, multi-centre trial comparing imlifidase and standard-of-care with standard-of-care alone in the treatment of severe anti-GBM antibody disease (Goodpasture disease)
Active, not recruitingCTIS2022-500121-33-01
Start: 2023-05-08Target: 35Updated: 2024-12-19
A prospective, post-authorisation long-term follow up trial of patients previously treated with imlifidase prior to kidney transplantation, including a non-comparative concurrent reference cohort
Active, not recruitingCTIS2022-502727-21-00
Start: 2023-07-03Target: 126Updated: 2026-01-27
A controlled, open-label post-authorisation efficacy and safety study in imlifidase desensitised kidney transplant patients with positive crossmatch against a deceased donor prior to imlifidase treatment, including non-comparative registry and concurrent reference cohorts
Active, not recruitingCTIS2024-511810-18-00
Start: 2022-04-19Target: 144Updated: 2025-12-17