Table of Contents
- Clinical trial overview
- Kidney transplant studies
- Rare disease studies
- What the trials measure
- Trial phases and study size
- Study status and who can join
Clinical trial overview
The source data show that Imlifidase is being studied in interventional clinical trials across several serious conditions, with a strong focus on transplant medicine and rare diseases.[1] These studies include patients with kidney transplant needs, highly sensitized children and adults, and people with specific immune-related or genetic diseases.[1][2]
The trials range from early research to later confirmatory studies, including Phase 1, Phase 2, and Phase 3 designs.[1] Some studies are completed, while others are authorised and still planned or ongoing in the source data.[1]
Kidney transplant studies
Several trials study kidney transplantation in people who are highly sensitized, meaning they have antibodies that can make it hard to find a compatible donor kidney.[2][4][8]
In 2022-500230-28-00, the study looks at highly sensitized paediatric patients with a positive crossmatch against a living or deceased donor kidney, and the main goal is to see whether treatment changes the crossmatch from positive to negative within 24 hours.[2]
In NCT06461546, the trial studies highly sensitized recipients with a living donor kidney and measures whether a positive virtual crossmatch becomes negative within 6 hours after treatment, up to two doses.[4]
In NCT05369975, the study focuses on highly sensitized patients with end stage chronic kidney disease who are waiting for a kidney transplant, and it measures 1-year graft failure-free survival after treatment-enabled transplantation.[8]
NCT05937750 is a long-term follow-up study for patients who already had kidney transplantation after Imlifidase, and it compares long-term graft failure-free survival in the Imlifidase-treated group with a non-comparative reference group of less sensitized transplanted patients.[5]
Rare disease studies
Some trials investigate rare immune or genetic diseases rather than transplantation alone.[1] In 2024-516727-13-00, the condition is ANCA-associated vasculitis with severe diffuse alveolar hemorrhage, and the study aims to see whether ANCA levels fall below the reference range within 24 hours.[3]
In NCT05679401, the study is a Phase 3 open-label, controlled, randomised, multi-centre trial in severe anti-GBM antibody disease, also called Goodpasture disease, and it measures kidney function using eGFR at 6 months.[6]
2024-517176-38-00 studies neuromyelitis optica spectrum disorder, also described in the data as devic disease, in people with severe optic neuritis and/or myelitis, and it measures whether pathogenic anti-AQP4 IgG antibodies are depleted below detection within 6 hours.[7]
2023-510405-18-00 studies severe Crigler-Najjar syndrome in participants aged 16 years and older with pre-existing anti-AAV8 antibodies, and the main outcome is the proportion with serum total bilirubin at or below 300 µmol/L at 48 weeks without phototherapy from Week 16.[9]
In NCT06241950, the trial is in Duchenne muscular dystrophy and evaluates gene transfer therapy after Imlifidase infusion in participants with pre-existing antibodies to rAAVrh74.[1]
What the trials measure
The main outcomes vary by disease, but many trials focus on whether treatment helps overcome antibody barriers or improve disease-related results.[2][3][4][6]
In the transplant studies, the key outcomes include conversion of a positive crossmatch or virtual crossmatch to negative, as well as graft failure-free survival after transplantation.[2][4][5][8]
In the disease studies, the outcomes include antibody reduction, kidney function, bilirubin levels, and muscle biopsy measures such as dystrophin protein expression and vector genome copies.[1][3][6][7][9]
Trial phases and study size
The data include one Phase 1 trial, several Phase 2 trials, and two Phase 3 trials.[1] This mix shows that Imlifidase is being tested both in early studies and in larger studies that look at longer-term benefit.[1]
The enrollment numbers are small in many studies, such as 3, 5, 7, or 10 participants, which is common in rare disease research or early transplant studies.[1] The larger studies in the data include 126 participants in the long-term follow-up trial and 150 participants in the Phase 3 kidney transplant study.[5][8]
Study status and who can join
According to the source data, some studies are marked Completed and others are Authorised.[1] This means the research is at different stages, from finished trials to studies that are approved to start or continue.[1]
Participation is limited to very specific groups, such as highly sensitized kidney transplant candidates, children with a positive crossmatch, patients with severe ANCA-associated vasculitis, and people with pre-existing antibodies in gene therapy studies.[1][2][3][7][9]
Because each study has a narrow target population and a different main outcome, the trials are not interchangeable and should be read as separate research projects.[1]
