In short
Clinical trials are investigating Imlifidase in several rare and serious conditions. These studies look at safety, early effectiveness, and longer-term outcomes in patients such as highly sensitized kidney transplant recipients, and people with DMD, ANCA-associated vasculitis, anti-GBM disease, neuromyelitis optica spectrum disorder, and Crigler-Najjar syndrome.
Key points
- Imlifidase is being studied in clinical trials for several rare and serious conditions, with the strongest focus on kidney transplantation in highly sensitized patients. The trial data also include studies in Duchenne muscular dystrophy, ANCA-associated vasculitis, anti-GBM antibody disease, neuromyelitis optica spectrum disorder, and Crigler-Najjar syndrome. The studies range from Phase 1 to Phase 3, showing both early testing and larger confirmatory research. Main outcomes include crossmatch conversion, antibody reduction, kidney function, protein expression in muscle, and long-term graft survival. Some trials are completed, while others are still authorised. The target groups are very specific, often involving patients with pre-existing antibodies or severe disease.
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. These studies include patients with kidney transplant needs, highly sensitized children and adults, and people with specific immune-related or genetic diseases.
The trials range from early research to later confirmatory studies, including Phase 1, Phase 2, and Phase 3 designs. Some studies are completed, while others are authorised and still planned or ongoing in the source data.
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.
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.
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.
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.
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.
Rare disease studies
Some trials investigate rare immune or genetic diseases rather than transplantation alone. 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.
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.
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.
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.
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.
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.
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.
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.
Trial phases and study size
The data include one Phase 1 trial, several Phase 2 trials, and two Phase 3 trials. This mix shows that Imlifidase is being tested both in early studies and in larger studies that look at longer-term benefit.
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. 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.
Study status and who can join
According to the source data, some studies are marked Completed and others are Authorised. This means the research is at different stages, from finished trials to studies that are approved to start or continue.
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.
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.