In short
Clinical trials investigating Azathioprine are studying how it is used in several diseases, including ulcerative colitis, Crohn’s disease, myasthenia gravis, lupus nephritis, vasculitis, and other immune-related conditions. These trials mainly look at how well it works, how safe it is, and which patients may benefit most.
Key points
- Clinical trials investigating Azathioprine are mainly studying immune-related diseases and transplant-related problems. Most of the studies are Phase 3 trials, with some Phase 2 and Phase 4 research. The target groups include adults, children, kidney transplant recipients, and people with conditions such as ulcerative colitis, Crohn’s disease, myasthenia gravis, vasculitis, lupus nephritis, myocarditis, and sarcoidosis. The trials are measuring outcomes like relapse, remission, disease control, organ function, and time to treatment response. Safety is also a major focus in several studies, especially in long-term or combination-treatment settings.
Trials by condition
Several trials are studying Azathioprine in ulcerative colitis, including a Phase 3 study comparing top-down mirikizumab therapy with standard care that includes Azathioprine, and another completed Phase 2 study in moderately to severely active ulcerative colitis where Azathioprine was part of the treatment options being compared.
In Crohn’s disease, one Phase 3 trial compares Azathioprine with methotrexate as combination treatment with adalimumab, and the main outcome is endoscopic response at week 26.
Azathioprine is also being studied in myelin oligodendrocyte glycoprotein antibody associated disease (MOGAD), where one Phase 3 trial in adults and another in children look at relapse prevention and time to first relapse.
Other trials include studies in myasthenia gravis, lupus nephritis, myocarditis and inflammatory cardiomyopathy, granulomatosis with polyangiitis, sarcoidosis, antisynthetase syndrome-related interstitial lung disease, and kidney transplant settings.
Study phases and designs
Most Azathioprine trials in the source data are Phase 3 studies, which usually means larger, later-stage research that compares treatments and checks how well they work in real patient groups.
There is also a Phase 2 trial in ulcerative colitis and a Phase 2 study in autosomal dominant polycystic kidney and liver disease, although that kidney and liver study was withdrawn.
One study is listed as Phase 4 in kidney transplant recipients who were admitted to intensive care, and one ANCA vasculitis study is listed as Low Intervention, meaning the research is closer to routine care than a full drug-testing trial.
Who is being studied
The trials include a wide range of patient groups, such as adults with active idiopathic inflammatory myopathy, adults with ulcerative colitis, children with MOGAD, and patients with kidney transplant complications.
Some studies focus on people with newly diagnosed disease, such as newly diagnosed moderate-to-severe ulcerative colitis, while others include patients with long-term or difficult-to-treat disease, such as refractory lupus nephritis or inadequate response in granulomatosis with polyangiitis.
Several trials also include patients already receiving other treatments, which shows that Azathioprine is often being studied as part of a broader treatment plan rather than on its own.
What outcomes are measured
The main outcomes are different from trial to trial, but many studies measure remission, relapse, or disease control over time.
Some trials use organ-specific measures, such as endoscopic response in Crohn’s disease, LVEF in myocarditis and inflammatory cardiomyopathy, or progression-free survival in antisynthetase syndrome-related interstitial lung disease.
Other studies measure time-related outcomes, such as time to first relapse, time to end of BKPyV-DNAemia, or time to the first severe prejudicial event in ANCA vasculitis with end-stage renal disease.
In some studies, the endpoint is a score or percentage, such as disability progression in multiple sclerosis, comprehensive disease control in ulcerative colitis, or the proportion of patients with major relapses in sarcoidosis maintenance strategies.
Safety and follow-up
Safety is a major part of many Azathioprine-related trials, especially in long-term studies and studies where Azathioprine is used with other medicines.
Common safety measures include treatment-emergent adverse events, serious adverse events, lab test changes, vital signs, and ECG findings, which help researchers see whether treatment causes harm or unexpected problems.
Some trials also track quality of life, symptoms, or treatment stopping because of side effects, which gives a fuller picture of how patients may feel during treatment.
How Azathioprine is used in comparisons
In several trials, Azathioprine is compared with another active treatment, with placebo, or with a treatment strategy that includes other medicines such as glucocorticoids, rituximab, methotrexate, infliximab, or tacrolimus.
This means the research is not only asking whether Azathioprine works, but also whether it is better, similar, or less helpful than other treatment plans for a specific disease.
Some studies also use Azathioprine as part of a maintenance plan after remission, while others study it in induction treatment, meaning the first phase of treatment meant to bring disease under control.