Table of contents
- Trials by condition
- Study phases and designs
- Who is being studied
- What outcomes are measured
- Safety and follow-up
- How Azathioprine is used in comparisons
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.[1][2]
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.[3]
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.[4][5]
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.[6][7][8][9][10][11][12]
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.[1][3]
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and a Phase 2 study in autosomal dominant polycystic kidney and liver disease, although that kidney and liver study was withdrawn.[2][13]
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.[14][15]
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.[16][1][5][12]
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.[1][7][11]
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.[3][8][10]
What outcomes are measured
The main outcomes are different from trial to trial, but many studies measure remission, relapse, or disease control over time.[4][5][1]
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.[3][10][9]
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.[4][15][8]
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.[1][14][12]
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.[16][13][10]
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.[16][13]
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.[13]
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.[1][3][4][8][11][12]
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.[1][3][11]
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.[12][14]
