Table of contents
- Trial overview
- Gout and high uric acid studies
- Blood cancer and transplant studies
- Cardiovascular risk study
- Study phases and main endpoints
- Who can participate
Trial overview
The trial data show that Allopurinol is being studied in several different settings, especially gout, high uric acid levels, cardiovascular risk, and blood cancers.[1][2][3]
These studies are mostly interventional trials, which means the researchers give a planned treatment and then measure what happens.[1][2]
The trials include both authorised and completed studies, with phases from Phase 1 to Phase 3.[1][2]
Gout and high uric acid studies
Several trials focus on gout and hyperuricemia, which means high uric acid levels in the blood.[3][4]
One Phase 3 study compares dotinurad with Allopurinol in adults with hyperuricemia associated with gout, and its main outcome is the proportion of participants with serum uric acid below 6.0 mg/dL at Week 24.[3]
Another large Phase 3 study compares tigulixostat with appropriately titrated Allopurinol, looking at the share of participants who keep serum uric acid below 6.0 mg/dL during Months 4, 5, and 6.[4]
A further Phase 3 trial in gout studies treatment strategy and asks whether patients meet adapted gout remission criteria during the last 6 months of a 24-month follow-up period.[5]
There is also a Phase 2 study in adult gout patients that tests whether stopping oral urate-lowering treatment is not worse than staying on treatment for the risk of flares after 2 years.[6]
Blood cancer and transplant studies
Allopurinol is also used in several trials for blood cancers, usually as part of a larger treatment plan rather than as the main study drug.[1][7][8]
In a Phase 2 study of young patients with high-risk chronic lymphocytic leukemia, the main goal is to measure uMRD, which means undetectable measurable residual disease, in blood and bone marrow at the end of treatment and after adding zanubrutinib in some patients.[1]
In relapsed or refractory diffuse large B-cell lymphoma, a Phase 2 trial measures objective response rate, meaning how many patients have their cancer shrink or disappear according to standard response rules.[7]
In acute lymphoid leukemia and acute lymphoblastic leukaemia studies, Allopurinol appears in treatment packages that also include cell therapy, chemotherapy, or other supportive drugs, and the main outcomes are response rate and measurable residual disease negativity.[8][9][10]
One Phase 1/2 study in relapsed or refractory AML, MDS/AML, or CMML measures safety first, then complete remission in Phase 2.[11]
A Phase 1 transplant study in patients with MDS, CMML, or secondary AML looks mainly at safety, including organ toxicity and grade III or higher adverse events after transplantation.[12]
Cardiovascular risk study
One Phase 3 study is testing Allopurinol in people with high or very high cardiovascular risk, including patients with hyperuricemia, hypertension, stroke, heart failure, peripheral artery disease, atrial fibrillation, and diabetes.[13]
The main endpoint is the occurrence of a major cardiovascular event, or MACE, which includes death, stroke, acute coronary syndrome, revascularization, unstable angina, and worsening heart failure.[13]
The brief summary says the study is looking at whether Allopurinol can reduce cardiovascular risk in the full study group and in people over 60 years of age.[13]
Study phases and main endpoints
The trial list includes Phase 1, Phase 1/2, Phase 2, and Phase 3 studies, so the research is at different stages of development.[1][11]
Phase 1 studies mainly focus on safety, side effects, and dose finding, while Phase 2 and Phase 3 studies look more closely at how well the treatment works.[11][3][13]
The main endpoints include serum uric acid control, gout remission, flare rates, response rates in cancer, measurable residual disease negativity, complete remission, and major cardiovascular events.[3][5][7][13]
Some trials also measure safety details such as adverse events, dose-limiting toxicities, changes in vital signs, and laboratory results.[2][11]
Who can participate
The target populations are different across studies, but the main groups include adults with gout, adults with high cardiovascular risk, young patients with chronic lymphocytic leukemia, children and adolescents with acute lymphoblastic leukaemia, and adults with several forms of blood cancer.[3][1][9][13]
Some studies are limited to people with relapsed or refractory disease, which means the cancer has come back or has not responded well to earlier treatment.[7][11]
Other studies include patients before stem cell transplantation or after transplant rejection, showing that Allopurinol is being used in complex treatment settings as part of broader study plans.[12][14]
