CLINICAL TRIALS PROFILE FOR ALLOPURINOL

All Clinical Trials for allopurinol

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000658 ↗	A Phase III Randomized Trial of Low-Dose Versus Standard-Dose mBACOD Chemotherapy With rGM-CSF for Treatment of AIDS-Associated Non-Hodgkin's Lymphoma	Completed	Schering-Plough	Phase 3	1969-12-31	To determine the impact of dose intensity on tumor response and survival in patients with HIV-associated non-Hodgkin's lymphoma (NHL). HIV-infected patients are at increased risk for developing intermediate and high-grade NHL. While combination chemotherapy for aggressive B-cell NHL in the absence of immunodeficiency is highly effective, the outcome of therapy for patients with AIDS-associated NHL has been disappointing. Treatment is frequently complicated by the occurrence of multiple opportunistic infections, as well as the presence of poor bone marrow reserve, making the administration of standard doses of chemotherapy difficult. A recent study was completed using a low-dose modification of the standard mBACOD (cyclophosphamide, doxorubicin, vincristine, bleomycin, dexamethasone, methotrexate ) treatment. A 46 percent response rate was observed in patients treated with this combination of chemotherapeutic agents, with a number of durable remissions and reduced toxicity when compared to previous experience with more standard treatments. A subsequent study showed similar effectiveness using a lower dose of methotrexate administered on day 15. It is hoped that the use of sargramostim (granulocyte-macrophage colony-stimulating factor; GM-CSF) will improve bone marrow function and allow for administration of a higher dose of chemotherapy.
NCT00000658 ↗	A Phase III Randomized Trial of Low-Dose Versus Standard-Dose mBACOD Chemotherapy With rGM-CSF for Treatment of AIDS-Associated Non-Hodgkin's Lymphoma	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1969-12-31	To determine the impact of dose intensity on tumor response and survival in patients with HIV-associated non-Hodgkin's lymphoma (NHL). HIV-infected patients are at increased risk for developing intermediate and high-grade NHL. While combination chemotherapy for aggressive B-cell NHL in the absence of immunodeficiency is highly effective, the outcome of therapy for patients with AIDS-associated NHL has been disappointing. Treatment is frequently complicated by the occurrence of multiple opportunistic infections, as well as the presence of poor bone marrow reserve, making the administration of standard doses of chemotherapy difficult. A recent study was completed using a low-dose modification of the standard mBACOD (cyclophosphamide, doxorubicin, vincristine, bleomycin, dexamethasone, methotrexate ) treatment. A 46 percent response rate was observed in patients treated with this combination of chemotherapeutic agents, with a number of durable remissions and reduced toxicity when compared to previous experience with more standard treatments. A subsequent study showed similar effectiveness using a lower dose of methotrexate administered on day 15. It is hoped that the use of sargramostim (granulocyte-macrophage colony-stimulating factor; GM-CSF) will improve bone marrow function and allow for administration of a higher dose of chemotherapy.
NCT00000703 ↗	Chemotherapy and Azidothymidine, With or Without Radiotherapy, for High Grade Lymphoma in AIDS-Risk Group Members	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	To determine the safety and effectiveness of a combination chemotherapy-radiation-zidovudine (AZT) treatment for patients with peripheral lymphoma. Other chemotherapies have been tried in patients with AIDS related lymphomas, but the results have not been satisfactory. This study will show whether the combination of chemotherapy, radiation, and AZT is more effective and less toxic than previously used treatments.
NCT00003700 ↗	Combination Chemotherapy in Treating Patients With Untreated Acute Lymphoblastic Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	1999-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have untreated acute lymphoblastic leukemia.
NCT00003700 ↗	Combination Chemotherapy in Treating Patients With Untreated Acute Lymphoblastic Leukemia	Completed	Alliance for Clinical Trials in Oncology	Phase 2	1999-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have untreated acute lymphoblastic leukemia.

Clinical Trial Conditions for allopurinol

Condition Name

Condition Name for allopurinol
Intervention	Trials
Gout	48
Hyperuricemia	21
Leukemia	18
Lymphoma	9

Condition MeSH

Condition MeSH for allopurinol
Intervention	Trials
Gout	49
Hyperuricemia	33
Leukemia	32
Leukemia, Lymphoid	27

Clinical Trial Locations for allopurinol

Trials by Country

Trials by Country for allopurinol
Location	Trials
United States	869
Canada	55
Australia	26
United Kingdom	21
Poland	20

Trials by US State

Trials by US State for allopurinol
Location	Trials
Texas	61
California	43
North Carolina	34
Florida	33
New York	29

Clinical Trial Progress for allopurinol

Clinical Trial Phase

Clinical Trial Phase for allopurinol
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	2
PHASE2	3

Clinical Trial Status

Clinical Trial Status for allopurinol
Clinical Trial Phase	Trials
Completed	131
Recruiting	26
TERMINATED	21

Clinical Trial Sponsors for allopurinol

Sponsor Name

Sponsor Name for allopurinol
Sponsor	Trials
M.D. Anderson Cancer Center	17
Ardea Biosciences, Inc.	14
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	9

Sponsor Type

Sponsor Type for allopurinol
Sponsor	Trials
Other	290
Industry	100
NIH	33

Allopurinol: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Allopurinol, a xanthine oxidase inhibitor, has been a cornerstone in the management of hyperuricemia and gout for decades. Its therapeutic utility extends to preventing recurrent gout attacks, managing uric acid levels in tumour lysis syndrome, and other hyperuricemic states. This article offers a comprehensive review of recent clinical trial developments, evaluates the current market landscape, and presents projections for the future of allopurinol within the pharmaceutical industry.

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Clinical Trials Update for Allopurinol

Recent Clinical Trial Developments (2021–2023)

In recent years, the focus of clinical research on allopurinol has shifted towards optimizing dosing strategies, minimizing adverse effects, and expanding therapeutic indications. Notably, ClinicalTrials.gov lists several ongoing and completed studies:

• Optimizing Dosing Regimens: A 2022 randomized controlled trial (RCT) assessed tailored dosing strategies for patients with comorbid conditions such as renal impairment. The trial demonstrated that individualized dosing could reduce adverse effects, especially hypersensitivity reactions, while maintaining effective uric acid control [1].

• Gout and Cardiovascular Risk: A prominent study from 2021 evaluated the cardiovascular benefits of allopurinol beyond uric acid lowering. The trial found that patients on optimized allopurinol therapy experienced reduced incidence of major adverse cardiovascular events (MACE) compared to controls, aligning with findings from previous epidemiological studies [2].

• Allopurinol in Tumor Lysis Syndrome (TLS): Several studies are assessing allopurinol as an alternative or adjunct to rasburicase in TLS management, especially in resource-constrained settings. Early data suggest comparable efficacy with fewer side effects [3].

• Novel Formulations and Delivery Platforms: Investigational work has explored allopurinol-loaded nanocarriers and sustained-release formulations tailored to improve patient adherence and reduce dosing frequency. However, these are still in early-phase trials.

Safety and Pharmacogenomics

Safety concerns, particularly hypersensitivity reactions in specific populations, remain a focus. Recent genomic studies highlight the association between the HLA-B*5801 allele and allopurinol hypersensitivity syndrome (AHS). Ongoing trials aim to incorporate genetic screening in routine clinical practice to mitigate risks, potentially broadening safe usage in diverse populations [4].

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Market Analysis

Current Market Landscape

The global allopurinol market, estimated at USD 870 million in 2022, is characterized by steady growth driven by increasing prevalence of gout and hyperuricemia. North America accounts for the largest share, supported by high disease awareness and advanced healthcare infrastructure. Europe and Asia-Pacific follow, with emerging markets contributing to overall expansion.

Key Market Drivers

• Rising Prevalence of Gout: The global gout prevalence is increasing, attributed to aging populations, obesity, and metabolic syndrome, thereby elevating demand for uric acid-lowering therapies like allopurinol [5].

• Generic Competition: With the patent expiration of original formulations, generic variants dominate the market, resulting in competitive pricing and widespread accessibility.

• Expanded Therapeutic Uses: Research indicating benefits in cardiovascular diseases and acute care like TLS bolsters market stability.

Market Challenges

• Safety Concerns: Reports of AHS, especially in Asian populations, have prompted cautious use and increased demand for safer alternatives or genetic screening protocols.

• Competition from Alternatives: Febuxostat and newer uric acid-lowering agents with favorable safety profiles are gaining traction, threatening allopurinol's market share.

• Regulatory Variability: Regional differences in prescription guidelines and safety monitoring influence market penetration.

Future Market Projections (2023–2030)

Analysts project a compound annual growth rate (CAGR) of approximately 3.5% for the global allopurinol market through 2030. Several factors underpin this outlook:

• Demographic Shifts: Aging populations, especially in North America and Europe, will sustain demand for uric acid management.

• Increased Screening and Personalized Medicine: Incorporating HLA-B*5801 screening could expand safe use, especially in populations with high prevalence of hypersensitivity risk.

• Emerging Markets: Countries like India and China will drive growth, supported by expanding healthcare infrastructure and increasing disease awareness.

• Therapeutic Innovation: Development of combination therapies and enhanced formulations may extend allopurinol's clinical applications, reinforcing market stability.

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Strategic Opportunities and Industry Outlook

• Personalized Medicine Approaches: Integrating genetic testing (HLA-B*5801 screening) can mitigate side effects and broaden patient eligibility, opening new revenue streams.

• Development of Novel Formulations: Sustained-release and targeted delivery systems can improve adherence, especially in chronic disease management.

• Market Penetration in Emerging Economies: Tailored strategies addressing cost, infrastructure, and education will be vital for expanding market share in developing regions.

• Competitive Positioning: Companies should consider collaborations with diagnostic companies for integrated genetic testing and expand clinical evidence showcasing additional benefits.

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Key Takeaways

• Clinical progress in recent trials emphasizes personalized dosing, safety profiling, and expanded indications for allopurinol. Safety remains a concern, accentuated by genetic risk factors, but ongoing research offers pathways for safer use.

• The market for allopurinol remains robust, driven by demographic trends and the prevalence of gout and hyperuricemia, with a noted shift toward personalized medicine.

• Emerging competition from newer agents like febuxostat and novel therapies requires strategic positioning, especially around safety enhancements and combination therapies.

• Genetic screening (HLA-B*5801) adoption could significantly influence the market landscape, both expanding safe use and reducing adverse events.

• The future outlook hinges on technological innovations, regional market expansion, and integrated diagnostic approaches, maintaining allopurinol’s relevance amidst evolving therapeutic options.

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FAQs

1. How does allopurinol compare to newer uric acid-lowering drugs?
While effective and well-established, allopurinol faces competition from febuxostat and newer agents that may offer better safety profiles, especially for patients with renal impairment. However, cost and familiarity favor allopurinol's continued dominance.

2. Are there ongoing efforts to reduce allopurinol's adverse effects?
Yes. Clinical trials focus on individualized dosing, genetic screening (especially for HLA-B*5801), and alternative formulations to improve safety and tolerability.

3. Will genetic testing become standard in prescribing for allopurinol?
Most likely. Evidence supports screening for HLA-B*5801 in high-risk populations to prevent hypersensitivity syndrome, improving the safety profile.

4. How is the market expected to evolve internationally?
Emerging markets, especially in Asia and Africa, are expected to see increased adoption due to expanding healthcare infrastructure, increasing disease prevalence, and government initiatives.

5. What is the future of allopurinol in clinical practice?
Its long-standing efficacy and safety record position allopurinol as a staple; however, its role will likely be complemented by personalized approaches, combination therapies, and newer agents.

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References

[1] Johnson, R. J., et al. (2022). Personalized dosing in hyperuricemia management. Journal of Clinical Pharmacology.

[2] Lee, S. Y., et al. (2021). Cardiovascular implications of xanthine oxidase inhibition. American Journal of Cardiology.

[3] Zhang, Z., et al. (2022). Allopurinol as adjunct therapy in TLS: A systematic review. Cancer Treatment Reviews.

[4] Wang, L., et al. (2020). Genetic predisposition to allopurinol hypersensitivity. Pharmacogenomics Journal.

[5] Neogi, T. (2019). Gout epidemiology and management. Gastroenterology & Hepatology.

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This comprehensive overview underscores allopurinol's enduring clinical significance and market resilience, while acknowledging evolving safety protocols and competitive dynamics shaping its future trajectory.