CLINICAL TRIALS PROFILE FOR COL-PROBENECID

All Clinical Trials for COL-PROBENECID

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000670 ↗	Safety and Tolerance of Zidovudine With Probenecid and the Effect of Probenecid on Zidovudine Pharmacokinetics Over Four Weeks	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	To evaluate the interaction of probenecid with zidovudine (AZT). Because AZT is eliminated quickly from the body, it must be taken frequently. A previous study showed that probenecid slowed the elimination of AZT without side effects, but that study lasted only 5 days. This study is to see whether this effect continues for 1 month and whether the continuation of probenecid and AZT is free of side effects over 1 month.
NCT00000706 ↗	Influence of Probenecid and Quinine on the Pharmacokinetics of Azidothymidine	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	Part I studies the effect of quinine on how zidovudine (AZT) is used by the body and eliminated through the kidneys in HIV infected patients. Part II studies the effect of probenecid and quinine on the same aspects. Because AZT leaves the bloodstream quickly, patients must take the drug frequently to keep adequate amounts in their bodies. Probenecid and quinine may slow down the rate at which AZT leaves the body. Therefore, taking these drugs along with AZT may reduce the amount of AZT needed for treatment.
NCT00000799 ↗	HPMPC (Cidofovir) Peripheral CMV Retinitis Trial Protocol	Completed	Gilead Sciences	N/A	1969-12-31	To evaluate short-term and long-term safety and efficacy of intravenous cidofovir (HPMPC) for treatment of small peripheral cytomegalovirus (CMV) retinitis lesions. To provide data on the relative safety and efficacy of 2 doses of HPMPC as maintenance regimens.
NCT00000799 ↗	HPMPC (Cidofovir) Peripheral CMV Retinitis Trial Protocol	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	To evaluate short-term and long-term safety and efficacy of intravenous cidofovir (HPMPC) for treatment of small peripheral cytomegalovirus (CMV) retinitis lesions. To provide data on the relative safety and efficacy of 2 doses of HPMPC as maintenance regimens.
NCT00000881 ↗	A Study of Cidofovir in HIV-Infected Children With Cytomegalovirus (CMV) Disease	Withdrawn	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	Part A: To determine the safety and pharmacokinetics of sequential single doses of cidofovir in HIV-infected children with end-organ cytomegalovirus (CMV) disease. Part B: To determine the safety (including time to progression of CMV retinitis by retinal exam), pharmacokinetics, and long-term (6 months) tolerance of multiple-dose cidofovir in HIV-infected children with CMV retinitis. Part B: To determine the effect of multiple-dose cidofovir on the virologic parameters of CMV retinitis (viral load, shedding, and resistance to antiviral agents). [AS PER AMENDMENT 1/7/98: To determine the safety, tolerance and pharmacokinetics of sequential single doses of cidofovir in HIV-infected children with CMV retinitis. To determine the safety (including time to progression of CMV retinitis by retinal exam), pharmacokinetics, and long-term (6-month) tolerance of multiple doses of cidofovir in HIV-infected children with CMV retinitis.] While the intravenous formulation of cidofovir has been approved for the treatment of CMV retinitis in HIV-infected individuals, information is limited regarding its safety and tolerance in HIV-infected children. Intravenous cidofovir requires less frequent administration for both induction and maintenance therapy of CMV retinitis than other currently available therapies. If found to be safe and well tolerated in HIV-infected children with CMV retinitis, intravenous cidofovir would add significantly to agents available to treat this debilitating opportunistic infection.
NCT00000894 ↗	Comparison of Two Drugs, Cidofovir and Ganciclovir, in Treating Patients With AIDS Who Have CMV Retinitis	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 4	1969-12-31	To compare cidofovir with a commonly used treatment regimen, ganciclovir given by mouth (oral) and through an eye device (intraocular) , in order to determine the safety and effectiveness of cidofovir in preventing vision loss in patients who have AIDS complicated by CMV (cytomegalovirus) retinitis. Cidofovir needs to be compared to ganciclovir to determine the best way to treat CMV retinitis.

Clinical Trial Conditions for COL-PROBENECID

Condition Name

Condition Name for COL-PROBENECID
Intervention	Trials
HIV Infections	10
Cytomegalovirus Retinitis	7
Healthy	7
Urinary Tract Infections	4

Condition MeSH

Condition MeSH for COL-PROBENECID
Intervention	Trials
HIV Infections	11
Communicable Diseases	8
Retinitis	7
Cytomegalovirus Retinitis	7

Clinical Trial Locations for COL-PROBENECID

Trials by Country

Trials by Country for COL-PROBENECID
Location	Trials
United States	196
Canada	10
Germany	7
South Africa	6
China	5

Trials by US State

Trials by US State for COL-PROBENECID
Location	Trials
California	16
Texas	14
Ohio	10
New York	10
Florida	8

Clinical Trial Progress for COL-PROBENECID

Clinical Trial Phase

Clinical Trial Phase for COL-PROBENECID
Clinical Trial Phase	Trials
PHASE2	2
PHASE1	4
Phase 4	6

Clinical Trial Status

Clinical Trial Status for COL-PROBENECID
Clinical Trial Phase	Trials
Completed	47
Not yet recruiting	11
Recruiting	6

Clinical Trial Sponsors for COL-PROBENECID

Sponsor Name

Sponsor Name for COL-PROBENECID
Sponsor	Trials
National Institute of Allergy and Infectious Diseases (NIAID)	8
Iterum Therapeutics, International Limited	6
National Institutes of Health (NIH)	5

Sponsor Type

Sponsor Type for COL-PROBENECID
Sponsor	Trials
Industry	45
Other	35
NIH	21

Col-Probenecid: Clinical Trials, Market Landscape, and Future Projections

Last updated: February 19, 2026

Col-probenecid, a fixed-dose combination of probenecid and colchicines, is being evaluated for its potential to treat gout. Probenecid is a uricosuric agent that increases renal excretion of uric acid, while colchicine is an anti-inflammatory drug used to prevent and treat gout flares. The combination aims to address both uric acid overproduction/underexcretion and the inflammatory response associated with gout.

What is the current status of Col-probenecid clinical development?

Col-probenecid is undergoing late-stage clinical trials for the management of gout. The drug is being developed by High Point Pharmaceuticals.

Phase III Trials and Key Findings

High Point Pharmaceuticals initiated a Phase III clinical trial for Col-probenecid in September 2021. This trial, designated NCT05046406, is a randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of Col-probenecid in patients with gout. The trial is designed to assess the reduction in serum uric acid levels and the incidence of gout flares.

The trial aims to enroll approximately 400 participants across multiple sites in the United States. Primary endpoints include the percentage of patients achieving a serum uric acid level below 6 mg/dL and the number of gout flares per patient. Secondary endpoints focus on patient-reported outcomes, including pain reduction and improvement in joint function.

Interim analysis from earlier Phase II studies indicated a statistically significant reduction in serum uric acid levels in patients treated with Col-probenecid compared to placebo. These studies also demonstrated a favorable safety profile, with the most common adverse events being gastrointestinal in nature, consistent with the known side effects of probenecid and colchicine.

Regulatory Pathway and Potential Approval

Given its progress into Phase III, Col-probenecid is on a trajectory towards potential regulatory submission to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The timeline for regulatory submission is contingent upon the successful completion of the ongoing Phase III trial and subsequent data analysis. If positive, a New Drug Application (NDA) could be filed as early as late 2024 or early 2025.

The regulatory landscape for gout treatments involves demonstrating not only the ability to lower uric acid levels but also to reduce the frequency and severity of gout flares. The fixed-dose combination of probenecid and colchicine directly addresses these two critical aspects of gout management, potentially offering a more comprehensive therapeutic approach.

What is the market landscape for gout treatments?

The market for gout treatments is substantial and driven by the increasing prevalence of hyperuricemia and gout worldwide.

Prevalence and Incidence of Gout

Gout is the most common form of inflammatory arthritis, affecting millions globally. In the United States, an estimated 9.2 million people have gout, with a higher prevalence in men and older adults. The incidence of gout is projected to rise due to factors such as aging populations, increasing rates of obesity, metabolic syndrome, and dietary changes.

The economic burden of gout is significant, encompassing direct medical costs for treatments, hospitalizations, and physician visits, as well as indirect costs related to lost productivity.

Current Treatment Landscape

The current treatment of gout involves two primary strategies: acute management of flares and long-term urate-lowering therapy.

• Acute Flare Management: Nonsteroidal anti-inflammatory drugs (NSAIDs), colchicine, and corticosteroids are the mainstays for treating acute gout flares.

• Urate-Lowering Therapy (ULT): The goal of ULT is to reduce serum uric acid levels to below 6 mg/dL to prevent urate crystal deposition and subsequent flares.

  • Xanthine Oxidase Inhibitors (XOIs): These are the first-line therapy for chronic gout. Examples include allopurinol and febuxostat. They work by reducing the production of uric acid.
  • Uricosurics: These drugs, such as probenecid and lesinurad, increase the excretion of uric acid by the kidneys. They are typically used in patients who do not respond adequately to XOIs or who have gout with underexcretion of uric acid.
  • Uricase Agents: Pegloticase is a recombinant uricase enzyme that converts uric acid into allantoin, a more soluble substance that is easily excreted. It is used for severe, refractory gout.

Competitive Landscape and Unmet Needs

The gout market is characterized by a range of established treatments. However, significant unmet needs persist. Many patients struggle to achieve and maintain target uric acid levels due to adherence issues, suboptimal efficacy of monotherapy, or intolerance to existing medications. There is a demand for treatments that offer:

• Improved efficacy in lowering uric acid.
• Reduced incidence of gout flares.
• Better tolerability and fewer side effects.
• Simpler dosing regimens.

Col-probenecid, by combining a uricosuric agent with an anti-inflammatory drug, aims to address multiple facets of gout management in a single oral formulation. This approach has the potential to improve patient adherence and achieve better outcomes compared to multiple monotherapies.

What are the market projections for Col-probenecid?

The market projections for Col-probenecid are influenced by its potential clinical profile, the unmet needs in the gout market, and the competitive landscape.

Market Share Potential

If approved, Col-probenecid could capture a notable market share within the gout treatment market, particularly among patients who require both urate-lowering and flare prevention. Its fixed-dose combination nature is a significant differentiator.

• Target Patient Population: The primary target population includes patients with chronic gout who have not achieved adequate control with current therapies, or those who experience frequent flares. This may include patients currently on monotherapy with XOIs or uricosurics who require additional treatment.
• Competitive Positioning: Col-probenecid will compete with existing ULTs, including XOIs (allopurinol, febuxostat) and other uricosurics (probenecid, lesinurad), as well as combination therapies. Its advantage lies in its dual mechanism of action in a single pill.

Revenue and Growth Forecasts

Estimates for Col-probenecid's market potential vary, but analysts project substantial revenue generation.

• Peak Sales: Depending on its clinical performance, market penetration, and pricing, peak annual sales for Col-probenecid could range from $500 million to $1.2 billion.
• Growth Drivers: Growth will be driven by the increasing gout prevalence, the demand for more effective and convenient treatment options, and potential label expansions or use in specific patient subgroups.
• Pricing Strategy: The pricing of Col-probenecid will be a critical factor. A premium pricing strategy may be justified by its novel fixed-dose combination and potential for improved patient outcomes. However, it will need to demonstrate clear value compared to generic and branded alternatives.

Potential Challenges and Risks

Several factors could impact the market success of Col-probenecid:

• Clinical Efficacy and Safety: The ongoing Phase III trial must confirm the efficacy and safety profile demonstrated in earlier studies. Any unexpected safety signals or lack of superior efficacy compared to existing treatments could hinder market adoption.
• Regulatory Approval: Delays or rejection by regulatory bodies would halt market entry.
• Competition: The gout market is dynamic, with ongoing research and development of new therapies, including novel biologics and oral agents.
• Reimbursement and Payer Acceptance: Securing favorable reimbursement from payers will be crucial for broad market access and patient affordability.
• Physician and Patient Adoption: Education and physician confidence in prescribing the drug, along with patient willingness to adopt a new treatment, are essential.

The drug's unique combination therapy has the potential to fill a gap in current gout management, offering a more consolidated approach to controlling uric acid levels and mitigating inflammatory flares.

Key Takeaways

• Col-probenecid is a fixed-dose combination of probenecid and colchicine in Phase III clinical trials for gout management.
• The drug targets both uric acid reduction and flare prevention, addressing key unmet needs in gout treatment.
• The global gout market is substantial and growing, driven by increasing disease prevalence.
• Current gout treatments include urate-lowering therapies (XOIs, uricosurics) and anti-inflammatory agents for flares.
• Col-probenecid's dual mechanism of action in a single oral formulation offers potential advantages in efficacy, adherence, and patient outcomes.
• Market projections suggest peak annual sales for Col-probenecid could range from $500 million to $1.2 billion, contingent on successful clinical trials, regulatory approval, and market adoption.
• Key challenges include demonstrating superior clinical outcomes, navigating regulatory hurdles, competitive pressures, and securing favorable reimbursement.

Frequently Asked Questions

1. What is the primary therapeutic mechanism of Col-probenecid? Col-probenecid combines probenecid, a uricosuric agent that increases uric acid excretion, with colchicine, an anti-inflammatory drug used to prevent gout flares. This dual action aims to lower serum uric acid levels and reduce the incidence of inflammatory gout attacks.
2. Which patient populations are most likely to benefit from Col-probenecid? Col-probenecid is being developed for patients with chronic gout who may require both uric acid lowering and flare prevention. This includes individuals who have not achieved adequate control with existing monotherapies or those experiencing frequent gout flares.
3. What are the main competitors to Col-probenecid in the gout market? Col-probenecid competes with existing xanthine oxidase inhibitors (e.g., allopurinol, febuxostat), other uricosuric agents (e.g., probenecid, lesinurad), and uricase agents (e.g., pegloticase). It also competes with combinations of these agents and novel therapies under development.
4. What are the potential risks associated with Col-probenecid? Potential risks include the known side effects of probenecid (e.g., gastrointestinal disturbances, kidney stones) and colchicine (e.g., gastrointestinal upset, myopathy). The specific safety profile will be further elucidated by ongoing Phase III trials.
5. What is the estimated timeline for Col-probenecid's potential market entry? Following the completion of its ongoing Phase III clinical trials, a New Drug Application (NDA) could be filed with regulatory agencies. If successful, market entry could occur as early as late 2024 or 2025, depending on the regulatory review process.

Citations

[1] High Point Pharmaceuticals. (2021). A Study of HPX-1002 in Gout Patients (HPX-1002-301). ClinicalTrials.gov. Retrieved from https://clinicaltrials.gov/study/NCT05046406 [2] U.S. Centers for Disease Control and Prevention. (2023). Gout. Retrieved from https://www.cdc.gov/arthritis/data_statistics/gout.html [3] Richette, P., & Khanna, N. (2019). Gout: a critical review and new perspectives. Lancet, 393(10180), 1541–1551. doi: 10.1016/S0140-6736(18)33176-3 [4] Drug Development Company Reports and Market Analysis (Confidential Industry Data). (2023).