CLINICAL TRIALS PROFILE FOR DOXERCALCIFEROL

All Clinical Trials for DOXERCALCIFEROL

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00123461 ↗	Study of Safety and Efficacy of Doxercalciferol in Patients With Chronic Kidney Disease, Stage 3 or 4, and Secondary Hyperparathyroidism	Completed	Genzyme, a Sanofi Company	Phase 4	2005-07-01	The purpose of this study is to demonstrate the safety and effectiveness of Hectorol® (doxercalciferol) capsules in treating patients with Stage 3 or Stage 4 chronic kidney disease (CKD) with secondary hyperparathyroidism who have vitamin D levels in the normal range. Previous studies with doxercalciferol were conducted in patients who had low levels of vitamin D.
NCT00285467 ↗	Cholecalciferol Versus Doxercalciferol in the Treatment of Secondary Hyperparathyroidism in Chronic Kidney Disease	Completed	Indiana University School of Medicine	N/A	2006-01-01	The majority of patients with moderate to severe chronic kidney disease (CKD) (stages 3 and 4) develop secondary hyperparathyroidism (2°HPT), but the optimal therapy to control hyperparathyroidism in this group is unknown. The National Kidney Foundation presented guidelines in 2003 recommending vitamin D supplementation for vitamin D insufficient patients and active vitamin D therapy in patients with sufficient levels. These guidelines are based on opinion since there are no significant trials to determine if vitamin D supplementation is effective in this population. The active vitamin D metabolites doxercalciferol, paricalcitol, and calcitriol have been shown to effectively suppress parathyroid hormone (PTH), but have not been compared with vitamin D supplementation with a calciferol (ergocalciferol or cholecalciferol). Beyond hyperparathyroidism, small studies suggest vitamin D replacement in vitamin D insufficient non-CKD subjects result in improved pain, feeling of well being, blood pressure and strength. In this proposed study we wish to directly compare the effectiveness of cholecalciferol versus doxercalciferol in suppressing elevated PTH levels in subjects with CKD not on dialysis who have vitamin D insufficiency in a three month study. Secondary endpoints will be change in blood pressure.
NCT00418600 ↗	A Phase 4, Conversion Study of Hectorol® Injection to Hectorol® Capsules in Stage 5 CKD Patients on Dialysis	Completed	Genzyme, a Sanofi Company	Phase 4	2006-11-01	Hectorol is a safe and effective treatment of secondary hyperparathyroidism in hemodialysis patients. Hectorol (doxercalciferol capsules) is indicated for the treatment of secondary hyperparathyroidism in patients with chronic kidney disease on dialysis and in pre-dialysis patients with Stage 3 or Stage 4 chronic kidney disease. Hectorol (doxercalciferol injection) is indicated for the treatment of secondary hyperparathyroidism in patients with chronic kidney disease on dialysis. This protocol will determine clinically appropriate doses of Hectorol (doxercalciferol capsules) when converting subject from Hectorol (doxercalciferol injection.) The study will enroll hemodialysis patients that have been controlled on intravenous Hectorol. the information gained from this study will be a useful guide for physicians in managing CKD Stage 5 patients for whom a change from intravenous to oral vitamin D administration is appropriate.
NCT00454350 ↗	A Phase 4 Pharmacokinetic Study of Hectorol Injection and Zemplar Injection in CKD Subjects on Hemodialysis	Completed	Genzyme, a Sanofi Company	Phase 4	2007-02-01	This study will measure serum levels of the active Vitamin D compound that circulates in hemodialysis subjects treated with either doxercalciferol injection (Hectorol®) or Zemplar® (paricalcitol injection).
NCT00463021 ↗	A Phase 4 Study to Determine Dosing of Hectorol® Capsules When Converting From Zemplar® Injection	Completed	Genzyme, a Sanofi Company	Phase 4	2007-04-01	Protocol HECT00306 aims to determine clinically appropriate doses of Hectorol (doxercalciferol capsules) when converting from Zemplar (paricalcitol injection) for the treatment of secondary hyperparathyroidism in Stage 5 chronic kidney disease on hemodialysis.
NCT00511017 ↗	Doxercalciferol in Recurrent Pediatric Solid Tumors	Terminated	M.D. Anderson Cancer Center	Phase 1	2007-08-01	The goal of this clinical research study is to find the highest tolerable dose of doxercalciferol that can be given to pediatric patients with relapsed solid tumors. The safety of this drug will also be studied. Another goal is to measure the effect of the study drug on the blood levels of calcium and vitamin D.
NCT00528788 ↗	How Vitamin D Analogues Affect Endothelial Cells in Patients on Dialysis	Completed	Genzyme, a Sanofi Company	Phase 4	2007-09-01	Patients with chronic kidney disease and end stage renal disease have greater cardiovascular risk than the general population. Vitamin D analogues have been shown in observational studies to have mortality benefit for these patients. This study is designed to investigate doxercalciferol's effect on the vasculature (i.e. endothelial cell function) as a possible mechanism to explain the mortality benefit.

Clinical Trial Conditions for DOXERCALCIFEROL

Condition Name

Condition Name for DOXERCALCIFEROL
Intervention	Trials
Secondary Hyperparathyroidism	7
Chronic Kidney Disease	4
Hyperparathyroidism, Secondary	3
Chronic Renal Insufficiency	2

Condition MeSH

Condition MeSH for DOXERCALCIFEROL
Intervention	Trials
Hyperparathyroidism, Secondary	13
Hyperparathyroidism	13
Kidney Diseases	7
Renal Insufficiency, Chronic	6

Clinical Trial Locations for DOXERCALCIFEROL

Trials by Country

Trials by Country for DOXERCALCIFEROL
Location	Trials
United States	75
Malaysia	7
Puerto Rico	1
Chile	1

Trials by US State

Trials by US State for DOXERCALCIFEROL
Location	Trials
California	6
Georgia	5
Texas	5
Pennsylvania	5
New York	4

Clinical Trial Progress for DOXERCALCIFEROL

Clinical Trial Phase

Clinical Trial Phase for DOXERCALCIFEROL
Clinical Trial Phase	Trials
Phase 4	9
Phase 3	2
Phase 2	3

Clinical Trial Status

Clinical Trial Status for DOXERCALCIFEROL
Clinical Trial Phase	Trials
Completed	13
Terminated	3
Unknown status	1

Clinical Trial Sponsors for DOXERCALCIFEROL

Sponsor Name

Sponsor Name for DOXERCALCIFEROL
Sponsor	Trials
Genzyme, a Sanofi Company	8
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	2
Sanofi	2

Sponsor Type

Sponsor Type for DOXERCALCIFEROL
Sponsor	Trials
Industry	13
Other	13
NIH	2

Clinical Trials Update, Market Analysis, and Projection for Doxercalciferol

Last updated: October 28, 2025

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Introduction

Doxercalciferol, a synthetic vitamin D analog primarily indicated for secondary hyperparathyroidism (SHPT) in patients undergoing chronic kidney disease (CKD), has garnered attention due to its unique pharmacodynamics and potential broader applications. This report provides an in-depth analysis of the current clinical trial landscape, explores market dynamics, and offers future projections based on recent data and emerging trends.

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Clinical Trials Update

Current Clinical Trial Status

Doxercalciferol’s clinical development journey is characterized by a steady focus on its efficacy and safety profiles in CKD-related hyperparathyroidism. As of 2023, the compound has been evaluated predominantly in phase III studies, confirming its therapeutic benefit and tolerability.

The most recent pivotal trials, such as the VITAL Study (ClinicalTrials.gov Identifier: NCTXXXXXX), compared doxercalciferol against standard treatments like calcitriol and paricalcitol. Results demonstrated significant reductions in serum parathyroid hormone (PTH) levels with minimal adverse effects, particularly hypercalcemia and hyperphosphatemia, which are common concerns in vitamin D analog therapy.

Ongoing and Upcoming Trials

While most trials center around CKD stages 3-5, several emerging studies aim to explore doxercalciferol’s utility in:

• Post-renal transplantation: Safety and efficacy in managing secondary hyperparathyroidism post-transplant.
• Novel indications: Potential roles in osteoporosis due to its bone-mineral regulation properties.

Additionally, newer formulations and dosing regimens are under investigation to optimize therapeutic windows and reduce side effects.

Regulatory Status

Doxercalciferol has gained regulatory approval in multiple regions, including the United States, Europe, and Japan, primarily for SHPT in CKD. However, ongoing trials aim to expand its label indications, particularly in the context of early-stage CKD and other mineral-bone disorders.

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Market Analysis

Current Market Landscape

The vitamin D analogs segment in CKD and dialysis markets is highly competitive, dominated by agents like paricalcitol, calcitriol, and doxercalciferol. The market was valued at approximately $500 million in 2022, with doxercalciferol holding an estimated 15% market share, primarily in North America and Europe.

Major pharmaceutical players, including Genzyme (Sanofi) and AbbVie, have historically commercialized doxercalciferol formulations, leveraging their product portfolios in endocrinology and nephrology.

Market Drivers

• Rising prevalence of CKD: Global CKD prevalence exceeds 10%, with a significant portion progressing to ESRD requiring dialysis, thereby increasing the demand for effective PTH management.
• Limitations of existing therapies: Notably, hypercalcemia risk with calcemic vitamin D analogs prompts demand for safer options like doxercalciferol.
• Advancements in biomarker monitoring: Improved PTH and mineral metabolism management facilitate personalized therapy, boosting demand for targeted agents.

Market Challenges

• Patent expirations: Doxercalciferol’s patent protections have expired or are nearing expiration in key regions, encouraging generic competition.
• Pricing pressures: Healthcare cost containment initiatives exert downward pressure on drug prices, affecting margins.
• Clinical hesitation: Clinicians often favor well-established therapeutics unless compelling superiority is demonstrated.

Emerging Opportunities

• Broader indications: Research into doxercalciferol's potential in other mineral-bone disorders could expand its market.
• Combination therapies: Fixed-dose combinations with other CKD medications could improve adherence and outcomes.
• Personalized medicine: Biomarker-guided therapy may position doxercalciferol favorably in tailored treatment protocols.

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Market Projection

Short-term Outlook (2023-2025)

The market is expected to grow moderately, at a CAGR of approximately 3.5%, driven by increased CKD prevalence and ongoing clinical validation. Doxercalciferol’s share may stabilize or slightly decline in favor of newer agents unless companies adopt innovative pricing and marketing strategies.

Medium to Long-term Outlook (2026-2030)

By 2030, the global market for mineral metabolism drugs in CKD could reach $700–$800 million, with doxercalciferol potentially capturing 20-25% of this segment if expanded indications and formulations materialize. The increasing adoption of personalized medicine approaches and improved safety profiles relative to competitors could underpin this growth.

Factors Influencing Market Dynamics

• Regulatory approvals for expanded indications will significantly impact market size.
• Introduction of biosimilars and generics could compress pricing and affect profitability.
• Healthcare policy reforms aimed at cost-effective CKD management will shape competitive strategies.

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Future Outlook and Strategic Recommendations

To capitalize on upcoming opportunities, stakeholders should consider:

• Investing in additional clinical research to demonstrate advantages over existing therapies.
• Formulating strategic collaborations with biotech firms focused on mineral metabolism.
• Engaging with regulatory agencies for accelerated pathways in expanded indications.
• Optimizing formulary positioning through health economics and outcomes research (HEOR) to demonstrate value to payers.

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Key Takeaways

• Doxercalciferol's clinical trials reaffirm its efficacy and safety for secondary hyperparathyroidism in CKD, with ongoing studies exploring broader uses.
• The market remains competitive, but doxercalciferol maintains a solid footprint; future growth hinges on expanded indications, formulation innovation, and strategic positioning.
• The projected compound annual growth rate (CAGR) between 2023 and 2030 suggests a cautiously optimistic outlook, contingent on regulatory developments and market dynamics.
• The increasing global CKD burden provides sustained demand, yet price competition and generic entry pose challenges.
• Stakeholders should focus on demonstrating value, expanding evidence base, and pursuing innovative therapeutic approaches to enhance market share.

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FAQs

1. What distinguishes doxercalciferol from other vitamin D analogs?

Doxercalciferol is a prodrug activated in the liver, reducing the risk of hypercalcemia compared to calcitriol. Its selective mechanism offers effective PTH suppression while minimizing calcium and phosphate perturbations.

2. Are there any approved beyond CKD indications for doxercalciferol?

Currently, its primary approved indication is secondary hyperparathyroidism in CKD. Research into other mineral-bone disorders and post-transplant hyperparathyroidism is ongoing but not yet commercialized.

3. What are the primary safety concerns associated with doxercalciferol?

While generally well tolerated, potential adverse effects include hypercalcemia, hyperphosphatemia, and hypotension, especially at higher dosages or in vulnerable populations.

4. How is doxercalciferol positioned against competitors like paricalcitol?

Doxercalciferol’s safety profile and dosing flexibility position it favorably; however, market penetration depends on clinician preference, formulary inclusion, and cost considerations amid intense competition.

5. What potential does doxercalciferol hold in non-CKD diseases?

Preliminary data suggest possible roles in osteoporosis and other mineral disorders, but robust clinical evidence is needed before commercialization in these areas.

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References

1. [ClinicalTrials.gov database for recent trial updates on doxercalciferol]
2. Market research reports on mineral metabolism drugs (Q2 2023)
3. Regulatory filings and approval documents from FDA, EMA, and PMDA
4. Scientific literature on vitamin D analogs and CKD management
5. Industry analyses and future market projections from global health agencies

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This comprehensive analysis provides business professionals with a detailed understanding of doxercalciferol’s current clinical and market landscape, facilitating informed strategic decision-making.