CLINICAL TRIALS PROFILE FOR CALCITRIOL

505(b)(2) Clinical Trials for Calcitriol

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT00055263 ↗	A New Formulation of Calcitriol (DN-101) in Patients With Advanced Malignancies	Completed	Novacea	Phase 1	2002-03-01	The purposes of this study are to: - Test the safety of DN-101 in patients with advanced malignancies - Understand how fast the body absorbs, processes, and eliminates DN-101 - Determine the highest dose of DN-101 that is well tolerated by cancer patients - Learn how fast the body absorbs, processes, and eliminates DN-101 compared to the approved product

All Clinical Trials for Calcitriol

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000412 ↗	Osteoporosis Prevention After Heart Transplant	Completed	Merck Sharp & Dohme Corp.	Phase 3	1997-09-01	During the first year after a heart transplant, people often rapidly lose bone from their spine and hips. About 35 percent of people who receive heart transplants will suffer broken bones during the first year after transplantation. This study will compare the safety and effectiveness of the drug alendronate (Fosamax) and the active form of vitamin D (calcitriol) in preventing bone loss at the spine and hip after a heart transplant. In this study, people who have had a successful heart transplant will receive either active alendronate and a "dummy pill" instead of calcitriol, or active calcitriol and a dummy pill instead of alendronate for the first year after their transplant, starting within 1 month after transplant surgery. We will measure bone density in the hip and spine at the start of the study and after 6 and 12 months, and will also check for broken bones in the spine. This research should lead to ways of preventing this crippling form of osteoporosis.
NCT00000412 ↗	Osteoporosis Prevention After Heart Transplant	Completed	National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)	Phase 3	1997-09-01	During the first year after a heart transplant, people often rapidly lose bone from their spine and hips. About 35 percent of people who receive heart transplants will suffer broken bones during the first year after transplantation. This study will compare the safety and effectiveness of the drug alendronate (Fosamax) and the active form of vitamin D (calcitriol) in preventing bone loss at the spine and hip after a heart transplant. In this study, people who have had a successful heart transplant will receive either active alendronate and a "dummy pill" instead of calcitriol, or active calcitriol and a dummy pill instead of alendronate for the first year after their transplant, starting within 1 month after transplant surgery. We will measure bone density in the hip and spine at the start of the study and after 6 and 12 months, and will also check for broken bones in the spine. This research should lead to ways of preventing this crippling form of osteoporosis.
NCT00000412 ↗	Osteoporosis Prevention After Heart Transplant	Completed	Columbia University	Phase 3	1997-09-01	During the first year after a heart transplant, people often rapidly lose bone from their spine and hips. About 35 percent of people who receive heart transplants will suffer broken bones during the first year after transplantation. This study will compare the safety and effectiveness of the drug alendronate (Fosamax) and the active form of vitamin D (calcitriol) in preventing bone loss at the spine and hip after a heart transplant. In this study, people who have had a successful heart transplant will receive either active alendronate and a "dummy pill" instead of calcitriol, or active calcitriol and a dummy pill instead of alendronate for the first year after their transplant, starting within 1 month after transplant surgery. We will measure bone density in the hip and spine at the start of the study and after 6 and 12 months, and will also check for broken bones in the spine. This research should lead to ways of preventing this crippling form of osteoporosis.

Clinical Trial Conditions for Calcitriol

Condition Name

Condition Name for Calcitriol
Intervention	Trials
Vitamin D Deficiency	12
Prostate Cancer	10
Chronic Kidney Disease	9

Condition MeSH

Condition MeSH for Calcitriol
Intervention	Trials
Kidney Diseases	32
Renal Insufficiency, Chronic	22
Hyperparathyroidism	16

Clinical Trial Locations for Calcitriol

Trials by Country

Trials by Country for Calcitriol
Location	Trials
United States	264
Canada	24
China	12
Italy	7
Malaysia	7

Trials by US State

Trials by US State for Calcitriol
Location	Trials
New York	32
California	18
Texas	15
Pennsylvania	12
Oregon	11

Clinical Trial Progress for Calcitriol

Clinical Trial Phase

Clinical Trial Phase for Calcitriol
Clinical Trial Phase	Trials
PHASE4	1
PHASE2	1
PHASE1	2

Clinical Trial Status

Clinical Trial Status for Calcitriol
Clinical Trial Phase	Trials
Completed	94
Unknown status	28
Terminated	21

Clinical Trial Sponsors for Calcitriol

Sponsor Name

Sponsor Name for Calcitriol
Sponsor	Trials
National Cancer Institute (NCI)	12
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	9
Novacea	8

Sponsor Type

Sponsor Type for Calcitriol
Sponsor	Trials
Other	182
Industry	55
NIH	34

Calcitriol: Clinical Trials, Market Outlook, and Future Projections

Last updated: October 27, 2025

---

Introduction

Calcitriol, the active form of vitamin D3, is widely used in the management of various medical conditions, notably chronic kidney disease (CKD), osteoporosis, and hypocalcemia. Derived from cholecalciferol (vitamin D3), calcitriol exerts its effects by regulating calcium and phosphate homeostasis, essential for bone health and metabolic stability. Given its clinical significance, ongoing research, evolving market dynamics, and strategic development efforts are critical to understanding calcitriol’s future trajectory.

---

Clinical Trials Landscape for Calcitriol

Current State of Clinical Trials

Recent years have seen a surge in clinical investigations exploring calcitriol's expanded therapeutic potential, particularly in renal and oncological domains. Data from clinicaltrials.gov indicates over 50 active or completed studies investigating calcitriol's efficacy and safety across diverse indications. Notably, these include:

• Chronic Kidney Disease (CKD): Multiple trials focus on calcitriol’s role in delaying renal disease progression, mitigating secondary hyperparathyroidism, and reducing fracture risk. For instance, NCT03925802 examined high-dose calcitriol's impact on mineral metabolism in CKD stage 3–4 patients.

• Oncology: Several Phase II trials assess calcitriol’s potential to enhance cancer therapy outcomes, capitalizing on its antiproliferative and differentiation-inducing properties. An example includes NCT02975856, exploring calcitriol as an adjunct in prostate cancer.

• Bone and Metabolic Disorders: Trials continue evaluating calcitriol's role in osteoporosis, especially in patients intolerant to bisphosphonates, with some studies investigating novel formulations for improved bioavailability.

Emerging Research Directions

Innovations are focusing on synthetic analogues of calcitriol with enhanced stability and reduced hypercalcemia risk, such as paricalcitol and doxercalciferol, to broaden therapeutic windows. Additionally, research aims to elucidate molecular pathways involving calcitriol, seeking to optimize dosing strategies and mitigate adverse effects.

Safety and Efficacy Metrics

Key focus areas include hypercalcemia management, cardiovascular safety, and pharmacokinetic optimization. The results from ongoing trials will influence regulatory positions, dosing guidelines, and therapeutic indications.

---

Market Analysis

Market Size & Dynamics

The global calcitriol market was valued at approximately USD 250 million in 2022 and is projected to grow at a CAGR of 6–8% over the next five years, driven by increasing prevalence of CKD, osteoporosis, and calcium deficiency disorders. The Asia-Pacific region is experiencing significant growth owing to rising health awareness and healthcare infrastructure expansion.

Key Market Segments

• Pharmaceuticals & Biotech: Major players include Sanofi, Abbott, and Teva. Several biosimilars and generics are entering markets, intensifying competition and driving pricing strategies.

• Formulations & Delivery Systems: Oral capsules remain dominant; however, injectable and novel transdermal systems are emerging, offering alternative administration routes and enhanced compliance.

Regulatory & Reimbursement Environment

Enhanced emphasis on safety profiles, especially hypercalcemia risks, influences regulatory approvals. Payer policies increasingly favor cost-effective therapies, prompting innovation in formulation and optimized dosing protocols. The burgeoning geriatric population further amplifies demand for calcitriol-based therapies.

Competitive Landscape

Patent expirations for some formulations have facilitated a proliferation of generics, reducing costs but intensifying competition. Innovators are focusing on developing analogues with improved safety profiles, aiming to capture niche markets, especially in oncology and rare metabolic conditions.

---

Future Market Projections

Growth Drivers

• Increased Disease Burden: Rising incidence of CKD, osteoporosis, and vitamin D deficiency in aging populations sustains demand.
• Innovative Formulations: Advances in drug delivery systems enhance patient adherence and safety, fostering market expansion.
• Expanded Therapeutic Claims: Clinical trials exploring anti-cancer and immunomodulatory roles of calcitriol may open new markets.

Challenges & Opportunities

• Safety Concerns: Hypercalcemia remains a significant barrier; addressing this through novel analogues and precise dosing is critical.
• Regulatory Hurdles: Demonstrating clear benefit-risk profiles influences approval prospects.
• Precision Medicine: Personalized dosing based on genetic and metabolic markers offers future growth avenues.

Market Outlook (2023–2030)

By 2030, the calcitriol market is anticipated to reach USD 400–500 million, with a compound annual growth rate supportive of sustained expansion. Key opportunities lie in developing targeted formulations, expanding indications, and leveraging digital health tools for monitoring therapy.

---

Strategic Insights for Industry Stakeholders

• Invest in R&D: Focus on developing analogues with improved safety profiles and novel delivery mechanisms.
• Navigate Regulatory Pathways: Engage proactively with regulators to streamline approval processes for new indications.
• Market Diversification: Explore emerging niches such as oncology adjunct therapy and personalized medicine.
• Partnerships & Licensing: Collaborate with biotech firms to co-develop innovative formulations and expand geographic reach.
• Patient-Centric Approaches: Emphasize tolerability and compliance to enhance therapeutic outcomes and market penetration.

---

Key Takeaways

• The clinical trial pipeline for calcitriol reflects expanding therapeutic interests beyond traditional use, especially in CKD and oncology.
• Market growth is driven by an aging population, rising disease prevalence, and innovations in drug formulation and delivery.
• Safety concerns, primarily hypercalcemia, necessitate continued development of safer analogues and personalized dosing strategies.
• Patent expirations and biosimilar entry are reshaping competitive dynamics, prompting innovation and pricing strategies.
• Strategic investments in research, regulatory engagement, and patient-centric formulations are vital for capturing emerging opportunities.

---

FAQs

1. What are the primary clinical indications for calcitriol today?
   Calcitriol is primarily used to manage secondary hyperparathyroidism in CKD, osteoporosis, and hypocalcemia, especially in patients with impaired renal function.

2. Are there ongoing clinical trials exploring calcitriol’s use in cancer?
   Yes, several trials are investigating calcitriol as an adjunctive therapy in prostate, breast, and other cancers, focusing on its potential antiproliferative and differentiation-inducing effects.

3. What safety concerns are associated with calcitriol therapy?
   Hypercalcemia is the main risk, which can lead to vascular calcification and renal issues. Monitoring serum calcium levels and developing safer analogues are ongoing priorities.

4. How is the market for calcitriol expected to evolve in the next decade?
   The market is projected to grow at a CAGR of approximately 6–8%, reaching USD 400–500 million by 2030, driven by new formulations and expanded indications.

5. What role do biosimilars and generics play in the calcitriol market?
   They increase accessibility and reduce costs but also intensify competition. This encourages innovation in formulations and therapeutic uses to maintain competitive advantage.

---

References

[1] ClinicalTrials.gov. Database of ongoing calcitriol studies.
[2] MarketResearchFuture. Global vitamin D analogs market report, 2022.
[3] Global Data. Therapeutic applications and market projections for calcitriol, 2022.
[4] Journal of Clinical Endocrinology & Metabolism. Safety profiles of calcitriol and analogues, 2021.