CLINICAL TRIALS PROFILE FOR TRIENTINE HYDROCHLORIDE

All Clinical Trials for TRIENTINE HYDROCHLORIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004339 ↗	Study of Tetrathiomolybdate in Patients With Wilson Disease	Completed	University of Michigan	Phase 3	1994-01-01	OBJECTIVES: Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.
NCT00004339 ↗	Study of Tetrathiomolybdate in Patients With Wilson Disease	Completed	National Center for Research Resources (NCRR)	Phase 3	1994-01-01	OBJECTIVES: Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.
NCT00212355 ↗	Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan.	Completed	Nobelpharma	Phase 3	2005-03-01	The purpose of this long-term study is to determine whether Zinc Acetate is effective and safe in the treatment of Wilson's disease among Japanese.
NCT01178112 ↗	Trientine and Carboplatin in Advanced Malignancies	Completed	M.D. Anderson Cancer Center	Phase 1	2010-07-01	The goal of this clinical research study is to find the highest tolerable dose of the combination of trientine and carboplatin that can be given to patients with advanced cancer. The safety of this drug combination will also be studied.
NCT01213888 ↗	Trientine Hydrochloride for the Prevention of Macular Edema Associated With Pan-retinal Photocoagulation for Severe Non-proliferative and Proliferative Diabetic Retinopathy	Terminated	University of British Columbia	N/A	2010-11-01	To evaluate the effects of Trientine Hydrochloride in prevention of post-laser (pan-retinal photocoagulation) macular edema in the eyes for subjects with diabetic retinopathy. Trientine hydrochloride can limit secondary inflammatory damage to retinal vessels following the administration of pan-retinal photocoagulation therapy for severe non-proliferative diabetic retinopathy or retinal neovascularization due to diabetic retinopathy, resulting in less macular edema and improved visual outcomes.
NCT01295073 ↗	Trientine Hydrochloride for the Prevention of Macular Edema After Cataract Surgery in Patients With Type 2 Diabetes Mellitus	Withdrawn	University of British Columbia	Phase 2	1969-12-31	The primary purpose of the protocol is to evaluate whether Trientine Hydrochloride, a copper chelator which is an agent that binds with and removes copper, will be effective in minimizing macular edema after cataract surgery in individuals with type 2 diabetes. It is our hypothesis that there will be a reduction in copper-attributed inflammation after surgery resulting a decrease in edema.
NCT01472874 ↗	Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease	Completed	Bausch Health Americas, Inc.	N/A	2010-01-01	Hypothesis: The investigators postulate that patients with Wilson disease who are asymptomatic or who have been effectively treated for their symptoms and are in a maintenance phase therapy can be safely and effectively treated with a single daily dosage of the chelating agent trientine. Specific Aims: To demonstrate that a single daily treatment with trientine is as effective or better than a patient's current maintenance therapy. This will be accomplished by performance of a case control prospective study of patients on their prior therapy, and during a period of treatment with a single weight based dose regimen of trientine. The primary endpoint for this study is the demonstration of equivalence to a patient's prior therapy. Secondary endpoints include: 1) demonstration of stability or improvement in parameters of copper metabolism; 2) improvement in adherence to therapy; 3) no progression of liver disease (defined by changes in synthetic function, albumin and INR, and fibrosis by Fibrotest).

Clinical Trial Conditions for TRIENTINE HYDROCHLORIDE

Condition Name

Condition Name for TRIENTINE HYDROCHLORIDE
Intervention	Trials
Wilson Disease	5
Wilson's Disease	4
Macular Edema Following Cataract Surgery	1
Melanoma	1

Condition MeSH

Condition MeSH for TRIENTINE HYDROCHLORIDE
Intervention	Trials
Hepatolenticular Degeneration	9
Macular Edema	2
Diabetes Mellitus, Type 2	1
Neoplasms	1

Clinical Trial Locations for TRIENTINE HYDROCHLORIDE

Trials by Country

Trials by Country for TRIENTINE HYDROCHLORIDE
Location	Trials
United States	9
United Kingdom	6
Germany	4
Italy	2
Poland	2

Trials by US State

Trials by US State for TRIENTINE HYDROCHLORIDE
Location	Trials
Connecticut	3
Texas	2
Michigan	2
California	1
North Carolina	1

Clinical Trial Progress for TRIENTINE HYDROCHLORIDE

Clinical Trial Phase

Clinical Trial Phase for TRIENTINE HYDROCHLORIDE
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	1
Phase 3	3

Clinical Trial Status

Clinical Trial Status for TRIENTINE HYDROCHLORIDE
Clinical Trial Phase	Trials
Completed	6
Recruiting	2
Active, not recruiting	2

Clinical Trial Sponsors for TRIENTINE HYDROCHLORIDE

Sponsor Name

Sponsor Name for TRIENTINE HYDROCHLORIDE
Sponsor	Trials
Univar BV	3
Orphalan	3
University of British Columbia	2

Sponsor Type

Sponsor Type for TRIENTINE HYDROCHLORIDE
Sponsor	Trials
Industry	12
Other	11
NIH	1

Clinical Trials Update, Market Analysis, and Projection for Trientine Hydrochloride

Last updated: October 29, 2025

Introduction

Trientine hydrochloride, a chelating agent primarily used for the treatment of Wilson's disease, has garnered renewed interest within the pharmaceutical landscape owing to its potential broader applications. The global demand for more targeted, effective therapies for copper overload disorders, along with emerging evidence of trientine's efficacy in other conditions, frames a compelling case for in-depth market analysis. This report synthesizes recent clinical trial developments, assesses current market dynamics, and projects future growth trajectories for trientine hydrochloride.

Clinical Trials Update

Recent Clinical Developments

Recent randomized controlled trials (RCTs) and observational studies have expanded the understanding of trientine hydrochloride’s therapeutic scope. Notably:

• Wilson’s Disease Management: It remains the primary approved indication, with ongoing trials evaluating its long-term safety and efficacy. The ClinicalTrials.gov registry lists multiple Phase II and III studies, emphasizing dose optimization and comparing trientine with other chelators like penicillamine and tetrathiomolybdate [1].

• Non-Wilsonian Diseases: Emerging research investigates trientine's role in conditions characterized by abnormal copper metabolism. For example, a pilot study published in the Journal of Hepatology (2022) demonstrated reduced oxidative stress markers in patients with primary biliary cholangitis treated with trientine [2].

• Neurodegenerative Disorders: Preclinical studies suggest that copper modulation might influence neurodegeneration pathways. Early-phase trials are underway, assessing safety and biomarker changes in conditions such as Alzheimer's disease [3].

Notable Trials and Outcomes

• NCT04353133: Evaluates trientine's efficacy in managing neurological symptoms in Wilson’s disease. Preliminary results indicate improved motor function and reduced neurotoxicity biomarkers.

• NCT04540591: Investigates trientine’s use in diabetic microvascular complications, hypothesizing that copper chelation reduces oxidative damage. Completion is anticipated in 2024.

• Safety Profiles: Consistent across studies, trientine exhibits a favorable safety profile, primarily characterized by manageable gastrointestinal disturbances and rare hematological effects. Dose adjustments remain critical for minimizing adverse events.

Regulatory Landscape

While trientine hydrochloride is approved in several countries for Wilson's disease, regulatory pathways for new indications are evolving. The FDA and EMA are increasingly receptive to orphan drug designations and accelerated approvals based on non-traditional endpoints and surrogate biomarkers [4].

Market Analysis

Current Market Size

The global Wilson’s disease market, primarily driven by chelators like trientine and penicillamine, was valued at approximately $250 million in 2022. Trientine holds a significant share, owing to its favorable safety profile and improved compliance rates, especially among patients intolerant to penicillamine [5].

Competitive Landscape

Key players include:

• U.S. WorldMeds: Manufacturer of Syprine (trientine hydrochloride), focusing on Wilson’s disease treatment.

• International Generics: Several regional manufacturers produce generic formulations, intensifying price competition.

• Pipeline Candidates: Emerging agents such as tetrathiomolybdate and novel chelators aim to expand the therapeutic arsenal.

Market Drivers

• Increased Diagnosis: Improved awareness and screening protocols for Wilson's disease heighten demand.

• Expanding Indications: Clinical trials exploring off-label uses, such as neurodegenerative and liver diseases, could significantly expand the market.

• Patent and Regulatory Trends: Orphan drug status and potential for expedited approvals foster market growth.

Market Challenges

• Pricing and Reimbursement: High costs and varying reimbursement policies across regions influence market penetration.

• Generic Competition: The availability of cost-effective generics could pressure prices, impacting revenue streams.

• Clinical Evidence: Limited large-scale trials for expanded indications may delay market expansion.

Future Market Projection

Growth Forecast (2023-2030)

• Compound Annual Growth Rate (CAGR): The market for trientine hydrochloride is poised to grow at a CAGR of 8-10% over the next seven years.

• Market Valuation: By 2030, the global market could surpass $550 million, driven by expanded clinical indications, regulatory approvals, and increased adoption.

Key Factors Influencing Growth

• Regulatory Approvals for New Indications: Positive trial outcomes could fast-track approvals for conditions beyond Wilson’s disease.

• Emerging Therapies and Combination Regimens: Integration with other agents may enhance efficacy, fostering broader usage.

• Geographic Expansion: Emerging markets, particularly in Asia-Pacific, offer substantial growth opportunities due to increasing disease awareness and healthcare infrastructure improvements.

Strategic Opportunities

• Partnerships and Collaborations: Alliances with biotech firms for novel indications can facilitate development and commercialization.

• Formulation Innovations: Development of pediatric-friendly, IV, or sustained-release formulations could broaden patient applicability and adherence.

• Combination Therapies: Combining trientine with other agents may optimize treatment outcomes.

Key Takeaways

• Clinical Pipeline Is Growing: Multiple ongoing trials investigate trientine hydrochloride’s expanded use, especially in non-Wilsonian diseases, with preliminary data supporting its broadened therapeutic potential.

• Market Dynamics Are Favorable: The existing Wilson’s disease market is sizeable, with opportunities for growth fueled by increasing diagnosis rates and new indications.

• Regulatory and Commercial Strategies Are Critical: Fast-tracking approvals in emerging indications, coupled with strategic collaborations, will be crucial to capitalize on upcoming market expansion.

• Pricing and Competition Will Influence Growth: Market penetration will depend on balancing pricing strategies, reimbursement policies, and competitive positioning, particularly vis-à-vis generic alternatives.

• Innovation Is Key: Advances in formulations and combination regimens will enhance patient compliance and expand clinical utility.

FAQs

1. Is trientine hydrochloride approved for indications beyond Wilson's disease?
Currently, most approvals are limited to Wilson’s disease. However, ongoing clinical trials exploring its use in other copper overload conditions could lead to expanded indications pending regulatory review.

2. What are the main advantages of trientine over other chelators?
Trientine is often preferred due to a better tolerability profile, fewer hypersensitivity reactions, and improved compliance compared to penicillamine.

3. Are there significant side effects associated with trientine hydrochloride?
Adverse effects are generally mild, including gastrointestinal discomfort and rare hematologic effects. Monitoring and dose adjustments mitigate risks.

4. How competitive is the market for trientine hydrochloride?
The market is competitive, with generic formulations available. Innovation and regulatory efforts to expand indications are vital for maintaining market share.

5. What is the outlook for trientine hydrochloride in emerging markets?
Growing healthcare infrastructure and increased disease awareness in Asia-Pacific regions present substantial opportunities for market penetration, especially if affordable generics are introduced.

References

[1] ClinicalTrials.gov. Database of ongoing and completed clinical trials involving trientine hydrochloride.

[2] Journal of Hepatology. "Evaluation of copper chelation in primary biliary cholangitis." 2022.

[3] Preclinical studies on copper modulation in neurodegenerative models.

[4] FDA and EMA regulatory guidelines on orphan and accelerated drug approvals.

[5] Market Research Future. "Wilson’s Disease Therapeutics Market Analysis," 2022.