CLINICAL TRIALS PROFILE FOR PENICILLAMINE

All Clinical Trials for penicillamine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00003751 ↗	Penicillamine, Low Copper Diet, and Radiation Therapy in Treating Patients With Glioblastoma	Completed	National Cancer Institute (NCI)	Phase 2	1999-03-01	RATIONALE: Penicillamine may stop the growth of glioblastomas by stopping blood flow to the tumor. A diet low in copper may interfere with the growth of brain tumor cells. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining these therapies may be effective in treating glioblastoma. PURPOSE: Phase II trial to study the effectiveness of penicillamine, a low copper diet, and radiation therapy in treating patients who have newly diagnosed glioblastoma.
NCT00003751 ↗	Penicillamine, Low Copper Diet, and Radiation Therapy in Treating Patients With Glioblastoma	Completed	Sidney Kimmel Comprehensive Cancer Center	Phase 2	1999-03-01	RATIONALE: Penicillamine may stop the growth of glioblastomas by stopping blood flow to the tumor. A diet low in copper may interfere with the growth of brain tumor cells. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining these therapies may be effective in treating glioblastoma. PURPOSE: Phase II trial to study the effectiveness of penicillamine, a low copper diet, and radiation therapy in treating patients who have newly diagnosed glioblastoma.
NCT00003751 ↗	Penicillamine, Low Copper Diet, and Radiation Therapy in Treating Patients With Glioblastoma	Completed	Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	Phase 2	1999-03-01	RATIONALE: Penicillamine may stop the growth of glioblastomas by stopping blood flow to the tumor. A diet low in copper may interfere with the growth of brain tumor cells. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining these therapies may be effective in treating glioblastoma. PURPOSE: Phase II trial to study the effectiveness of penicillamine, a low copper diet, and radiation therapy in treating patients who have newly diagnosed glioblastoma.
NCT00161148 ↗	Probiotics in Patients With Primary Sclerosing Cholangitis	Unknown status	UMC Utrecht	Phase 3	2005-01-01	PSC is a progressive liver disease without effective medical treatment. There is often co-existent ulcerative colitis. Probiotics (bacterial food supplements) have been shown to benefit patients with ulcerative colitis. In the current protocol potential beneficial effects of probiotics on liver biochemistry and liver related symptoms as pruritus are being assessed in 12 PSC patients in a randomized controlled cross over study (3 months probiotics, 1 one wash-out and 3 months placebo).
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.

Clinical Trial Conditions for penicillamine

Condition Name

Condition Name for penicillamine
Intervention	Trials
Wilson Disease	4
Wilson's Disease	2
Scleroderma	2
Vitamin D Deficiency	1

Condition MeSH

Condition MeSH for penicillamine
Intervention	Trials
Hepatolenticular Degeneration	6
Arthritis	2
Scleroderma, Systemic	2
Arthritis, Rheumatoid	2

Clinical Trial Locations for penicillamine

Trials by Country

Trials by Country for penicillamine
Location	Trials
United States	31
United Kingdom	4
China	3
Poland	3
Germany	3

Trials by US State

Trials by US State for penicillamine
Location	Trials
Connecticut	3
Texas	3
Pennsylvania	3
Georgia	2
Florida	2

Clinical Trial Progress for penicillamine

Clinical Trial Phase

Clinical Trial Phase for penicillamine
Clinical Trial Phase	Trials
Phase 4	3
Phase 3	4
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for penicillamine
Clinical Trial Phase	Trials
Completed	9
Recruiting	2
Active, not recruiting	2

Clinical Trial Sponsors for penicillamine

Sponsor Name

Sponsor Name for penicillamine
Sponsor	Trials
University of Pennsylvania	1
Ankara University	1
Sidney Kimmel Comprehensive Cancer Center	1

Sponsor Type

Sponsor Type for penicillamine
Sponsor	Trials
Industry	12
Other	11
NIH	2

Penicillamine Market Analysis and Financial Projection

Penicillamine: Clinical Trials, Market Analysis, and Projections

Introduction to Penicillamine

Penicillamine, also known by its brand name Cuprimine, is a chelating agent used primarily in the treatment of Wilson's disease, a genetic disorder that leads to copper accumulation in the body. It is also utilized in the treatment of other conditions such as rheumatoid arthritis, kidney stones, and heavy metal poisoning.

Clinical Trials and Efficacy

Comparison with Trientine Tetrahydrochloride

A recent phase 3 trial, known as the CHELATE study, compared the efficacy and safety of penicillamine with trientine tetrahydrochloride (TETA4) in patients with Wilson's disease. This randomized, open-label, non-inferiority trial involved 53 patients who were stable on penicillamine and were switched to either continue penicillamine or switch to TETA4. The results showed that TETA4 was non-inferior to penicillamine in maintaining serum non-caeruloplasmin-bound copper (NCC) levels and urinary copper excretion over 24 and 48 weeks. The study also highlighted that TETA4 had a better safety profile, with fewer serious adverse events compared to penicillamine[1].

Dosage and Safety in Other Conditions

Another clinical trial assessed the efficacy and safety of high versus low doses of D-penicillamine in patients with primary biliary cirrhosis. The trial found no significant differences in liver test results between the high-dose (750 mg) and low-dose (250 mg) groups, although patients on the higher dose experienced more side effects, such as rash and dysgeusia. This led to an early conclusion of the trial due to the severity of side effects[3].

Market Analysis

Current Market Size and Growth

The global penicillamine market was valued at US$ 83 million in 2023 and is projected to reach US$ 91 million by 2030, growing at a Compound Annual Growth Rate (CAGR) of 1.3% during the forecast period of 2024-2030[2].

Market Dynamics

The growth of the penicillamine market is driven by several factors, including the increasing prevalence of chronic diseases such as Wilson's disease and rheumatoid arthritis, as well as the growing aging population globally. The presence of a robust pipeline of novel therapeutic agents also contributes to the market's expansion. However, potential side effects associated with penicillamine, such as bone marrow suppression, vomiting, diarrhea, hepatotoxicity, and aplastic anemia, are expected to restrain market growth[5].

Market Segmentation

The penicillamine market is segmented based on type (penicillamine capsules and tablets), application (Wilson's disease, arsenic poisoning, rheumatoid arthritis), and end-user (hospitals, clinics, and others). The market is also analyzed across various regions, including North America, Europe, Asia-Pacific, Middle East and Africa, and South & Central America[5].

Regional Framework

The penicillamine market varies significantly across different regions due to factors such as disease prevalence, healthcare infrastructure, and regulatory environments. For instance, North America and Europe are expected to be significant markets due to their well-established healthcare systems and higher awareness of Wilson's disease and other conditions treated with penicillamine. The Asia-Pacific region is also anticipated to show substantial growth due to its large population and increasing healthcare expenditure[5].

Competitive Landscape

The penicillamine market is characterized by a few key players, including those manufacturing branded and generic versions of the drug. Companies must navigate strict regulatory requirements and ensure the quality and safety of their products to maintain market share. The market also sees competition from alternative treatments, such as trientine hydrochloride (Syprine), which is indicated for patients intolerant to penicillamine[4].

Challenges and Opportunities

Side Effects and Safety Concerns

One of the major challenges facing the penicillamine market is the drug's side effect profile. Common adverse events include bone marrow suppression, hepatotoxicity, and idiosyncrasy, which can lead to treatment discontinuation and affect patient compliance. However, ongoing research and the development of new formulations may help mitigate these issues[5].

Emerging Therapies and Pipeline

The pipeline of novel therapeutic agents for Wilson's disease and other conditions treated with penicillamine presents both opportunities and challenges. New treatments could potentially replace or complement penicillamine, but they also offer the possibility of improved efficacy and safety profiles, which could drive market growth[5].

Key Takeaways

• Clinical Efficacy: Penicillamine remains a viable treatment option for Wilson's disease, although it faces competition from newer agents like trientine tetrahydrochloride.
• Market Growth: The global penicillamine market is expected to grow at a CAGR of 1.3% from 2024 to 2030.
• Regional Variations: Market dynamics vary significantly across regions, with North America and Europe being key markets.
• Safety Concerns: Penicillamine's side effect profile is a significant concern and a potential restraint on market growth.
• Competitive Landscape: The market is characterized by a mix of branded and generic products, with competition from alternative treatments.

FAQs

What is the primary use of penicillamine?

Penicillamine is primarily used in the treatment of Wilson's disease, a genetic disorder that leads to copper accumulation in the body. It is also used in treating rheumatoid arthritis, kidney stones, and heavy metal poisoning.

What are the common side effects of penicillamine?

Common side effects include bone marrow suppression, vomiting, diarrhea, hepatotoxicity, idiosyncrasy, and aplastic anemia.

How does the market size of penicillamine project to grow?

The global penicillamine market is projected to grow from US$ 83 million in 2023 to US$ 91 million by 2030, at a CAGR of 1.3%.

What are the main drivers of the penicillamine market?

The market is driven by the increasing prevalence of chronic diseases such as Wilson's disease and rheumatoid arthritis, as well as the growing aging population globally.

What are the regional variations in the penicillamine market?

The market varies significantly across regions, with North America and Europe being key markets due to their well-established healthcare systems and higher awareness of treated conditions.

Sources

1. The Lancet Gastroenterology & Hepatology: "Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomised, open-label, non-inferiority, phase 3 trial"[1].
2. Valuates Reports: "Global Penicillamine Market Research Report 2024"[2].
3. PubMed: "A prospective clinical trial of D-penicillamine in the treatment of primary biliary cirrhosis"[3].
4. Blue Cross Blue Shield of Louisiana: "Penicillamine (Cuprimine®)/Trientine (Syprine®, Cuvrior™)"[4].
5. The Insight Partners: "Penicillamine Market SWOT Analysis by 2031"[5].