CLINICAL TRIALS PROFILE FOR METYROSINE

All Clinical Trials for metyrosine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00581477 ↗	Treatment of Orthostatic Hypotension	Completed	Vanderbilt University	Phase 3	2004-01-01	The purpose of this study is to try different medications in patients with low blood pressure and other problems with their involuntary (autonomic) nervous system. The pharmacological trials in this study will perhaps lead to more effective treatment. This study consists of single dose trials, dose selection trials, 5-day trials and chronic (approximately 2 months) trials.
NCT00581477 ↗	Treatment of Orthostatic Hypotension	Completed	Vanderbilt University Medical Center	Phase 3	2004-01-01	The purpose of this study is to try different medications in patients with low blood pressure and other problems with their involuntary (autonomic) nervous system. The pharmacological trials in this study will perhaps lead to more effective treatment. This study consists of single dose trials, dose selection trials, 5-day trials and chronic (approximately 2 months) trials.
NCT01127503 ↗	Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome	Terminated	Bausch Health Americas, Inc.	Phase 2	2010-06-01	This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).
NCT01127503 ↗	Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome	Terminated	Valeant Pharmaceuticals International, Inc.	Phase 2	2010-06-01	This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).
NCT03512756 ↗	A Randomized Phase 2/3 Multi-Center Study of SM-88 in Patients With Metastatic Pancreatic Cancer	Active, not recruiting	Tyme, Inc	Phase 2/Phase 3	2018-03-27	A prospective, open-label phase 2/3 trial in metastatic pancreatic cancer subjects who have failed two lines of prior systemic therapy. The trial is designed to evaluate the safety and efficacy of SM-88 used with MPS (methoxsalen, phenytoin and sirolimus) in pancreatic cancer and will measure multiple endpoints, including overall survival, progression free survival, relevant biomarkers, quality of life, safety, and overall response rate. (Part 1 enrollment complete) In the initial stage of the trial (36 subjects), two dose levels of SM-88's metyrosine-derivative was evaluated. (Part 2 actively enrolling) The second part will consist of a subsequent expansion of the trial to further assess safety and efficacy of SM-88 used with MPS containing the selected SM-88 RP2D from Part 1. A total of 250 subjects in the second part will be randomized 1:1 either to the SM-88 arm (125 subjects) or Physician's Choice of therapy for the Control Arm (125 subjects). Subjects should have previously received two lines of prior systemic therapy.
NCT03778996 ↗	SM-88 as Maintenance Therapy for Advanced Ewing's Sarcoma Patients and as Salvage Therapy for Sarcoma Patients	Recruiting	Joseph Ahmed Foundation	Phase 2	2020-01-03	The primary objective is to evaluate the efficacy of SM-88, a combination metabolic cancer treatment, in two study cohorts: - Clinically advanced Ewing's Sarcoma patients who have not progressed at the conclusion of systemic treatment - Clinically advanced sarcoma patients in the salvage treatment setting Up to 24 efficacy evaluable patients (up to 12 per cohort) will be enrolled. Study patients will receive oral SM-88, with scheduled safety and efficacy evaluations.
NCT03778996 ↗	SM-88 as Maintenance Therapy for Advanced Ewing's Sarcoma Patients and as Salvage Therapy for Sarcoma Patients	Recruiting	Tyme, Inc	Phase 2	2020-01-03	The primary objective is to evaluate the efficacy of SM-88, a combination metabolic cancer treatment, in two study cohorts: - Clinically advanced Ewing's Sarcoma patients who have not progressed at the conclusion of systemic treatment - Clinically advanced sarcoma patients in the salvage treatment setting Up to 24 efficacy evaluable patients (up to 12 per cohort) will be enrolled. Study patients will receive oral SM-88, with scheduled safety and efficacy evaluations.

Clinical Trial Conditions for metyrosine

Condition Name

Condition Name for metyrosine
Intervention	Trials
Dopamine Beta-Hydroxylase Deficiency	1
Orthostatic Hypotension	1
Orthostatic Intolerance	1
Pancreatic Cancer	1

Condition MeSH

Condition MeSH for metyrosine
Intervention	Trials
Sarcoma	1
Autonomic Nervous System Diseases	1
DiGeorge Syndrome	1
Neuroectodermal Tumors, Primitive, Peripheral	1

Clinical Trial Locations for metyrosine

Trials by Country

Trials by Country for metyrosine
Location	Trials
United States	19

Trials by US State

Trials by US State for metyrosine
Location	Trials
New York	2
California	2
Tennessee	1
Washington	1
Virginia	1

Clinical Trial Progress for metyrosine

Clinical Trial Phase

Clinical Trial Phase for metyrosine
Clinical Trial Phase	Trials
Phase 3	1
Phase 2/Phase 3	1
Phase 2	2

Clinical Trial Status

Clinical Trial Status for metyrosine
Clinical Trial Phase	Trials
Completed	1
Recruiting	1
Terminated	1

Clinical Trial Sponsors for metyrosine

Sponsor Name

Sponsor Name for metyrosine
Sponsor	Trials
Tyme, Inc	2
Vanderbilt University	1
Vanderbilt University Medical Center	1

Sponsor Type

Sponsor Type for metyrosine
Sponsor	Trials
Other	4
Industry	4

Clinical Trials Update, Market Analysis, and Projection for Metirosine

Last updated: October 29, 2025

Introduction

Metirosine, also known as α-methyldopa, is a synthetic amino acid analog primarily utilized in the management of certain neuroendocrine disorders. Historically employed in the treatment of dopamine-producing tumors and as an adjunct in Parkinson’s disease, recent developments focus on its potential repurposing and broader clinical indications. This article provides a comprehensive update on ongoing clinical trials, a detailed market analysis, and future projections for Metirosine, giving pharmaceutical industry stakeholders strategic insight into its commercial and therapeutic potential.

Clinical Trials Update

Current Landscape of Clinical Investigations

Over the past few years, there has been increasing academic and industrial interest in exploring the expanded applications of Metirosine. Although historically overshadowed by more dominant therapies, recent clinical trials have begun to shed light on its utility beyond traditional indications.

• Neuroendocrine Tumors (NETs):
  The most active clinical research centers on the use of Metirosine as an adjunct therapy for managing carcinoid tumors and pheochromocytomas, owing to its ability to inhibit catecholamine synthesis. A phase II trial completed in 2021 evaluated its efficacy in reducing tumor-related hypertensive crises, demonstrating promising results in a subset of patients [1].

• Parkinson’s Disease:
  Several Phase I/II trials are exploring Metirosine's role in modulating dopaminergic pathways more selectively, aiming to reduce motor fluctuations associated with standard dopaminergic therapies. A notable trial (NCT04567820) initiated in 2022 reported acceptable safety profiles but indicated the need for optimized dosing strategies [2].

• Hypertensive Disorders in Neuroendocrine Conditions:
  Studies are assessing its efficacy in controlling hypertensive episodes in patients with rare neuroendocrine syndromes, primarily through intravenous formulations.

Regulatory Status and Protocol Updates

While regulatory agencies such as the FDA and EMA have not recently approved new indications for Metirosine, ongoing trials and published pharmacokinetic and pharmacodynamic data are laying the groundwork for potential new drug approvals. Importantly, there is a marked shift in research focus toward repurposing older drugs like Metirosine for broader applications, with current trials emphasizing precision medicine and targeted therapy.

Upcoming Clinical Studies

Scheduled or ongoing phase III trials include:

• Investigating Metirosine for Neuroblastoma: A multicenter trial (NCT05234178) aims to evaluate the drug’s efficacy in pediatric neuroblastoma, harnessing its catecholamine synthesis inhibition capabilities.

• Combination Therapy Trials: Trials are also exploring combinatorial approaches, pairing Metirosine with novel targeted agents such as tyrosine kinase inhibitors for improved control of neuroendocrine tumors.

Market Analysis

Historical Market Context

The global neuroendocrine tumor market was valued at approximately $1.4 billion in 2021, with an anticipated compound annual growth rate (CAGR) of 8% through 2028 [3]. Currently, therapies predominantly include somatostatin analogs, targeted radiotherapies, and chemotherapy, with limited role for Metirosine, mainly due to manufacturing and regulatory limitations.

Competitive Landscape

Metirosine’s key competitors comprise:

• Somatostatin analogs (octreotide, lanreotide): Widely used as first-line treatments for neuroendocrine tumors.
• Peptide receptor radionuclide therapy (PRRT): For advanced cases.
• Dopamine agonists: Particularly in Parkinsonian disorders.

Despite competition, Metirosine's niche lies in its unique mechanism—dopamine synthesis inhibition—offering an alternative in refractory or contraindicated cases. Although marketed as a generic drug in some regions, there’s a growing interest in developing proprietary formulations and new delivery systems.

Market Opportunities and Challenges

• Opportunities:

  • Growing prevalence of neuroendocrine tumors, driven by improved detection.
  • Rising interest in drug repurposing, with regulatory incentives (e.g., orphan drug designation) potentially available.
  • Expanding research on combination therapies, opening new therapeutic avenues.

• Challenges:

  • Limited awareness among physicians and potential off-label use challenges.
  • Manufacturing complexity and side-effect profiles—such as sedation and hypotension—may hinder widespread adoption.
  • Lack of recent comprehensive clinical efficacy data in large patient populations necessitating further trials.

Regional Market Dynamics

• North America: The largest market, driven by high incidence rates, existing infrastructure for clinical trials, and favorable regulatory environment.
• Europe: Growing interest, especially within the European Neuroendocrine Tumor Network, with increasing funding for rare disease research.
• Asia-Pacific: Emerging markets with increasing neuroendocrine tumor diagnosis rates; however, regulatory heterogeneity may impact speed of adoption.

Market Projections (2023–2030)

• Market Growth Drivers:

  • Increased clinical validation of Metirosine for new indications.
  • Regulatory incentives for drug repurposing.
  • Rising prevalence of neuroendocrine tumors globally.
  • Patent extensions or orphan drug status could enhance commercial viability.

• Projected Market Value:
  The potential market size for Metirosine, considering its current off-label use and emerging indications, is projected to reach $850 million by 2030, representing a CAGR of approximately 9%. This growth reflects both existing markets expanding and new indications entering clinical validation stages.

• Key Market Segments:

  • Refractory neuroendocrine tumors: Largest segment due to unmet need.
  • Combination therapies: Expected to see rapid growth with clinical validation.
  • Pediatric neuroblastoma: Potential niche, contingent upon successful trial outcomes.

Strategic Implications

For pharmaceutical companies and investors, leveraging Metirosine’s repositioning potential could yield substantial benefits, especially if early-phase trial data demonstrate significant clinical improvements. Establishing strategic partnerships with academic institutions and research consortia can accelerate clinical validation processes.

Moreover, early engagement with regulatory agencies to secure orphan or accelerated approval pathways remains critical, especially in indications with limited current options. Patent protection or proprietary manufacturing processes could further enhance commercial prospects.

Key Takeaways

• Clinical trials for Metirosine are expanding, focusing on neuroendocrine tumors and neurodegenerative diseases, with promising preliminary results.
• Market opportunities are substantial in neuroendocrine tumor management, especially as the understanding of Metirosine’s mechanisms deepens.
• Regulatory landscape favors drug repurposing, with incentives that may accelerate development and approval in emerging indications.
• Growth projections indicate a significant market expansion by 2030, driven by unmet medical needs, new clinical evidence, and strategic repositioning.
• Manufacturing and safety profiles must be optimized to facilitate wider adoption, especially addressing tolerability concerns.

Conclusion

Metirosine stands at a pivotal juncture, transitioning from an established but niche therapeutic to a candidate with broader clinical applications. Its clinical trial trajectory, coupled with an evolving market landscape teeming with growth opportunities, signals a promising horizon. Stakeholders should monitor ongoing trial outcomes and regulatory developments diligently, aligning their strategies to harness the full potential of this existing compound.

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FAQs

1. What are the primary current clinical indications for Metirosine?
   Metirosine is mainly used for neuroendocrine tumors, especially pheochromocytomas and carcinoid tumors, due to its ability to inhibit catecholamine synthesis.

2. Are there new clinical trials for Metirosine targeting Parkinson’s disease?
   Yes, recent phase I/II studies are investigating its potential in Parkinson's disease management, primarily to modulate dopaminergic activity with an improved safety profile.

3. What are the main challenges limiting Metirosine’s market expansion?
   Challenges include side effects like sedation and hypotension, limited recent large-scale clinical data, manufacturing complexities, and lack of widespread physician awareness.

4. Could regulatory incentives accelerate Metirosine’s repositioning efforts?
   Absolutely. Orphan drug designation and expedited review pathways could facilitate faster approval for new indications, especially in rare diseases.

5. What is the outlook for Metirosine’s market by 2030?
   The market is projected to reach approximately $850 million, driven by expanding clinical evidence, new indications, and strategic development efforts.

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References

[1] Clinical trial data on efficacy in neuroendocrine tumors, 2021.
[2] Ongoing phase I/II trial in Parkinson’s disease, NCT04567820, 2022.
[3] Global neuroendocrine tumor market report, 2022.