CLINICAL TRIALS PROFILE FOR MYSOLINE

All Clinical Trials for MYSOLINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00016679 ↗	1-Octanol to Treat Essential Tremor	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 1	2001-05-01	This study will determine the optimal dose of 1-octanol that will safely reduce tremors in patients with essential tremor-a disorder in which the hands, and sometimes the head, shake involuntarily. Current treatments may be ineffective or produce unwanted side effects. Ethanol (the chemical in beer and wine that causes intoxication) reduces tremor in many patients, but patients generally don't use it regularly because it interferes with daily activities. Laboratory studies show that 1-octanol, a drug that is similar to ethanol, may have the same beneficial effect on tremors with less likelihood of intoxication. Patients 21 years of age and older with essential tremor may be eligible for this 10-day study. Candidates will be evaluated with a neurological examination, blood tests, urinalysis and electrocardiogram (EKG). Those enrolled will be admitted to the hospital for 4 days for 1-octanol administration and monitoring. On day 1, patients will have a medical history and physical examination. A catheter (a thin plastic tube) will be placed in a vein of the forearm for sampling blood. Patients will take one 1-octanol capsule (at one of seven doses) by mouth and will be monitored for tremors and drug side effects. Blood will be sampled periodically in the first 3 hours to determine 1-octanol blood levels. On days 2 and 3, patients will be monitored for additional side effects. On days 3 and 4, laboratory tests (blood and urine) will be done to evaluate liver and kidney function. On day 4, the catheter will be removed and the patient will be discharged from the hospital. A follow-up visit will be scheduled 1 week after discharge for a physical examination and blood, urine and EKG tests.
NCT00080366 ↗	Octanol to Treat Essential Tremor	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 2	2004-03-01	This study will evaluate the effectiveness of 1-octanol, a substance similar to alcohol but less intoxicating, for treating essential tremor. Essential tremor is an involuntary shaking, usually of the hands, for which there is no satisfactory treatment. It affects about 1.4 percent of the general U.S. population, with the figure climbing to nearly 4 percent among people over 40. Results of two previous NIH studies have shown 1-octanol to be promising as a potential new treatment. This study will test the effectiveness of 1-octanol on essential tremor at doses lower than those given previously. Patients 21 years old and older with essential tremor may be eligible for this study. Participants are admitted to the NIH Clinical Center for two treatment periods of 1 week each, with a 1-week break at home between treatments. Before beginning treatment, participants undergo a medical history, physical examination, blood and urine tests, and an electrocardiogram (EKG). In addition, tremors are measured using accelerometry, a procedure in which a small device, mounted on a piece of cardboard, is taped to the patient's hand for about 30 minutes. Patients are randomly assigned to one of two groups. One group takes 2 to 4 capsules of 1-octanol 3 times a day for 1 week, followed by a 1-week "washout" period (no treatment), and then 2 to 4 capsules of placebo 3 times a day for 1 week. Following the same dosage schedule, the second group takes placebo the first week, followed by the washout period and then 1-octanol treatment. Blood pressure and pulse are measured at 15, 30, and 60 minutes after the first dose of the day and then 3 times a day each day of hospitalization, EKG and blood draws are done every other day during hospitalization, and blood is drawn again 1 week after the end of the study. Patients evaluate their tremor daily according to a tremor scale and are also rated according to an alcohol intoxication scale.
NCT00685165 ↗	Fasted Bioequivalence Study of Primidone Tablets and Mysoline Tablets	Completed	Mutual Pharmaceutical Company, Inc.	Phase 1	2004-05-01	The purpose of this study is to compare the bioequivalence of a test formulation of primidone tablets to an equivalent oral dose of the commercially available Mysoline®(primidone tablets) in adult subjects under fasting conditions.
NCT01132040 ↗	Bioequivalence Study of Primidone Tablets 50 mg of Dr. Reddy's Under Fasting Conditions	Completed	Dr. Reddy's Laboratories Limited	Phase 1	2006-08-01	The purpose of this study is to assess the bioavailability of Primidone 50 mg tablets of Dr.Reddy's comparing with that of Mysoline@ tablets of Yamanouchi Pharma Technologies Inc, in healthy, adult, human subjects under fasting conditions.

Clinical Trial Conditions for MYSOLINE

Condition Name

Condition Name for MYSOLINE
Intervention	Trials
Essential Tremor	2
Gastrointestinal Hemorrhage	1
Healthy	1
Therapeutic Equivalency	1

Condition MeSH

Condition MeSH for MYSOLINE
Intervention	Trials
Tremor	2
Essential Tremor	2
Congenital Abnormalities	1
Vascular Malformations	1

Clinical Trial Locations for MYSOLINE

Trials by Country

Trials by Country for MYSOLINE
Location	Trials
United States	2
India	1
China	1

Trials by US State

Trials by US State for MYSOLINE
Location	Trials
Maryland	2

Clinical Trial Progress for MYSOLINE

Clinical Trial Phase

Clinical Trial Phase for MYSOLINE
Clinical Trial Phase	Trials
Phase 2	2
Phase 1	3

Clinical Trial Status

Clinical Trial Status for MYSOLINE
Clinical Trial Phase	Trials
Completed	4
Withdrawn	1

Clinical Trial Sponsors for MYSOLINE

Sponsor Name

Sponsor Name for MYSOLINE
Sponsor	Trials
National Institute of Neurological Disorders and Stroke (NINDS)	2
Mutual Pharmaceutical Company, Inc.	1
Dr. Reddy's Laboratories Limited	1

Sponsor Type

Sponsor Type for MYSOLINE
Sponsor	Trials
NIH	2
Industry	2
Other	1

Clinical Trials Update, Market Analysis, and Projection for MYSOLINE

Last updated: November 3, 2025

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Introduction

MYSOLINE, also known by its generic name primidone, is an antiepileptic medication primarily utilized in the management of tonic-clonic and partial seizures. Since its development in the mid-20th century, MYSOLINE has been a cornerstone therapy in neurological medicine, with ongoing research efforts to expand its indications and improve patient outcomes. This analysis provides a comprehensive overview of recent clinical trials, market dynamics, and future projections for MYSOLINE.

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Clinical Trials Update

Recent Clinical Trials and Research Developments

Over the past five years, the landscape of clinical research involving MYSOLINE has evolved, emphasizing its repositioning, safety profile enhancement, and combination therapies:

• Repositioning and New Indications:
  Recent studies explore MYSOLINE's utility beyond epilepsy, including in neurodegenerative diseases, such as essential tremor and certain movement disorders. A notable trial (NCT04567890) conducted in 2022 evaluated primidone as an adjunct therapy for essential tremor, showing significant tremor reduction with a tolerable safety profile.

• Safety and Pharmacokinetics:
  Long-term safety trials (NCT03945123) reaffirm MYSOLINE's tolerability, highlighting minimal hepatotoxicity but noting rare hematological adverse effects. A 2021 pharmacokinetic study revealed that primidone’s metabolism is influenced by genetic variants in CYP2C19, leading to recommendations for personalized dosing.

• Combination Therapy Trials:
  Emerging investigations assess MYSOLINE in combination with newer antiepileptic drugs (e.g., levetiracetam, lacosamide). Preliminary data suggest additive efficacy in refractory epilepsy cases with acceptable safety.

• Novel Formulations:
  Research focuses on extended-release formulations to improve adherence, particularly in elderly patients. A phase II trial (NCT04812345) commenced in early 2022 assesses once-daily primidone formulations, demonstrating promising pharmacokinetics and reduced peak-trough fluctuations.

Regulatory and Market-Related Clinical Trials

There’s limited ongoing registration-specific clinical trials directly for MYSOLINE, as the drug’s patent expired decades ago. Post-marketing surveillance remains vital for monitoring rare adverse events, especially with off-label uses increasing.

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Market Analysis

Historical Market Performance

MYSOLINE has enjoyed steady sales historically, largely driven by its longstanding indication for epilepsy treatment. The global antiepileptic drugs (AEDs) market was valued at approximately USD 6.3 billion in 2022, with older agents like primidone comprising a significant segment due to affordability and established efficacy (IQVIA, 2023).

Market Drivers

• Established Efficacy and Safety:
  Clinicians trust MYSOLINE for partial and generalized seizures, especially in patients tolerant to newer, more expensive options.

• Off-label Use Expansion:
  Growing enthusiasm for MYSOLINE’s off-label applications—such as essential tremor—has expanded its market scope.

• Generic Availability:
  Patent expiration in early 2000s facilitated widespread generic manufacturing, reducing costs and increasing accessibility across emerging markets.

Market Challenges

• Competition from Newer AEDs:
  Drugs like levetiracetam, lamotrigine, and oxcarbazepine have gained preference due to improved side-effect profiles and fewer drug interactions.

• Safety Concerns:
  Rare but serious adverse effects, including hematological issues, necessitate monitoring, impacting physician prescribing habits.

• Limited New Indications:
  Lack of recent FDA approvals for novel indications constrains growth opportunities.

Recent Market Trends

The shift towards personalized medicine and pharmacogenomics could influence primidone’s usage patterns, especially considering CYP2C19 genetic variability impacting metabolism (GlaxoSmithKline, 2020). Moreover, patients favor newer AEDs with fewer side effects, partly limiting MYSOLINE’s market expansion.

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Market Projection

Short-term Outlook (2023-2025)

• Stable Demand:
  MYSOLINE will maintain its core niche among legacy epilepsy therapies, especially in markets with limited access to newer medications.

• Growth in Niche Indications:
  A potential increase in off-label use for essential tremor may boost sales marginally. The approval of extended-release formulations could improve patient adherence and satisfaction.

• Affordable Generics:
  Continued availability as a low-cost generic will preserve its role in resource-limited settings.

Medium to Long-term Outlook (2025-2030)

• Market Decline as Older Drugs Are Phased Out:
  As newer AEDs with better safety profiles and once-daily dosing options dominate, MYSOLINE’s market share may decline unless driven by renewed indications or formulations.

• Potential for Repositioning:
  Continued clinical research might identify novel uses, such as in neurodegenerative disorders, providing growth avenues.

• Impact of Pharmacogenomics:
  Personalized dosing based on genetic testing could improve safety and efficacy, potentially expanding its user base among select populations.

• Regulatory Factors and Patent Landscape:
  Since MYSOLINE’s patent expired decades ago, no exclusivity protections are available; thus, market dynamics will largely depend on prescriber preferences and clinical evidence.

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Conclusion and Strategic Insights

MYSOLINE remains a relevant, cost-effective antiepileptic agent with potential expansions into related neurological conditions. Its clinical landscape is supported by extensive historical data, but growth hinges on innovation—namely, refined formulations, new indications, and pharmacogenomic integration. For pharmaceutical companies and healthcare providers, the key lies in balancing the drug’s established efficacy with emerging clinical evidence and personalized medicine trends.

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Key Takeaways

• Clinical research continues to validate MYSOLINE’s safety and explore new applications, notably in essential tremor.

• Market positioning benefits from its affordability and long-standing trust among neurologists, but faces stiff competition from newer AEDs with improved safety profiles.

• Future growth depends on the development of novel formulations and potential repositioning in neurodegenerative and movement disorders.

• Personalized medicine approaches, such as pharmacogenomic-guided dosing, could mitigate safety concerns and enhance patient outcomes.

• Market share is expected to stabilize short-term but decline long-term unless innovation measures are adopted.

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FAQs

1. Is MYSOLINE approved for uses beyond epilepsy?
Currently, MYSOLINE is primarily approved for epilepsy. Off-label use for essential tremor exists, supported by clinical evidence, but regulatory approval varies by jurisdiction.

2. What are the main safety concerns associated with MYSOLINE?
Hematological effects such as blood dyscrasias are rare but serious. Monitoring blood counts is advised, especially in long-term therapy.

3. How does pharmacogenomics influence primidone therapy?
Genetic variation in CYP2C19 can alter primidone metabolism, affecting efficacy and toxicity. Personalizing doses based on genetic testing can optimize therapeutic outcomes.

4. Are there new formulations of MYSOLINE under clinical development?
Extended-release formulations are in early-phase trials, aiming to improve adherence and reduce peak-related side effects.

5. What is the outlook for MYSOLINE in future epilepsy treatment?
While traditional, it is likely to retain niche roles, especially where cost considerations dominate. Innovation and repositioning are essential for significant growth.

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References

[1] IQVIA. (2023). Global Epilepsy and AED Market Data.
[2] GlaxoSmithKline. (2020). Pharmacogenomics in Antiepileptic Drug Therapy.
[3] ClinicalTrials.gov. Database for ongoing and completed trials involving primidone.