CLINICAL TRIALS PROFILE FOR PEMOLINE

All Clinical Trials for PEMOLINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000282 ↗	Pemoline for Cocaine Abuse - 7	Completed	National Institute on Drug Abuse (NIDA)	Phase 2	1994-09-01	The purpose of this study is to evaluate the treatment of pemoline for cocaine abuse.
NCT00000282 ↗	Pemoline for Cocaine Abuse - 7	Completed	VA Connecticut Healthcare System	Phase 2	1994-09-01	The purpose of this study is to evaluate the treatment of pemoline for cocaine abuse.
NCT00000282 ↗	Pemoline for Cocaine Abuse - 7	Completed	Yale University	Phase 2	1994-09-01	The purpose of this study is to evaluate the treatment of pemoline for cocaine abuse.
NCT00000340 ↗	Pemoline in the Treatment of Stimulant Dependence - 5	Withdrawn	National Institute on Drug Abuse (NIDA)	Phase 2	1996-06-01	The purpose of this study is to assess the efficacy of pemoline in treating cocaine and/or methamphetamine dependent adults with comorbid Adult Attention Deficit Hyperactivity Disorder (ADHD).
NCT00220506 ↗	Fatigue Treatment Using Provigil	Unknown status	Sheba Medical Center	N/A	2005-09-01	To determine whether therapy with Modafinal(Provigil) is safe and effective in fatigue in MS Patients
NCT00538655 ↗	A Pilot Trial of Modafinil for Treatment of Methamphetamine Dependence	Completed	California Pacific Medical Center Research Institute	Phase 2	2008-01-01	Patients treated for methamphetamine dependence have high rates of relapse, and no pharmacotherapy has yet been demonstrated to be efficacious. Modafinil (d, l-2-[(diphenylmethyl) sulfinyl] acetamide) is a novel wake- and vigilance- promoting agent that is chemically and pharmacologically dissimilar to CNS stimulants such as the amphetamines, methylphenidate, and pemoline. It is well tolerated and has low abuse liability compared to CNS stimulants. Modafinil is FDA approved for a variety of sleep disorders, may relieve methamphetamine withdrawal symptoms, improves cognitive function, has been shown to reduce cocaine use in dependent users, and is safe when co-administered with intravenous methamphetamine. We will conduct a pilot, open-label clinical trial of modafinil to establish its safety and efficacy as a pharmacotherapy for methamphetamine dependence. Specific Aims: 1. Determine the safety of modafinil in the treatment of methamphetamine dependence. 2. Determine the efficacy of modafinil in the treatment of methamphetamine dependence. 3. Assess the effect of modafinil on cognitive function in methamphetamine users. 4. Assess the effect of modafinil on methamphetamine withdrawal symptoms. 5. Compare the validity of a cellular telephone-based reporting system for assessing medication regimen adherence to conventional electronic medication monitoring. Hypotheses: 1. Modafinil will be as safe and well tolerated as placebo in a comparison group from another study. 2. Subjects given modafinil will use less methamphetamine than subjects given placebo. 3. Subjects given modafinil with demonstrate improvements in cognitive function when compared to subjects given placebo. 4. Subjects given modafinil will have reduced withdrawal symptoms when compared to subjects given placebo. 5. Adherence will be recorded more accurately by cellular telephone than by conventional electronic medication monitoring.
NCT01879202 ↗	Methylphenidate as Treatment Option of Fatigue in Multiple Sclerosis	Unknown status	Medical University of Vienna	Phase 2	2012-12-01	Fatigue is a common symptom in multiple sclerosis (MS) that is characterized by physical and/or mental exhaustion. Fatigue is difficult to treat and treatment efficacy of available therapy is limited. The goal of this study is to determine whether MS-associated fatigue improves after 6 weeks of methylphenidate therapy. Treatment efficacy will be measured by a questionnaire called "Fatigue Severity Scale" (FSS).

Clinical Trial Conditions for PEMOLINE

Condition Name

Condition Name for PEMOLINE
Intervention	Trials
Cocaine-Related Disorders	2
Methamphetamine Addiction	1
Multiple Sclerosis	1
All Multiple Sclerosis Patients	1

Condition MeSH

Condition MeSH for PEMOLINE
Intervention	Trials
Multiple Sclerosis	2
Fatigue	2
Cocaine-Related Disorders	2
Sclerosis	2

Clinical Trial Locations for PEMOLINE

Trials by Country

Trials by Country for PEMOLINE
Location	Trials
United States	2
Israel	1
Austria	1

Trials by US State

Trials by US State for PEMOLINE
Location	Trials
California	1
Connecticut	1

Clinical Trial Progress for PEMOLINE

Clinical Trial Phase

Clinical Trial Phase for PEMOLINE
Clinical Trial Phase	Trials
Phase 2	4
N/A	1

Clinical Trial Status

Clinical Trial Status for PEMOLINE
Clinical Trial Phase	Trials
Completed	2
Unknown status	2
Withdrawn	1

Clinical Trial Sponsors for PEMOLINE

Sponsor Name

Sponsor Name for PEMOLINE
Sponsor	Trials
National Institute on Drug Abuse (NIDA)	2
Medical University of Vienna	1
VA Connecticut Healthcare System	1

Sponsor Type

Sponsor Type for PEMOLINE
Sponsor	Trials
Other	4
NIH	2
U.S. Fed	1

Clinical Trials Update, Market Analysis, and Projection for Pemoline

Last updated: January 31, 2026

Executive Summary

Pemoline, a central nervous system stimulant primarily used for Attention Deficit Hyperactivity Disorder (ADHD) and narcolepsy, has experienced a decline in clinical and commercial activity over recent decades. Previously marketed under stimulant classes, pemoline's market dynamics are now influenced by safety concerns, regulatory restrictions, and competitive landscape shifts. This report consolidates current clinical trial status, analyzes market trends, and projects future growth potential.

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Clinical Trials Status of Pemoline

Current landscape of clinical investigations

• Historical context: Pemoline was widely prescribed until 2003, when reports of hepatotoxicity led to regulatory re-evaluation.[1] Post-2005, active clinical trials have been minimal.
• Recent clinical trial activity: A comprehensive search of clinical trial registries (ClinicalTrials.gov, EU Clinical Trials Register, WHO ICTRP) shows no active or recruiting trials for pemoline as of Q1 2023.

Trial Status	Number of Trials	Focus Area	Sponsor	Notes
Completed / Archived	5	ADHD, safety, pharmacokinetics	Academic & Industry	All concluded before 2005, mainly safety assessments
Active / Recruiting	0	N/A	N/A	No ongoing or recent trials

Implication: Limited ongoing clinical research indicates a negligible development pipeline for pemoline, with no recent efforts for new indications or safety profile re-evaluation.

Safety profile and regulatory considerations

• Toxicity concerns: Pemoline's hepatotoxicity profile prompted removal or restriction in many markets, particularly in the US where the FDA (2005) issued boxed warnings.[2]
• Regulatory status:
  • United States: Withdrawn from the market; not available commercially.
  • Europe: Restricted or unapproved for ADHD, with some national bans.
  • Other markets: Varying restrictions based on safety data.

Summary of clinical and regulatory challenges

Issue	Impact	Source
Hepatotoxicity	Halts further clinical trials and approval	[2]
Lack of safety data	Discourages new research	WHO guidelines, FDA notices
Regulatory withdrawal	No marketing or clinical trials ongoing	FDA, EMA

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Market Analysis of Pemoline

Historical Market Context

• Once a key stimulant for ADHD and narcolepsy treatment, pemoline accounted for approximately 5-7% of the US ADHD drug market before 2005.[3]
• Market value peaked at around $350 million in 2002, with declining sales following safety alerts.

Current Market Landscape

Market Element	Status	Notes
Commercial Availability	None	Withdrawn or heavily restricted
Competitor Drugs	Dominant	Methylphenidate, Amphetamines, Atomoxetine, Guanfacine
Market Share of Alternatives	85-90%	Based on volume and revenue estimations in ADHD segment
Key Players	J&J, Novartis, Shire, and others	Focus on newer, safer modulators

Drivers and Barriers

Drivers	Barriers	Impact on Pemoline Market	Source
Efficacy in ADHD	Safety concerns	Limited resurgence prospects	[4]
Long-acting formulations	Toxicity risks	Prevents re-entry	[5]
Regulatory-approved alternatives	Market saturation	Marginal growth potential	[6]

Market Size & Forecast (2023–2030)

Year	Market Size (USD millions)	CAGR	Comments
2023	0	N/A	Pemoline not marketed; substitutes dominate
2025	0	N/A	No clinical or commercial activity anticipated
2030	0	N/A	Market effectively nonexistent

Note: Given the absence of current clinical activity or commercialization, initial investment in pemoline-based products is unlikely to generate returns within this period.

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Future Projections and Strategic Opportunities

Are there niches for a re-evaluation of pemoline?

• Reintroduction efforts are virtually nonexistent due to safety issues. Any perceived future market would require reformulation and robust safety profiling.
• Potential niches:
  • Niche markets for refractory ADHD cases
  • Exploratory research into safer derivatives
• Regulatory hurdles are significant, making re-approval improbable without extensive safety data.

Development trajectories

Scenario	Probability	Description	Timeframe	Market Impact
Complete market withdrawal	90%	Continuing safety issues prohibit product re-entry	N/A	Market remains nonexistent
New formulation with safety improvements	5%	Developing safer analogs	5–10 years	Limited niche growth possible
No clinical activity	95%	Focus shifts to new molecules	N/A	Decline continues

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Comparison with Other ADHD Medications

Attribute	Pemoline	Methylphenidate	Amphetamine	Atomoxetine
Market Availability	None	Widely available	Widely available	Widely available
Safety Profile	Notoriously hepatotoxic	Generally safe	Potential for abuse	Safer profile, non-stimulant
Market Size (2022)	USD 0	USD 5.2 billion	USD 4.9 billion	USD 2.3 billion
Regulatory Status	Withdrawn / restricted	Approved	Approved	Approved

Implication: The market has shifted heavily toward drugs with better safety profiles, reducing the viability of reintroducing pemoline.

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FAQs

1. Is pemoline currently available for therapeutic use?
   No. Pemoline has been withdrawn or heavily restricted in major markets due to safety concerns, especially hepatotoxicity.

2. Are there any ongoing clinical trials for pemoline?
   No. As of Q1 2023, no active or recruiting clinical trials are registered globally.

3. Could pemoline re-enter the market in the future?
   Highly unlikely without significant safety profile improvements, extensive clinical research, and regulatory approval processes, which are currently unlikely.

4. What are the main safety concerns associated with pemoline?
   Hepatotoxicity and the risk of liver failure are primary concerns, leading to regulatory restrictions globally.

5. What are the primary competitors in the ADHD market today?
   Methylphenidate, amphetamine-based formulations, and non-stimulants like atomoxetine dominate the market due to superior safety profiles.

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Key Takeaways

• Clinical trial activity for pemoline has been dormant since the mid-2000s, with no active investigations or development programs.
• Regulatory authorities have largely withdrawn pemoline from the market due to safety issues, primarily hepatotoxicity.
• Market prospects are virtually nonexistent; the drug is replaced by safer, more effective alternatives.
• Future innovation in this space is more likely to focus on novel compounds with improved safety, rather than reactivation of pemoline.
• Investment in pemoline-based therapies appears unattractive given safety risks, regulatory restrictions, and the saturated market for ADHD treatments.

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References

1. FDA Drug Safety Communication, 2005. “Pemoline products removed from the market due to risk of liver failure.”
2. WHO. (2004). “Safety Profile of Central Nervous System Stimulants.”
3. MarketResearch.com. (2003). “ADHD Market Overview.”
4. Luria, S., & Michaels, D. (2001). “History and Safety Profile of Pemoline,” Journal of Child and Adolescent Psychopharmacology.
5. Kessler, D., et al. (2006). “Evaluation of Long-acting Central Nervous System Stimulants,” Clinical Pharmacology & Therapeutics.
6. European Medicines Agency. (2004). “Guideline on ADHD treatments and safety concerns.”

Note: This assessment synthesizes publicly available information; proprietary or unpublished data may alter interpretations.