CLINICAL TRIALS PROFILE FOR RILUTEK

All Clinical Trials for RILUTEK

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00026052 ↗	Riluzole to Treat Major Depression	Completed	National Institute of Mental Health (NIMH)	Phase 2	2001-11-01	This study will examine the safety and effectiveness of the drug riluzole (Rilutek® (Registered Trademark)) for short-term treatment of depression symptoms, such as depressed mood, psychomotor retardation, and excessive sleeping. Despite the availability of a wide range of antidepressant drugs, studies indicate that 30 to 40 percent of patients with major depression do not respond to first-line antidepressant treatment with drugs such as fluoxetine, upropion, venlafaxine and others. Riluzole, which is approved by the Food and Drug Administration (FDA) for amyotrophic lateral sclerosis (ALS), causes chemical changes in the brain that may also have antidepressant properties. Patients between 18 and 70 years of age with major depressive disorder without psychotic features may be eligible for this 2-stage 7-week study. Candidates will be screened with a medical history and physical examination, including an electrocardiogram (EKG), blood and urine tests, and a psychiatric evaluation. A blood or urine sample will be tested for illegal drugs.Women of childbearing potential will have a pregnancy test. Participants will complete stage 1 of the study, which lasts 1 week, and may then continue with stage 2 for an additional 6 weeks. At the start of the study, patients will be tapered off all psychiatric medicines and will begin treatment with a placebo (a sugar pill formulated to look like the active drug). At some point, they will be switched from placebo to riluzole. In addition, participants will undergo the following procedures: - Physical examination and electrocardiograms (EKG) at the beginning and end of the study, with vital signs (temperature, blood pressure and heart rate) checked daily - Weekly 1-hour interviews consisting of psychiatric and psychomotor rating scales to assess treatment response - Weekly blood tests to measure blood levels of riluzole and evaluate drug side effects At the end of the study, participants' psychiatric status will be reassessed and appropriate long-term psychiatric treatment arranged. Patients, ages 18 to 70 with a diagnosis of major depression without psychotic features, will in this pilot study (single arm, single blind) receive riluzole (50-200 mg/day) for a period of 6 weeks. Acute efficacy will be determined by demonstrating a greater response rate using specified criteria. Approximately 25 patients will enter the study to obtain 22 subjects who complete the 6 weeks of acute riluzole treatment. Therefore, if 7/22 patients or greater have greater than 50% improvement on the primary efficacy measure, then based on statistically guidelines from the Optimal Two Stage Design for Clinical Trials, a controlled trial would be indicated to scientifically confirm the signal observed in the single arm trial.
NCT00047723 ↗	Minocycline to Treat Amyotrophic Lateral Sclerosis	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 3	2003-01-01	The purpose of this trial is to test the safety, tolerability, and effectiveness of minocycline compared to placebo in patients with amyotrophic lateral sclerosis (ALS).
NCT00054704 ↗	Riluzole to Treat Depression in Bipolar Disorder	Terminated	National Institute of Mental Health (NIMH)	Phase 2	2003-02-01	This study examines if Riluzole, FDA approved for ALS, will improve symptoms of depression in Bipolar Disorder. Purpose: This study will examine the safety and effectiveness of riluzole (Rilutek trademark) for short-term treatment of depression symptoms, such as depressed mood, psychomotor retardation, and excessive sleeping in patients with bipolar disease. Riluzole is approved by the Food and Drug Administration (FDA) to treat amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease). Preliminary findings of a study using riluzole to treat acute depression in patients with unipolar depression indicate that it may have antidepressant properties in some patients. Patients between 18 and 70 years of age with bipolar I or II disorder without psychosis may be eligible for this 8-week study. Candidates must be currently depressed, must have had at least one previous major depressive episode, and must have failed to improve with prior treatment with at least one antidepressant. They will be screened with a medical history, physical examination, electrocardiogram (EKG), blood and urine tests, and psychiatric evaluation. A blood or urine sample will be analyzed for illegal drugs. Women of childbearing potential will have a pregnancy test. Participants will begin an 8-week course of treatment, starting with a placebo (a sugar pill formulated to look like the active drug) and, at some point, switching to riluzole. In addition to drug treatment, participants will undergo the following procedures: Physical examination and electrocardiogram (EKG) at the beginning and end of the study; Weekly check of vital signs (temperature, blood pressure and heart rate); Weekly 1-hour interviews consisting of psychiatric and psychomotor rating scales to assess treatment response; Weekly blood tests to measure blood levels of riluzole and evaluate drug side effects. At the end of the study, participants' psychiatric status will be reassessed and appropriate long-term psychiatric treatment arranged. Atendemos pacientes de habla hispana. We enroll eligible participants locally and from around the country. Travel arrangements are provided and costs covered by the National Institute of Mental Health (NIMH). (Arrangements vary by distance and by specific study.) After completing the study participants receive short-term follow-up care while transitioning back to a provider.

Clinical Trial Conditions for RILUTEK

Condition Name

Condition Name for RILUTEK
Intervention	Trials
Amyotrophic Lateral Sclerosis	9
Depression	2
Bipolar Disorder	2

Condition MeSH

Condition MeSH for RILUTEK
Intervention	Trials
Amyotrophic Lateral Sclerosis	12
Sclerosis	11
Motor Neuron Disease	11

Clinical Trial Locations for RILUTEK

Trials by Country

Trials by Country for RILUTEK
Location	Trials
United States	80
Canada	19
Spain	3
Germany	3
France	3

Trials by US State

Trials by US State for RILUTEK
Location	Trials
Maryland	8
Massachusetts	4
Ohio	4
New York	4
Georgia	3

Clinical Trial Progress for RILUTEK

Clinical Trial Phase

Clinical Trial Phase for RILUTEK
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	1
Phase 4	3

Clinical Trial Status

Clinical Trial Status for RILUTEK
Clinical Trial Phase	Trials
Completed	21
Recruiting	5
Active, not recruiting	2

Clinical Trial Sponsors for RILUTEK

Sponsor Name

Sponsor Name for RILUTEK
Sponsor	Trials
National Institute of Mental Health (NIMH)	4
Stanley Medical Research Institute	2
University of Calgary	2

Sponsor Type

Sponsor Type for RILUTEK
Sponsor	Trials
Other	39
Industry	11
NIH	7

Clinical Trials Update, Market Analysis, and Projection for Rilutek (Riluzole)

Last updated: November 2, 2025

Introduction

Rilutek (riluzole) is a pioneering drug approved for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. Since its approval by the FDA in 1995, riluzole has established itself as a cornerstone in ALS management, offering modest but meaningful extension of survival. This article provides a comprehensive update on current clinical trials involving riluzole, evaluates its market landscape, and projects future trends based on recent developments.

Clinical Trials Update

Historical and Ongoing Research Landscape

Riluzole was initially approved based on clinical data demonstrating a survival extension of approximately 2-3 months in ALS patients. Since then, research focus has shifted towards optimizing its efficacy, exploring combination therapies, and understanding its broader mechanisms of neuroprotection.

Recent and Ongoing Clinical Trials

Recent clinical trials encompass both phase I-III studies and real-world evidence evaluations:

1. Combination Therapy Trials: Investigations have sought to assess riluzole's synergy with other neuroprotective agents. For instance, trials combining riluzole with edaravone — another FDA-approved ALS drug — aim to determine additive benefits on disease progression. A notable phase II trial (NCT04588541) initiated in 2021 examined safety and efficacy of combined riluzole and edaravone therapy, although results are pending.

2. Extended Use and Off-label Applications: Several ongoing studies explore riluzole’s potential in other neurodegenerative disorders, such as multiple sclerosis and spinal cord injury recovery, though these are mostly early-phase trials with limited published data.

3. Dose Optimization and Safety Studies: Additional trials, including NCT04627755, investigate long-term safety profiles and optimal dosing regimens, especially given concerns about hepatic toxicity associated with riluzole.

4. Biomarkers and Personalized Medicine: Emerging research aims to identify biomarkers that predict response to riluzole, facilitating individualized therapy strategies. For instance, genetic polymorphism studies (e.g., in EAAT2 transporter genes) are underway to optimize patient selection.

Key Findings from Recent Trials

• Efficacy Stability: Riluzole continues to demonstrate a modest but statistically significant benefit in survival and functional decline slowing, consistent with historical data.
• Safety Profile: Adverse events remain manageable, with hepatic function monitoring recommended due to hepatic enzyme elevations.
• Combination Therapy Promise: Early-phase data suggest potential synergistic effects when riluzole is combined with other agents, though definitive conclusions await phase III trial results.

Regulatory Developments

Regulatory agencies have shown interest in expanding riluzole’s therapeutic indications. For instance, the European Medicines Agency (EMA) has approved riluzole for primary lateral sclerosis, an ALS variant, indicating recognition of its neuroprotective role.

Market Analysis

Current Market Landscape

The global ALS drug market, estimated at USD 84 million in 2022, is relatively niche but growing steadily, driven by increased diagnosis rates and ongoing research.

Key Market Players

Riluzole remains the dominant therapy, with Teva Pharmaceutical Industries being the primary manufacturer, holding the patent portfolio and marketing rights predominantly in North America and Europe. Generic versions of riluzole are available in numerous markets, significantly affecting pricing and market share dynamics.

Market Drivers

• Aging Population & Rising ALS Incidence: ALS incidence is approximately 2 per 100,000 globally, with higher prevalence among individuals aged 55–75. Aging demographics are projected to augment market demand.
• Unmet Needs & Limited Therapeutic Options: While riluzole prolongs survival, it offers limited symptomatic relief. The lack of curative treatments sustains demand for existing therapies and incentivizes novel drug development.
• Regulatory Incentives: Orphan drug designation and fast-track approvals encourage pipeline investments and market expansion.

Market Challenges

• Modest Efficacy: The limited clinical benefit constrains market growth and prompts the need for more effective therapies.
• Pricing & Reimbursement: Cost containment pressures and reimbursement uncertainties influence adoption rates.
• Patent Expiry & Generics: Patent expiration in key markets has led to increased generic availability, intensifying price competition.

Growth Opportunities

• Combination Therapies: Research integrating riluzole with novel agents, such as gene therapies and neurotrophic factors, could rejuvenate its market position.
• New Indications: Potential expansion into other neurodegenerative conditions broadens market scope.
• Geographic Expansion: Emerging markets, notably Asia-Pacific, demonstrate increasing diagnosis and treatment rates, representing growth avenues.

Market Projections (2023-2030)

Based on current trends, the ALS drug market is expected to grow at a CAGR of approximately 4.2%, reaching USD 112 million by 2030. Riluzole is projected to maintain a significant share, driven by its status as the first-line treatment, with incremental growth from combination therapy approvals and expanded indications.

Future Outlook and Strategic Considerations

While riluzole's clinical efficacy remains modest, its enduring position as the first FDA-approved ALS therapy grants it sustained market relevance. Ongoing clinical research exploring next-generation combination therapies and personalized approaches could further enhance its therapeutic value. Moreover, increasing awareness and diagnosis rates globally reinforce the continued demand.

Pharmaceutical companies should focus on partnerships for combination treatments, expedite biomarker discovery for precision medicine, and advocate for expanded indication approvals to secure competitive advantage.

Key Takeaways

• Clinical Stability with Growth Potential: Riluzole remains clinically relevant, with ongoing trials aiming to optimize its use and explore new indications.
• Market Dynamics: The market, dominated by generics, faces pricing pressures but benefits from ongoing ALS incidence growth and regulatory incentives.
• Innovation Focus: Future growth hinges on combination therapies and personalized treatment strategies that can surpass riluzole's modest benefits.
• Regulatory and Geographic Expansion: Expanding approvals in broader neurodegenerative indications and emerging markets will support long-term demand.
• Strategic Opportunities: Companies investing in biomarker research, combination therapy development, and global market penetration are positioned for sustained growth.

FAQs

1. What is the current clinical indication for riluzole?
Riluzole is primarily indicated for the treatment of amyotrophic lateral sclerosis (ALS) to extend survival and delay disease progression.

2. Are there ongoing clinical trials to improve riluzole’s efficacy?
Yes, multiple trials are exploring combination therapies, dose optimization, and biomarker-guided personalized treatments aimed at enhancing clinical benefits.

3. How does riluzole’s market share compare globally?
While it remains the standard-of-care in many regions, patent expiry and generic versions have reduced its market share in some markets, though demand persists due to lack of better alternatives.

4. What are the main challenges facing riluzole’s market?
Limited efficacy, competition from emerging therapies, pricing pressures, and generic market entries are primary challenges.

5. Is riluzole being investigated for any other neurodegenerative diseases?
Preclinical studies and early-phase trials are investigating riluzole's potential in conditions like multiple sclerosis and spinal cord injuries, but these are not yet established indications.

References

[1] Excerpted from recent clinical trial registries and regulatory agency updates.
[2] Market data sourced from GlobalData and IQVIA reports.
[3] FDA and EMA approvals and indications.