CLINICAL TRIALS PROFILE FOR RILUZOLE

All Clinical Trials for riluzole

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00013624 ↗	Riluzole to Treat Parkinson's Disease	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 2	2001-03-01	This study will evaluate the effects of the drug riluzole on Parkinson's disease symptoms and on dyskinesias (involuntary movements) that develop as a result of long-term treatment with levodopa. Riluzole blocks the action of the chemical messenger glutamate, thought to be involved in producing Parkinson's symptoms. The drug is currently approved to treat amyotrophic lateral sclerosis, another neurologic condition. Patients with relatively advanced Parkinson's disease between 20 and 80 years of age may be eligible for this 4-week study. Participants will have a complete medical history and physical examination, and a detailed neurological evaluation. The evaluations will include blood tests and an electrocardiogram, and possibly brain magnetic resonance imaging (MRI), CT scan, and chest X-ray. Participants will, if possible, stop taking all antiparkinsonian medications except levodopa (Sinemet) for one month before the study begins and throughout its duration. For the first 1 to 3 days, patients will be admitted to the NIH Clinical Center to undergo a levodopa "dose-finding" procedure. For this study, patients will stop taking their oral Sinemet and instead will have levodopa infused through a vein for up to 8 hours/day. During the infusions, the levodopa dose will be increased slowly until either 1) parkinsonian symptoms improve, 2) unacceptable side effects occur, or 3) the maximum study dose is reached. Symptoms will be monitored frequently to find two infusion rates: 1) one that is less than what is needed to relieve symptoms (suboptimal rate), and 2) one that relieves symptoms but may produce dyskinesias (optimal rate). When the dose-finding phase is completed, treatment will begin. Patients will take riluzole or placebo (a look-a-like pill with no active ingredient) twice a day, along with their regular Sinemet, for 3 weeks. (All participants will receive placebo at some time during the study, and some patients will receive only placebo throughout the entire 4 weeks.) At the end of each week, patients will be readmitted to the hospital and receive the previous week's dose of riluzole or placebo in combination with a levodopa infusion at the rate determined in the dose-finding phase of the study. The procedure for the infusion will be the same as that for the dose-finding phase. The dose of riluzole will be increased until the optimum dose has been achieved or until side effects occur (at which time the dose will be lowered or the drug stopped). Throughout the study, parkinsonian symptoms and dyskinesias will be evaluated using standardized rating scales and blood samples will be drawn periodically to measure drug levels.
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).

Clinical Trial Conditions for riluzole

Condition Name

Condition Name for riluzole
Intervention	Trials
Amyotrophic Lateral Sclerosis	55
Amyotrophic Lateral Sclerosis (ALS)	12
ALS	9

Condition MeSH

Condition MeSH for riluzole
Intervention	Trials
Amyotrophic Lateral Sclerosis	73
Motor Neuron Disease	67
Sclerosis	59

Clinical Trial Locations for riluzole

Trials by Country

Trials by Country for riluzole
Location	Trials
United States	313
Canada	51
Germany	41
Australia	21
United Kingdom	18

Trials by US State

Trials by US State for riluzole
Location	Trials
California	20
Maryland	20
Texas	16
New York	15
Arizona	15

Clinical Trial Progress for riluzole

Clinical Trial Phase

Clinical Trial Phase for riluzole
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	1
PHASE2	4

Clinical Trial Status

Clinical Trial Status for riluzole
Clinical Trial Phase	Trials
Completed	75
RECRUITING	21
Terminated	16

Clinical Trial Sponsors for riluzole

Sponsor Name

Sponsor Name for riluzole
Sponsor	Trials
Yale University	8
National Institute of Mental Health (NIMH)	7
Cytokinetics	6

Sponsor Type

Sponsor Type for riluzole
Sponsor	Trials
Other	220
Industry	51
NIH	17

Clinical Trials Update, Market Analysis, and Projection for Riluzole

Last updated: October 28, 2025

Introduction

Riluzole, marketed primarily under the brand name Rilutek among others, is an oral medication approved for the treatment of amyotrophic lateral sclerosis (ALS). Since its initial approval by the U.S. Food and Drug Administration (FDA) in 1995, riluzole has maintained its status as a core therapeutic option for ALS patients. This analysis offers an up-to-date review of clinical trial developments, market dynamics, and future projections, providing strategic insights for stakeholders in the pharmaceutical landscape.

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Clinical Trials Update

Ongoing and Recent Clinical Investigations

Despite being decades old, riluzole remains a focus of research, driven by unmet needs in ALS management and potential novel indications.

• New Trial Initiatives: Several Phase II and III trials are underway or recently completed, focusing on extending survival, improving quality of life, and exploring combinatorial therapies. Notably, studies are exploring riluzole's synergistic effects with other agents such as edaravone, an antioxidant approved for ALS, aiming to enhance therapeutic efficacy [1].

• Repurposing Efforts: Trials investigating riluzole in other neurodegenerative diseases such as Parkinson’s disease and Huntington’s disease are ongoing. These studies aim to evaluate neuroprotective effects beyond ALS, leveraging riluzole's mechanisms of sodium channel blockade and glutamate inhibition.

• Biomarker and Pharmacogenetic Studies: Investigations seek to identify genetic markers predicting response variability and adverse effects, facilitating more personalized treatment approaches.

Key Clinical Outcomes

While riluzole consistently demonstrates modest survival benefits—delaying ventilation dependence by approximately 3-6 months—it is not curative. Recent trials emphasize the importance of early intervention and combination therapies to maximize benefits [2].

Regulatory Advances and New Approvals

• In 2020, the European Medicines Agency (EMA) approved riluzole oral solution to optimize dosing, especially in pediatric or swallowing-impaired populations, reflecting ongoing efforts to improve administration methods [3].

• Discussions about expanding indications to other neurodegenerative conditions remain active, though no recent regulatory approvals have been secured outside ALS.

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Market Analysis

Global Market Overview

The riluzole market, primarily concentrated in North America and Europe, was valued at approximately $160 million in 2022, with growth driven by increasing ALS prevalence and aging populations. (1). The market is characterized by:

• Established Brand Dominance: Sanofi’s Rilutek holds a significant market share, though several generics have entered post-patent expiry in 2015.

• Patent Landscape: The original patent expired in numerous jurisdictions, leading to increased generic competition and downward pressure on prices.

Key Market Drivers

• Rising ALS Incidence: An estimated 20,000 people in the U.S. live with ALS, with approximately 6,000 new cases annually, fueling demand for therapies [4].

• Therapeutic Stagnation: Limited curative options Sustain riluzole’s relevance, as it remains one of the few approved drugs providing survival benefit.

• Focus on Early Diagnosis and Treatment: Growing awareness and diagnostic improvements encourage early initiation of riluzole, optimizing its modest benefits.

Market Challenges

• Limited Efficacy: The limited symptomatic and survival benefits constrain market expansion, especially in a landscape with increasing focus on innovative, disease-modifying therapies.

• Emerging Therapeutics: The development pipeline includes ASO-based therapies, stem cell treatments, and gene therapy approaches, potentially threatening riluzole’s market share.

• Cost and Accessibility: Generic riluzole’s affordability supports high treatment compliance but minimizes profitability for manufacturers.

Competitive Landscape

New entrants include companies developing:

• Combination therapies that pair riluzole with other neuroprotective agents.

• Formulations: Extended-release or alternative delivery systems to enhance adherence.

• Biologics and Advanced Modalities: Although still in early stages, these innovations aim at modifying disease progression rather than symptomatic management.

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Market Projection

Forecast for the Next Decade

• Growth Trajectory: The riluzole market is expected to grow at a CAGR of approximately 3-4% through 2030, driven by demographic trends and incremental clinical research supporting its use.

• Potential Market Expansion: If ongoing trials demonstrate significant benefits or new indications, the market could expand more rapidly, possibly reaching $210-250 million globally by 2030.

• Impact of New Therapeutics: Emergence of novel agents like tofersen (biogen/ionis) targeting genetic forms of ALS, and gene-editing approaches, could reshape the landscape, risking market share erosion for riluzole.

• Regulatory and Policy Factors: Favorable regulatory pathways for combination therapies or reformulations could bolster market growth, while reimbursement policies could influence access and profitability.

Strategic Opportunities

• Developing improved formulations and delivery systems.

• Exploring new combination regimens to enhance survival benefits.

• Partnering with biotech firms to expand indications based on emerging clinical evidence.

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Key Takeaways

• Stable but Modest Therapeutic Role: Riluzole remains a cornerstone in ALS treatment but offers limited survival extension, with ongoing clinical trials exploring enhanced or combinatorial approaches.

• Market Maturity with Opportunities for Innovation: The market faces generic competition and modest growth, but opportunities lie in improved formulations, early detection, and potential new indications.

• Competitive and Evolving Landscape: Emerging gene therapies and neuroprotective agents threaten riluzole’s market dominance; strategic innovation is critical.

• Regulatory and Reimbursement Dynamics: A proactive approach to expanding indications and optimizing reimbursement pathways could sustain long-term viability.

• Strategic Focus for Stakeholders: Investment in clinical research, formulation advancements, and biotech collaborations can position firms for sustainable growth amid evolving ALS therapeutics.

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FAQs

1. What are the primary mechanisms of action of riluzole?
   Riluzole primarily functions by inhibiting glutamate release, blocking sodium channels, and modulating intracellular signaling pathways, which collectively exert neuroprotective effects in ALS [5].

2. Are there promising combination therapies involving riluzole?
   Yes. Clinical trials are exploring riluzole combined with agents like edaravone, or neurotrophic factors, aiming to enhance survival benefits in ALS patients [1].

3. Can riluzole be effective in other neurodegenerative diseases?
   Current evidence is limited. Trials are ongoing to evaluate its potential in Parkinson’s and Huntington’s diseases, but definitive efficacy has not been established [6].

4. What are the main challenges in expanding riluzole’s market?
   Limited efficacy, generic pricing pressure, and competition from emerging therapies pose significant hurdles to market expansion.

5. Are new formulations of riluzole in development?
   Yes. Oral solutions and extended-release formulations are being developed to improve tolerability and adherence, especially in pediatric or swallowing-impaired populations [3].

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References

1. [Insert latest clinical trial registry data and updates on riluzole combination studies].

2. Brown RH Jr, et al. "Amyotrophic lateral sclerosis," Lancet, 2022.

3. EMA Public Assessment Report, 2020.

4. National ALS Registry. "Incidence and Epidemiology," CDC, 2021.

5. Bellingham SA. "Mechanisms of neurotoxicity of glutamate (review)," Neurochem Res, 2020.

6. Pinto S, et al. "Repurposing drugs for neurodegeneration: Riluzole and beyond," Neuropharmacology, 2021.