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).
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.
NCT00202397 ↗	Effect of Riluzole as a Symptomatic Approach in Patients With Chronic Cerebellar Ataxia	Completed	S. Andrea Hospital	Phase 2	2005-06-01	Cerebellar disorders are often disabling and symptomatic therapies are limited to few options that are partially effective. It seems therefore appropriate to search for additional approaches. Purkinje cells are the sole output of the cerebellar cortex: they project inhibitory signals to the deep cerebellar nuclei (DCN), which have a critical role in cerebellar function and motor performance. DCN neurons fire spontaneously in the absence of synaptic input from Purkinje neurons and modulation of the DCN response by Purkinje input is believed to be responsible for coordination of movement. Recent evidences support the notion that an increase in DCN excitability may be an important step in the development of cerebellar ataxia and point to the underlying molecular mechanisms: the inhibition of small-conductance calcium-activated potassium (SK) channels, that causes an increase of the firing frequency in DCN, correlates with cerebellar ataxia. The rationale of the present project is that SK channel openers, such as riluzole, may have a beneficial effect on cerebellar ataxia. The researchers propose to perform a pilot study investigating safety and efficacy of riluzole, an approved treatment for amyotrophic lateral sclerosis, as a symptomatic approach in patients with chronic cerebellar ataxia.
NCT00211224 ↗	Neuroprotection and Natural History in Parkinson's Plus Syndromes (NNIPPS)	Terminated	Assistance Publique - Hôpitaux de Paris	Phase 3	2000-04-01	NNIPPS is a clinical trial of riluzole (a drug previously shown to slow down the rate of progression og amyotrophic lateral sclerosis-ALS; Lou Gehrig's disease) involving nearly 800 people diagnosed with the 'parkinson plus' syndromes of multiple system atrophy (MSA) and progressive supranuclear plasy (PSP). In addition to showing whether riluzole is helpful in MSA and PSP, NNIPPS will improve criteria for making an accurate and early diagnosis, for assessing the rate of progression, and will advance understanding of the biology of these disabling and progressive neurodegenerative diseases.

Clinical Trial Conditions for riluzole

Condition Name

Condition Name for riluzole
Intervention	Trials
Amyotrophic Lateral Sclerosis	56
Amyotrophic Lateral Sclerosis (ALS)	12
ALS	9
Obsessive-Compulsive Disorder	7

Condition MeSH

Condition MeSH for riluzole
Intervention	Trials
Amyotrophic Lateral Sclerosis	74
Motor Neuron Disease	67
Sclerosis	59
Disease	14

Clinical Trial Locations for riluzole

Trials by Country

Trials by Country for riluzole
Location	Trials
United States	313
Canada	52
Germany	41
Australia	21
United Kingdom	18

Trials by US State

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

Clinical Trial Progress for riluzole

Clinical Trial Phase

Clinical Trial Phase for riluzole
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	2
PHASE2	5

Clinical Trial Status

Clinical Trial Status for riluzole
Clinical Trial Phase	Trials
Completed	75
Recruiting	22
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	221
Industry	54
NIH	17

Riluzole Clinical Trials Update, Market Analysis, and Projection

Last updated: April 24, 2026

What is riluzole, and what does the clinical pipeline look like?

Riluzole is an oral glutamate-modulating therapy used for amyotrophic lateral sclerosis (ALS). Current commercial use is anchored to the established ALS indication; the meaningful “pipeline” for riluzole today is largely driven by (i) new formulations (to improve dosing or tolerability), (ii) new combinations (to address unmet efficacy gaps in ALS subtypes and progression patterns), and (iii) label-expansion attempts outside ALS.

Clinical development reality check (2018-2025): the riluzole “trial picture” is dominated by smaller, sponsor-specific studies and reformulation/adjunct research rather than large, late-stage registration programs typical of a pipeline franchise. The net effect for investors is that near-term incremental value creation tends to depend on incremental clinical signals rather than new global label expansions.

Recent and ongoing trial themes

Across publicly indexed trial registries and ALS-focused academic-industry collaborations, riluzole studies cluster into these categories:

• Combination trials in ALS: riluzole plus other agents targeting inflammation, glutamate homeostasis, mitochondrial pathways, or neuroprotection.
• Reformulation and dosing optimization: alternate release profiles, improved adherence, and tolerability.
• Biomarker-driven studies: neurofilament light (NfL), neuroimaging endpoints, and survival/progression proxies to reduce trial duration.
• Non-ALS exploration: studies outside ALS are present but rarely translate into major commercial endpoints.

Key read-through: if a sponsor does not have a clearly differentiated formulation or a combination with reproducible phase-level effect, the trial updates usually do not change the commercial curve materially. Riluzole remains a “core standard-of-care anchor” rather than a blockbuster-style growth story.

Which data points define the market for riluzole today?

Regulatory positioning and addressable use

• Indication anchor: ALS treatment (established standard of care in multiple geographies).
• Competitive context: riluzole competes primarily against other ALS disease-modifying and symptomatic regimens, and against payor preference for branded vs generic supply.

Supply and pricing dynamics

Riluzole is widely available as generic in multiple markets. That structure typically:

• compresses branded pricing,
• shifts commercial strategy to pack economics, channel contracting, and pharmacovigilance footprint,
• limits revenue growth unless a sponsor holds differentiation via formulation patents, branded exclusivity, or payer access advantages.

Market drivers

Riluzole demand is supported by:

• stable prevalence/incidence of ALS with ongoing diagnosis,
• guideline inclusion in ALS standard care,
• payer reimbursement for ALS therapies once patients meet diagnostic criteria.

Primary headwinds

• Generic competition in most developed markets.
• ALS drug category competition from therapies with differentiated efficacy or patient selection.
• Switching risk to other glutamate pathway agents or combination regimens that payors prefer.

How should investors project riluzole revenue and utilization through the next cycle?

Projection framework

For riluzole, revenue projection is more sensitive to:

1. price erosion rate (generic penetration and channel pricing),
2. treatment persistence (patients remain on therapy as long as tolerated and as long as clinicians keep it),
3. net ALS treated population (diagnosis and survival trends),
4. share of differentiated supply (if any patent-protected formulation exists or if a branded product maintains payer access).

Base case projection (structure, not guesswork)

Because riluzole is widely generic, the market typically behaves like a commodity within ALS standard care:

• Utilization follows ALS diagnosis volumes and persistence.
• Revenue follows a decline-to-flat path as unit prices fall, then stabilizes when pricing floors and reimbursement anchor channels form.

A credible projection therefore models riluzole revenues as:

• Revenue = (ALS treated patients on riluzole) x (net price per patient-day or per pack)
• Net price trends: dominated by generics in most markets, with potential stabilization where branded generics dominate contracts.

Scenario outcomes

• Bull case (value upside):
  • sustained share due to improved formulation or demonstrably better tolerability,
  • payer preference from contracting advantages,
  • incremental clinical adoption via combination guidance.
• Base case:
  • continued volume stability with ongoing price erosion,
  • incremental growth limited to net treated population changes.
• Bear case:
  • accelerated replacement by newer ALS standards in key geographies,
  • sharper unit price compression as more low-cost suppliers enter or expand coverage.

What changes the curve most?

The projection meaningfully changes only if riluzole gains any of the following:

• a new validated clinical positioning that changes guideline placement (beyond existing ALS use),
• a new formulation with a defensible regulatory and commercial moat (not only reformulation),
• a combination that becomes standard-of-care through evidence strong enough to move practice and payor behavior.

Absent such events, the market curve remains largely persistence-driven.

Where does riluzole sit in the ALS competitive landscape?

Positioning

Riluzole’s competitive role is:

• foundational therapy in ALS medication schemas,
• part of early disease management logic,
• often continued alongside other agents depending on patient profile and clinician practice.

Competitive pressure

• Other ALS therapies that show differentiated outcomes can shift initiation and continuation behavior.
• Payors may steer patients to preferred agents based on cost-effectiveness, evidence, and budget impact.

Implication for market projection: riluzole is less exposed to “trial-risk volatility” than novel ALS assets. The risk for riluzole is mainly economic substitution rather than clinical failure.

Clinical trials update: what matters for near-term decisions?

For business and investment decisions, the near-term questions are not “is riluzole studied,” but whether trial updates show:

• a new endpoint signal strong enough to shift practice,
• a phase-to-phase reproducibility pattern in combination regimens,
• a regulatory path that plausibly leads to label updates or payer-relevant differentiation.

Decision-grade checkpoints

• Any late-stage trial that demonstrates a clinically meaningful outcome versus best supportive care when used as a backbone.
• Any reformulation that shows better adherence or tolerability leading to lower discontinuation.
• Any evidence that changes treatment sequencing (initiation timing, add-on behavior, or continuation criteria).

Market analysis summary by region

United States

• Generic availability is the dominant economic feature.
• Revenue is primarily driven by treatment persistence and contracted supply economics.
• Growth comes from stable treated population rather than pricing.

Europe

• Similar generic dynamics.
• Pricing and reimbursement are shaped by national health systems, which tend to favor lower-cost supply once generic entry is established.

Rest of world

• Generic penetration varies by country.
• Some markets retain branded share for longer periods due to slower entry, tender structures, and reimbursement access.

Quantitative snapshot: what to use for modeling

Because the request requires a clinical trials update and a market analysis/projection, the modeling inputs that typically control outcomes are:

• Patient counts: ALS treated population and switching rates to other ALS standards.
• Persistence: discontinuation trends due to disease progression, tolerability, and clinician practice.
• Price: net price per pack (or per mg-day equivalent), including discounts and reimbursement.
• Supply structure: number of generic entrants and contract concentration.
• Regulatory events: approvals for any new formulation or label expansion.

Key Takeaways

• Riluzole remains anchored to long-established ALS use, and today’s trial activity is weighted toward combinations, biomarkers, and reformulation rather than major registration programs that re-rate the franchise.
• The market is structurally generic-driven, so revenue projection hinges on persistence and ALS treated volumes more than on clinical upside.
• Near-term value creation requires evidence that shifts guideline practice or payer behavior, typically via a differentiated formulation or a clearly superior combination backbone.

FAQs

1) Is riluzole still being actively studied in ALS?

Yes. Trial activity continues, with most programs focusing on combination regimens, biomarkers, and dosing/formulation optimization.

2) What is the biggest determinant of riluzole revenue in mature markets?

Net price erosion from generic competition and treatment persistence among ALS patients.

3) What trial results would most change riluzole’s market trajectory?

Late-stage evidence that changes standard-of-care sequencing or increases continuation rates due to improved clinical outcomes or tolerability.

4) Does riluzole face “patent-style” risk like novel ALS assets?

The primary risk is economic substitution by other ALS standards and payor preferred agents, with incremental patent effects depending on any reformulation differentiation.

5) Is non-ALS expansion a near-term market driver?

Non-ALS programs exist, but they rarely translate into large commercial shifts unless they produce clear, regulatory-grade outcomes that change labeling and prescribing patterns.

References

[1] ClinicalTrials.gov. “Riluzole” (search results and study records). U.S. National Library of Medicine.
[2] FDA. Drug labels and pharmacology reviews for riluzole-containing products (as applicable). U.S. Food and Drug Administration.
[3] EMA. European public assessment reports and product information for riluzole-containing products (as applicable). European Medicines Agency.