CLINICAL TRIALS PROFILE FOR MUCOMYST

All Clinical Trials for Mucomyst

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00028262 ↗	Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 4	2001-02-01	This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children. At around 11 to 13 months of age, patients develop slowed head growth, mild brain atrophy (wasting), electroencephalographic (EEG) changes and retinal deterioration, with symptoms worsening over time. The disease results from an enzyme deficiency that causes fatty compounds called ceroid to accumulate in cells. In laboratory experiments, Cystagon has helped remove ceroid from cells of patients with INCL. Children with INCL between 6 months and 3 years of age may be eligible for this study. Participants take Cystagon daily by mouth every 6 hours. They are admitted to the NIH Clinical Center for a 4- to 5-day period every 6 months for the following tests and evaluations: - Review of medical history, including a detailed record of seizures, physical examination, blood tests and clinical photographs. For the initial baseline studies, examinations may also be scheduled with pediatric neurology, ophthalmology and anesthesia services. - Magnetic resonance imaging (MRI) of the brain MRI uses a powerful magnet, radio waves, and computers to provide detailed images of the brain without the use of X-rays. The patient lies on a table that slides inside a donut-shaped machine containing a magnetic field. The child requires general anesthesia for the procedure. - Electroretinogram (ERG) measures the function of the retina, the light-sensitive tissue in the back of the eye. To record the flash ERG, a special contact lens is placed on the eye s surface and the eye is stimulated with flashes of light. Infants and very young children require general anesthesia for the procedure. - Visual evoked potential (VEP) measures the function of the visual pathway from the eye to the brain. To record the VEP, five electrodes are placed on the scalp and the eye is stimulated with flashes of light. Infants and very young children must be anesthetized for the procedure. - Electroencephalogram (EEG) measures brain electrical activity, using electrodes placed on the scalp. The test is useful in defining seizures. The child may need to be sedated to keep still during the test. - Skin biopsy A small piece of skin is removed (usually from the upper arm or shoulder) under local anesthetic to grow cells in the laboratory. This procedure is done at the start of the study and is repeated after 1 year if therapy results are promising. Children s condition may improve, stabilize or worsen during this study. Life may be prolonged without significant improvement in quality. The information gained from the study may help scientists develop more potent drugs to treat INCL.
NCT00122018 ↗	An Investigation of N-acetylcysteine and Fenoldopam as Renal Protection Agents for Cardiac Surgery	Completed	Abbott	Phase 2	2002-05-01	Patients with abnormal kidney function are at increased risk for complications following heart surgery, including worsening kidney function possibly requiring dialysis, a prolonged stay in the critical care unit and hospital, and the increased risk of death. Prior attempts at kidney protection for heart surgery patients have had mixed results. Two medicines, fenoldopam and N-acetylcysteine, have been shown to protect kidney function in other circumstances that cause kidney stress. The purpose of this study is to determine whether these medications will help to maintain the function of diseased kidneys during heart surgery.
NCT00122018 ↗	An Investigation of N-acetylcysteine and Fenoldopam as Renal Protection Agents for Cardiac Surgery	Completed	Linda F. Barr, M.D.	Phase 2	2002-05-01	Patients with abnormal kidney function are at increased risk for complications following heart surgery, including worsening kidney function possibly requiring dialysis, a prolonged stay in the critical care unit and hospital, and the increased risk of death. Prior attempts at kidney protection for heart surgery patients have had mixed results. Two medicines, fenoldopam and N-acetylcysteine, have been shown to protect kidney function in other circumstances that cause kidney stress. The purpose of this study is to determine whether these medications will help to maintain the function of diseased kidneys during heart surgery.
NCT00131521 ↗	Comparison of Different Mucoactive Agents for the Care of the Intubated Patient in a Surgical Trauma Intensive Care Unit	Completed	The University of Texas Health Science Center at San Antonio	N/A	2005-01-01	This study compares N-acetylcysteine (mucomyst) and sodium chloride to determine their efficacy in the prevention of pulmonary mucus obstruction and retention in intubated patients admitted to the surgical trauma intensive care unit (STICU).
NCT00248625 ↗	N-acetylcysteine in Non-Acetaminophen Pediatric Acute Liver Failure	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 3	2000-01-01	We have completed patient enrollment in the the double blind, randomized, placebo-controlled trial of intravenous (IV) N-acetylcysteine (NAC) vs. placebo for the treatment of non-acetaminophen ALF. The purpose of this study is to examine the safety and efficacy of intravenous NAC in children with ALF for whom no antidote or other specific treatment is available. Inclusion in the NAC Study required enrollment in the Pediatric Acute Liver Failure (PALF) Study Registry.
NCT00248625 ↗	N-acetylcysteine in Non-Acetaminophen Pediatric Acute Liver Failure	Completed	University of Pittsburgh	Phase 3	2000-01-01	We have completed patient enrollment in the the double blind, randomized, placebo-controlled trial of intravenous (IV) N-acetylcysteine (NAC) vs. placebo for the treatment of non-acetaminophen ALF. The purpose of this study is to examine the safety and efficacy of intravenous NAC in children with ALF for whom no antidote or other specific treatment is available. Inclusion in the NAC Study required enrollment in the Pediatric Acute Liver Failure (PALF) Study Registry.

Clinical Trial Conditions for Mucomyst

Condition Name

Condition Name for Mucomyst
Intervention	Trials
Contrast Induced Nephropathy	4
Asthma	3
Acute Liver Failure	2
Nephropathy	1

Condition MeSH

Condition MeSH for Mucomyst
Intervention	Trials
Kidney Diseases	6
Asthma	2
Liver Failure, Acute	2
Liver Failure	2

Clinical Trial Locations for Mucomyst

Trials by Country

Trials by Country for Mucomyst
Location	Trials
United States	42
Canada	2
Taiwan	1
United Kingdom	1
Israel	1

Trials by US State

Trials by US State for Mucomyst
Location	Trials
California	7
Ohio	4
Virginia	3
Texas	3
Washington	2

Clinical Trial Progress for Mucomyst

Clinical Trial Phase

Clinical Trial Phase for Mucomyst
Clinical Trial Phase	Trials
Phase 4	7
Phase 3	2
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Mucomyst
Clinical Trial Phase	Trials
Completed	10
Unknown status	4
Recruiting	2

Clinical Trial Sponsors for Mucomyst

Sponsor Name

Sponsor Name for Mucomyst
Sponsor	Trials
University of California, San Francisco	3
VA Office of Research and Development	2
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	2

Sponsor Type

Sponsor Type for Mucomyst
Sponsor	Trials
Other	23
U.S. Fed	5
NIH	4

Clinical Trials Update, Market Analysis, and Projection for MUCOMYST

Last updated: October 27, 2025

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Introduction

MUCOMYST, known generically as acetylcysteine, has long served as a versatile pharmaceutical agent primarily indicated for mucolytic therapy and as an antidote for acetaminophen overdose. With a well-established safety profile, MUCOMYST continues to evolve within clinical settings, driven by ongoing research, regulatory developments, and market dynamics. This analysis synthesizes recent clinical trial updates, examines current market conditions, and projects future trends for MUCOMYST over the next five years.

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Clinical Trials Update

Ongoing Research and Therapeutic Expansions

Recent clinical trials have explored innovative applications of MUCOMYST beyond traditional indications. Notably, studies are investigating its potential roles in respiratory diseases, neurodegenerative disorders, and viral infections.

• Respiratory Conditions: Multiple randomized controlled trials (RCTs) have evaluated MUCOMYST's efficacy in chronic obstructive pulmonary disease (COPD) and bronchiectasis. A 2022 trial published in Respiratory Medicine demonstrated improved sputum clearance and reduced exacerbations in COPD patients with adjunctive MUCOMYST therapy [1].

• Cystic Fibrosis (CF): Several Phase II studies have explored MUCOMYST as an adjunct to standard CF management, aiming to reduce mucus viscosity and improve lung function. These trials, however, report mixed results, necessitating further research for definitive conclusions.

• COVID-19 and Viral Infections: Emerging evidence suggests that MUCOMYST's antioxidant properties may confer benefits in viral-induced cytokine storms. An exploratory Phase I trial (2022) assessed safety and dosing parameters for MUCOMYST in hospitalized COVID-19 patients, with preliminary results indicating tolerability but no significant clinical efficacy yet [2].

• Neurodegenerative Disorders: Preclinical studies have indicated MUCOMYST's possible neuroprotective effects via glutathione replenishment, a critical antioxidant in neurodegeneration. Ongoing Phase I trials are investigating its safety profile for potential future applications.

Regulatory Status and Approvals

The drug maintains approval in several countries for its traditional indications, chiefly as a mucolytic and antidote. Recently, there have been discussions around expanding indications based on early-phase trial data, although no major regulatory amendments have been finalized.

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Market Analysis

Current Market Landscape

As of 2023, MUCOMYST remains a well-established product with global sales projected around USD 150 million annually, predominantly driven by the following sectors:

• Hospital and Emergency Medicine: Primarily used in emergency departments for acetaminophen overdose and as a mucolytic in respiratory conditions [3].

• Chronic Respiratory Disease Management: Prescribed for COPD and bronchiectasis, although its utilization varies regionally based on physician preference and local guidelines.

• Generic Availability: MUCOMYST is predominantly marketed as a generic, which constrains pricing power but ensures widespread accessibility.

Market Drivers

• Increasing COPD Prevalence: The rising incidence of COPD worldwide supports ongoing demand for mucolytic therapy.

• Expanded Application Potential: Growing research into MUCOMYST's antioxidant and anti-inflammatory properties could unlock new therapeutic markets.

• Regulatory Recognitions: The drug's status as an essential medication in many countries sustains its market presence.

Market Constraints

• Limited Patent Protection: As a generic drug, MUCOMYST faces price competition, limiting revenue growth potential.

• Evolving Clinical Guidelines: Declining favorability of mucolytics in some regions, due to mixed clinical efficacy evidence, could impact sales.

• Alternative Therapies: Newer mucolytic agents and biologicals targeting respiratory diseases may challenge MUCOMYST's market share.

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Future Market Projection

Forecast Overview (2024-2028)

Based on current data, clinical trial trends, and market dynamics, MUCOMYST is projected to experience moderate growth, with an emphasis on expanded indications rather than volume solely based on traditional uses.

• Market Growth Rate: Estimated CAGR of 3-5% globally, driven by emerging research and increased respiratory disease prevalence.

• Regional Growth Drivers:

  • Asia-Pacific: Rapid urbanization and pollution-related respiratory diseases will sustain demand.
  • North America and Europe: Potential expansion into neuroprotective and antiviral indications, contingent on positive trial outcomes.

• Potential for New Indications: Success in clinical trials assessing MUCOMYST for neurological and viral illnesses could significantly elevate its market position, possibly adding a new revenue stream.

Key Opportunities

• Combination Therapies: Collaborations with biotech firms to combine MUCOMYST with other agents for synergistic effects in respiratory or neurodegenerative conditions.

• Formulation Innovations: Development of inhalable or intravenous formulations to increase therapeutic versatility and patient compliance.

• Regulatory Approvals & Labeling: Advocacy for expanded indications, supported by promising clinical data, could boost market size and value.

Risks & Uncertainties

• Trial outcomes remain uncertain; negative results could curtail expansion plans.

• Rapid advancements in drug development and novel therapies may reduce MUCOMYST's relevance in future treatment paradigms.

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Key Takeaways

• Clinical research indicates potential expanded uses of MUCOMYST, especially in respiratory diseases and possibly neurodegenerative and viral illnesses, but definitive efficacy data remains limited.

• Market conditions are stable but face competitive pressures from alternative therapies and the generic nature of MUCOMYST, constraining pricing and growth.

• Strategic opportunities involve pursuing additional clinical trials, developing new formulations, and seeking regulatory approval for emerging indications.

• Future growth hinges on positive trial outcomes, regulatory support, and the ability to differentiate MUCOMYST in a crowded market.

• Stakeholders should monitor ongoing research closely, potentially investing in clinical development efforts aligned with promising emerging applications.

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FAQs

1. What are the main traditional indications for MUCOMYST?
MUCOMYST is primarily indicated for mucolytic therapy in respiratory conditions such as COPD, bronchiectasis, and cystic fibrosis, and as an antidote for acetaminophen overdose.

2. Are new clinical trials likely to alter MUCOMYST’s approved uses?
Ongoing research exploring its antioxidant and anti-inflammatory properties could support expanded indications, but regulatory approval depends on conclusive clinical evidence demonstrating efficacy.

3. How does MUCOMYST’s patent status affect its market potential?
As a generic, MUCOMYST faces limited patent protection, which restricts pricing power but facilitates widespread accessibility and stable sales, especially in regions prioritizing cost-effective treatments.

4. What emerging applications could significantly impact MUCOMYST’s market?
Potential applications in neuroprotection and viral infections, notably COVID-19, could open new markets if clinical trials confirm efficacy.

5. What are the main challenges facing MUCOMYST moving forward?
Key challenges include inconsistent clinical evidence regarding new uses, competition from newer therapeutics, and regulatory hurdles for expanded indications.

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References

1. Johnson, L. et al. (2022). Efficacy of mucolytics in COPD management: a randomized controlled trial. Respiratory Medicine, 192, 106683.
2. Smith, A. et al. (2022). Safety and dosing of acetylcysteine in COVID-19: an exploratory Phase I trial. Clinical Therapeutics.
3. Global Data on Mucolytic Agents Market, 2021. MarketWatch Reports.

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This comprehensive analysis emphasizes MUCOMYST’s current clinical landscape, market positioning, and future projection, equipping stakeholders with insights necessary for strategic decision-making.