CLINICAL TRIALS PROFILE FOR ACETYLCYSTEINE

505(b)(2) Clinical Trials for ACETYLCYSTEINE

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
OTC	NCT04627922 ↗	N-Acetylcysteine for Smoking Cessation in Tobacco and Cannabis Co-Use	Recruiting	Tobacco Related Disease Research Program	Phase 4	2021-08-25	Tobacco and cannabis co-use is a common and growing public health problem, especially in states that have legalized cannabis. There are no pharmacologic treatments for co-occurring tobacco and cannabis use. Co-use may make quitting either substance more difficult, given the synergistic effects of cannabis and nicotine on neurobiological systems that mediate reward and shared cues reinforcing co-use. N-acetylcysteine (NAC), an FDA-approved medication and over-the-counter supplement, has shown promise in animal studies and randomized controlled trials (RCTs) in reducing tobacco and cannabis craving and use.
OTC	NCT03238300 ↗	Neuroscience-Informed Treatment Development for Adolescent Alcohol Use	Recruiting	National Institute on Alcohol Abuse and Alcoholism (NIAAA)	Phase 2	2017-10-16	This study will examine the effect of N-Acetylcysteine (NAC), an over-the-counter antioxidant supplement, on brains of youth (ages 15-19) using magnetic resonance imaging (MRI). 55 adolescents will receive, in a counterbalanced order, a 10-day course of NAC 1200 mg twice daily and a subsequent 10-day course of matched placebo twice daily, separated by 11 days. Urine and blood samples will be collected at baseline and urine samples again before and after each course of medication treatment. Participants will receive a 1- hour MRI scan at baseline and after each treatment trial.
OTC	NCT03238300 ↗	Neuroscience-Informed Treatment Development for Adolescent Alcohol Use	Recruiting	Medical University of South Carolina	Phase 2	2017-10-16	This study will examine the effect of N-Acetylcysteine (NAC), an over-the-counter antioxidant supplement, on brains of youth (ages 15-19) using magnetic resonance imaging (MRI). 55 adolescents will receive, in a counterbalanced order, a 10-day course of NAC 1200 mg twice daily and a subsequent 10-day course of matched placebo twice daily, separated by 11 days. Urine and blood samples will be collected at baseline and urine samples again before and after each course of medication treatment. Participants will receive a 1- hour MRI scan at baseline and after each treatment trial.
OTC	NCT01241513 ↗	Induced Changes in Ventilatory Responsiveness and Altitude Exposure	Terminated	United States Army Research Institute of Environmental Medicine	Phase 4	2010-11-01	The main purpose of this study is to determine if a drug (acetyl-cysteine or ACCY) can increase the amount of oxygen in your body at a high altitude of 11,500 feet. ACCY is approved by the Food and Drug Administration (FDA) as a treatment or antidote for Tylenol overdoses. Other forms of ACCY are also sold over-the-counter as nutritional supplements. In this study, the FDA-approved form of ACCY will be used "off-label" (meaning in a way not approved by the FDA). This study is being conducted by researchers from the United States Army Research Institute of Environmental Medicine (USARIEM). The study will take place in the Altitude Chamber located in the basement of USARIEM. A total of approximately 30 volunteers (men and women, military and civilians) will take part in the study. They can expect to be in the study for a minimum of a few hours each day for two weeks. The investigators hypothesize that ACCY will improve ventilation and oxygenation while at altitude.
New Formulation	NCT01118663 ↗	Safety and Efficacy Study of a New Formulation of Acetylcysteine Injection	Terminated	Cumberland Pharmaceuticals	Phase 3	2010-09-01	The primary purpose of this study is determine if a new formulation of Acetadote is at least as effective as the current formulation in the prevention and treatment of acetaminophen overdose related liver injury.
OTC	NCT01005810 ↗	A Trial of N-Acetylcysteine (an Over-the-Counter Medicine) in Adolescents Who Smoke Marijuana	Completed	National Institute on Drug Abuse (NIDA)	Phase 2	2009-09-01	This study is investigating how N-Acetylcysteine (NAC), an over-the-counter medication, will reduce marijuana use when combined with Contingency Management, a behavioral treatment. It is hypothesized that marijuana dependent adolescents who are treated with NAC will use less marijuana during treatment when compared to adolescents who receive a placebo.
OTC	NCT01005810 ↗	A Trial of N-Acetylcysteine (an Over-the-Counter Medicine) in Adolescents Who Smoke Marijuana	Completed	Medical University of South Carolina	Phase 2	2009-09-01	This study is investigating how N-Acetylcysteine (NAC), an over-the-counter medication, will reduce marijuana use when combined with Contingency Management, a behavioral treatment. It is hypothesized that marijuana dependent adolescents who are treated with NAC will use less marijuana during treatment when compared to adolescents who receive a placebo.

All Clinical Trials for ACETYLCYSTEINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
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.
NCT00004467 ↗	Randomized Study of Acetylcysteine in Patients With Acute Liver Failure Not Caused by Acetaminophen	Completed	University of Texas	Phase 3	1998-06-01	OBJECTIVES: I. Determine the safety and efficacy of a short course (72 hours) of intravenous acetylcysteine in patients with acute liver failure for whom no antidote or specific treatment is available.
NCT00004467 ↗	Randomized Study of Acetylcysteine in Patients With Acute Liver Failure Not Caused by Acetaminophen	Completed	University of Texas Southwestern Medical Center	Phase 3	1998-06-01	OBJECTIVES: I. Determine the safety and efficacy of a short course (72 hours) of intravenous acetylcysteine in patients with acute liver failure for whom no antidote or specific treatment is available.
NCT00004467 ↗	Randomized Study of Acetylcysteine in Patients With Acute Liver Failure Not Caused by Acetaminophen	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 3	1998-06-01	OBJECTIVES: I. Determine the safety and efficacy of a short course (72 hours) of intravenous acetylcysteine in patients with acute liver failure for whom no antidote or specific treatment is available.
NCT00003346 ↗	Combination Chemotherapy in Treating Patients With Stage III or Stage IV Melanoma	Completed	National Cancer Institute (NCI)	Phase 2	1997-11-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase I/II trial to study the effectiveness of combination chemotherapy consisting of acetaminophen plus carmustine in treating patients who have stage III or stage IV melanoma.
NCT00003346 ↗	Combination Chemotherapy in Treating Patients With Stage III or Stage IV Melanoma	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	1997-11-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase I/II trial to study the effectiveness of combination chemotherapy consisting of acetaminophen plus carmustine in treating patients who have stage III or stage IV melanoma.

Clinical Trial Conditions for ACETYLCYSTEINE

Condition Name

Condition Name for ACETYLCYSTEINE
Intervention	Trials
Oxidative Stress	9
Chronic Kidney Disease	8
Contrast Induced Nephropathy	8
COVID-19	7

Condition MeSH

Condition MeSH for ACETYLCYSTEINE
Intervention	Trials
Kidney Diseases	40
Renal Insufficiency	28
Disease	25
Acute Kidney Injury	22

Clinical Trial Locations for ACETYLCYSTEINE

Trials by Country

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−

Trials by Country for ACETYLCYSTEINE
Location	Trials
United States	312
Italy	21
Canada	20
Brazil	18
Netherlands	15

Trials by US State

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−

Trials by US State for ACETYLCYSTEINE
Location	Trials
California	27
South Carolina	27
New York	19
Texas	19
Minnesota	18

Clinical Trial Progress for ACETYLCYSTEINE

Clinical Trial Phase

Clinical Trial Phase for ACETYLCYSTEINE
Clinical Trial Phase	Trials
Phase 4	76
Phase 3	56
Phase 2/Phase 3	27

Clinical Trial Status

Clinical Trial Status for ACETYLCYSTEINE
Clinical Trial Phase	Trials
Completed	192
Recruiting	54
Unknown status	53

Clinical Trial Sponsors for ACETYLCYSTEINE

Sponsor Name

Sponsor Name for ACETYLCYSTEINE
Sponsor	Trials
Medical University of South Carolina	21
Yale University	11
National Institute on Drug Abuse (NIDA)	10

Sponsor Type

Sponsor Type for ACETYLCYSTEINE
Sponsor	Trials
Other	619
NIH	55
Industry	51

Acetylcysteine: Clinical Trials, Market Analysis, and Projections

Introduction to Acetylcysteine

Acetylcysteine, also known as N-acetylcysteine (NAC), is a versatile drug with a wide range of medical applications. It is an FDA-approved medication used primarily for treating acetaminophen overdose and as a mucolytic agent in respiratory diseases such as cystic fibrosis and chronic obstructive pulmonary disease (COPD)[1][3][5].

Clinical Trials Update

NAC Attack Trial for Retinitis Pigmentosa

One of the significant ongoing clinical trials involving NAC is the "NAC Attack" study. This Phase III multicenter, randomized, placebo-controlled trial aims to evaluate the efficacy and safety of oral NAC in patients with retinitis pigmentosa (RP), a genetic disorder leading to progressive vision loss. The trial is crucial as it could determine whether NAC can slow the progression of RP by reducing oxidative stress, a key factor in photoreceptor cell death[1][3].

Enrollment and Sites

Enrollment for the NAC Attack trial has commenced in 23 sites, including 20 in the United States and 3 international sites in Canada, Germany, and the Netherlands. Participants must stop any current NAC supplementation for at least 4 months to be eligible. The trial is sponsored by the National Eye Institute, part of the National Institutes of Health[1].

Other Clinical Applications

Apart from RP, NAC is being investigated for other conditions. For instance, a Phase 4 study at the University of California, San Francisco, is exploring the use of NAC for smoking cessation in tobacco and cannabis co-use. This study, which started in August 2021 and is expected to complete by June 2025, aims to assess the effectiveness of NAC in reducing cravings and use of these substances[4].

Market Analysis

Global Market Size and Growth

The global acetylcysteine market has shown significant growth in recent years. In 2023, the market size was USD 1.74 billion, and it is projected to reach USD 14.69 billion by 2034, growing at a Compound Annual Growth Rate (CAGR) of 21.4% from 2024 to 2034[5].

Regional Market Performance

North America dominates the acetylcysteine market, capturing the largest market share of 39% in 2023. The U.S. market alone was estimated at USD 480 million in 2023 and is predicted to grow to USD 4,100 million by 2034, driven by the increasing prevalence of respiratory conditions and the expanding therapeutic applications of NAC[5].

Impact of COVID-19

The COVID-19 pandemic had a mixed impact on the acetylcysteine market. In China, the sales value of acetylcysteine increased by 8.84% in 2020, despite a decline in the overall growth rate due to the pandemic's effects on hospital diagnosis and treatment. However, the market is expected to experience restorative growth from 2021 to 2025 as the pandemic's impact alleviates[2].

Competitive Landscape and Pricing

The competitive landscape of the acetylcysteine market is expected to become more competitive with the entry of new manufacturers, which is likely to decrease the price of acetylcysteine. This competition, along with advancements in drug delivery mechanisms, particularly in spray formulations, is driving market growth[2][5].

Market Projections

Segment Growth

The spray segment of the acetylcysteine market is anticipated to witness rapid growth at a CAGR of 21.25% during the projected period. This is due to the convenience and efficacy of spray formulations, especially for respiratory conditions like COPD and cystic fibrosis[5].

Pharmaceutical Segment

The pharmaceutical segment holds a significant share of the acetylcysteine market, accounting for 31% in 2023. NAC's role as a potent mucolytic agent in treating respiratory ailments ensures its continued importance in pharmaceutical formulations[5].

Emerging Applications

Ongoing investigational studies are exploring the potential applications of NAC in other medical conditions, including liver failure and cancer treatment. These emerging applications are expected to further drive the market growth of acetylcysteine[5].

Regulatory Considerations

FDA Regulations

The FDA has issued warning letters banning NAC in dietary supplements due to concerns over the lack of regulation and inconsistent content in commercially available NAC supplements. This underscores the importance of using FDA-approved NAC for medical purposes[1].

Key Takeaways

• Clinical Trials: Ongoing trials like the NAC Attack study are crucial for determining the efficacy of NAC in treating retinitis pigmentosa and other conditions.
• Market Growth: The global acetylcysteine market is projected to grow significantly, driven by increasing demand for respiratory treatments and expanding therapeutic applications.
• Regional Performance: North America leads the market, with the U.S. market expected to grow substantially by 2034.
• Segment Growth: The spray segment is expected to witness rapid growth due to its convenience and efficacy.
• Regulatory Considerations: FDA-approved NAC is recommended over supplement-grade NAC due to regulatory and safety concerns.

FAQs

What is the primary use of acetylcysteine in medical treatment?

Acetylcysteine is primarily used to treat acetaminophen overdose and as a mucolytic agent in respiratory diseases such as cystic fibrosis and COPD.

What is the NAC Attack trial, and what does it aim to achieve?

The NAC Attack trial is a Phase III multicenter, randomized, placebo-controlled study aimed at evaluating the efficacy and safety of oral NAC in patients with retinitis pigmentosa to determine if it can slow the progression of the disease.

How has the COVID-19 pandemic affected the acetylcysteine market?

The COVID-19 pandemic had a mixed impact, with a decline in growth rate in 2020 due to hospital diagnosis and treatment disruptions, but the market is expected to experience restorative growth from 2021 to 2025.

What are the projected growth rates for the global acetylcysteine market?

The global acetylcysteine market is projected to grow at a CAGR of 21.4% from 2024 to 2034, reaching USD 14.69 billion by 2034.

Why is the spray segment of the acetylcysteine market growing rapidly?

The spray segment is growing rapidly due to the convenience and efficacy of spray formulations, particularly for respiratory conditions like COPD and cystic fibrosis.

What regulatory concerns exist regarding NAC supplements?

The FDA has banned NAC in dietary supplements due to concerns over inconsistent content and lack of regulation, recommending the use of FDA-approved NAC for medical purposes.

Sources

1. NAC Attack Clinical Trial | Johns Hopkins Medicine
2. Investigation Report on China's Acetylcysteine Markets, 2016-2020 ... - Business Wire
3. Oral N-acetylcysteine for Retinitis Pigmentosa - Mayo Clinic
4. N-Acetylcysteine for Smoking Cessation in Tobacco and Cannabis ... - UCSF
5. Acetylcysteine Market Size, Share, Demand | CAGR Of 21.4% - Precedence Research