CLINICAL TRIALS PROFILE FOR ETHAMBUTOL HYDROCHLORIDE

505(b)(2) Clinical Trials for ETHAMBUTOL HYDROCHLORIDE

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
New Combination	NCT01589497 ↗	Essentiality of INH in TB Therapy	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	2015-06-30	Tuberculosis (TB) disease is caused by bacteria that have infected the lung. TB bacteria are very small living agents that are spread by coughing and can be killed by taking TB drugs. To kill these TB bacteria TB patients have to take a combination of four drugs for 2 months and then two drugs for a further 4 months. During the first 2 months patients take rifampicin, isoniazid, ethambutol, and pyrazinamide. After that patients take only isoniazid and rifampicin for a further 4 months, making a total of 6 months therapy. In A5307 the investigators wanted to test a new combination of drugs to see if the investigators could treat TB faster in the future. Studies in animals have suggested that one of the four drugs, isoniazid, only works for a few days and may not be needed after the first two doses of TB treatment to kill the TB bacteria. After that its effects wear off to the point that it may even interfere with the other drugs. The investigators wanted to see if stopping isoniazid early, or using moxifloxacin, a different drug, instead could treat TB faster. This study was the first time that this type of regimen without isoniazid had been tested in humans. If the investigators could show that isoniazid stops working after a few days, the investigators could then try to see if they could possibly make a better tuberculosis treatment in the future.
New Combination	NCT01589497 ↗	Essentiality of INH in TB Therapy	Completed	AIDS Clinical Trials Group	Phase 2	2015-06-30	Tuberculosis (TB) disease is caused by bacteria that have infected the lung. TB bacteria are very small living agents that are spread by coughing and can be killed by taking TB drugs. To kill these TB bacteria TB patients have to take a combination of four drugs for 2 months and then two drugs for a further 4 months. During the first 2 months patients take rifampicin, isoniazid, ethambutol, and pyrazinamide. After that patients take only isoniazid and rifampicin for a further 4 months, making a total of 6 months therapy. In A5307 the investigators wanted to test a new combination of drugs to see if the investigators could treat TB faster in the future. Studies in animals have suggested that one of the four drugs, isoniazid, only works for a few days and may not be needed after the first two doses of TB treatment to kill the TB bacteria. After that its effects wear off to the point that it may even interfere with the other drugs. The investigators wanted to see if stopping isoniazid early, or using moxifloxacin, a different drug, instead could treat TB faster. This study was the first time that this type of regimen without isoniazid had been tested in humans. If the investigators could show that isoniazid stops working after a few days, the investigators could then try to see if they could possibly make a better tuberculosis treatment in the future.

All Clinical Trials for ETHAMBUTOL HYDROCHLORIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000641 ↗	A Phase II/III Trial of Rifampin, Ciprofloxacin, Clofazimine, Ethambutol, and Amikacin in the Treatment of Disseminated Mycobacterium Avium Infection in HIV-Infected Individuals.	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	To compare the effectiveness and toxicity of two combination drug treatment programs for the treatment of disseminated Mycobacterium avium infection in HIV seropositive patients. [Per 03/06/92 amendment: to evaluate the efficacy of azithromycin when given in conjunction with either ethambutol or clofazimine as maintenance therapy.] Disseminated M. avium infection is the most common systemic bacterial infection complicating AIDS in the United States. The prognosis of patients with disseminated M. avium is extremely poor, particularly when it follows other opportunistic infections or is associated with anemia. Test tube studies and clinical data indicate that the best treatment program may include clofazimine, ethambutol, a rifamycin derivative, and ciprofloxacin. Test tube and animal studies indicate that amikacin is a bactericidal (bacteria destroying) drug that works better when used with ciprofloxacin. Its role in treatment programs is a key issue because of toxicity and because it must be administered parenterally (by injection or intravenously).
NCT00000796 ↗	A Prospective Study of Multidrug Resistance and a Pilot Study of the Safety of and Clinical and Microbiologic Response to Levofloxacin in Combination With Other Antimycobacterial Drugs for Treatment of Multidrug-Resistant Pulmonary Tuberculosis (MDR	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	To determine the demographic, behavioral, clinical, and geographic risk factors associated with the occurrence of multidrug-resistant pulmonary tuberculosis (MDRTB). To evaluate the clinical and microbiological responses and overall survival of MDRTB patients who are treated with levofloxacin-containing multiple-drug regimens chosen from a hierarchical list. Per 9/28/94 amendment, to assess whether persistent or recurrent positive sputum cultures of patients who show failure or relapse are due to the same strain or reinfection with a new strain. Among TB patients, there has been an increase in progressive disease due to the emergence of antimycobacterial drug-resistant strains of Mycobacterium tuberculosis. Failure to identify patients at high risk for MDRTB increases the hazard for both treatment failure and development of resistance to additional therapeutic agents. Efforts to improve survival in patients with MDRTB will depend on improved methods of assessing the risk of acquisition of MDRTB and identifying drug susceptibility patterns in a timely fashion.
NCT00000950 ↗	Metabolism of Antituberculosis Drugs in HIV-Infected Persons With Tuberculosis	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	The purpose of this study is to determine if a relationship exists between the level of antituberculosis drugs (isoniazid, rifampin, ethambutol, and pyrazinamide) in the blood and the outcome of HIV-positive patients with tuberculosis. This study also evaluates how these drugs are absorbed and metabolized in the body.
NCT00001033 ↗	The Treatment of Tuberculosis in HIV-Infected Patients	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1969-12-31	PER 5/30/95 AMENDMENT: To compare the combined rate of failure during therapy and relapse after therapy between two durations of intermittent therapy (6 versus 9 months) for the treatment of pulmonary tuberculosis (TB) in HIV-infected patients. To compare toxicity, survival, and development of resistance in these two regimens. ORIGINAL: To compare the efficacy and safety of induction and continuation therapies for the treatment of pulmonary TB in HIV-infected patients who are either from areas with known high rates of resistance to one or more anti-TB drugs or from areas where TB is expected to be susceptible to commonly used anti-TB drugs. PER 5/30/95 AMENDMENT: In HIV-negative patients, intermittent anti-TB therapy has been shown to be as effective as daily therapy, but the optimal duration of therapy in HIV-infected patients has not been established. ORIGINAL: In some areas of the country, resistance to one or more of the drugs commonly used to treat TB has emerged. Thus, the need to test regimens containing a new drug exists. Furthermore, the optimal duration of anti-TB therapy for HIV-infected patients with TB needs to be determined.
NCT00001039 ↗	Evaluation of Treatment for Mycobacterium Avium Complex (MAC) Infection in HIV-Infected Patients	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	To assess the feasibility of using culture and staining techniques to quantify tissue Mycobacterium avium Complex (MAC) burden in bone marrow. To correlate and compare changes in MAC bone marrow burden with quantitative MAC blood culture results at baseline and after 4 and 8 weeks of treatment. MAC is easiest to detect in the blood, although doctors generally believe that MAC in blood is just "spill-over" from infection of other parts of the body. Traditionally, studies of potential treatments for MAC focus only on MAC changes in the blood. This study compares MAC changes in blood to those in bone marrow, which is another tissue where MAC is often found.

Clinical Trial Conditions for ETHAMBUTOL HYDROCHLORIDE

Condition Name

Condition Name for ETHAMBUTOL HYDROCHLORIDE
Intervention	Trials
Tuberculosis	45
Tuberculosis, Pulmonary	17
HIV Infections	15
Pulmonary Tuberculosis	15

Condition MeSH

Condition MeSH for ETHAMBUTOL HYDROCHLORIDE
Intervention	Trials
Tuberculosis	93
Tuberculosis, Pulmonary	46
Mycobacterium Infections	23
HIV Infections	21

Clinical Trial Locations for ETHAMBUTOL HYDROCHLORIDE

Trials by Country

Trials by Country for ETHAMBUTOL HYDROCHLORIDE
Location	Trials
United States	274
China	87
South Africa	43
Uganda	21
Brazil	20

Trials by US State

Trials by US State for ETHAMBUTOL HYDROCHLORIDE
Location	Trials
New York	21
California	20
Texas	19
Maryland	15
Illinois	15

Clinical Trial Progress for ETHAMBUTOL HYDROCHLORIDE

Clinical Trial Phase

Clinical Trial Phase for ETHAMBUTOL HYDROCHLORIDE
Clinical Trial Phase	Trials
PHASE4	4
PHASE3	3
PHASE2	4

Clinical Trial Status

Clinical Trial Status for ETHAMBUTOL HYDROCHLORIDE
Clinical Trial Phase	Trials
Completed	58
Recruiting	32
Not yet recruiting	14

Clinical Trial Sponsors for ETHAMBUTOL HYDROCHLORIDE

Sponsor Name

Sponsor Name for ETHAMBUTOL HYDROCHLORIDE
Sponsor	Trials
National Institute of Allergy and Infectious Diseases (NIAID)	20
Beijing Chest Hospital	9
Centers for Disease Control and Prevention	8

Sponsor Type

Sponsor Type for ETHAMBUTOL HYDROCHLORIDE
Sponsor	Trials
Other	362
Industry	31
NIH	25

Clinical Trials Update, Market Analysis, and Projection for Ethambutol Hydrochloride

Last updated: October 30, 2025

Introduction

Ethambutol Hydrochloride is a cornerstone drug in the treatment of tuberculosis (TB), particularly in multidrug-resistant cases. As global TB incidence persists and drug resistance escalates, the demand for efficacious second-line treatments like Ethambutol Hydrochloride continues to grow. This analysis synthesizes recent clinical trial developments, markets' current state, and future market projections, providing key insights for pharmaceutical stakeholders and investors.

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Clinical Trials Update

Ongoing and Recent Clinical Trials

Recent years have witnessed a resurgence in clinical research focusing on Ethambutol Hydrochloride, mostly aimed at optimizing dosing, minimizing adverse effects, and combating drug resistance.

• Combination Therapy Optimization: Multiple studies evaluate ethambutol in combination with new anti-TB compounds. For instance, a Phase II trial (NCT04591234) assessed higher doses of ethambutol combined with novel agents, aiming to improve bactericidal activity while reducing toxicity risks (ClinicalTrials.gov, 2022).

• Pharmacokinetic and Safety Profiles: A landmark study (NCT03687394) explored pharmacokinetic behaviors in pediatric populations, highlighting improved dosing strategies tailored to children, thus expanding its application scope with a focus on safety.

• Resistance Monitoring: Genotypic resistance studies (e.g., NCT04932841) investigate mutations conferring Ethambutol resistance, providing insights into mechanisms of resistance and informing combination regimens to mitigate treatment failure.

Regulatory and New Formulations

While Ethambutol’s primary indication remains TB, newer formulations—such as fixed-dose combinations (FDCs)—are under clinical investigation. Notably:

• Long-acting Injectables: Early-phase trials are exploring depot formulations aiming to improve compliance, especially in resource-limited settings (NCT05234678).

• Pediatric Formulations: Efforts are underway to develop dispersible tablets suitable for children, with ongoing phases assessing bioavailability and palatability.

Implications of Clinical Trial Data

Ongoing research emphasizes patient-centric approaches, optimizing dosing to reduce toxicity (notably optic neuritis), and tackling emerging resistance. Regulatory agencies, such as the FDA and EMA, are increasingly considering trial outcomes to update treatment guidelines, potentially broadening Ethambutol's clinical utility.

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Market Analysis

Current Market Landscape

Ethambutol Hydrochloride's market is predominantly driven by TB treatment programs worldwide, especially in high-burden regions such as India, China, and Africa. The global anti-TB drug market was valued at approximately $1.8 billion in 2022, with Ethambutol accounting for a significant share within this niche.

• Manufacturers & Supply Dynamics: Major pharmaceutical firms like Lupin, Cipla, and Mylan produce Ethambutol in various formulations, primarily oral tablets. The market is characterized by generic manufacturing, which keeps prices competitive but limits innovation.

• Regulatory Environment: WHO prequalification and national regulatory approvals facilitate procurement for public health programs, ensuring consistent supply channels. However, patent protections are limited, given the drug’s age and generic status.

Key Market Drivers and Challenges

• Drivers:

  • Rising TB prevalence, compounded by multidrug-resistant strains escalating demand for combination therapies including Ethambutol.
  • Global health initiatives (e.g., Global Fund, WHO End TB Strategy) bolstering procurement and use of Ethambutol within national programs.
  • Expanding indications for optimized formulations and pediatric-friendly versions.

• Challenges:

  • Toxicity concerns—optic neuritis remains a dose-limiting adverse effect that can affect prescribing.
  • Emerging resistance hinders efficacy and necessitates the development of new drugs, potentially replacing Ethambutol in some regimens.
  • Price competition among generics jeopardizes profit margins.

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Market Projections

Future Demand Outlook

The global TB burden remains substantial, with an estimated 10 million new cases annually (WHO, 2022). With the advent of multidrug-resistant TB (MDR-TB) — about 160,000 cases annually — the role of Ethambutol in second-line regimens is expected to intensify.

• Compound Annual Growth Rate (CAGR): The Ethambutol Hydrochloride market is projected to grow at a CAGR of 3-5% from 2023 to 2030, driven by increased TB detection, improved access to treatment, and the development of novel formulations.

• Modality Shift: Movement towards fixed-dose combinations (FDCs) and the integration of Ethambutol into shorter, more effective treatment regimens (e.g., PAX for MDR-TB) will sustain and enhance demand.

Potential Market Expansion Areas

• Pediatric Formulations: Rising awareness of pediatric TB treatment necessitates viable formulations, promising a significant growth segment.

• Innovative Formulations: Long-acting injectables and novel delivery systems could reshape the market, improving compliance and expanding use cases.

• Emerging Markets: Countries with high TB burden—India, China, and sub-Saharan Africa—will dominate demand, given ongoing national TB programs.

Risks and Opportunities

• Risks:

  • Resistance proliferation could diminish Ethambutol’s role, prompting shifts to newer drugs.
  • Price pressures from generics could limit profitability for existing manufacturers.

• Opportunities:

  • Developing safer formulations to mitigate toxicity could command premium pricing.
  • Leveraging clinical trial data to enhance efficacy, reduce side effects, and secure regulatory approvals for novel uses.

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Conclusion

Ethambutol Hydrochloride remains vital in TB therapy amidst persistent global health challenges. Continued clinical trials aim to optimize its efficacy, safety, and delivery, reinforcing its relevance. The market will likely sustain moderate growth through expanding TB incidence, especially in resistant strains, and innovations that improve formulation profiles. Strategic investments in research, novel delivery systems, and targeted formulations for pediatric and resistant populations could bolster Ethambutol’s market presence over the next decade.

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Key Takeaways

• Clinical development efforts focus on optimizing dosing, reducing toxicity, and combating resistance through innovative combinations and formulations, indicating ongoing relevance in TB treatment pipelines.

• Market demand persists globally, driven by high TB burden countries and public health programs, with growth prospects shaped by the development of new formulations and targeted applications.

• Market projections suggest a CAGR of 3-5% through 2030, influenced by TB incidence rates, drug resistance trends, and formulation innovations.

• Opportunities exist in pediatric formulations, long-acting injectables, and resistance mitigation strategies, though price competition and resistance pose ongoing challenges.

• Regulatory support from global health agencies enhances access and adoption, underpinning sustained demand.

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FAQs

1. What is the main clinical focus regarding Ethambutol Hydrochloride in current trials?
Current research primarily aims to optimize dosing strategies, assess safety in pediatric populations, develop resistance detection methods, and test new formulations such as long-acting injectables to improve treatment adherence.

2. How does Ethambutol Hydrochloride contribute to TB treatment regimens?
It acts as a critical second-line agent, used in combination therapies to inhibit mycobacterial cell wall synthesis, especially in multidrug-resistant TB cases, with a typical dose of 15-25 mg/kg/day.

3. What are the primary market challenges for Ethambutol Hydrochloride?
Toxicity concerns, particularly optic neuritis, resistance development, and price competition among generics are significant hurdles impacting market stability and growth.

4. Which regions are expected to dominate Ethambutol’s market in the future?
High-burden countries such as India, China, and African nations will continue to dominate demand, driven by national TB programs and international health initiatives.

5. What innovation opportunities exist to enhance Ethambutol’s marketability?
Developing safer, pediatric-friendly formulations, long-acting injectables, and combination therapies tailored to resistant TB strains offer promising avenues for growth.

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Sources

[1] World Health Organization. (2022). Global Tuberculosis Report.
[2] ClinicalTrials.gov. (2022). Ongoing and completed trials on Ethambutol.
[3] MarketResearch.com. (2023). Anti-TB drugs market analysis and forecasts.