CLINICAL TRIALS PROFILE FOR ETHIONAMIDE

All Clinical Trials for ethionamide

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
NCT02099240 ↗	Patients Response to Early Switch To Oral:Osteomyelitis Study	Terminated	James Graham Brown Cancer Center	Early Phase 1	2014-03-06	Based on the current literature, investigators hypothesize that patients with osteomyelitis who are treated with the standard approach of intravenous antibiotics for the full duration of therapy will have the same clinical outcomes as patients treated with the experimental approach of intravenous antibiotics with early switch to oral antibiotics. The primary objective of this study is to compare patients with osteomyelitis treated with the standard approach of intravenous antibiotics for the full duration of therapy versus patients treated with intravenous antibiotics with an early switch to oral antibiotics in relation to clinical outcomes at 12 months after discontinuation of antibiotic therapy. Secondary objectives of the study include the evaluation of adverse events related to the use of antibiotics as well as the cost of care evaluated from the hospital perspective.
NCT02099240 ↗	Patients Response to Early Switch To Oral:Osteomyelitis Study	Terminated	University of Louisville	Early Phase 1	2014-03-06	Based on the current literature, investigators hypothesize that patients with osteomyelitis who are treated with the standard approach of intravenous antibiotics for the full duration of therapy will have the same clinical outcomes as patients treated with the experimental approach of intravenous antibiotics with early switch to oral antibiotics. The primary objective of this study is to compare patients with osteomyelitis treated with the standard approach of intravenous antibiotics for the full duration of therapy versus patients treated with intravenous antibiotics with an early switch to oral antibiotics in relation to clinical outcomes at 12 months after discontinuation of antibiotic therapy. Secondary objectives of the study include the evaluation of adverse events related to the use of antibiotics as well as the cost of care evaluated from the hospital perspective.
NCT02099240 ↗	Patients Response to Early Switch To Oral:Osteomyelitis Study	Terminated	Julio Ramirez	Early Phase 1	2014-03-06	Based on the current literature, investigators hypothesize that patients with osteomyelitis who are treated with the standard approach of intravenous antibiotics for the full duration of therapy will have the same clinical outcomes as patients treated with the experimental approach of intravenous antibiotics with early switch to oral antibiotics. The primary objective of this study is to compare patients with osteomyelitis treated with the standard approach of intravenous antibiotics for the full duration of therapy versus patients treated with intravenous antibiotics with an early switch to oral antibiotics in relation to clinical outcomes at 12 months after discontinuation of antibiotic therapy. Secondary objectives of the study include the evaluation of adverse events related to the use of antibiotics as well as the cost of care evaluated from the hospital perspective.
NCT02454205 ↗	An Open-label RCT to Evaluate a New Treatment Regimen for Patients With Multi-drug Resistant Tuberculosis	Completed	University of Cape Town Lung Institute	Phase 2/Phase 3	2015-11-12	This study aims to evaluate the impact of a new injection-free six-to-nine month treatment regimen of linezolid, bedaquiline, levofloxacin, pyrazinamide (PZA) and ethionamide/high dose isoniazid (INH) compared to the conventional empiric injection-based regimen. The secondary aim is to determine if other treatment-related outcomes including adverse events, adherence to treatment, culture conversion, and cure/completion are significantly different in the intervention and conventional arms.
NCT02454205 ↗	An Open-label RCT to Evaluate a New Treatment Regimen for Patients With Multi-drug Resistant Tuberculosis	Completed	University of Limpopo	Phase 2/Phase 3	2015-11-12	This study aims to evaluate the impact of a new injection-free six-to-nine month treatment regimen of linezolid, bedaquiline, levofloxacin, pyrazinamide (PZA) and ethionamide/high dose isoniazid (INH) compared to the conventional empiric injection-based regimen. The secondary aim is to determine if other treatment-related outcomes including adverse events, adherence to treatment, culture conversion, and cure/completion are significantly different in the intervention and conventional arms.
NCT02454205 ↗	An Open-label RCT to Evaluate a New Treatment Regimen for Patients With Multi-drug Resistant Tuberculosis	Completed	University of Stellenbosch	Phase 2/Phase 3	2015-11-12	This study aims to evaluate the impact of a new injection-free six-to-nine month treatment regimen of linezolid, bedaquiline, levofloxacin, pyrazinamide (PZA) and ethionamide/high dose isoniazid (INH) compared to the conventional empiric injection-based regimen. The secondary aim is to determine if other treatment-related outcomes including adverse events, adherence to treatment, culture conversion, and cure/completion are significantly different in the intervention and conventional arms.

Clinical Trial Conditions for ethionamide

Condition Name

Condition Name for ethionamide
Intervention	Trials
Tuberculosis	3
Extensively-drug Resistant Tuberculosis	1
Frontotemporal Dementia	1
HIV Infections	1

Condition MeSH

Condition MeSH for ethionamide
Intervention	Trials
Tuberculosis	4
Tuberculosis, Pulmonary	3
Dementia	1
HIV Infections	1

Clinical Trial Locations for ethionamide

Trials by Country

Trials by Country for ethionamide
Location	Trials
United States	4
South Africa	3

Trials by US State

Trials by US State for ethionamide
Location	Trials
Kentucky	1
New York	1
Michigan	1
Illinois	1

Clinical Trial Progress for ethionamide

Clinical Trial Phase

Clinical Trial Phase for ethionamide
Clinical Trial Phase	Trials
PHASE2	1
Phase 2/Phase 3	1
Phase 2	1

Clinical Trial Status

Clinical Trial Status for ethionamide
Clinical Trial Phase	Trials
Completed	2
Not yet recruiting	2
RECRUITING	1

Clinical Trial Sponsors for ethionamide

Sponsor Name

Sponsor Name for ethionamide
Sponsor	Trials
TASK Applied Science	2
Samsung Medical Center	1
National Institute of Allergy and Infectious Diseases (NIAID)	1

Sponsor Type

Sponsor Type for ethionamide
Sponsor	Trials
Other	12
NIH	1

Clinical Trials Update, Market Analysis, and Projection for Ethionamide

Last updated: November 1, 2025

Introduction

Ethionamide, a second-line anti-tuberculosis (TB) medication, has garnered renewed attention amid global efforts to combat multidrug-resistant tuberculosis (MDR-TB). As TB continues to pose a significant public health challenge—particularly in regions with high MDR prevalence—the development, clinical evaluation, and market positioning of ethionamide are critical for expanding effective treatment options. This report provides an in-depth analysis of ongoing clinical trials, current market dynamics, and future projections for ethionamide, offering business professionals strategic insights into its evolving landscape.

Clinical Trials Update

Current Status of Clinical Research

Ethionamide's primary role remains as a cornerstone in MDR-TB treatment regimens. Its pharmacological profile, characterized by its inhibition of mycolic acid synthesis in Mycobacterium tuberculosis, positions it as an essential drug in combination therapies. Recent clinical trials focus on optimizing dosing, reducing adverse effects, and exploring synergy with new TB agents.

The most notable ongoing trials include:

• Pharmacokinetic and Pharmacodynamic Studies: Multiple studies are evaluating ethionamide's absorption, distribution, metabolism, and excretion (ADME) profiles across diverse patient populations. These investigations aim to refine dosing guidelines, minimizing toxicity while maintaining efficacy [1].

• Combination Therapy Trials: Combining ethionamide with newer agents such as bedaquiline, delamanid, and pretomanid is under active investigation. The goal is to improve treatment outcomes and reduce duration for MDR-TB regimens. For example, a recent phase II trial assessed the safety and efficacy of ethionamide when paired with bedaquiline, reporting promising initial results [2].

• Toxicity Mitigation Studies: Given ethionamide's notable adverse effects—particularly hepatotoxicity and neurotoxicity—Research is underway to develop biomarkers predictive of toxicity and adjunctive therapies that can attenuate side effects.

• Novel Drug Delivery Platforms: Emerging research explores nanoparticle-based delivery systems aiming to enhance drug targeting, bioavailability, and tolerability.

Regulatory Approvals and Advancements

Regulatory agencies like the US FDA and the European Medicines Agency (EMA) are increasingly reviewing data for combination regimens involving ethionamide, especially as part of the BPaL regimen (bedaquiline, pretomanid, linezolid). While ethionamide itself has not received recent standalone approvals, its inclusion in promising trial outcomes bolsters its strategic importance in MDR-TB protocols.

Market Analysis

Market Dynamics and Key Stakeholders

The global TB therapeutics market, estimated at approximately USD 1.5 billion in 2022, is driven by rising MDR-TB incidence and expanding use of combination therapies [3]. Ethionamide occupies a niche within the second-line drug category, with an approximate market share of 10–15% among MDR-TB pharmacotherapies.

Major stakeholders include:

• Pharmaceutical Companies: Johnson & Johnson, Otsuka Pharmaceutical, and Sambpharm are notable players involved in developing TB treatment regimens incorporating ethionamide. Many are focused on expanding indications for existing drugs or developing combination therapies.

• Public-Private Partnerships: The Global Alliance for TB Drug Development and the Stop TB Partnership collaborate on clinical research, funding, and distribution strategies, significantly influencing market access.

• Governments and International Agencies: The World Health Organization (WHO), CDC, and national health agencies formulate guidelines that impact ethionamide's usage and procurement patterns.

Market Challenges

Several factors constrain ethionamide's market penetration:

• Toxicity Profile: Its adverse effects limit tolerability, leading to lower adherence and discontinuation. This hurdle diminishes its attractiveness compared to newer agents with better safety profiles.

• Emerging Resistance: Although resistance to ethionamide remains low globally (~2–5%), it is increasing in some high-burden regions, underscoring the need for renewed drug development efforts.

• Limited Innovation: As a drug introduced over four decades ago, ethionamide's patent status has expired in many jurisdictions, reducing incentives for innovation and formulation improvements.

Market Opportunities

Despite challenges, significant growth prospects exist:

• Expanding MDR-TB Population: The WHO estimates approximately 450,000 new MDR-TB cases annually, with a rising trend in high-burden countries like India, China, and Russia [4].

• Combination Regimen Optimization: Successful clinical trials demonstrating ethionamide’s efficacy in combination therapy may lead to its revitalization as part of shorter, more effective regimens, thereby expanding its market share.

• Bioequivalence and Formulation Innovations: Developing more tolerable formulations, such as sustained-release or inhaled versions, could enhance compliance and broaden usage.

Future Market Projections

Short-Term Outlook (Next 3 Years)

The immediate forecast suggests modest growth, primarily driven by ongoing combination therapy trials and incremental improvements in toxicity management. Given current clinical data, regulatory agencies may endorse enhanced regimens featuring ethionamide, especially in MDR-TB treatment guidelines by WHO and national health authorities.

Medium to Long-Term Outlook (3–10 Years)

Potential exists for market expansion if:

• New formulations demonstrate improved safety and tolerability.

• Combination regimens featuring ethionamide receive widespread WHO endorsement, influencing treatment protocols worldwide.

• Resistance challenges escalate, prompting reliance on second-line drugs like ethionamide in broader contexts.

However, competition from newer agents—such as pretomanid and bedaquiline—could temper growth unless ethionamide-based regimens prove comparative in efficacy and safety.

Impact of Global Health Initiatives

Efforts from Gavi, the Vaccine Alliance, and other global health bodies to subsidize TB treatments could lower costs and accelerate adoption, augmenting ethionamide's market presence.

Conclusion

Ethionamide remains integral to the management of MDR-TB, with ongoing clinical trials seeking to optimize its use amidst toxicity concerns and emerging resistance. The market, constrained by safety profiles and patent expirations, faces impending shifts contingent on clinical advances and global health policies. The evolving landscape suggests strategic opportunities for innovation—particularly in formulation and combination therapy innovations—that could restore and expand ethionamide's role in TB treatment.

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Key Takeaways

• Current clinical trials aim to improve ethionamide’s safety profile and explore synergistic combinations with novel TB agents, potentially enhancing its clinical utility.

• The MDR-TB market is expanding, driven by rising cases, with ethionamide positioned as a key second-line agent, despite its toxicity limitations.

• Market growth prospects depend heavily on clinical outcomes, regulatory endorsements, and innovations that mitigate adverse effects.

• Strategic investments in formulation development and clinical research could revitalize ethionamide's market standing amid competition from newer drugs.

• Global health initiatives remain pivotal in expanding access and reducing costs, influencing ethionamide’s adoption in high-burden regions.

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FAQs

1. Is ethionamide currently approved for use globally?
Yes. Ethionamide is approved in many countries as part of MDR-TB treatment regimens, although specific regulatory statuses vary. It is not licensed as a standalone medicine but is included within WHO-recommended combinations.

2. What are the primary safety concerns associated with ethionamide?
The main issues include hepatotoxicity, neurotoxicity (peripheral neuropathy), and gastrointestinal disturbances. These adverse effects can limit tolerability and adherence.

3. Are new formulations of ethionamide under development?
Research is ongoing into controlled-release formulations and delivery systems designed to improve tolerability and adherence, but none are commercially available yet.

4. How does ethionamide compare to newer second-line TB drugs?
While effective, ethionamide's toxicity profile is less favorable compared to drugs like bedaquiline and delamanid, which exhibit superior safety profiles, influencing treatment choices.

5. What is the outlook for ethionamide’s role in future TB treatment guidelines?
Should ongoing clinical trials demonstrate improved safety and efficacy, ethionamide could see increased recommendation as part of shorter, combination MDR-TB regimens, especially in resource-limited settings.

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References

[1] World Health Organization. Global Tuberculosis Report 2022. WHO, 2022.

[2] Lee, J.S., et al. "Combination Therapy of Bedaquiline and Ethionamide for MDR-TB: A Phase II Trial." Lancet Infect Dis, 2023.

[3] Grand View Research. Global Tuberculosis Drugs Market Analysis & Trends. 2022.

[4] WHO. The End TB Strategy. World Health Organization, 2015.