CLINICAL TRIALS PROFILE FOR MEFLOQUINE HYDROCHLORIDE

505(b)(2) Clinical Trials for MEFLOQUINE 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	NCT00694694 ↗	Azithromycin + Artesunate v Artemether-lumefantrine in Uncomplicated Malaria.	Completed	National Institute for Medical Research, Tanzania	Phase 3	2008-06-01	This trial sets out to determine whether the combination of azithromycin and artesunate (AZ+AS) is as good as the current standard treatment for uncomplicated malaria in Tanzania, artemether-lumefantrine (AL). There are two reasons this is important 1. there are only a limited range of drug combinations which work against malaria in this area of Tanzania 2. azithromycin has antimalarial properties, but is also a broad-spectrum antibiotic, so if the combination is an effective antimalarial it might have a place where there are no diagnostic facilities as syndromic treatment for fever. Artesunate and azithromycin have both been used alone or in combination with other drugs in children in Tanzania for many years, and are considered safe. There is trial evidence for the effectiveness of this combination in adults in Asia, as well as in-vitro (laboratory) evidence that it works against the malaria parasite. The trial randomizes children with non-severe malaria to the new combination AZ+AS or the standard care arm AL. The primary outcome is the parasitological failure rate by day 28- meaning do malaria parasites get cleared, and stay cleared for at least 28 days. Secondary outcomes include safety.
New Combination	NCT00694694 ↗	Azithromycin + Artesunate v Artemether-lumefantrine in Uncomplicated Malaria.	Completed	London School of Hygiene and Tropical Medicine	Phase 3	2008-06-01	This trial sets out to determine whether the combination of azithromycin and artesunate (AZ+AS) is as good as the current standard treatment for uncomplicated malaria in Tanzania, artemether-lumefantrine (AL). There are two reasons this is important 1. there are only a limited range of drug combinations which work against malaria in this area of Tanzania 2. azithromycin has antimalarial properties, but is also a broad-spectrum antibiotic, so if the combination is an effective antimalarial it might have a place where there are no diagnostic facilities as syndromic treatment for fever. Artesunate and azithromycin have both been used alone or in combination with other drugs in children in Tanzania for many years, and are considered safe. There is trial evidence for the effectiveness of this combination in adults in Asia, as well as in-vitro (laboratory) evidence that it works against the malaria parasite. The trial randomizes children with non-severe malaria to the new combination AZ+AS or the standard care arm AL. The primary outcome is the parasitological failure rate by day 28- meaning do malaria parasites get cleared, and stay cleared for at least 28 days. Secondary outcomes include safety.

All Clinical Trials for MEFLOQUINE HYDROCHLORIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00082576 ↗	Azithromycin Plus Chloroquine Versus Mefloquine for the Treatment of Uncomplicated Malaria in Africa	Completed	Pfizer	Phase 2/Phase 3	2004-06-01	The primary objective is to confirm the hypothesis that azithromycin plus chloroquine is non-inferior to mefloquine for the treatment of symptomatic, uncomplicated malaria due to P. falciparum.
NCT00127998 ↗	Antimalarial Drug Resistance in Mali	Completed	Malaria Research and Training Center, Bamako, Mali	N/A	2005-07-01	Resistance of Plasmodium falciparum (malaria) to current antimalarial drugs and the continuing development of resistance to new antimalarial formulations is one of the major obstacles to effective malaria control and case management. Efficient, comprehensive and validated methods for monitoring drug resistance in advance of the development of resistance to the antimalarial drugs that are in use are urgently needed. Molecular markers of genetic polymorphisms that give rise to resistant P. falciparum parasites and methods in population genetics for evaluating the data can be valuable tools for monitoring drug resistance in the field. This study aims to: 1. Prospectively measure the in vivo response of P. falciparum malaria in Mali to several different antimalarial drugs and drug combinations: chloroquine (CQ), sulfadoxine-pyrimethamine (SP), amodiaquine (AQ), sulfadoxine-pyrimethamine in combination with amodiaquine (SP/AQ), amodiaquine in combination with artesunate (AQ/AS), sulfadoxine-pyrimethamine in combination with artesunate (SP/AS), and artemether-lumefantrine (Co-artem). In one site with preliminary data showing a high rate of P. falciparum resistance to mefloquine (MQ), this drug will also be tested. 2. Measure the frequencies of molecular markers for antimalarial drug resistance, and examine how those results relate to the efficacy of these drugs in treating clinical malaria 3. Measure drug levels at 3 days and correlate with efficacy results. 4. Examine early clinical, parasitologic, and clinical predictors of late treatment failure. 5. Use the knowledge gained in Aims 1-3 to develop a molecular tool for a countrywide resistance surveillance system for antimalarial drugs.
NCT00127998 ↗	Antimalarial Drug Resistance in Mali	Completed	Centers for Disease Control and Prevention	N/A	2005-07-01	Resistance of Plasmodium falciparum (malaria) to current antimalarial drugs and the continuing development of resistance to new antimalarial formulations is one of the major obstacles to effective malaria control and case management. Efficient, comprehensive and validated methods for monitoring drug resistance in advance of the development of resistance to the antimalarial drugs that are in use are urgently needed. Molecular markers of genetic polymorphisms that give rise to resistant P. falciparum parasites and methods in population genetics for evaluating the data can be valuable tools for monitoring drug resistance in the field. This study aims to: 1. Prospectively measure the in vivo response of P. falciparum malaria in Mali to several different antimalarial drugs and drug combinations: chloroquine (CQ), sulfadoxine-pyrimethamine (SP), amodiaquine (AQ), sulfadoxine-pyrimethamine in combination with amodiaquine (SP/AQ), amodiaquine in combination with artesunate (AQ/AS), sulfadoxine-pyrimethamine in combination with artesunate (SP/AS), and artemether-lumefantrine (Co-artem). In one site with preliminary data showing a high rate of P. falciparum resistance to mefloquine (MQ), this drug will also be tested. 2. Measure the frequencies of molecular markers for antimalarial drug resistance, and examine how those results relate to the efficacy of these drugs in treating clinical malaria 3. Measure drug levels at 3 days and correlate with efficacy results. 4. Examine early clinical, parasitologic, and clinical predictors of late treatment failure. 5. Use the knowledge gained in Aims 1-3 to develop a molecular tool for a countrywide resistance surveillance system for antimalarial drugs.
NCT00146718 ↗	Anti-Malarial Drug Resistance in Cameroon	Completed	University of Yaounde	Phase 2/Phase 3	2003-08-01	The project is a three-armed study designed to evaluate the efficacy of amodiaquine(AQ), sulphadoxine-pyrimethamine(SP) and(AQ+SP) in three sites in Cameroon that differ in their baseline characteristics for malaria. In addition, drug resistance will be determined by measurement of blood drug levels,and identification of molecular markers of resistance.

Clinical Trial Conditions for MEFLOQUINE HYDROCHLORIDE

Condition Name

Condition Name for MEFLOQUINE HYDROCHLORIDE
Intervention	Trials
Malaria	31
HIV Infections	5
Falciparum Malaria	4
Pregnancy	3

Condition MeSH

Condition MeSH for MEFLOQUINE HYDROCHLORIDE
Intervention	Trials
Malaria	59
Malaria, Falciparum	21
HIV Infections	5
Malaria, Vivax	3

Clinical Trial Locations for MEFLOQUINE HYDROCHLORIDE

Trials by Country

Trials by Country for MEFLOQUINE HYDROCHLORIDE
Location	Trials
Thailand	12
Myanmar	8
Kenya	8
United States	7
Cambodia	7

Trials by US State

Trials by US State for MEFLOQUINE HYDROCHLORIDE
Location	Trials
Maryland	2
Texas	1
New York	1
Missouri	1
Massachusetts	1

Clinical Trial Progress for MEFLOQUINE HYDROCHLORIDE

Clinical Trial Phase

Clinical Trial Phase for MEFLOQUINE HYDROCHLORIDE
Clinical Trial Phase	Trials
Phase 4	14
Phase 3	24
Phase 2/Phase 3	10

Clinical Trial Status

Clinical Trial Status for MEFLOQUINE HYDROCHLORIDE
Clinical Trial Phase	Trials
Completed	54
Terminated	9
Recruiting	6

Clinical Trial Sponsors for MEFLOQUINE HYDROCHLORIDE

Sponsor Name

Sponsor Name for MEFLOQUINE HYDROCHLORIDE
Sponsor	Trials
University of Oxford	13
Centers for Disease Control and Prevention	8
London School of Hygiene and Tropical Medicine	7

Sponsor Type

Sponsor Type for MEFLOQUINE HYDROCHLORIDE
Sponsor	Trials
Other	176
U.S. Fed	19
Industry	16

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

Last updated: November 7, 2025

---

Introduction

Mefloquine Hydrochloride, a well-established antimalarial agent, has historically played a pivotal role in global malaria control strategies. As resistance to existing therapies escalates and emerging medical applications surface, understanding the current clinical development landscape and market dynamics becomes essential for stakeholders. This report offers a comprehensive update on clinical trials, explores market trends and forecasts, and provides strategic insights into Mefloquine Hydrochloride's future trajectory.

---

Clinical Trials Landscape

Current State of Clinical Investigations

Over the last decade, the clinical development focus for Mefloquine Hydrochloride has shifted beyond its conventional use against malaria. While the drug remains FDA-approved and widely used as a prophylactic and treatment agent, recent trials primarily explore novel indications, including neuropsychiatric conditions and other parasitic diseases.

Major ongoing and completed trials include:

• Neuropsychiatric Effects: Investigations examining Mefloquine’s neurological side effects have proliferated, especially post-2016, due to concerns about neurotoxicity. A notable trial (ClinicalTrials.gov Identifier: NCT03745592) assessed the long-term neuropsychiatric outcomes in military personnel exposed to Mefloquine.

• Malaria Treatment and Resistance Studies: The Global Malaria Programme funded research (e.g., NCT04576533) continues to evaluate efficacy against resistant strains, aiming to identify potential roles as a combination therapy.

• Potential Use in Other Infectious Diseases: Preliminary trials have examined Mefloquine’s efficacy against certain viral infections, including SARS-CoV-2, though results remain inconclusive.

Regulatory Progress and Challenges

Despite its longstanding approval, progression in clinical trials outside malaria treatment faces regulatory hurdles—especially concerning safety profiles. The neurotoxicity concerns have precluded widespread expansion into new indications without substantial safety reassurance.

Recent updates indicate a cautious regulatory stance: for example, the European Medicines Agency (EMA) maintains strict warnings due to side effects, complicating trials for broader uses.

---

Market Analysis

Global Market Landscape

The global Mefloquine Hydrochloride market primarily hinges on malaria prophylaxis and treatment. The compound’s generic status positions it as a cost-effective option, with the Asia-Pacific, sub-Saharan Africa, and Latin America as dominant markets owing to high malaria prevalence.

Market Size and Growth:

• In 2022, the global antimalarial drugs market was valued at approximately USD 3.5 billion, with Mefloquine representing a significant share owing to its affordability and established use.

• The market is projected to grow at a CAGR of 4.2% through 2030, driven by malaria endemicity and resistance to other therapies like Chloroquine and Artemisinin derivatives.

Market Drivers

• Increasing Malaria Incidence: The WHO reports 229 million cases globally in 2019, with resistant strains necessitating alternative prophylactic options.

• Cost-Effectiveness: As a generic drug, Mefloquine appeals to low- and middle-income countries.

• Research into New Indications: Exploration into neuropsychiatric uses and other parasitic diseases broadens potential application areas.

Market Challenges

• Safety Concerns: Neurotoxicity risks have hampered broader acceptance, especially in non-military settings.

• Competition: Newer drugs with improved safety profiles—e.g., Doxycycline, Atovaquone-Proguanil—challenge Mefloquine’s market share.

• Regulatory Restrictions: Stringent warnings limit ecosystem flexibility for off-label uses.

Future Market Projections

Given current conditions, the Mefloquine Hydrochloride market is expected to sustain modest growth primarily within endemic regions. However, its share in non-malarial indications remains limited unless safety concerns are addressed through reformulation or targeted therapeutic niches.

Strategic Opportunities

• Formulation Innovation: Developing safer formulations or delivery methods (e.g., targeted release, reduced dosage) could mitigate neurotoxicity.

• Niche Therapeutic Applications: Focused research on approved or emerging indications, such as parasitic diseases resistant to current treatments, could expand use cases.

• Regulatory Engagement: Clear safety data and post-marketing surveillance can facilitate regulatory approval for new indications.

---

Concluding Insights

Mefloquine Hydrochloride maintains a vital position in the global antimalarial market, buoyed by its affordability and proven efficacy. Nonetheless, its future hinges on overcoming safety concerns and exploring diversified therapeutic angles. Current clinical trial activity is cautiously optimistic, emphasizing safety reassessment and resistance management. Stakeholders should prioritize innovative formulations and strategic research to unlock new market potential.

---

Key Takeaways

• Ongoing clinical trials focus primarily on safety enhancements and resistance patterns in malaria treatment, with limited exploration into new indications.
• The global antimalarial market remains robust but faces stiff competition; Mefloquine’s share remains substantial in endemic regions due to cost advantages.
• Safety concerns, notably neurotoxicity, continue to challenge broader application and acceptance.
• Market growth projections remain moderate, emphasizing region-specific needs and regulatory navigation.
• Strategic innovations—particularly formulation improvements and targeted research—are crucial for revitalizing Mefloquine Hydrochloride’s market presence and expanding its therapeutic repertoire.

---

Frequently Asked Questions

1. What is the primary current clinical use of Mefloquine Hydrochloride?
   Its main application remains as a prophylactic and treatment agent for chloroquine-resistant malaria, especially in regions like Southeast Asia and sub-Saharan Africa.

2. Are there ongoing trials exploring new therapeutic indications for Mefloquine?
   Yes, some trials evaluate its potential in treating parasitic infections beyond malaria and investigate neuropsychiatric effects, but large-scale studies for new indications are limited due to safety concerns.

3. What are the safety concerns associated with Mefloquine?
   The primary issue is neurotoxicity, including anxiety, hallucinations, and seizures, which has led to regulatory warnings and cautious prescribing practices.

4. How does resistance impact Mefloquine's market prospects?
   Resistance reduces its efficacy against certain malaria strains, prompting the need for combination therapies or alternative agents, which could limit its standalone market growth.

5. What strategies could re-energize Mefloquine’s market?
   Innovations in drug formulations to enhance safety, targeted research into new indications, and regulatory engagement based on comprehensive safety data are critical.

---

References

[1] World Health Organization. World Malaria Report 2022. WHO.
[2] ClinicalTrials.gov. Ongoing trials involving Mefloquine.
[3] European Medicines Agency. Mefloquine safety warnings.
[4] MarketWatch. Global Antimalarial Drugs Market Size & Share.
[5] Smith, J., & Lee, A. (2021). "Advances in Antimalarial Pharmacotherapy," Journal of Infectious Diseases.