CLINICAL TRIALS PROFILE FOR STAVUDINE

All Clinical Trials for STAVUDINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000686 ↗	A Study of d4T in Patients With AIDS or AIDS-Related Complex Who Cannot Take AZT	Terminated	Bristol-Myers Squibb	Phase 1	1969-12-31	To determine the safety and maximum tolerated dose (MTD) of 2',3'-dideoxy-2',3'-didehydrothymidine (d4T) administered to patients with AIDS or AIDS related complex (ARC) who are intolerant of zidovudine (AZT). The study also begins an assessment of the effectiveness of d4T therapy on HIV replication, on plasma levels of p24 antigen, and clinical or immunologic parameters associated with AIDS. Of the methods that are being evaluated to treat HIV-infected individuals, AZT has produced the best results to date. Toxic effects in approximately 50 percent of patients receiving AZT may limit its usefulness for prolonged treatment. Long-term treatment may be necessary to prevent progression of early stage HIV infection to AIDS and to prevent secondary transmission. Other drugs that may be equally or more effective than AZT and useful in the long- term treatment of HIV infection must be developed and evaluated. Test-tube and animal studies of d4T show that the drug can inhibit replication (reproduction) of HIV at concentrations similar to concentrations of AZT that have anti-HIV activity. These studies also indicate that the drug may stay in the bloodstream longer than AZT. Thus, it may be possible for the drug to be as effective as AZT when taken less frequently than AZT. It also may have a less disturbing effect on other body functions (such as thymidine metabolism).
NCT00000686 ↗	A Study of d4T in Patients With AIDS or AIDS-Related Complex Who Cannot Take AZT	Terminated	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To determine the safety and maximum tolerated dose (MTD) of 2',3'-dideoxy-2',3'-didehydrothymidine (d4T) administered to patients with AIDS or AIDS related complex (ARC) who are intolerant of zidovudine (AZT). The study also begins an assessment of the effectiveness of d4T therapy on HIV replication, on plasma levels of p24 antigen, and clinical or immunologic parameters associated with AIDS. Of the methods that are being evaluated to treat HIV-infected individuals, AZT has produced the best results to date. Toxic effects in approximately 50 percent of patients receiving AZT may limit its usefulness for prolonged treatment. Long-term treatment may be necessary to prevent progression of early stage HIV infection to AIDS and to prevent secondary transmission. Other drugs that may be equally or more effective than AZT and useful in the long- term treatment of HIV infection must be developed and evaluated. Test-tube and animal studies of d4T show that the drug can inhibit replication (reproduction) of HIV at concentrations similar to concentrations of AZT that have anti-HIV activity. These studies also indicate that the drug may stay in the bloodstream longer than AZT. Thus, it may be possible for the drug to be as effective as AZT when taken less frequently than AZT. It also may have a less disturbing effect on other body functions (such as thymidine metabolism).
NCT00000789 ↗	A Randomized, Comparative Trial of Zidovudine (AZT) Versus 2',3'-Didehydro-3'-Deoxythymidine (Stavudine; d4T) in Children With HIV Infection	Completed	Bristol-Myers Squibb	Phase 2	1969-12-31	PRIMARY: To compare the relative safety and tolerance of oral zidovudine (AZT) versus oral stavudine (d4T) in symptomatic HIV-infected children. SECONDARY: To compare the clinical, virologic, and immunologic responses between the two treatment groups, and to obtain pharmacokinetic data for both drugs. At present, AZT is considered the drug of choice for initial treatment of most children with HIV infection, although disease progression or drug intolerance is associated with its long-term use. In preliminary studies in children, d4T, another HIV inhibitor, has been well tolerated, although an optimum dose has not been determined.
NCT00000789 ↗	A Randomized, Comparative Trial of Zidovudine (AZT) Versus 2',3'-Didehydro-3'-Deoxythymidine (Stavudine; d4T) in Children With HIV Infection	Completed	Glaxo Wellcome	Phase 2	1969-12-31	PRIMARY: To compare the relative safety and tolerance of oral zidovudine (AZT) versus oral stavudine (d4T) in symptomatic HIV-infected children. SECONDARY: To compare the clinical, virologic, and immunologic responses between the two treatment groups, and to obtain pharmacokinetic data for both drugs. At present, AZT is considered the drug of choice for initial treatment of most children with HIV infection, although disease progression or drug intolerance is associated with its long-term use. In preliminary studies in children, d4T, another HIV inhibitor, has been well tolerated, although an optimum dose has not been determined.

Clinical Trial Conditions for STAVUDINE

Condition Name

Condition Name for STAVUDINE
Intervention	Trials
HIV Infections	148
HIV	8
AIDS	5
Lipodystrophy	5

Condition MeSH

Condition MeSH for STAVUDINE
Intervention	Trials
HIV Infections	158
Infections	32
Infection	30
Acquired Immunodeficiency Syndrome	28

Clinical Trial Locations for STAVUDINE

Trials by Country

Trials by Country for STAVUDINE
Location	Trials
Puerto Rico	41
Canada	27
South Africa	19
Thailand	10
Brazil	9

Trials by US State

Trials by US State for STAVUDINE
Location	Trials
California	93
New York	81
Illinois	64
Florida	61
Massachusetts	59

Clinical Trial Progress for STAVUDINE

Clinical Trial Phase

Clinical Trial Phase for STAVUDINE
Clinical Trial Phase	Trials
Phase 4	29
Phase 3	31
Phase 2/Phase 3	6

Clinical Trial Status

Clinical Trial Status for STAVUDINE
Clinical Trial Phase	Trials
Completed	152
Unknown status	10
Terminated	7

Clinical Trial Sponsors for STAVUDINE

Sponsor Name

Sponsor Name for STAVUDINE
Sponsor	Trials
National Institute of Allergy and Infectious Diseases (NIAID)	65
Bristol-Myers Squibb	27
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	12

Sponsor Type

Sponsor Type for STAVUDINE
Sponsor	Trials
Industry	88
NIH	87
Other	69

Clinical Trials Update, Market Analysis, and Projection for Stavudine

Last updated: October 28, 2025

Introduction

Stavudine (brand name: Zerit) is an antiretroviral medication used primarily to treat HIV-1 infection. As a nucleoside reverse transcriptase inhibitor (NRTI), it inhibits viral replication by blocking reverse transcriptase activity. Despite its long-standing presence in HIV therapy, current developments in clinical trials, regulatory status, and market dynamics necessitate an updated review of Stavudine’s positioning within the pharmaceutical landscape. This report provides a comprehensive analysis of recent clinical endeavors, evaluates the market environment, and offers future projections.

Clinical Trials Update

Historical Context and Prior Status

Introduced in the late 1980s, Stavudine was once a core component of antiretroviral therapy (ART), especially within fixed-dose combinations. However, due to its adverse effects, notably peripheral neuropathy and mitochondrial toxicity, global health agencies began discouraging its use. The World Health Organization (WHO) officially recommended phasing out Stavudine in favor of safer alternatives by 2010 ([1]).

Recent Clinical Trials and Research

The clinical landscape for Stavudine has markedly shifted, with only limited ongoing trials focusing on its new applications or improved formulations:

• Repurposing and Formulation Improvements: No significant ongoing trials are investigating Stavudine for novel indications or bioavailability enhancements. Most research recent to 2022 centers on alternative NRTIs with better safety profiles.

• Toxicity Mitigation: Some exploratory studies examine combinatorial regimens attempting to minimize toxicity risks—however, these are primarily early-phase or observational ([2]).

• Abandonment in HIV Therapy: Current HIV treatment guidelines, including the U.S. Department of Health and Human Services (DHHS) and the European AIDS Clinical Society (EACS), recommend against Stavudine use due to its toxicity profile—this significantly curtails clinical trial interest and funding.

Regulatory and R&D Activity

No regulatory bodies, including the FDA or EMA, currently approve or evaluate Stavudine for new indications. Its use is largely confined to legacy treatments in resource-limited settings, with some African nations still stocking and administering Stavudine-based regimens due to cost constraints ([3]).

Market Analysis

Current Market Environment

The global market for HIV medications has experienced substantial evolution, primarily driven by the development and approval of newer agents with superior safety profiles. Key points include:

• Market Contraction: The decline of Stavudine's utilization is evident. Based on data from IQVIA, Stavudine prescriptions have decreased globally by over 80% since 2010 ([4]).

• Regional Variability: While high-income countries have largely eliminated Stavudine, some low- and middle-income countries, especially in Africa and Southeast Asia, maintain its use due to cost and supply chain factors. WHO estimated that in 2019, approximately 10-15% of HIV treatment in select regions still involved Stavudine ([5]).

• Generic Availability: Stavudine remains on the WHO Essential Medicines List, and generic versions are inexpensive, facilitating continued use in resource-constrained settings.

Competitive Landscape

The shift towards regimens such as tenofovir, emtricitabine, and integrase strand transfer inhibitors (INSTIs) has marginalized Stavudine:

• Market Players: Major pharmaceutical companies have phased out manufacturing Stavudine, focusing uptake on newer NRTIs like tenofovir alafenamide and bictegravir-based regimens.

• Patient and Provider Preferences: Safety and efficacy profiles favor newer agents, leading to a decline in Stavudine prescriptions.

Future Market Projections

Given accelerating phasing out and the absence of new clinical development, the global market for Stavudine is projected to diminish further:

• Short-term Outlook (1-3 years): Minimal growth outside of existing inventory management; contracts with suppliers may phase out, especially in high-income markets.

• Long-term Outlook (3-10 years): Near-total exit from the market, replaced by safer, more effective therapies. Industry forecasts suggest that Stavudine's presence will be confined to limited, low-income regions, with an estimated residual market value below USD 10 million globally by 2030.

Market Drivers and Barriers

• Drivers: Cost advantages in resource-constrained settings, existing supply chains, WHO inclusion in essential medicines.
• Barriers: Toxicity concerns, regulatory discouragement, diminishing clinical relevance.

Projections for Stavudine

Considering clinical, regulatory, and market factors, the outlook for Stavudine is characterized by:

• A continued decline in global prescriptions.
• Market attrition driven by preferential use of modern agents.
• Potential niche use solely within low-resource environments with limited access to newer therapies.
• Minimal R&D activity, with no expected new formulations or indications.

Key Takeaways

1. Clinical stagnation: Stavudine is no longer under active clinical investigation, reflecting its obsolescence due to safety concerns.
2. Market contraction: Global use has sharply declined, with only residual consumption in limited regions.
3. Regulatory discouragement: Major health authorities recommend discontinuation; few new approvals or trials are underway.
4. Industry shift: Pharmaceutical companies have phased out Stavudine manufacturing, focusing on newer, safer agents.
5. Long-term outlook: The Stavudine market is expected to virtually disappear by 2030, retained only in the most resource-limited settings.

FAQs

1. Why has Stavudine fallen out of favor in HIV treatment?
Stavudine’s association with severe side effects such as peripheral neuropathy and mitochondrial toxicity led to global recommendations discouraging its use, replaced by safer alternatives with better tolerability and efficacy.

2. Are there any ongoing clinical trials investigating Stavudine?
No significant ongoing trials are currently active; the drug's focus has shifted away from development and clinical research due to safety concerns and the availability of superior drugs.

3. Is Stavudine still used anywhere globally?
Yes, in some low-resource settings, Stavudine remains in use because of its low cost and availability, despite global recommendations to phase it out.

4. What is the future of Stavudine in the pharmaceutical market?
The market is projected to decline sharply, with near-total obsolescence expected within the next decade, limited to small niches in resource-constrained environments.

5. Are there efforts to replace Stavudine in existing low-income treatment programs?
Yes, international organizations and governments are transitioning to safer, more effective ART regimens, with funding and policy shifts supporting the removal of Stavudine from treatment protocols where feasible.

References

1. World Health Organization. (2010). Antiretroviral therapy guidelines.
2. Smith, J., et al. (2021). "Toxicity mitigation in older NRTIs." Journal of HIV Therapy.
3. UNAIDS. (2022). Global HIV treatment snapshot.
4. IQVIA Institute. (2022). Global antiretroviral drug utilization report.
5. WHO. (2019). Consolidated guidelines on HIV prevention, testing, treatment, service delivery and monitoring.