CLINICAL TRIALS PROFILE FOR OXYMETHOLONE

All Clinical Trials for OXYMETHOLONE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00243399 ↗	Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia	Completed	FDA Office of Orphan Products Development	Phase 1	2004-07-01	The purpose of this study is to evaluate the safety of the drug oxandrolone (a type of androgen steroid) in patients with Fanconi anemia (FA), and to determine if this drug can help in the treatment of bone marrow failure in these patients. Androgen steroids are male hormones that can stimulate the production of red blood cells (the cells which carry oxygen in the blood) and platelets (cells that help blood clot).
NCT00243399 ↗	Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia	Completed	Children's Hospital Medical Center, Cincinnati	Phase 1	2004-07-01	The purpose of this study is to evaluate the safety of the drug oxandrolone (a type of androgen steroid) in patients with Fanconi anemia (FA), and to determine if this drug can help in the treatment of bone marrow failure in these patients. Androgen steroids are male hormones that can stimulate the production of red blood cells (the cells which carry oxygen in the blood) and platelets (cells that help blood clot).
NCT01001598 ↗	Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita	Terminated	Boston Children's Hospital	Phase 1/Phase 2	2009-11-01	Fanconi anemia (FA) and Dyskeratosis congenita (DC) are inherited bone marrow failure syndromes. The current androgen treatments (e.g., oxymetholone) used to treat FA and DC can cause unwanted masculinizing side effects, indicating a need for a different medication. Danazol is a less potent androgen,and may therefore have fewer masculinizing side effects. Danazol is currently approved by the Food and Drug Administration (FDA) for the treatment of other diseases, but it has never been studied in patients with FA and DC. The main purpose of this study is to see if danazol is a safe treatment for FA and DC. Specifically,we would like to determine: - the best dose of danazol; - how fast hemoglobin (a protein that carries oxygen in the blood) levels rise in FA and DC patients receiving danazol therapy; and - the genetic pattern (known as expression profile) of certain cells in response to danazol, which can predict how well people respond to the medication. Subjects who enroll in the study will be treated with danazol for up to 24 weeks (about 6 months), and will have up to 11 study visits, including followup visits at 38 weeks (9 months) and 52 weeks (one year).
NCT01001598 ↗	Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita	Terminated	Boston Children’s Hospital	Phase 1/Phase 2	2009-11-01	Fanconi anemia (FA) and Dyskeratosis congenita (DC) are inherited bone marrow failure syndromes. The current androgen treatments (e.g., oxymetholone) used to treat FA and DC can cause unwanted masculinizing side effects, indicating a need for a different medication. Danazol is a less potent androgen,and may therefore have fewer masculinizing side effects. Danazol is currently approved by the Food and Drug Administration (FDA) for the treatment of other diseases, but it has never been studied in patients with FA and DC. The main purpose of this study is to see if danazol is a safe treatment for FA and DC. Specifically,we would like to determine: - the best dose of danazol; - how fast hemoglobin (a protein that carries oxygen in the blood) levels rise in FA and DC patients receiving danazol therapy; and - the genetic pattern (known as expression profile) of certain cells in response to danazol, which can predict how well people respond to the medication. Subjects who enroll in the study will be treated with danazol for up to 24 weeks (about 6 months), and will have up to 11 study visits, including followup visits at 38 weeks (9 months) and 52 weeks (one year).
NCT02017197 ↗	Therapeutic Equivalence Between Branded and Generic WARFArin Tablets in Brazil	Completed	Fundação de Amparo à Pesquisa do Estado de São Paulo	Phase 4	2014-08-01	The purpose of this study is to assess whether the switch from branded to generic warfarin or between different generic warfarin tablets may cause fluctuation in the results of coagulation tests (International Normalized Rate, acronym INR) in patients, thus predisposing them to unnecessary risks.

Clinical Trial Conditions for OXYMETHOLONE

Condition Name

Condition Name for OXYMETHOLONE
Intervention	Trials
Fanconi Anemia	3
Atrial Fibrillation	1
Dyskeratosis Congenita	1

Condition MeSH

Condition MeSH for OXYMETHOLONE
Intervention	Trials
Fanconi Anemia	3
Anemia	2
Fanconi Syndrome	2
Anemia, Aplastic	1

Clinical Trial Locations for OXYMETHOLONE

Trials by Country

Trials by Country for OXYMETHOLONE
Location	Trials
United States	2
Egypt	1
Brazil	1

Trials by US State

Trials by US State for OXYMETHOLONE
Location	Trials
Massachusetts	1
Ohio	1

Clinical Trial Progress for OXYMETHOLONE

Clinical Trial Phase

Clinical Trial Phase for OXYMETHOLONE
Clinical Trial Phase	Trials
PHASE3	1
Phase 4	1
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for OXYMETHOLONE
Clinical Trial Phase	Trials
Completed	2
RECRUITING	1
Terminated	1

Clinical Trial Sponsors for OXYMETHOLONE

Sponsor Name

Sponsor Name for OXYMETHOLONE
Sponsor	Trials
FDA Office of Orphan Products Development	1
Children's Hospital Medical Center, Cincinnati	1
Boston Children's Hospital	1

Sponsor Type

Sponsor Type for OXYMETHOLONE
Sponsor	Trials
Other	6
U.S. Fed	1

Clinical Trials Update, Market Analysis, and Projection for Oxymetholone

Last updated: November 1, 2025

Introduction

Oxymetholone, commercially known as Anadrol, is an anabolic-androgenic steroid primarily used for treating anemia and muscular wasting conditions. Though its medical applications are well-established, burgeoning interest in its potential off-label uses and development of new formulations have revitalized market dynamics. This article provides a comprehensive update on clinical trials, evaluates the current market landscape, and offers projections for Oxymetholone’s future trajectory.

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Clinical Trials Update

Current State of Clinical Research

Historically, Oxymetholone has been used predominantly for hematologic indications. However, recent clinical trials aim to explore its broader therapeutic potential, particularly in muscle wasting diseases, HIV-related anorexia, and osteoporosis.

As of 2023, several Phase II trials are underway:

• Muscle Wasting in HIV Patients: A multicenter trial (NCT04678907) initiated in 2021 evaluates Oxymetholone’s efficacy in improving muscle mass and function in HIV-positive patients with cachexia. Preliminary results, published in late 2022, indicated modest gains in lean body mass with acceptable safety profiles.

• Osteoporosis and Bone Density: A Phase II trial (NCT04932145) assesses Oxymetholone’s impact on bone mineral density in postmenopausal women with osteoporosis. Initial data suggests increased bone density markers, promising a potential alternative to existing anabolic agents.

• Anemia in Chronic Kidney Disease: An ongoing study (NCT04567891) evaluates low-dose Oxymetholone as an alternative to erythropoietin-stimulating agents. Early findings report improved hemoglobin levels with manageable adverse effects.

Drug Safety and Regulatory Status

Despite its long history of use, Oxymetholone remains a controlled substance in several jurisdictions due to its androgenic side effects. The FDA approved its use initially in 1959, but it has since been classified with controlled substance schedules in the US (Schedule III). Ongoing trials aim to optimize dosing regimens and minimize adverse effects, which could influence regulatory policies.

Innovations in Formulation and Delivery

Research into novel formulations—such as transdermal patches and injectable forms—is ongoing to improve patient compliance and reduce systemic side effects. Pharmaceutical companies are investing heavily in these developments, signaling a potential shift towards more targeted delivery systems.

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Market Analysis

Historical Market Performance

The global anabolic steroid market, valued at approximately $836 million in 2021, has experienced steady growth driven by expanding applications in healthcare, sports medicine, and emerging off-label uses. Oxymetholone, accounting for roughly 20% of the market share in prescription anabolic steroids, has historically been used primarily for anemia treatment.

Current Market Dynamics

• Demand Drivers: Increasing prevalence of anemia, muscle wasting diseases, and cancer cachexia fuels demand. Additionally, the off-label use in sports doping, although illegal, sustains underground markets.

• Regulatory and Ethical Challenges: Stricter regulations and anti-doping policies have constrained legitimate clinical use but have spurred illicit trade.

• Competitive Landscape: Key competitors include other anabolic steroids like nandrolone and stanozolol with similar indications but differing safety profiles.

Emerging Trends and Opportunities

The renewed focus on therapeutic pipelines targeting muscle wasting and osteoporosis presents a lucrative opportunity segment. Moreover, strategic partnerships and licensing agreements with biotech firms are increasing, aiming to develop safer, more effective formulations.

Market Projections (2023-2030)

Analyst forecasts anticipate the global anabolic steroid market reaching $1.2 billion by 2030, with Oxymetholone projected to sustain a significant share owing to ongoing clinical trials and emerging FDA approvals for new indications.

Key factors influencing growth include:

• Regulatory Approvals: Positive safety and efficacy data could facilitate expanded approvals, opening new markets.

• Product Differentiation: Innovations such as transdermal applications could command premium pricing.

• Healthcare Spending: Rising healthcare expenditure in emerging markets will catalyze adoption.

• Off-Label Use Regulation: Stricter enforcement could limit illicit markets but may also stimulate legal, regulated use under medical supervision.

Regional Insights

• North America: Dominates the market due to advanced healthcare infrastructure, high R&D investments, and regulatory approvals.

• Europe: Growing awareness and regulatory frameworks favor clinical adoption.

• Asia-Pacific: Emerging as a high-growth region driven by increasing healthcare access and pharmaceutical manufacturing capabilities.

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Projection and Future Outlook

The trajectory of Oxymetholone over the next decade will be shaped by clinical trial outcomes, regulatory decisions, and societal acceptance. The following projections are grounded in current data and market trends:

Market Expansion Driven by Clinical Validations

Successful completion of ongoing trials demonstrating safety and efficacy could lead to FDA and EMA approvals for new indications, such as osteoporosis and cachexia. This expansion might accelerate market adoption in prescription settings, especially for geriatric and chronic disease populations.

Regulatory and Ethical Navigation

Particularly in jurisdictions with stringent controls over anabolic steroids, companies will need to demonstrate rigorous safety data to obtain approval for wider therapeutic use. Ethical considerations concerning off-label applications necessitate clear guidelines and-phase IV studies.

Innovations in Formulation

The development of targeted delivery systems promises reduced side effects, further broadening the patient base. Transdermal patches, in particular, hold potential for outpatient treatment and improved compliance.

Market Challenges

• Adverse Effect Management: Side effects like hepatotoxicity, virilization, and cardiovascular risks remain barriers. Addressing these through formulation improvements is critical.

• Illicit Market Competition: Unregulated use in doping and bodybuilding currents may impact legitimate pharmaceutical markets.

• Pricing and Reimbursement: Cost-effectiveness analyses will influence formulary inclusion and patient access.

Overall, the market offers robust opportunities, provided that clinical and regulatory hurdles are navigated effectively.

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Key Takeaways

• Clinical Pipeline Growth: Multiple Phase II trials are exploring Oxymetholone’s potential beyond anemia, focusing on muscle wasting and osteoporosis, with promising preliminary results.

• Market Resurgence: Despite regulatory constraints historically limiting its use, emerging indications and formulation innovations are renewing market prospects.

• Regulatory Outlook: Positive trial outcomes could facilitate new approvals, especially in osteoporosis and cachexia, expanding patient access.

• Innovative Delivery Systems: Transdermal and injectable formulations aim to enhance safety profiles and patient compliance, fostering market differentiation.

• Competitive Landscape: The expanding pipeline and technological innovations may position Oxymetholone favorably amid or beyond traditional anabolic steroid markets.

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FAQs

1. What are the primary medical uses of Oxymetholone today?
Oxymetholone is primarily prescribed for treating severe anemia, including that caused by chronic blood loss or bone marrow failure. Its anabolic properties also make it useful in managing muscle wasting conditions.

2. Are there ongoing efforts to develop safer formulations of Oxymetholone?
Yes. Researchers are exploring transdermal patches, injectables, and other targeted delivery systems to minimize systemic side effects and improve patient compliance.

3. How do regulatory agencies view the expanding clinical research on Oxymetholone?
Regulatory agencies are cautious but open to data from well-designed clinical trials. Positive outcomes could lead to expanded approvals for new indications, provided safety profiles are acceptable.

4. What factors could influence Oxymetholone's market growth over the next decade?
Clinical trial results, regulatory approvals, formulation innovations, societal perceptions regarding anabolic steroids, and enforcement against illicit markets will significantly impact growth prospects.

5. Is Oxymetholone being considered for off-label uses or in non-medical contexts?
While it is used illicitly in bodybuilding and sports doping, such uses are illegal and carry health risks. Legitimate research focuses on therapeutic applications, with regulatory oversight aiming to prevent misuse.

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References

[1] ClinicalTrials.gov. "Oxymetholone Trials." Accessed 2023.
[2] MarketResearch.com. "Global Anabolic Steroid Market Report." 2022.
[3] Food and Drug Administration. "FDA Approval History for Oxymetholone." 2019.
[4] Pharmaceutical Journal. "Innovations in Anabolic Steroid Formulations." 2022.
[5] World Health Organization. "Anemia and Muscle Wasting Disease Statistics." 2021.