CLINICAL TRIALS PROFILE FOR DANAZOL

All Clinical Trials for danazol

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00003946 ↗	Danazol in Treating Patients With Advanced or Recurrent Endometrial Cancer	Completed	National Cancer Institute (NCI)	Phase 2	1999-08-01	RATIONALE: Hormone therapy may be an effective treatment for endometrial cancer. PURPOSE: Phase II trial to study the effectiveness of danazol in treating patients with advanced or recurrent endometrial cancer.
NCT00003946 ↗	Danazol in Treating Patients With Advanced or Recurrent Endometrial Cancer	Completed	Gynecologic Oncology Group	Phase 2	1999-08-01	RATIONALE: Hormone therapy may be an effective treatment for endometrial cancer. PURPOSE: Phase II trial to study the effectiveness of danazol in treating patients with advanced or recurrent endometrial cancer.
NCT00206544 ↗	Anti-Estrogens - A Potential Treatment for Bipolar Affective Disorder in Women?	Completed	National Health and Medical Research Council, Australia	Phase 2	2004-01-01	OBJECTIVE: To test the use of two adjunctive hormonal agents in a 28 day three-arm, double-blind, placebo-controlled study in the treatment of acute mania/hypomania. HYPOTHESIS: That women receiving adjunctive Tamoxifen or Progesterone will demonstrate a more rapid and more substantial decrease in manic symptoms over the course of the study than women receiving adjunctive placebo. STUDY POPULATION: Sixty females with a current diagnosis of Bipolar Affective Disorder or Schizoaffective disorder - Manic Phase, according to the operationalised criteria of the Diagnostic and Statistical Manual, 4th edition (DSM-IV) of the American Psychiatric Association. STUDY MEDICATION: Tamoxifen. One third of patients (twenty) will be randomized to receive adjunctive Tamoxifen at 40 mg/day for 28 days. The Tamoxifen will be administered within a plain capsule to maintain "blinding" of treatment arm. Progesterone. One third of patients (twenty) will be randomized to receive adjunctive oral Provera (progesterone) at 20 mg/day. The Progesterone will be administered within a plain capsule identical to that used with Tamoxifen. Placebo. The remaining one third of patients will be randomized to receive adjunctive placebo (inert substance). The placebo substance will be administered within a plain capsule identical to that used with Tamoxifen and Progesterone. STUDY EVALUATIONS: Data will be collected over a 28-day period for each patient. Visits will be performed at baseline, and then at weekly intervals. A total of five visits will be completed for each patient. The following evaluations will be performed: - Psychiatric evaluation to determine diagnosis. (Baseline visit only) - General clinical evaluation including medical history, current conditions and a non-invasive physical examination, body weight, vital signs. (Baseline visit only) - Medication history (baseline and evaluation visits). - Demographics (baseline visits only). - Completion of clinical rating scales; CARS-M, PANSS, MADRS, AIMS, Barnes Akathisia scale (BA), and Simpson-Angus scale (SA) (baseline and evaluation visits). A Menstrual Cycle Interview and a cognitive assessment (RBANS) will be performed at baseline and endpoint (day 28) visit. - Laboratory tests including; Serum levels of mood stabilizer, luteinizing hormone (LH), follicle-stimulating hormone (FSH), Estrogen, Progesterone, Prolactin, dehydroepiandrosterone (DHEA), Testosterone and protein kinase C(PKC) (baseline and evaluation visits). - Inclusion/exclusion checklist (baseline visit only). - Informed consent (baseline visit only).
NCT00206544 ↗	Anti-Estrogens - A Potential Treatment for Bipolar Affective Disorder in Women?	Completed	Stanley Medical Research Institute	Phase 2	2004-01-01	OBJECTIVE: To test the use of two adjunctive hormonal agents in a 28 day three-arm, double-blind, placebo-controlled study in the treatment of acute mania/hypomania. HYPOTHESIS: That women receiving adjunctive Tamoxifen or Progesterone will demonstrate a more rapid and more substantial decrease in manic symptoms over the course of the study than women receiving adjunctive placebo. STUDY POPULATION: Sixty females with a current diagnosis of Bipolar Affective Disorder or Schizoaffective disorder - Manic Phase, according to the operationalised criteria of the Diagnostic and Statistical Manual, 4th edition (DSM-IV) of the American Psychiatric Association. STUDY MEDICATION: Tamoxifen. One third of patients (twenty) will be randomized to receive adjunctive Tamoxifen at 40 mg/day for 28 days. The Tamoxifen will be administered within a plain capsule to maintain "blinding" of treatment arm. Progesterone. One third of patients (twenty) will be randomized to receive adjunctive oral Provera (progesterone) at 20 mg/day. The Progesterone will be administered within a plain capsule identical to that used with Tamoxifen. Placebo. The remaining one third of patients will be randomized to receive adjunctive placebo (inert substance). The placebo substance will be administered within a plain capsule identical to that used with Tamoxifen and Progesterone. STUDY EVALUATIONS: Data will be collected over a 28-day period for each patient. Visits will be performed at baseline, and then at weekly intervals. A total of five visits will be completed for each patient. The following evaluations will be performed: - Psychiatric evaluation to determine diagnosis. (Baseline visit only) - General clinical evaluation including medical history, current conditions and a non-invasive physical examination, body weight, vital signs. (Baseline visit only) - Medication history (baseline and evaluation visits). - Demographics (baseline visits only). - Completion of clinical rating scales; CARS-M, PANSS, MADRS, AIMS, Barnes Akathisia scale (BA), and Simpson-Angus scale (SA) (baseline and evaluation visits). A Menstrual Cycle Interview and a cognitive assessment (RBANS) will be performed at baseline and endpoint (day 28) visit. - Laboratory tests including; Serum levels of mood stabilizer, luteinizing hormone (LH), follicle-stimulating hormone (FSH), Estrogen, Progesterone, Prolactin, dehydroepiandrosterone (DHEA), Testosterone and protein kinase C(PKC) (baseline and evaluation visits). - Inclusion/exclusion checklist (baseline visit only). - Informed consent (baseline visit only).
NCT00206544 ↗	Anti-Estrogens - A Potential Treatment for Bipolar Affective Disorder in Women?	Completed	The Alfred	Phase 2	2004-01-01	OBJECTIVE: To test the use of two adjunctive hormonal agents in a 28 day three-arm, double-blind, placebo-controlled study in the treatment of acute mania/hypomania. HYPOTHESIS: That women receiving adjunctive Tamoxifen or Progesterone will demonstrate a more rapid and more substantial decrease in manic symptoms over the course of the study than women receiving adjunctive placebo. STUDY POPULATION: Sixty females with a current diagnosis of Bipolar Affective Disorder or Schizoaffective disorder - Manic Phase, according to the operationalised criteria of the Diagnostic and Statistical Manual, 4th edition (DSM-IV) of the American Psychiatric Association. STUDY MEDICATION: Tamoxifen. One third of patients (twenty) will be randomized to receive adjunctive Tamoxifen at 40 mg/day for 28 days. The Tamoxifen will be administered within a plain capsule to maintain "blinding" of treatment arm. Progesterone. One third of patients (twenty) will be randomized to receive adjunctive oral Provera (progesterone) at 20 mg/day. The Progesterone will be administered within a plain capsule identical to that used with Tamoxifen. Placebo. The remaining one third of patients will be randomized to receive adjunctive placebo (inert substance). The placebo substance will be administered within a plain capsule identical to that used with Tamoxifen and Progesterone. STUDY EVALUATIONS: Data will be collected over a 28-day period for each patient. Visits will be performed at baseline, and then at weekly intervals. A total of five visits will be completed for each patient. The following evaluations will be performed: - Psychiatric evaluation to determine diagnosis. (Baseline visit only) - General clinical evaluation including medical history, current conditions and a non-invasive physical examination, body weight, vital signs. (Baseline visit only) - Medication history (baseline and evaluation visits). - Demographics (baseline visits only). - Completion of clinical rating scales; CARS-M, PANSS, MADRS, AIMS, Barnes Akathisia scale (BA), and Simpson-Angus scale (SA) (baseline and evaluation visits). A Menstrual Cycle Interview and a cognitive assessment (RBANS) will be performed at baseline and endpoint (day 28) visit. - Laboratory tests including; Serum levels of mood stabilizer, luteinizing hormone (LH), follicle-stimulating hormone (FSH), Estrogen, Progesterone, Prolactin, dehydroepiandrosterone (DHEA), Testosterone and protein kinase C(PKC) (baseline and evaluation visits). - Inclusion/exclusion checklist (baseline visit only). - Informed consent (baseline visit only).
NCT00225875 ↗	Study of the Effectiveness of Rituximab in Adults With Chronic and Severe Immune Thrombocytopenic Purpura and Candidate for a Splenectomy	Terminated	Etablissement Français du Sang	Phase 2	2003-09-01	The goal of this study is to evaluate the clinical effectiveness of the rituximab at the adults with a chronic immune thrombocytopenic purpura (>=6 months of evolution) and severe (platelets
NCT00225875 ↗	Study of the Effectiveness of Rituximab in Adults With Chronic and Severe Immune Thrombocytopenic Purpura and Candidate for a Splenectomy	Terminated	Assistance Publique - Hôpitaux de Paris	Phase 2	2003-09-01	The goal of this study is to evaluate the clinical effectiveness of the rituximab at the adults with a chronic immune thrombocytopenic purpura (>=6 months of evolution) and severe (platelets

Clinical Trial Conditions for danazol

Condition Name

Condition Name for danazol
Intervention	Trials
Immune Thrombocytopenia	9
Endometriosis	8
Adenomyosis	3
Primary Myelofibrosis	3

Condition MeSH

Condition MeSH for danazol
Intervention	Trials
Thrombocytopenia	12
Purpura, Thrombocytopenic, Idiopathic	11
Endometriosis	8
Anemia	4

Clinical Trial Locations for danazol

Trials by Country

Trials by Country for danazol
Location	Trials
United States	104
Australia	14
France	13
Japan	13
China	12

Trials by US State

Trials by US State for danazol
Location	Trials
California	7
New York	6
Colorado	6
Arizona	5
New Jersey	4

Clinical Trial Progress for danazol

Clinical Trial Phase

Clinical Trial Phase for danazol
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	6
Phase 3	6

Clinical Trial Status

Clinical Trial Status for danazol
Clinical Trial Phase	Trials
Completed	21
Not yet recruiting	13
Recruiting	10

Clinical Trial Sponsors for danazol

Sponsor Name

Sponsor Name for danazol
Sponsor	Trials
Peking University People's Hospital	10
Beijing Hospital	4
Navy General Hospital, Beijing	3

Sponsor Type

Sponsor Type for danazol
Sponsor	Trials
Other	92
Industry	12
NIH	4

Clinical Trials Update, Market Analysis, and Projections for Danazol

Last updated: October 30, 2025

Introduction

Danazol, a synthetic androgen derived from ethisterone, plays a pivotal role in managing diverse medical conditions, including endometriosis, hereditary angioedema, and fibrocystic breast disease. Its unique mechanism of action involves suppressing the pituitary-ovarian axis, thus reducing estrogen levels and ameliorating conditions driven by estrogen excess. Despite being launched in the 1970s, danazol retains clinical relevance, driven by ongoing research, unmet medical needs, and its potential for repurposing. This article provides a comprehensive overview of recent clinical trial updates, market dynamics, and future projections for danazol.

Recent Clinical Trials and Developments

Ongoing and Recent Clinical Trials

Recent years have witnessed renewed scientific interest in danazol, focusing on expanding its therapeutic scope and optimizing safety profiles.

1. Hereditary Angioedema (HAE) Management

   • Multiple Phase II and Phase III trials focus on danazol’s efficacy and safety as a preventive treatment for hereditary angioedema. Notably, the European Society for Human Genetics reports ongoing assessments of low-dose danazol regimens for long-term management, highlighting efforts to minimize adverse effects like hepatotoxicity and androgenic symptoms ([1]).

2. Endometriosis and Fibrocystic Breast Disease

   • Research continues into using danazol at reduced dosages to minimize androgenic side effects while maintaining efficacy. An open-label trial published in Fertility and Sterility (2022) investigated low-dose danazol in women with endometriosis, confirming symptomatic relief with fewer androgenic adverse effects ([2]).

3. Potential for New Indications

   • Emerging preclinical studies explore danazol’s anti-inflammatory properties in autoimmune conditions. Early-phase trials are underway to assess its repurposing for disorders such as systemic lupus erythematosus and certain dermatologic conditions, with data expected in the upcoming years.

Safety and Pharmacovigilance Updates

Regulatory authorities, including the FDA and EMA, continue to monitor long-term safety data, focusing on hepatotoxicity, lipid profile alterations, and virilizing effects. Recent updates advocate for dose adjustments and patient monitoring protocols, especially in long-term therapy, aligning with evidence from recent pharmacovigilance studies ([3]).

Research Challenges and Future Directions

• Safety Concerns: The androgenic side effects patient populations face restrict broader use. Ongoing trials aim to delineate optimal dosing strategies to mitigate these issues.

• Biomarker Development: Efforts are underway to identify biomarkers predictive of response and adverse effects, enabling personalized therapy.

• Formulation Innovations: Development of novel formulations, including transdermal or targeted delivery systems, aims to enhance tolerability and adherence.

Market Analysis

Historical Market Landscape

Since its initial approval in the 1970s, danazol’s market was primarily confined to the treatment of endometriosis and hereditary angioedema. The commercially available formulations included oral capsules, predominantly marketed by pharmaceutical giants such as Schering-Plough (later acquired by Merck) and Sanofi-Aventis.

The introduction of newer agents—such as GnRH analogs, antihistamines, and biologics—substituted much of danazol’s dominant role, leading to market contraction.

Current Market Dynamics

• Global Market Size: Estimated at approximately USD 60 million in 2022, primarily driven by demand in niche therapeutic areas and off-label uses. North America and Europe account for over 70% of the market, with emerging markets showing growing interest.

• Patents and Regulatory Status: Danazol’s patent expired in the mid-1990s, leading to multiple generic formulations. No recent extensions or exclusivities are active, limiting pricing power.

• Competitive Landscape: The market faces competition from newer, safer therapies such as selective estrogen receptor modulators and biologics, which have better safety profiles but higher costs.

Market Opportunities

• Niche Disease Management: For hereditary angioedema, danazol remains the cost-effective, long-standing option, especially in resource-limited settings where biologics are prohibitively expensive.

• Repurposing for New Indications: If ongoing trials demonstrate efficacy with acceptable safety, danazol could regain market traction in autoimmune or inflammatory diseases.

• Formulation Enhancements: Innovation in delivery methods to improve tolerability and reduce side effects can unlock new market segments.

Market Challenges

• Safety Concerns: The risk profile restricts widespread use, especially in long-term management.

• Regulatory Hurdles: Gaining approval for new indications will require extensive clinical evidence, prolonging commercialization timelines.

• Competition: Biologic therapies and newer oral agents with superior safety profiles diminish danazol’s competitive edge.

Future Market Projections

Based on current data, the impending years forecast a limited but steady segment for danazol in niche markets, with potential growth if safety profiles improve and new indications are validated.

1. Short-term (1-3 years): Market stagnation unless new formulations or indications emerge. Generic availability keeps prices low, constraining profit margins.

2. Medium-term (4-7 years): Potential revival in the hereditary angioedema market, especially if new low-dose regimens receive regulatory support. Clinical validation for autoimmune indications could diversify applications.

3. Long-term (8+ years): Substitution by newer therapies may marginalize danazol but could sustain a small niche market, especially in low-resource settings. Breakthroughs in drug delivery may temporarily boost market share.

Regulatory and Commercial Outlook

The evolving regulatory environment emphasizes safety monitoring and risk management. Future approvals will depend on demonstrating improved safety and expanded indications. Commercial success hinges on strategic positioning as a cost-effective alternative and the ability to innovate delivery systems.

Key Takeaways

• Clinical trials are ongoing exploring danazol’s expanded indications, with some recent studies indicating efficacy in low-dose regimens to mitigate side effects.

• Safety concerns remain paramount; long-term hepatotoxicity and androgenic adverse effects limit its use, underscoring the need for optimized dosing and formulations.

• Market size is stable but niche-oriented, driven mainly by hereditary angioedema management and off-label uses, with limited growth potential absent significant innovation.

• Opportunities lie in drug repurposing and formulation innovations that can improve safety profiles and patient adherence.

• Regulatory pathways will require robust evidence for new indications, but danazol’s low-cost profile sustains its relevance in resource-constrained settings.

Conclusion

Danazol maintains a specialized position in the pharmaceutical landscape. While its legacy is rooted in decades-old applications, current clinical research and reformulation efforts could revitalize its market presence. Business strategies that focus on safety optimization, new therapeutic niches, and cost-effective delivery systems will be critical to leveraging its full potential in the evolving biotech environment.

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FAQs

1. What are the main indications for danazol currently?
Danazol is primarily used for hereditary angioedema prophylaxis, endometriosis, and fibroadenomas. Off-label and research applications include autoimmune and inflammatory disorders.

2. Are there recent advancements in danazol formulations?
Research is exploring transdermal and targeted delivery systems aimed at reducing androgenic side effects and improving tolerability.

3. What are the primary safety concerns associated with danazol?
Long-term hepatotoxicity, virilization in women and children, lipid profile alterations, and potential carcinogenicity pose safety challenges.

4. How does danazol compare to newer therapies in terms of efficacy and safety?
While effective, danazol’s safety profile is less favorable compared to biologics and modern hormonal therapies, limiting its use primarily to resource-limited settings or niche indications.

5. Could danazol’s market resurgence occur in the future?
Yes, especially if ongoing trials demonstrate safe, effective low-dose regimens and new formulations emerge, enabling broader and safer applications.

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Sources

[1] European Society for Human Genetics. Clinical management guidelines for hereditary angioedema. 2021.

[2] Johnson, L., et al. (2022). Low-dose danazol for endometriosis: efficacy and safety. Fertility and Sterility, 117(2), 350-358.

[3] Pharmacovigilance Data Reports, EMA. (2022). Danazol safety assessment update.