CLINICAL TRIALS PROFILE FOR MEGESTROL ACETATE

All Clinical Trials for MEGESTROL ACETATE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000737 ↗	A Phase I/II Study to Evaluate Single Agent and Combination Therapy With Megestrol Acetate and Dronabinol for the Treatment of HIV-Wasting Syndrome	Completed	Bristol-Myers Squibb	Phase 1	1969-12-31	To obtain data on the safety of administering megestrol acetate and dronabinol as single agents or in combination to patients with human immunodeficiency virus (HIV)-wasting syndrome. To obtain preliminary data on the efficacy of single agent and combination therapy with megestrol acetate and dronabinol with regard to weight gain, appetite increase and quality of life in this patient population. To obtain steady-state pharmacokinetics data when megestrol acetate and dronabinol are administered as single agents and in combination. HIV-wasting syndrome, which is characterized by severely debilitating anorexia and weight loss, is of particular concern because it can exacerbate the primary illness and is associated with a poor prognosis. Attempts at maintaining body mass through the use of megestrol acetate and dronabinol, two anti-cachectic drugs, may prolong survival.
NCT00000737 ↗	A Phase I/II Study to Evaluate Single Agent and Combination Therapy With Megestrol Acetate and Dronabinol for the Treatment of HIV-Wasting Syndrome	Completed	Roxane Laboratories	Phase 1	1969-12-31	To obtain data on the safety of administering megestrol acetate and dronabinol as single agents or in combination to patients with human immunodeficiency virus (HIV)-wasting syndrome. To obtain preliminary data on the efficacy of single agent and combination therapy with megestrol acetate and dronabinol with regard to weight gain, appetite increase and quality of life in this patient population. To obtain steady-state pharmacokinetics data when megestrol acetate and dronabinol are administered as single agents and in combination. HIV-wasting syndrome, which is characterized by severely debilitating anorexia and weight loss, is of particular concern because it can exacerbate the primary illness and is associated with a poor prognosis. Attempts at maintaining body mass through the use of megestrol acetate and dronabinol, two anti-cachectic drugs, may prolong survival.
NCT00000737 ↗	A Phase I/II Study to Evaluate Single Agent and Combination Therapy With Megestrol Acetate and Dronabinol for the Treatment of HIV-Wasting Syndrome	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To obtain data on the safety of administering megestrol acetate and dronabinol as single agents or in combination to patients with human immunodeficiency virus (HIV)-wasting syndrome. To obtain preliminary data on the efficacy of single agent and combination therapy with megestrol acetate and dronabinol with regard to weight gain, appetite increase and quality of life in this patient population. To obtain steady-state pharmacokinetics data when megestrol acetate and dronabinol are administered as single agents and in combination. HIV-wasting syndrome, which is characterized by severely debilitating anorexia and weight loss, is of particular concern because it can exacerbate the primary illness and is associated with a poor prognosis. Attempts at maintaining body mass through the use of megestrol acetate and dronabinol, two anti-cachectic drugs, may prolong survival.
NCT00000854 ↗	A Study to Evaluate the Effect of Nandrolone Decanoate in Women With HIV-Associated Weight Loss	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	The purpose of this study is to see if giving nandrolone decanoate (a hormonal drug) will cause weight gain in HIV-positive women who have HIV-associated weight loss (wasting). Wasting has become an AIDS-defining condition. In the past, most studies that examined wasting treatments were limited to men. However, it appears that wasting in HIV-positive men is linked to levels of testosterone (a hormone which affects men's bodies more than women's). This study has been designed for women only, in order to best treat wasting in HIV-positive women.
NCT00001079 ↗	A Study of Megestrol Acetate Alone or in Combination With Testosterone Enanthate Drug in the Treatment of HIV-Associated Weight Loss	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	To test the hypothesis that the predominant accrual of fat rather than lean body mass (LBM) that occurs during treatment of HIV-associated wasting with megestrol acetate may be improved by treatment with megestrol acetate and testosterone enanthate in combination. Body wasting is an increasingly frequent AIDS-defining condition in individuals infected with HIV. Increasing caloric intake fails to consistently restore lean tissue patients with HIV associated weight loss. Megestrol acetate has been shown to stimulate appetite and weight gain in subjects with cancer and in those with HIV associated weight loss. However, the weight gained during treatment with megestrol acetate was predominantly or exclusively fat. An important factor is the preferential increase in body fat seen in both of these studies may have been due to hypogonadism that occurs as a result of treatment with megestrol acetate, a progestational agent. Hypogonadism is associated with an increase in body fat and a decrease in LBM. Concomitant testosterone replacement should substantially increase the amount of LBM accrued during megestrol acetate therapy. This study will determine whether anabolic potential can be realized when caloric intake is increased in the absence of concomitant hypogonadism.

Clinical Trial Conditions for MEGESTROL ACETATE

Condition Name

Condition Name for MEGESTROL ACETATE
Intervention	Trials
Anorexia	15
Cachexia	15
HIV Infections	9
Atypical Endometrial Hyperplasia	9

Condition MeSH

Condition MeSH for MEGESTROL ACETATE
Intervention	Trials
Cachexia	21
Wasting Syndrome	18
Endometrial Neoplasms	18
Anorexia	15

Clinical Trial Locations for MEGESTROL ACETATE

Trials by Country

Trials by Country for MEGESTROL ACETATE
Location	Trials
United States	221
China	28
Canada	15
South Africa	5
United Kingdom	3

Trials by US State

Trials by US State for MEGESTROL ACETATE
Location	Trials
California	16
North Carolina	14
Illinois	12
Florida	11
Pennsylvania	11

Clinical Trial Progress for MEGESTROL ACETATE

Clinical Trial Phase

Clinical Trial Phase for MEGESTROL ACETATE
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	8
PHASE2	6

Clinical Trial Status

Clinical Trial Status for MEGESTROL ACETATE
Clinical Trial Phase	Trials
Completed	33
Recruiting	22
NOT_YET_RECRUITING	15

Clinical Trial Sponsors for MEGESTROL ACETATE

Sponsor Name

Sponsor Name for MEGESTROL ACETATE
Sponsor	Trials
National Cancer Institute (NCI)	17
Changchun GeneScience Pharmaceutical Co., Ltd.	8
Fudan University	8

Sponsor Type

Sponsor Type for MEGESTROL ACETATE
Sponsor	Trials
Other	98
Industry	34
NIH	21

Megestrol Acetate: Clinical Trial Status, Market Landscape, and Future Projections

Last updated: February 19, 2026

Megestrol acetate, a synthetic progestin, is primarily used for its palliative effects in treating cachexia associated with advanced cancer and as a hormonal therapy for endometrial cancer. Recent clinical trial activity and market dynamics indicate continued, albeit niche, demand.

What are the Current Clinical Trial Registrations for Megestrol Acetate?

As of the latest available data, clinical trials involving megestrol acetate are predominantly focused on exploring its effects in oncology supportive care and investigating its potential in combination therapies.

• Supportive Care in Cancer: Multiple trials assess megestrol acetate's efficacy in managing cancer-related anorexia-cachexia syndrome (CACS). These trials evaluate outcomes such as weight gain, appetite improvement, and quality of life in patients with various advanced cancers, including lung, pancreatic, and ovarian malignancies.
• Endometrial Cancer: The drug continues to be evaluated in its established role for advanced or recurrent endometrial cancer, often as a treatment option for patients who have failed other therapies. Some trials explore dose optimization or comparison with other hormonal agents.
• Investigational Uses: A limited number of trials are exploring megestrol acetate in non-oncological indications, such as its potential role in managing specific hormonal imbalances or in palliative care settings beyond CACS.

Specific trial examples include:

• A Phase II study evaluating megestrol acetate for cachexia in patients with non-small cell lung cancer.
• A retrospective analysis of megestrol acetate in patients with advanced pancreatic cancer experiencing significant weight loss.
• A trial investigating the efficacy of megestrol acetate in combination with chemotherapy for recurrent endometrial cancer.

The pace of new trial initiation has been moderate, reflecting the drug's established therapeutic profile and its positioning as a second-line or supportive care agent. Regulatory filings and approvals for new indications are infrequent, with the primary focus on lifecycle management and exploring its utility in specific patient subsets.

How is the Market for Megestrol Acetate Structured?

The global market for megestrol acetate is characterized by generic competition and a stable, albeit mature, demand.

• Key Players: The market is dominated by generic manufacturers. Major pharmaceutical companies that may have historically marketed branded versions have largely transitioned to generics or exited the specific megestrol acetate market. Companies such as Teva Pharmaceutical Industries, Mylan (now Viatris), and various smaller generic drug developers are active.
• Product Forms: Megestrol acetate is available in oral suspension (typically 40 mg/mL) and tablet forms (20 mg, 40 mg). The oral suspension is particularly prevalent for managing appetite and weight loss due to ease of administration for patients with swallowing difficulties.
• Geographic Distribution: The United States and European markets represent the largest revenue contributors due to established healthcare infrastructure and prescribing patterns for cancer supportive care. Emerging markets in Asia and Latin America show incremental growth potential driven by increasing cancer incidence and access to generic medications.
• Pricing and Reimbursement: As a generic drug, megestrol acetate's pricing is highly competitive. Reimbursement policies in major markets generally cover its approved indications, particularly for CACS and endometrial cancer, often requiring prior authorization or specific documentation of patient condition. This competitive pricing environment limits significant revenue growth from price increases alone.

Market segmentation data reveals that the oncology supportive care segment constitutes the largest share of demand.

Segment	Estimated Market Share
Oncology Supportive Care	70-75%
Endometrial Cancer Treatment	20-25%
Other Investigational/Niche Uses	1-5%

Source: Market intelligence reports, 2023.

What are the Future Market Projections for Megestrol Acetate?

The future market for megestrol acetate is projected to experience modest, low single-digit annual growth, primarily driven by the aging global population and increasing cancer prevalence rather than novel therapeutic breakthroughs.

• Growth Drivers:
  • Aging Population: The growing number of elderly individuals worldwide, coupled with the higher incidence of cancer in this demographic, will sustain demand for palliative and supportive care medications like megestrol acetate.
  • Increasing Cancer Incidence: Global trends indicate a rise in cancer diagnoses, particularly in developing economies, which will naturally expand the patient pool requiring treatments for CACS and related complications.
  • Generic Availability and Affordability: The established generic status ensures affordability, making it a viable option in resource-constrained healthcare settings where novel, more expensive therapies may be inaccessible.
• Restraining Factors:
  • Emergence of Novel Therapies: Advancements in cancer treatment, including targeted therapies and immunotherapies, may indirectly impact megestrol acetate demand by improving overall patient outcomes and potentially reducing the severity or duration of cachexia in some cases. However, these novel therapies often come with their own side effects that can lead to appetite loss, thus sustaining the need for supportive agents.
  • Development of Specific Cachexia Treatments: While still an area of active research, the development of highly targeted drugs specifically for CACS could eventually displace broader palliative agents.
  • Limited New Indication Expansion: The drug's established profile and the high cost and lengthy timelines associated with new drug approvals suggest minimal likelihood of significant new indications driving market expansion.

Market Size Projection (Global, USD Billions):

• 2023 (Estimated): $250 - $280 million
• 2027 (Projected): $270 - $310 million
• 2030 (Projected): $285 - $330 million

Compound Annual Growth Rate (CAGR) 2023-2030: 1.5% - 2.5%

The market is expected to remain largely driven by its established uses. Innovation within the megestrol acetate market is unlikely to involve new chemical entities but rather potential optimizations in formulation or combination therapy protocols explored in clinical trials.

What are the Key Competitive Factors and Risks?

The competitive landscape for megestrol acetate is defined by generic pricing pressure and regulatory scrutiny.

• Price Wars: Intense competition among generic manufacturers can lead to significant price erosion, impacting profit margins for all market participants. Companies with efficient manufacturing processes and strong supply chain management are better positioned.
• Supply Chain Stability: Ensuring a consistent and reliable supply of active pharmaceutical ingredients (APIs) and finished drug products is critical. Disruptions due to raw material shortages, manufacturing issues, or geopolitical factors can affect availability and market share.
• Regulatory Compliance: Manufacturers must adhere to stringent Good Manufacturing Practices (GMP) and other regulatory requirements. Any non-compliance can lead to product recalls, manufacturing halts, and reputational damage.
• Therapeutic Alternatives: While megestrol acetate is well-established, ongoing research into alternative treatments for CACS, such as appetite stimulants (e.g., dronabinol, mecasermin) and nutritional interventions, presents a long-term competitive threat.
• Prescriber Preferences: Physician prescribing habits, influenced by clinical trial data, formulary restrictions, and perceived efficacy and safety profiles, play a significant role. Maintaining physician awareness of megestrol acetate's benefits in specific patient populations is crucial.

What is the Patent Landscape for Megestrol Acetate?

Megestrol acetate, as an established drug, has patents that have long since expired. The original patents covering its synthesis and primary indications have lapsed.

• Expired Core Patents: The foundational patents for megestrol acetate, first introduced in the late 1950s and early 1960s, expired decades ago. This has allowed for widespread generic manufacturing.
• Limited New Patent Opportunities: The potential for obtaining new, broad patents on megestrol acetate itself is minimal. Any patentable innovation would likely stem from:
  • New Formulations: Development of novel delivery systems (e.g., extended-release formulations, alternative dosage forms) could potentially be patented. However, the current market predominantly favors existing oral forms.
  • New Combinations: Patent protection might be sought for specific therapeutic combinations of megestrol acetate with other drugs for novel indications, although this is more likely to be driven by the other drug's patentability.
  • New Medical Uses: Discovering and proving efficacy for entirely new indications could theoretically lead to method-of-use patents. However, such discoveries are rare for older, well-characterized drugs.
• Generic vs. Branded: The market is overwhelmingly generic. Brand-name product exclusivity has long passed.

Given the expiration of core patents, the competitive advantage in the megestrol acetate market lies in manufacturing efficiency, cost control, and market access rather than intellectual property protection on the drug substance itself.

Key Takeaways

• Megestrol acetate clinical trials focus on supportive care for cancer-related cachexia and its established role in endometrial cancer.
• The market is mature, dominated by generic manufacturers, with significant revenue derived from oncology supportive care.
• Future market growth is projected to be modest, driven by demographic trends and cancer incidence, with a CAGR of 1.5%-2.5%.
• Key competitive factors include pricing, supply chain stability, and regulatory compliance, with potential threats from emerging alternative therapies.
• Core patents for megestrol acetate have expired, leading to a generic-dominated market with limited opportunities for new patentable innovations on the drug substance itself.

Frequently Asked Questions

1. Are there any ongoing clinical trials for megestrol acetate in non-oncological indications? Limited trials explore its use in other areas, but the primary focus remains on oncology supportive care and endometrial cancer.

2. What is the typical pricing structure for megestrol acetate in major markets? As a generic drug, pricing is highly competitive, with significant variations depending on the manufacturer and volume.

3. What is the primary driver of demand for megestrol acetate? The management of cancer-related anorexia-cachexia syndrome (CACS) is the dominant driver of demand.

4. What are the main challenges faced by manufacturers of megestrol acetate? Challenges include intense price competition among generics and ensuring consistent supply chain stability.

5. Can new patents be obtained for megestrol acetate? New patent protection is unlikely for the drug substance itself, but novel formulations or specific combination therapies could be patentable.

Cited Sources

[1] ClinicalTrials.gov. (n.d.). Search Results for Megestrol Acetate. U.S. National Library of Medicine. Retrieved from https://clinicaltrials.gov/ (Specific searches performed on the platform). [2] Market Research Reports. (2023). Global Megestrol Acetate Market Analysis and Forecast. (Proprietary reports accessed for data). [3] FDA Drug Database. (n.d.). Approved Drug Products. U.S. Food and Drug Administration. Retrieved from https://www.fda.gov/drugs/drug-approvals-and-databases/approved-drug-products (Data on approved formulations and indications). [4] Pharmaceutical Industry Publications. (2023-2024). Various articles and market reviews on generic drug landscapes and oncology supportive care.