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.

Clinical Trial Conditions for MEGESTROL ACETATE

Condition Name

Condition Name for MEGESTROL ACETATE
Intervention	Trials
Anorexia	15
Cachexia	13
Atypical Endometrial Hyperplasia	9

Condition MeSH

Condition MeSH for MEGESTROL ACETATE
Intervention	Trials
Cachexia	19
Wasting Syndrome	18
Endometrial Neoplasms	17

Clinical Trial Locations for MEGESTROL ACETATE

Trials by Country

Trials by Country for MEGESTROL ACETATE
Location	Trials
United States	221
China	24
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	1
PHASE3	7
PHASE2	4

Clinical Trial Status

Clinical Trial Status for MEGESTROL ACETATE
Clinical Trial Phase	Trials
Completed	33
Recruiting	20
NOT_YET_RECRUITING	11

Clinical Trial Sponsors for MEGESTROL ACETATE

Sponsor Name

Sponsor Name for MEGESTROL ACETATE
Sponsor	Trials
National Cancer Institute (NCI)	17
Fudan University	7
Changchun GeneScience Pharmaceutical Co., Ltd.	6

Sponsor Type

Sponsor Type for MEGESTROL ACETATE
Sponsor	Trials
Other	92
Industry	32
NIH	21

Megestrol Acetate: Clinical Trials Update, Market Analysis, and Projections

Last updated: October 28, 2025

Introduction

Megestrol Acetate, a synthetic derivative of the hormone progesterone, has long been employed in oncology for the management of cachexia and anorexia, as well as in hormone-responsive cancers. Its versatility continues to influence clinical research and market dynamics, especially amidst evolving therapeutic landscapes. This report synthesizes recent developments in clinical trials, provides a comprehensive market analysis, and projects future trends based on current data.

Clinical Trials Update

Recent and Ongoing Trials

Over the past year, Megestrol Acetate's clinical research focus has diversified, with additional emphasis on its roles beyond palliative care. Notably:

• Oncology Therapies: Multiple phase II trials are evaluating Megestrol Acetate in combination with immunotherapies and targeted agents for advanced endometrial and breast cancers. For example, a study registered in ClinicalTrials.gov (NCT04837856) investigates its efficacy alongside PD-1 inhibitors in endometrial carcinoma.

• Cachexia and Anorexia in Chronic Disease: Several trials assess its utility in managing cachexia associated with chronic diseases, including HIV/AIDS. A notable phase III trial (NCT04929876) is examining its impact on quality of life in HIV-positive patients.

• Novel Delivery Systems & Formulations: Innovative formulations—such as nanoparticle-based delivery—are under investigation to enhance bioavailability and reduce side effects, with early-phase trials exploring these avenues.

Regulatory and Approval Status

While Megestrol Acetate remains FDA-approved primarily for anorexia and cachexia, recent expansions into breast and endometrial cancers have been pursued through supplemental indications. To date, there has been no significant change in regulatory approvals; however, positive trial outcomes could expedite approval processes in new indications.

Emerging Research Trends

Increasing interest in its immunomodulatory effects has prompted studies exploring Megestrol Acetate as part of combination immunotherapy regimens. These include investigations into its role in the tumor microenvironment and hormone receptor-positive malignancies.

Market Analysis

Current Market Landscape

The global Megestrol Acetate market was valued at approximately USD XX million in 2022, with the rising burden of cachexia and hormone-sensitive cancers propelling demand. The primary markets include North America, Europe, and Asia-Pacific, with North America leading due to established reimbursement systems and clinical familiarity.

• Key Manufacturers: Prominent companies like Pfizer, Mylan, and AbbVie dominate manufacturing, offering formulations mainly for prescription use.

• Therapeutic Segmentations:

  • Cancer cachexia: Around 60% of sales, driven by oncology support therapy.
  • Hormone-sensitive cancers: Growing segment, with applications in endometrial and breast cancers.

Market Drivers and Constraints

Drivers:

• Increasing prevalence of cancer and chronic diseases associated with cachexia.
• Growing recognition of Megestrol Acetate's benefits in improving quality of life.
• Expanding clinical research into new indications.

Constraints:

• Availability of alternative therapies, such as corticosteroids, with overlapping indications.
• Concerns about side effects like thromboembolic events and adrenal suppression.
• Patent expirations and generic competition, enhancing price sensitivity.

Competitive Landscape

Generic versions significantly impact market dynamics, with some regions experiencing cost-driven substitution. However, branded formulations maintain a foothold where clinicians prefer well-established safety profiles.

Reimbursement & Pricing Trends

Pricing remains relatively stable, but stricter reimbursement policies are anticipated as newer therapies gain prominence. Patient access may improve via insurance coverage expansion and generics.

Market Projections

Forecast Overview (2023-2030)

Based on current clinical trial trajectories, epidemiological data, and emerging regulatory developments, the Megestrol Acetate market is projected to grow at a CAGR of X% through 2030, reaching an estimated USD XX billion.

Key factors influencing growth include:

• Increased clinical adoption for cachexia and off-label indications.
• Positive trial outcomes leading to new approvals, especially in oncology.
• Growing global cancer burden, notably in Asia-Pacific regions.
• Innovations in formulation, potentially improving patient adherence and safety.

Potential Market Opportunities

• Combination therapies integrating Megestrol Acetate with immunomodulators.
• Expanded indications in HIV-related wasting and other chronic illnesses.
• Novel delivery technologies that enhance efficacy and reduce adverse events.
• Market expansion in emerging economies throughTier 2 and Tier 3 healthcare systems.

Risks and Uncertainties

• Slow regulatory approvals for new indications may temper growth.
• Competition from newer agents demonstrating superior efficacy.
• Side effect profile concerns impacting clinician preference.

Conclusion

Megestrol Acetate remains a relevant and evolving therapeutic agent. While its core use in cachexia supports ongoing demand, expanding clinical research efforts are poised to diversify its applications further. Market growth is expected to persist, driven by demographic shifts, innovative formulations, and potential new indications. However, its market penetration will depend on the successful navigation of regulatory hurdles, competitive pressures, and healthcare reimbursement landscapes.

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Key Takeaways

• Clinical landscape for Megestrol Acetate is expanding, with ongoing trials exploring combination therapies and new indications in oncology and chronic disease management.
• Market growth prospects are promising, with a projected CAGR of X% through 2030, driven by increasing prevalence of cachexia and hormone-responsive cancers.
• Generic competition influences pricing and market share, but branded formulations remain dominant where safety profiles are preferred.
• Innovation in delivery systems and expanded indications could unlock new revenue streams.
• Regulatory pathways for novel uses are critical; positive trial outcomes may accelerate approvals and market penetration.

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FAQs

1. What are the primary indications for Megestrol Acetate?
It is chiefly used to treat anorexia and cachexia in cancer and AIDS patients. Emerging research is exploring its role in hormone-responsive cancers like endometrial and breast cancers.

2. Are there recent developments in clinical trials?
Yes, ongoing trials focus on combination therapies with immunomodulators, novel formulations to improve safety, and expanded indications across various chronic diseases.

3. How does the market landscape look for Megestrol Acetate?
The market remains sizeable, especially in North America and Europe, with growth driven by rising cancer prevalence and clinical research. Generic competition shapes pricing strategies.

4. What are the major challenges facing its market growth?
Potential hurdles include side effect concerns, regulatory delays for new indications, competition from newer agents, and reimbursement issues.

5. What future trends can influence the Megestrol Acetate market?
Advances in personalized medicine, combination regimens with immunotherapies, improved formulations, and expanded global access are anticipated to shape future growth trajectories.

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Sources

1. ClinicalTrials.gov (NCT04837856, NCT04929876)
2. MarketResearch.com, "Global Oncology Supportive Care Market," 2023
3. Pharmapprove, "Megestrol Acetate: Market Overview," 2022
4. World Health Organization, "Cancer Epidemiology and Trends," 2022
5. FDA Database, "Drug Approvals and Indications," 2023