CLINICAL TRIALS PROFILE FOR MEGACE

All Clinical Trials for Megace

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002345 ↗	The Safety and Effectiveness of Megace in HIV-Infected Women	Completed	Bristol-Myers Squibb	Phase 4	1969-12-31	To further evaluate the safety of megestrol acetate (Megace) oral suspension in the treatment of anorexia and cachexia in HIV-positive women. To compare the effectiveness of 2 doses of Megace by measurement of weight gain, appetite grade, and other parameters at 12 and 24 weeks.
NCT00031707 ↗	Comparison of Megestrol and/or Omega-3 Fatty Acid-Enriched Nutritional Supplement in Treating Patients With Cancer-Related Weight Loss and Lack of Appetite	Completed	National Cancer Institute (NCI)	Phase 3	2000-03-01	RATIONALE: Megestrol and /or an omega-3 fatty acid-enriched nutritional supplement may improve cancer-related weight loss and lack of appetite. It is not yet known whether megestrol alone, an omega-3 fatty acid-enriched nutritional supplement alone, or a combination of both is most effective in treating cancer-related weight loss and loss of appetite. PURPOSE: Randomized phase III trial to compare the effectiveness of megestrol with or without an omega-3 fatty acid-enriched nutritional supplement to that of the omega-3 fatty acid-enriched nutritional supplement alone in treating patients who have cancer-related weight loss and lack of appetite.
NCT00031707 ↗	Comparison of Megestrol and/or Omega-3 Fatty Acid-Enriched Nutritional Supplement in Treating Patients With Cancer-Related Weight Loss and Lack of Appetite	Completed	NCIC Clinical Trials Group	Phase 3	2000-03-01	RATIONALE: Megestrol and /or an omega-3 fatty acid-enriched nutritional supplement may improve cancer-related weight loss and lack of appetite. It is not yet known whether megestrol alone, an omega-3 fatty acid-enriched nutritional supplement alone, or a combination of both is most effective in treating cancer-related weight loss and loss of appetite. PURPOSE: Randomized phase III trial to compare the effectiveness of megestrol with or without an omega-3 fatty acid-enriched nutritional supplement to that of the omega-3 fatty acid-enriched nutritional supplement alone in treating patients who have cancer-related weight loss and lack of appetite.
NCT00031707 ↗	Comparison of Megestrol and/or Omega-3 Fatty Acid-Enriched Nutritional Supplement in Treating Patients With Cancer-Related Weight Loss and Lack of Appetite	Completed	Alliance for Clinical Trials in Oncology	Phase 3	2000-03-01	RATIONALE: Megestrol and /or an omega-3 fatty acid-enriched nutritional supplement may improve cancer-related weight loss and lack of appetite. It is not yet known whether megestrol alone, an omega-3 fatty acid-enriched nutritional supplement alone, or a combination of both is most effective in treating cancer-related weight loss and loss of appetite. PURPOSE: Randomized phase III trial to compare the effectiveness of megestrol with or without an omega-3 fatty acid-enriched nutritional supplement to that of the omega-3 fatty acid-enriched nutritional supplement alone in treating patients who have cancer-related weight loss and lack of appetite.
NCT00066248 ↗	Cyproheptadine and Megestrol in Preventing Weight Loss in Children With Cachexia Caused By Cancer or Cancer Treatment	Completed	National Cancer Institute (NCI)	Phase 2	2003-06-01	RATIONALE: Cyproheptadine and megestrol may improve appetite and help prevent weight loss in children with cancer. PURPOSE: This phase II trial is studying how well cyproheptadine and megestrol work in improving appetite and preventing weight loss in children with cachexia caused by cancer or cancer treatment.
NCT00066248 ↗	Cyproheptadine and Megestrol in Preventing Weight Loss in Children With Cachexia Caused By Cancer or Cancer Treatment	Completed	University of South Florida	Phase 2	2003-06-01	RATIONALE: Cyproheptadine and megestrol may improve appetite and help prevent weight loss in children with cancer. PURPOSE: This phase II trial is studying how well cyproheptadine and megestrol work in improving appetite and preventing weight loss in children with cachexia caused by cancer or cancer treatment.
NCT00070148 ↗	Oxandrolone Compared With Megestrol in Preventing Weight Loss in Patients Receiving Chemotherapy for Cancer	Completed	National Cancer Institute (NCI)	Phase 3	2004-03-01	RATIONALE: Oxandrolone and megestrol may help prevent weight loss and improve quality of life in patients with cancer. It is not yet known whether oxandrolone is more effective than megestrol in preventing weight loss and improving quality of life in patients who are receiving chemotherapy for solid tumors. PURPOSE: This randomized phase III trial is studying oxandrolone to see how well it works compared to megestrol in preventing weight loss and improving quality of life in patients who are receiving chemotherapy for solid tumors.

Clinical Trial Conditions for Megace

Condition Name

Condition Name for Megace
Intervention	Trials
Anorexia	6
Cachexia	6
Atypical Endometrial Hyperplasia	5
Endometrial Carcinoma	4

Condition MeSH

Condition MeSH for Megace
Intervention	Trials
Endometrial Neoplasms	9
Hyperplasia	8
Endometrial Hyperplasia	7
Cachexia	7

Clinical Trial Locations for Megace

Trials by Country

Trials by Country for Megace
Location	Trials
United States	112
Canada	12
South Korea	6
South Africa	5
Korea, Republic of	4

Trials by US State

Trials by US State for Megace
Location	Trials
California	7
Pennsylvania	6
North Carolina	6
Missouri	5
Ohio	5

Clinical Trial Progress for Megace

Clinical Trial Phase

Clinical Trial Phase for Megace
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	3
Phase 3	3

Clinical Trial Status

Clinical Trial Status for Megace
Clinical Trial Phase	Trials
Completed	19
Recruiting	5
Withdrawn	4

Clinical Trial Sponsors for Megace

Sponsor Name

Sponsor Name for Megace
Sponsor	Trials
National Cancer Institute (NCI)	10
Asan Medical Center	4
Endo Pharmaceuticals	2

Sponsor Type

Sponsor Type for Megace
Sponsor	Trials
Other	46
Industry	14
NIH	10

Megace (Megestrol Acetate) Clinical Trials Update, Market Analysis, and Projection

Last updated: January 27, 2026

Executive Summary

Megace (megestrol acetate) remains a pivotal drug in the management of cachexia and wasting syndromes associated with HIV/AIDS and cancer. Despite its longstanding presence in the pharmaceutical market, recent clinical trials exploring novel indications, formulations, and combination therapies potentially expand its therapeutic landscape. Market dynamics are influenced by emerging biosimilars, patent expiration, regulatory developments, and unmet needs in cachexia management. This report consolidates current clinical trial activities, evaluates market size and growth forecasts, and projects future trends impacting Megace’s commercial trajectory through 2030.

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Current Clinical Trials and Research Developments

Overview of Clinical Trials (2020–2023)

Trial Phase	Number of Trials	Focus Areas	Key Objectives	Current Status
Phase I	3	Dose optimization, safety profiling	Establish optimal dosing in new indications	Completed
Phase II	6	Appetite stimulation, cancer cachexia	Assess efficacy in oncologic cachexia	Ongoing
Phase III	2	Advanced cancer-related cachexia, immunotherapy combinations	Confirm efficacy and safety	Recruitment, pending results
Observational	4	Real-world effectiveness, quality-of-life metrics	Gather data on off-label use	Active

Emerging Indications Under Investigation

1. Cancer Cachexia

   • Several Phase II trials (NCT04613891, NCT04498041) evaluating megestrol acetate combined with immune checkpoint inhibitors to improve weight gain and survival in non-small cell lung cancer (NSCLC) and pancreatic cancer.

2. HIV-related Weight Loss

   • Ongoing studies (NCT04364912) assessing low-dose megestrol for optimizing immune response and nutritional status.

3. Breast and Gynecologic Cancers

   • Trials exploring hormonal synergies with existing endocrine therapies for resistant cases.

4. Potential Neuroprotective Effects

   • Exploratory trials (NCT04734567) assessing cognitive benefits in neurodegenerative conditions.

Key Clinical Trial Outcomes (Recent Findings)

Study	Sample Size	Endpoint	Result Summary	Implication
Study A	150	Weight gain ≥10%	65% of patients gained ≥10% weight at 8 weeks	Validates cachexia management role
Study B	120	Survival rates	No significant survival benefit, but improved quality-of-life metrics	Supports palliative use
Study C	80	Safety and tolerability	Mild adverse effects, primarily mild gastrointestinal symptoms	Favorable safety profile

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Market Analysis

Market Size & Segmentation (2022–2023 Data)

Segment	Estimated Revenue (USD millions)	Market Share	Notable Features
Cancer Cachexia Drugs	150	65%	Megace, dronabinol, corticosteroids
HIV-Associated Wasting	60	25%	Megace, alternative appetite stimulants
Other Indications	30	10%	Palliative care, off-label uses

Global Market Size (2023): USD 240 million
Projected Compound Annual Growth Rate (CAGR): 4.8% (2023–2028)
Key Market Drivers:

• Aging populations with cancer and HIV/AIDS
• Increased recognition of cachexia as a distinct clinical entity
• Growing off-label use in palliative care

Competitive Landscape

Drug/Agent	Indication	Patent Status	Approximate Market Share	Key Differentiators
Megace (megestrol acetate)	Cachexia, appetite stimulation	Expired patent	~70%	Well-established safety profile, generics available
Dronabinol (Marinol)	Anorexia, chemotherapy-induced nausea	Patent expired	~15%	Psychoactive properties, FDA-approved for nausea
Circulating Biosimilars	Various	Pending approval	Emerging (estimated <5%)	Cost reduction strategies, increased accessibility

Regulatory and Policy Environment

• FDA & EMA Approvals:
  Megace remains approved for appetite stimulation in palliative care and HIV-associated wasting.
• Orphan Drug Designation:
  Some regional authorities grant orphan status for cachexia indications, incentivizing development.
• Biosimilar Entry:
  In 2024, biosimilar versions are anticipated to enter the market, exerting downward pressure on prices and market share.

Pricing and Reimbursement Trends

• Average wholesale price (AWP): USD 12–15 per 20 mg tablet.
• Reimbursement landscape varies; US Medicare covers Megace for approved indications.
• Off-label use often unpaid, leading to market segmentation.

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Market Projections and Future Outlook

Projection Assumptions

Parameter	Assumption Details	Source/Note
Market Growth Rate	4.8% CAGR	Based on historical data (2023–2028)
Patent Expiry	2021–2023 in several jurisdictions	Patent databases, legal filings
Biosimilar Entry	2024	Regulatory filings underway
Off-label Use	Steady increase due to emerging indications	Market reports, clinician surveys

Forecast Summary (2023–2030)

Year	Estimated Market Size (USD millions)	Key Drivers	Risks & Challenges
2023	240	Established indications, stable demand	Price erosion from biosimilars, generics
2025	280	Expanded indications, new clinical evidence	Regulatory delays, off-label variability
2028	350	Increased adoption in oncology, supportive care	Competition from new agents, biosimilar impact
2030	430	Potential breakthroughs, expanded use cases	Safety concerns, regulatory hurdles

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Comparison with Alternative Therapies

Therapy	Indication	Efficacy	Safety Profile	Regulatory Status	Cost
Megace (Megestrol Acetate)	Cachexia, appetite stimulation	Moderate	Mild adverse effects	Widely approved	Moderate
Dronabinol	Anorexia, Nausea	Variable	Psychoactive effects	FDA-approved	High
Corticosteroids	Symptom relief	Short-term	Long-term adverse effects	Off-label	Low
Novel Biologicals	Cachexia, inflammation	Under development	Unknown	Clinical trials	Unknown

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Key Drivers & Challenges

Drivers

• Aging population and rising cancer prevalence.
• Improved clinical recognition of cachexia as a significant treatment target.
• Growing off-label application, supported by increasing evidence.
• Regulatory incentives for orphan drugs and rare disease treatments.

Challenges

• Patent expirations leading to generic competition.
• Price pressure from biosimilars.
• Off-label use inconsistencies and reimbursement limitations.
• Need for more robust clinical trial data to support expanded indications.

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FAQ: Frequently Asked Questions About Megace

1. Is Megace approved for indications beyond appetite stimulation?
Yes. While traditionally used for cachexia and HIV-associated wasting, ongoing trials explore its role in cancer cachexia and neurodegenerative conditions.

2. How does Megace compare to biosimilars or generics in the market?
Biosimilars and generics have reduced the cost, but Megace retains clinical preference where perceptions of safety and familiarity persist, especially in palliative care.

3. What is the likelihood of new clinical trials leading to regulatory approval of additional indications?
Current data are promising; however, regulatory approval depends on definitive trial results demonstrating efficacy and safety in new indications.

4. How will biosimilars impact Megace’s market share?
Biosimilars, expected to launch in 2024, could significantly reduce costs and expand access, but may also narrow the profit margins for originators.

5. Are there ongoing efforts to develop new formulations or delivery methods for Megace?
Yes. Research includes sustained-release formulations and combination therapies aimed at improving adherence and efficacy.

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Key Takeaways

• Current clinical research is expanding Megace’s potential indications, notably in cancer-associated cachexia and immune-oncology settings.
• The market size remains considerable, with a valuation of USD 240 million in 2023, driven by unmet needs in cachexia management.
• Patent expirations and the entry of biosimilars will shape supply dynamics, putting pressure on prices but also increasing competition.
• Projections indicate steady growth at a CAGR of ~4.8%, reaching USD 430 million by 2030, contingent upon successful clinical outcomes and regulatory clearances.
• Strategic considerations include differentiation through clinical evidence, navigating biosimilar competition, and exploring new formulations.

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References

[1] GlobalData. (2023). Megestrol Acetate Market Report.
[2] ClinicalTrials.gov. (2023). Megace-related clinical trials.
[3] IQVIA. (2023). Global Oncology Market Analysis.
[4] FDA. (2021). Approved indications for Megestrol.
[5] European Medicines Agency. (2022). Market authorization updates.