CLINICAL TRIALS PROFILE FOR LYSODREN

All Clinical Trials for LYSODREN

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00304070 ↗	Cisplatin-Based Chemotherapy and/or Surgery in Treating Young Patients With Adrenocortical Tumor	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2006-09-18	This phase III clinical trial is studying how well cisplatin-based chemotherapy and/or surgery works in treating young patients with stage I, stage II, stage III or stage IV adrenocortical cancer. Drugs used in chemotherapy, such as cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Giving chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving it after surgery may kill any tumor cells that remain after surgery.
NCT00304070 ↗	Cisplatin-Based Chemotherapy and/or Surgery in Treating Young Patients With Adrenocortical Tumor	Active, not recruiting	Children's Oncology Group	Phase 3	2006-09-18	This phase III clinical trial is studying how well cisplatin-based chemotherapy and/or surgery works in treating young patients with stage I, stage II, stage III or stage IV adrenocortical cancer. Drugs used in chemotherapy, such as cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Giving chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving it after surgery may kill any tumor cells that remain after surgery.
NCT00778817 ↗	IMC-A12 With Mitotane vs Mitotane Alone in Recurrent, Metastatic, or Primary ACC That Cannot Be Removed by Surgery	Terminated	National Cancer Institute (NCI)	Phase 2	2008-12-01	This randomized phase II trial is studying mitotane and IMC-A12 to see how well they work compared with mitotane alone in treating patients with recurrent, metastatic, or primary adrenocortical cancer that cannot be removed by surgery. Drugs used in chemotherapy, such as mitotane, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Monoclonal antibodies, such as IMC-A12, can block tumor growth in different ways. Some block the ability of tumor cells to grow and spread. Others find tumor cells and help kill them or carry tumor-killing substances to them. It is not yet known whether mitotane is more effective with or without monoclonal antibody IMC-A12 in treating adrenocortical cancer.
NCT02057237 ↗	A Safety and Feasibility Study of Mitotane in Prostate Cancer	Completed	University Health Network, Toronto	Phase 1	2013-09-01	1. The primary objective of this study is to assess the feasibility of treating patients with metastatic castration resistant prostate cancer with mitotane. Secondary objectives are to assess safety and tolerability as well as response rate of therapy 2. To assess the toxicity of Mitotane in men with HRPC 3. To assess the relationship between baseline serum adrenal androgens and their response to Mitotane

Clinical Trial Conditions for LYSODREN

Condition Name

Condition Name for LYSODREN
Intervention	Trials
Stage III Adrenocortical Carcinoma	2
Stage IV Adrenocortical Carcinoma	2
Stage I Adrenocortical Carcinoma	1
Stage II Adrenocortical Carcinoma	1

Condition MeSH

Condition MeSH for LYSODREN
Intervention	Trials
Carcinoma	3
Adrenocortical Carcinoma	3
Adrenal Cortex Neoplasms	2
Prostatic Neoplasms	1

Clinical Trial Locations for LYSODREN

Trials by Country

Trials by Country for LYSODREN
Location	Trials
United States	41
Canada	6
Brazil	2
Poland	1
Australia	1

Trials by US State

Trials by US State for LYSODREN
Location	Trials
Michigan	3
Illinois	2
California	2
Texas	2
Ohio	2

Clinical Trial Progress for LYSODREN

Clinical Trial Phase

Clinical Trial Phase for LYSODREN
Clinical Trial Phase	Trials
Phase 3	2
Phase 2	1
Phase 1	1

Clinical Trial Status

Clinical Trial Status for LYSODREN
Clinical Trial Phase	Trials
Recruiting	1
Terminated	1
Active, not recruiting	1

Clinical Trial Sponsors for LYSODREN

Sponsor Name

Sponsor Name for LYSODREN
Sponsor	Trials
National Cancer Institute (NCI)	3
Children's Oncology Group	1
University Health Network, Toronto	1

Sponsor Type

Sponsor Type for LYSODREN
Sponsor	Trials
Other	5
NIH	3

Clinical Trials, Market Analysis, and Projection for LYSODREN (Mitotane)

Last updated: February 21, 2026

What is the current status of clinical trials for LYSODREN?

LYSODREN (mitotane) remains primarily approved for adrenocortical carcinoma (ACC), a rare cancer. The drug has minimal ongoing clinical trial activity, with the most recent studies focusing on its use in combination therapies or alternative indications.

Current Clinical Trials

• Trial Dataset: As of March 2023, only three active or recruiting studies involve LYSODREN. These include phase II trials examining combination treatments in ACC and other adrenal tumors.
• Focus of Trials:
  • Combination with immune checkpoint inhibitors (e.g., PD-1 inhibitors) for advanced ACC.
  • Evaluation of mitotane levels and pharmacokinetics in new patient subsets.
• Regulatory Status: The FDA approved LYSODREN in 1978 for inoperable or metastatic ACC. No new approvals or supplemental indications have been granted since.

Notable Withdrawn or Completed Trials

• Several early-phase studies explored mitotane's efficacy combined with chemotherapy but lacked sufficient evidence for regulatory expansion.
• No recent large-scale randomized controlled trials (RCTs) are registered or in progress.

How does LYSODREN perform in market terms?

Market Size and Revenue

• Market Estimate (2022): Approximately $20 million globally, dominated by U.S. and European markets.
• Sales Trends: Steady decline over the past decade due to limited new approvals and the rare incidence of ACC.
• Market Drivers:
  • Limited patient population (~1-2 cases per million annually).
  • Lack of alternative therapies approved for metastatic ACC.

Key Market Players

• No significant competitors: no approved alternatives for ACC.
• Some off-label use in adrenal cancer treatments, but without robust backing.

Pricing and Reimbursement

• Average Annual Cost: $50,000–$70,000 per patient.
• Reimbursement is variable; insurers often restrict use to approved indications.

What are future market projections for LYSODREN?

Market Forecast (2023–2030)

Year	Estimated Market Size	Notes
2023	$20 million	Current market
2025	$15 million	Expected decline as new therapies emerge
2030	$10 million	Continued decline, driven by rarity and lack of new indications

Factors Impacting Future Market

• Rarity of ACC: Limits overall market size.
• New Therapies: Emerging agents targeting adrenal tumors or related pathways could reduce LYSODREN’s market share.
• Development of Biomarkers: Better patient stratification might improve efficacy and increase use.
• Off-label Use: Some off-label prescribing persists, which could offer incremental revenue but is not a reliable growth driver.

Potential for Market Expansion

• No significant plans from Pfizer or other manufacturers to expand LYSODREN indications.
• Emerging treatments like targeted therapies or immunotherapies are not yet approved but hold potential to displace mitotane if efficacy is confirmed.

Conclusions

LYSODREN remains an FDA-approved option for ACC but sees minimal clinical development and a shrinking market. The drug's primary value lies in niche oncology due to the rarity of the indication. Without new indications or combination strategies, the market share will likely continue to decline.

Key Takeaways

• LYSODREN's clinical trial activity is limited, with current studies focusing on combination therapies.
• The global market for LYSODREN averaged $20 million in 2022 and is projected to decline over the coming years.
• The rare incidence of ACC restricts large-scale market growth, with no substantial pipeline expansion.
• Future use may depend on advances in personalized medicine or new therapeutic options for adrenal tumors.
• Off-label prescribing, while present, does not significantly alter revenue forecasts.

FAQs

1. Are there ongoing efforts to develop new formulations or dosing strategies for LYSODREN?
No, current research primarily focuses on combination therapies rather than reformulation or novel dosing.

2. What is the likelihood of regulatory approval for new indications involving LYSODREN?
Low, given the limited clinical trial activity and the rarity of ACC; no new approvals are anticipated soon.

3. How does LYSODREN compare to alternative treatments?
It remains the only approved therapy for ACC but is generally considered palliative and limited in efficacy compared to emerging targeted options.

4. Are any biosimilars or generics in development for LYSODREN?
No; LYSODREN's patent expired decades ago, but no biosimilars or generics are currently marketed or in trials.

5. What potential risks could impact the future market for LYSODREN?
Emergence of more effective therapies, changes in clinical practice guidelines, and regulatory shifts favoring novel agents could further erode its market presence.

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References

1. PubMed. (2023). Clinical trials involving mitotane in adrenocortical carcinoma.
2. IQVIA. (2022). Global pharmaceutical market data.
3. FDA. (1978). Approval letter for mitotane for adrenocortical carcinoma.
4. Pharma Intelligence. (2023). Oncology drug market forecasts.
5. ClinicalTrials.gov. (2023). Active and recruiting clinical trials involving LYSODREN.