CLINICAL TRIALS PROFILE FOR ISOTRETINOIN

All Clinical Trials for ISOTRETINOIN

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000764 ↗	Chemoprevention of Anal Neoplasia Arising Secondary to Anogenital Human Papillomavirus Infection in Persons With HIV Infection.	Completed	Hoffmann-La Roche	Phase 1	1969-12-31	PRIMARY: In Phase I, to define a broadly tolerable dose of isotretinoin that can be used in combination with interferon alfa-2a (IFN alfa-2a). In Phase II, to determine trends in efficacy of isotretinoin alone or in combination with IFN alfa-2a as chemoprevention (preventing progression or recurrence) of anal intraepithelial neoplasia ( AIN ) / squamous intraepithelial lesions ( SIL ) in patients with HIV infection. SECONDARY: To evaluate the effects of isotretinoin alone or in combination with IFN alfa-2a on immune function markers, human papillomavirus (HPV) type, and HPV DNA levels. Patients with HIV infection have a significant risk of recurrence following local ablation of intraepithelial neoplasia; thus, anogenital epithelial may become an increasingly important cause of morbidity, and possibly mortality, as the HIV epidemic matures. Clinical studies of non-HIV-infected subjects have established that synthetic retinoids inhibit the progression of epithelial preneoplastic conditions and some neoplastic states.
NCT00000764 ↗	Chemoprevention of Anal Neoplasia Arising Secondary to Anogenital Human Papillomavirus Infection in Persons With HIV Infection.	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	PRIMARY: In Phase I, to define a broadly tolerable dose of isotretinoin that can be used in combination with interferon alfa-2a (IFN alfa-2a). In Phase II, to determine trends in efficacy of isotretinoin alone or in combination with IFN alfa-2a as chemoprevention (preventing progression or recurrence) of anal intraepithelial neoplasia ( AIN ) / squamous intraepithelial lesions ( SIL ) in patients with HIV infection. SECONDARY: To evaluate the effects of isotretinoin alone or in combination with IFN alfa-2a on immune function markers, human papillomavirus (HPV) type, and HPV DNA levels. Patients with HIV infection have a significant risk of recurrence following local ablation of intraepithelial neoplasia; thus, anogenital epithelial may become an increasingly important cause of morbidity, and possibly mortality, as the HIV epidemic matures. Clinical studies of non-HIV-infected subjects have established that synthetic retinoids inhibit the progression of epithelial preneoplastic conditions and some neoplastic states.
NCT00001073 ↗	Safety and Effectiveness of Giving Isotretinoin to HIV-Infected Women to Treat Cervical Tumors	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1969-12-31	The purpose of this study is to see if it is safe and effective to give isotretinoin to HIV-infected women with cervical tumors to prevent these tumors from becoming cancerous. Cervical tumors are found in both HIV-infected and HIV-negative women. However, HIV-infected women are at a greater risk, and often their tumors become cancerous more quickly than those in HIV-negative women. Isotretinoin may be able to prevent this from happening. However, since these tumors tend to disappear over time, many doctors are hesitant to give their patients isotretinoin since this drug causes birth defects. This study looks at whether it is better to treat cervical tumors in HIV-infected women or to wait and see if they will disappear by themselves.
NCT00002506 ↗	Isotretinoin Plus Interferon in Treating Patients With Recurrent Cancer	Completed	Cancer Biotherapy Research Group	Phase 2	1992-08-01	RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. The use of isotretinoin may be an effective way to prevent cancer or stop cancer from growing. Interferon alfa may interfere with the growth of cancer cells. Combining isotretinoin and interferon may be an effective treatment for some recurrent cancers. PURPOSE: Phase II trial to study the effectiveness of interferon alfa plus isotretinoin in treating patients with recurrent cancer.
NCT00002506 ↗	Isotretinoin Plus Interferon in Treating Patients With Recurrent Cancer	Completed	Hoag Memorial Hospital Presbyterian	Phase 2	1992-08-01	RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. The use of isotretinoin may be an effective way to prevent cancer or stop cancer from growing. Interferon alfa may interfere with the growth of cancer cells. Combining isotretinoin and interferon may be an effective treatment for some recurrent cancers. PURPOSE: Phase II trial to study the effectiveness of interferon alfa plus isotretinoin in treating patients with recurrent cancer.
NCT00002737 ↗	Interferon Alfa With or Without Isotretinoin in Treating Patients With Metastatic Kidney Cancer	Completed	European Organisation for Research and Treatment of Cancer - EORTC	Phase 3	1996-03-01	RATIONALE: Interferon alfa may interfere with the growth of the cancer cells and slow the growth of kidney cancer. Isotretinoin may help kidney cancer cells develop into normal cells. It is not yet known whether interferon alfa plus isotretinoin is more effective than interferon alfa alone for kidney cancer. PURPOSE: Randomized phase III trial to compare the effectiveness of interferon alfa with or without isotretinoin in treating patients who have metastatic kidney cancer.

Clinical Trial Conditions for ISOTRETINOIN

Condition Name

Condition Name for ISOTRETINOIN
Intervention	Trials
Neuroblastoma	29
Acne Vulgaris	22
Acne	10
Recurrent Neuroblastoma	8

Condition MeSH

Condition MeSH for ISOTRETINOIN
Intervention	Trials
Neuroblastoma	41
Acne Vulgaris	32
Leukemia	7
COVID-19	7

Clinical Trial Locations for ISOTRETINOIN

Trials by Country

Trials by Country for ISOTRETINOIN
Location	Trials
United States	691
Canada	61
Australia	28
France	13
Egypt	13

Trials by US State

Trials by US State for ISOTRETINOIN
Location	Trials
California	34
Pennsylvania	33
Texas	28
New York	28
Massachusetts	26

Clinical Trial Progress for ISOTRETINOIN

Clinical Trial Phase

Clinical Trial Phase for ISOTRETINOIN
Clinical Trial Phase	Trials
PHASE4	5
PHASE3	4
PHASE1	2

Clinical Trial Status

Clinical Trial Status for ISOTRETINOIN
Clinical Trial Phase	Trials
Completed	76
RECRUITING	22
Not yet recruiting	18

Clinical Trial Sponsors for ISOTRETINOIN

Sponsor Name

Sponsor Name for ISOTRETINOIN
Sponsor	Trials
National Cancer Institute (NCI)	39
Children's Oncology Group	11
M.D. Anderson Cancer Center	7

Sponsor Type

Sponsor Type for ISOTRETINOIN
Sponsor	Trials
Other	175
NIH	46
Industry	36

Clinical Trials Update, Market Analysis, and Projection for Isotretinoin

Last updated: October 27, 2025

Introduction

Isotretinoin, a potent oral retinoid primarily prescribed to treat severe acne refractory to other therapies, continues to be a mainstay in dermatological medicine. Developed decades ago, it remains under active investigation, with ongoing clinical trials exploring broader indications and optimizing safety profiles. This article provides a comprehensive overview of recent clinical trial activity, market dynamics, and future projections for isotretinoin.

Clinical Trials Update

Recent Clinical Trial Developments

Over the past three years, clinical research involving isotretinoin has increasingly focused on expanding its indications beyond severe acne. Notably, the following areas have seen significant trial activity:

• Psychological Impact and Neuropsychiatric Safety: Multiple studies assess the neuropsychiatric adverse effects linked to isotretinoin. For instance, randomized controlled trials (RCTs) such as the NEARNET study evaluate the correlation between isotretinoin and depression, anxiety, or mood disturbances. Results remain nuanced; some data suggest an association, while others attribute mental health issues to pre-existing conditions or confounding factors [1].

• Use in Granulomatous Conditions: Randomized trials are exploring isotretinoin's efficacy in treating granulomatous dermatologic conditions like hidradenitis suppurativa and granulomatous rosacea, offering potential off-label applications.

• Combination Regimens: Trials are investigating isotretinoin combined with other agents such as antibiotics, hormonal therapies, or biologics to optimize efficacy and reduce dosage-related adverse effects.

Safety and Pharmacovigilance Trials

Enhancing safety profiles remains a focus, with ongoing pharmacovigilance studies examining teratogenicity, hepatotoxicity, and mucocutaneous side effects. For example, the iPLEDGE risk management program continues to refine pregnancy prevention strategies associated with isotretinoin use.

Novel Formulations and Delivery Methods

Clinicians and researchers are exploring novel formulations—such as sustained-release microspheres and topical combinations—to improve tolerability and adherence [2]. Early-phase trials suggest these innovations could mitigate some adverse effects and expand patient eligibility.

Summary of Clinical Trial Trends

Aspect	Status	Notable Trials / Initiatives
Expanded Indications	Increased activity	Acne, granulomatous conditions, neuropsychological effects
Safety Profile Optimization	Active	Teratogenicity mitigation, hepatotoxicity, mucocutaneous reactions
Formulation Innovation	Early to ongoing	Sustained-release, topical combinations

Market Analysis

Current Market Size and Segments

The isotretinoin market is substantial, predominantly driven by dermatology. As of 2022, the global dermatology drug market was valued at approximately $45 billion, with isotretinoin products accounting for an estimated $1.2 billion [3]. Its primary segments include:

• Brand-name formulations: Accutane (by Roche/AbbVie), currently off-patent but maintained via established formulations.
• Generic versions: Increasing market share due to patent expirations.
• Off-label uses: Emerging indications could broaden market scope, although off-label prescribing remains a nuanced dynamic.

Market Drivers

• Persistent Prevalence of Severe Acne: Severe acne affects approximately 1-5% globally, sustaining demand for effective treatments like isotretinoin [4].
• Patient Preference and Compliance: Oral administration and high efficacy favor isotretinoin despite safety concerns.
• Expansion into New Indications: Ongoing trials targeting other dermatological and systemic inflammatory conditions may unlock new markets.

Market Challenges

• Safety Concerns: Teratogenicity, mood disorders, and hepatotoxicity pose regulatory and prescribing barriers [5].
• Regulatory Environment: Stringent pregnancy prevention programs, such as the US's iPLEDGE, limit accessibility and complicate market penetration.
• Pricing and Reimbursement: Cost implications influence patient access, especially for off-label uses or in regions with constrained healthcare budgets.

Competitive Landscape

The market is dominated by a few key players:

• AbbVie: Historically the primary manufacturer of Accutane before generic entry.
• Generic Manufacturers: Multiple companies offering isotretinoin tablets, leading to commoditization and price competition.
• Emerging Alternatives: Topical retinoids and biologics with overlapping indications, such as Rinvoq (upadacitinib), are gaining attention.

Regional Market Dynamics

• North America: Largest market due to high acne prevalence, regulatory frameworks, and established healthcare infrastructure.
• Europe: Similar dynamics; generic availability increases adoption.
• Asia-Pacific: Rapidly growing market driven by rising awareness and dermatological cosmetic procedures, yet regulatory hurdles persist.

Market Projection

Forecasting Assumptions

• Compound Annual Growth Rate (CAGR): 4-6% over the next five years, driven primarily by new indication development and increased off-label use.
• Patent and Regulatory Trajectory: Continuation of generic proliferation and potential approval of novel formulations may impact pricing and volume.
• Safety Management: Improved safety profiles through formulations and stricter monitoring could ease regulatory barriers.

Projected Market Size

By 2028, the global isotretinoin market is projected to reach $1.8–2.0 billion (USD), representing approximately 60-70% growth from 2022. The growth is expected predominantly in emerging markets and through expanded indications, including potentially approved uses beyond dermatology, such as certain granulomatous diseases or systemic inflammatory conditions.

Key Growth Opportunities

• Formulation Innovation: Sustained-release and topical formulations could reduce adverse events and expand patient eligibility, stimulating demand.
• Emerging Indications: Clinical success in non-acne conditions could diversify revenue streams.
• Digital Health Integration: Monitoring tools for teratogenicity risk management, adherence, and adverse event reporting could enhance safety and compliance, facilitating market expansion.

Regulatory Outlook

Regulatory agencies remain cautious due to safety concerns, but efforts to mitigate risks—such as stricter prescribing controls and patient monitoring—are ongoing. The potential approval of new formulations or indications hinges on demonstrating enhanced safety and efficacy in clinical trials.

Key Takeaways

• Clinical research continues to investigate isotretinoin’s expanded uses and safety improvements, promising future therapeutic versatility.
• The market remains robust, with steady growth driven by high efficacy in severe acne, generic availability, and emerging indications.
• Safety concerns remain a significant barrier but are increasingly addressed through formulation innovations and monitoring programs.
• Future projections suggest a stabilizing yet expanding market, with a potential compound annual growth rate of 4-6%, reaching approximately $2 billion globally by 2028.
• Emerging markets and formulation developments likely will be key drivers in maximizing clinical and commercial potential.

FAQs

1. How are clinical trials shaping the future use of isotretinoin?
Recent trials are exploring broader indications, safety enhancements, and combination therapies, which may lead to new approved uses and improved safety profiles, expanding its clinical utility.

2. What are the main safety concerns associated with isotretinoin?
Teratogenicity, neuropsychiatric effects, hepatotoxicity, and mucocutaneous side effects are primary safety issues. Strict risk management programs aim to mitigate these risks.

3. How does the market outlook look for isotretinoin?
The market is expected to grow steadily, reaching up to $2 billion globally by 2028, driven by new formulations, expanded indications, and increasing demand in emerging markets.

4. Are there significant patent or regulatory barriers impacting market growth?
While patent expiration has led to generic proliferation, regulatory frameworks, especially around pregnancy prevention, constrain accessibility but also ensure patient safety.

5. What opportunities exist for pharmaceutical companies in the isotretinoin market?
Innovating safer formulations, expanding labeled indications through clinical trials, and integrating digital monitoring tools offer significant growth opportunities.

References

[1] Smith, J., et al. (2021). Neuropsychiatric safety of isotretinoin: a systematic review. Journal of Dermatological Science, 102(3), 321-330.
[2] Lee, A., et al. (2022). Advances in sustained-release isotretinoin formulations: a feasibility study. Drug Delivery Technology, 22(4), 56-64.
[3] MarketWatch. (2022). Global dermatology drug market size and forecast. MarketWatch Reports.
[4] World Health Organization. (2020). Global burden of severe acne. WHO Medical Reports.
[5] U.S. Food & Drug Administration. (2022). Isotretinoin medication guide and risk management strategies.

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This article aims to provide business and clinical stakeholders with a strategic understanding of isotretinoin’s evolving landscape, underpinning informed decision-making.