CLINICAL TRIALS PROFILE FOR MIFEPRISTONE

All Clinical Trials for MIFEPRISTONE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001849 ↗	New Imaging Techniques in the Evaluation of Patients With Ectopic Cushing Syndrome	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	1999-05-20	Cushing Syndrome is an endocrine disorder causing an over production of the hormone cortisol. Cortisol is produced in the adrenal gland as a response to the production of corticotropin (ACTH) in the pituitary gland. Between 10% and 20% of patients with hypercortisolism (Cushing Syndrome) have ectopic production of the hormone ACTH. Meaning, the hormone is not being released from the normal site, the pituitary gland. In many cases the ectopic ACTH is being produced by a tumor of the lung, thymus, or pancreas. However, in approximately 50% of these patients the source of the ACTH cannot be found even with the use of extensive imaging studies such as computed tomography (CT) scans, magnetic resonance imaging (MRI), and nuclear scans (111-indium pentetreotide). The ability of these tests to locate the source of the hormone production is dependent on the changes of anatomy and / or the dose and adequate uptake of the radioactive agent. The inability to detect the source of ectopic ACTH production often results in unnecessary pituitary surgery or irradiation. Unlike the previously described tests, positron emission tomography (PET scan) has the ability to detect pathologic tissue based on physiologic and biochemical processes within the abnormal tissue. This study will test whether fluorine-18-fluorodeoxyglucose (FDG), fluorine-18-dihydroxyphenylalanine (F-DOPA) or use of a higher dose of 111-indium pentetreotide can be used to successfully localize the source of ectopic ACTH production.
NCT00043654 ↗	Clinical Trial of Mifepristone for Bipolar Depression	Completed	National Institute of Mental Health (NIMH)	Phase 2	2002-08-07	Bipolar Depression is a severe illness with high rates of psychiatric comorbidity and increased mortality related to suicide and medical illness. Hypothalamic pituitary axis (HPA) hyperactivity are found in bipolar disorder related to depression and mixed states. Patients with bipolar disorder also have cognitive difficulties and endocrine disturbances may contribute to such dysfunction. Antiglucorticoid therapies are novel treatments of mood disorder. Preliminary data in psychotic depression suggesting that mifepristone (RU-486), a glucocorticoid receptor antagonist, has antidepressant and salutary cognitive effects in a matter of days. In this study we examine the effects of mifepristone in severe bipolar depression in a parallel, double blind placebo controlled experiment. Bipolar subjects maintained on either lithium or valproate, after washout or prior antidepressants have a detailed neuroendocrine assessment. Patients approximately or almost 75 will receive eight days of mifepristone versus placebo after which patients are blindly crossed over to the opposite arm. Patients and a group of matched controls approximately or almost 35 will be compared with neuroendocrine, cognitive, and neurophysiologic testing to fully characterize their phenotype and explore biomarkers of response. It is hypothesized that stigmata of HPA axis hyperactivity and cognitive impairment will be predictive of response to antiglucocorticoid therapy with mifepristone.
NCT00044876 ↗	Treatment of Uterine Fibroids With CDB-2914, an Experimental Selective Progesterone Receptor Antagonist	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	2002-09-02	Uterine leiomyomata (fibroids) are a common benign tumor of the uterine muscle in premenopausal women. These tumors may cause bleeding, pelvic pain and pressure. Because fibroids grow in the presence of estrogen, medical therapies that decrease estrogen levels (like GnRH analog) cause fibroids to shrink and so may relieve symptoms. However, such medication can only be given short-term and has inconvenient side effects such as hot-flushes. Thus, many women with symptomatic fibroids choose to have them removed surgically, either individually or by removing the uterus via hysterectomy. The study evaluates a new medical treatment for fibroids using the progesterone receptor modulator CDB-2914. A similar compound, mifepristone (Registered Trademark), reduced fibroid size when given for twelve weeks. This study will compare fibroid size, hormone levels and symptoms before and during daily administration of CDB-2914 (10 or 25 mg) or placebo for 10 - 14 weeks. To do this, women will undergo MRI and a saline hysterosonogram (ultrasound with fluid) of the uterus before and at the end of the treatment; they will have blood drawn every 7 - 14 days, and will fill out a symptom calendar at home. Hysterectomy will be performed at the end of the treatment to evaluate the effects of the medication on the uterine and fibroid tissues, and to provide treatment for the study participant. Women will be randomly assigned to the treatment groups; during the treatment period neither the participants nor the investigators will know the type of treatment that a woman receives. ...
NCT00046553 ↗	Brain Receptor Function in Post-Traumatic Stress Disorder	Completed	National Institute of Mental Health (NIMH)		2002-09-01	The purpose of this study is to examine the function of cortisol receptors in post-traumatic stress disorder (PTSD). Patients with PTSD have neurobiological dysregulation of the hypothalamic-pituitary-adrenal (HPA) axis function. High corticotrophin releasing hormone (CRH) levels and decreased hippocampal volume are major features of the disorder. The mechanisms responsible for these alterations are not known. This study will evaluate the function of cortisol receptors to determine their roles in maintaining PTSD HPA axis dysregulation. Three groups of subjects will take part in the study: Patients with PTSD, healthy control subjects who were exposed to trauma in the past and remained healthy and healthy control subjects who were never traumatized At study entry, the cerebral spinal fluid (CSF) of all participants will be sampled and evaluated. Participants will also undergo a magnetic resonance imaging (MRI) scan of the brain as well as eye blink trace conditioning and neuropsychological tests. Participants will be admitted to the Clinical Center for two nights on three different occasions. At each overnight visits, blood levels of stress hormones will be measured every hour for 26 hours after medication or placebo are given. This will be the end of the study for both groups of healthy control subjects, with the exception that they may be asked to repeat neuropsychologic and eye blink tests after 12 weeks. Participants with PTSD will receive paroxetine for 10 weeks. After 10 weeks these participants will be reevaluated in exactly the same way as before treatment (except they will not repeat the MRI scan).
NCT00099645 ↗	Anti-HIV Activity and Safety of 3 Different Doses of Mifepristone in HIV Infected People	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1/Phase 2	1969-12-31	The purpose of this study is to determine the anti-HIV activity and safety of 3 different doses of mifepristone (also known as VGX-410 and RU486) in HIV infected people. Hypothesis: Mifepristone will be generally safe (no serious adverse effects) and well tolerated.
NCT00105105 ↗	Mifepristone as Adjunctive Therapy in Alzheimer's Disease	Terminated	Institute for the Study of Aging (ISOA)	Phase 2	2003-04-01	The purpose of this study is to evaluate the effects of C-1073 (Mifepristone) on cognition in patients with Alzheimer's disease (AD) who are also taking an acetylcholinesterase inhibitor (Aricept, Exelon or Reminyl).

Clinical Trial Conditions for MIFEPRISTONE

Condition Name

Condition Name for MIFEPRISTONE
Intervention	Trials
Abortion, Induced	12
Medical Abortion	8
Major Depressive Disorder	6
Cushing's Syndrome	5

Condition MeSH

Condition MeSH for MIFEPRISTONE
Intervention	Trials
Depression	13
Depressive Disorder	12
Abortion, Spontaneous	11
Leiomyoma	11

Clinical Trial Locations for MIFEPRISTONE

Trials by Country

Trials by Country for MIFEPRISTONE
Location	Trials
United States	302
China	21
Vietnam	9
Netherlands	9
Georgia	8

Trials by US State

Trials by US State for MIFEPRISTONE
Location	Trials
California	37
New York	26
Pennsylvania	22
Illinois	21
Massachusetts	18

Clinical Trial Progress for MIFEPRISTONE

Clinical Trial Phase

Clinical Trial Phase for MIFEPRISTONE
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	1
PHASE2	2

Clinical Trial Status

Clinical Trial Status for MIFEPRISTONE
Clinical Trial Phase	Trials
Completed	115
Terminated	28
RECRUITING	25

Clinical Trial Sponsors for MIFEPRISTONE

Sponsor Name

Sponsor Name for MIFEPRISTONE
Sponsor	Trials
Gynuity Health Projects	37
Corcept Therapeutics	24
Stanford University	10

Sponsor Type

Sponsor Type for MIFEPRISTONE
Sponsor	Trials
Other	292
Industry	35
NIH	21

Clinical Trials Update, Market Analysis, and Projection for Mifepristone

Last updated: October 30, 2025

Introduction

Mifepristone, widely known as RU-486, is a synthetic steroid with primary applications in medical termination of early pregnancy and management of hyperglycemia associated with endogenous Cushing's syndrome. Since its development in the 1980s, mifepristone has undergone extensive clinical evaluation, regulatory scrutiny, and market evolution. This report synthesizes recent clinical trial updates, analyzes market dynamics, and offers projections concerning mifepristone's future trajectory within the pharmaceutical landscape.

Clinical Trials Update

Recent Clinical Trial Landscape

Over the past few years, mifepristone has garnered renewed attention driven by expanding indications, changing regulatory policies, and evolving public health needs. The main domains of active research include:

• Expanded Maternal Health Applications: Several ongoing trials explore off-label uses, such as medical management of pregnancy complications beyond early termination, including postpartum hemorrhage and early pregnancy loss management. For instance, a phase II trial (NCT04563251) initiated in late 2021 investigates mifepristone in combination with misoprostol for second-trimester pregnancy termination, indicating ongoing interest in extending its clinical utility.

• Cushings Disease and Syndrome: The SEISMIC trial (NCT01643496), a pivotal phase III study published in 2017, demonstrated the efficacy of higher-dose mifepristone in controlling hyperglycemia and improving quality of life in Cushing's syndrome. Subsequently, several small-scale studies and retrospective analyses have assessed long-term safety, setting the stage for potential regulatory revisions.

• Reproductive and Gynecological Disorders: Emerging research evaluates mifepristone’s role in conditions like uterine fibroids and endometriosis. For example, phase II studies are examining lower-dose regimens for fibroid symptom relief, with preliminary data suggesting favorable safety profiles.

Regulatory Status and Clinical Trial Considerations

Despite multiple regulatory approvals—most notably, the FDA's approval of mifepristone for pregnancy termination up to 10 weeks gestation in combination with misoprostol—the drug's clinical trial landscape remains highly dynamic. Several trials face delays or modifications due to regulatory challenges, especially outside the United States, where access restrictions influence trial designs.

Furthermore, ongoing trial results are pivotal in addressing safety concerns, particularly regarding adverse effects such as bleeding, infection, and rare but serious complications. The expanding evidence base supports the potential for broader indications, contingent on successful trial outcomes and regulatory acceptance.

Market Analysis

Current Market Overview

The global mifepristone market has historically been segmented into three primary categories:

• Reproductive Health: Dominant in North America, Europe, and parts of Asia-Pacific, driven by regulatory approvals for medical termination of pregnancy and reproductive health management.

• Endocrinology (Cushing's Syndrome): Niche but expanding segment, with mifepristone’s use in controlling hyperglycemia linked to endogenous cortisol excess.

• Off-Label and Future Applications: Potential new markets involve fibroids, endometriosis, and other gynecological conditions, although commercial penetration remains limited pending robust clinical validation.

The global market value was estimated at USD 220 million in 2022, with North America holding approximately 70% share owing to favorable regulatory landscape and high adoption rates.

Market Drivers

• Regulatory Shifts: Changes in abortion legislation in various countries influence mifepristone’s marketability. Recent developments signal relaxations in restrictions in some European countries and Latin America, expanding access.

• Expanding Indications: Growing clinical evidence supports new therapeutic areas—particularly hypercortisolism management—potentially broadening market scope.

• Pandemic Influence: COVID-19 has catalyzed telemedicine platforms, facilitating remote prescriptions and expanding access, especially for early pregnancy termination where restrictions are eased.

Market Challenges

• Regulatory and Political Barriers: Legislation remains a significant obstacle. For example, in the U.S., ongoing legal battles over FDA's REMS restrictions potentially limit distribution.

• Market Competition: Though mifepristone is currently a leader in medical termination, alternatives such as misoprostol alone or surgical methods exist, affecting market share.

• Supply Chain and Accessibility: Manufacturing permissions and geopolitical stability influence availability, particularly in low- and middle-income countries.

Market Projection

Forecasted Trends (2023-2030)

• Compound Annual Growth Rate (CAGR): The market is projected to grow at a CAGR of approximately 8-10%, driven predominantly by regulatory liberalization and expansion into new clinical indications.

• Regional Expansion: Asia-Pacific is anticipated to be the fastest-growing segment, supported by increasing acceptance of medical abortion and growing healthcare infrastructure.

• Indication Diversification: The inclusion of mifepristone for conditions beyond pregnancy termination, such as endometrial hyperplasia and selected endocrine disorders, could contribute an incremental USD 150-200 million to market size by 2030.

• Regulatory Approvals: Anticipated global approval for expanded indications, including Cushing's syndrome management, could stimulate significant revenue, with peak sales reaching USD 500 million globally by 2030.

• Commercial Strategies: Companies may leverage patent expirations, combination therapy development, and novel delivery systems (e.g., sustained-release formulations) to sustain market growth.

Strategic Implications

Manufacturers and stakeholders should prioritize ongoing clinical trials for new indications, navigate geopolitical and legislative landscapes adeptly, and invest in supply chain robustness. Collaborations with regulatory agencies and healthcare providers are also critical to expanding access, particularly in traditionally restricted markets.

Key Takeaways

• Clinical development of mifepristone is active, with ongoing trials probing new indications, particularly in gynecological and endocrine medicine, promising expanded therapeutic roles.

• Market dynamics are shaped by regulatory environments, with liberalization in some regions fostering growth, while legislative hurdles in others dampen potential.

• Projected market growth is robust, driven by new indications, regional expansion, and evolving healthcare delivery models, with an estimated CAGR of 8-10% through 2030.

• Emerging trends such as telemedicine and combination therapies will influence supply, distribution, and commercial strategies.

• Strategic investments in clinical research, regulatory engagement, and global access initiatives will be essential to capitalize on mifepristone’s full market potential.

FAQs

1. What are the recent advancements in clinical trials involving mifepristone?
Recent trials focus on extending its indications to treat conditions like uterine fibroids and Cushing's syndrome, with evidence supporting safety and efficacy. Notably, phase II studies are evaluating lower-dose regimens for fibroid management, and ongoing research into combination therapies aims to optimize outcomes.

2. How do regulatory changes impact mifepristone's market availability?
In regions where regulations become more permissive, access expands, stimulating market growth. Conversely, legislative restrictions, such as those enforced by restrictive abortion laws or strict REMS protocols, limit distribution, affecting sales and market penetration.

3. What is the forecasted market size for mifepristone by 2030?
Projected to reach approximately USD 500 million globally, driven by expanded indications, regional acceptance, and technological innovations in drug delivery.

4. Which regions are expected to lead mifepristone market growth?
North America remains dominant but growth is fastest in Asia-Pacific, supported by increasing acceptance and healthcare infrastructure expansion.

5. What are potential challenges facing mifepristone market expansion?
Legislative restrictions, political opposition, supply chain disruptions, and competition from alternative therapies pose ongoing challenges.

References

[1] U.S. Food and Drug Administration. Mifepristone label and REMS information. 2021.
[2] ClinicalTrials.gov. Ongoing trials involving mifepristone. 2023.
[3] Market Research Future. Global Mifepristone Market Analysis & Forecast. 2022.
[4] National Institutes of Health. Studies on Cushing's syndrome management. 2017.
[5] Grand View Research. Reproductive health pharmaceuticals market overview. 2023.