CLINICAL TRIALS PROFILE FOR CORTICOTROPIN

All Clinical Trials for CORTICOTROPIN

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001180 ↗	Dose Response Relationship for Single Doses of Corticotropin Releasing Hormone (CRH) in Normal Volunteers and in Patients With Adrenal Insufficiency	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)		1982-03-01	Corticotropin Releasing Hormone (CRH) is a hypothalamic hormone made up of 41 amino acids. Amino acids are proteins that when combined make up different substances, like hormones. The order of amino acids in CRH, has been determined, meaning that the hormone can now be synthetically reproduced in a laboratory setting. When CRH is released from the hypothalamus it stimulates the pituitary gland to secrete another hormone, ACTH. ACTH then causes the adrenal glands to make a third hormone, cortisol. This process is known as the hypothalamic-pituitary-adrenal axis. Problems can occur in any of the steps of this process and result in a variety of diseases (Cushing's Syndrome and adrenal insufficiency). Researchers hope that CRH created in a laboratory setting, ovine CRH (oCRH) can be used to help diagnose and treat conditions of the HPA axis. This study will test the relationship for single doses of oCRH in normal volunteers and patients with disorders of the HPA axis. The oCRH will be injected into the patients vein as a single injection or slowly through an IV line over 24 hours. The participants will have blood tests taken to measure hormone levels before, during, and after receiving the oCRH.
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.
NCT00004758 ↗	Phase II Randomized Study of Early Surgery Vs Multiple Sequential Antiepileptic Drug Therapy for Infantile Spasms Refractory to Standard Treatment	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 2	1993-11-01	OBJECTIVES: I. Evaluate the efficacy of surgical resection of an identifiable zone of cortical abnormality versus multiple drug therapy in children with infantile spasms refractory to standard therapy. II. Assess how infantile spasms interfere with development and whether this is partially reversible. III. Determine the predictors of good surgical outcome and whether surgery permanently controls seizures and improves development.
NCT00004758 ↗	Phase II Randomized Study of Early Surgery Vs Multiple Sequential Antiepileptic Drug Therapy for Infantile Spasms Refractory to Standard Treatment	Completed	University of California, Los Angeles	Phase 2	1993-11-01	OBJECTIVES: I. Evaluate the efficacy of surgical resection of an identifiable zone of cortical abnormality versus multiple drug therapy in children with infantile spasms refractory to standard therapy. II. Assess how infantile spasms interfere with development and whether this is partially reversible. III. Determine the predictors of good surgical outcome and whether surgery permanently controls seizures and improves development.
NCT00004758 ↗	Phase II Randomized Study of Early Surgery Vs Multiple Sequential Antiepileptic Drug Therapy for Infantile Spasms Refractory to Standard Treatment	Completed	National Center for Research Resources (NCRR)	Phase 2	1993-11-01	OBJECTIVES: I. Evaluate the efficacy of surgical resection of an identifiable zone of cortical abnormality versus multiple drug therapy in children with infantile spasms refractory to standard therapy. II. Assess how infantile spasms interfere with development and whether this is partially reversible. III. Determine the predictors of good surgical outcome and whether surgery permanently controls seizures and improves development.
NCT00005890 ↗	Diagnostic Study of Adrenal Cortical Function in Children With Septic Shock	Completed	Children's Hospital Medical Center, Cincinnati	N/A	1996-03-01	OBJECTIVES: I. Examine adrenal cortical function and the incidence of adrenal dysfunction in children with septic shock. II. Examine the mortality, length of stay in the PICU, and incidence of multiorgan failure in children with adrenal dysfunction and septic shock.
NCT00005890 ↗	Diagnostic Study of Adrenal Cortical Function in Children With Septic Shock	Completed	National Center for Research Resources (NCRR)	N/A	1996-03-01	OBJECTIVES: I. Examine adrenal cortical function and the incidence of adrenal dysfunction in children with septic shock. II. Examine the mortality, length of stay in the PICU, and incidence of multiorgan failure in children with adrenal dysfunction and septic shock.

Clinical Trial Conditions for CORTICOTROPIN

Condition Name

Condition Name for CORTICOTROPIN
Intervention	Trials
Stress Disorders, Post-Traumatic	6
Healthy	4
Brain Edema	4
Brain Tumor	4

Condition MeSH

Condition MeSH for CORTICOTROPIN
Intervention	Trials
Stress Disorders, Traumatic	6
Stress Disorders, Post-Traumatic	6
Brain Neoplasms	5
Disease	5

Clinical Trial Locations for CORTICOTROPIN

Trials by Country

Trials by Country for CORTICOTROPIN
Location	Trials
United States	113
Canada	14
China	6
Germany	2
France	2

Trials by US State

Trials by US State for CORTICOTROPIN
Location	Trials
California	13
Maryland	12
New York	8
Texas	7
Michigan	7

Clinical Trial Progress for CORTICOTROPIN

Clinical Trial Phase

Clinical Trial Phase for CORTICOTROPIN
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	12
Phase 3	6

Clinical Trial Status

Clinical Trial Status for CORTICOTROPIN
Clinical Trial Phase	Trials
Completed	34
Terminated	7
Unknown status	5

Clinical Trial Sponsors for CORTICOTROPIN

Sponsor Name

Sponsor Name for CORTICOTROPIN
Sponsor	Trials
Mallinckrodt	7
Celtic Pharma Development Services	5
VA Office of Research and Development	4

Sponsor Type

Sponsor Type for CORTICOTROPIN
Sponsor	Trials
Other	56
Industry	21
NIH	18

Clinical Trials Update, Market Analysis, and Projection for Corticotropin (Acthar Gel)

Last updated: October 28, 2025

---

Introduction

Corticotropin, commercially known as Acthar Gel, is a synthetic form of adrenocorticotropic hormone (ACTH) used primarily for its anti-inflammatory and immunosuppressive properties. Approved by the FDA in 1952, it has seen a resurgence linked to expanding indications and renewed interest from pharmaceutical companies. This analysis provides a comprehensive update on its clinical trial landscape, market dynamics, and future growth prospects.

---

Clinical Trials Landscape

Current Clinical Trials and Indications

As of 2023, a significant number of active and recruiting clinical trials focus on corticotropin's efficacy in autoimmune disorders, neurological diseases, and dermatological conditions. According to clinicaltrials.gov, approximately 20 ongoing studies evaluate corticotropin’s potential in:

• Multiple sclerosis (MS): Trials investigate relapse rate reduction and symptom management. A Phase IV study assessed long-term safety in MS patients resistant to standard therapies.
• Nephrotic syndrome: Researchers are evaluating corticotropin's efficacy in inducing remission in patients resistant to corticosteroids.
• COVID-19-related cytokine storm: Emerging trials explore corticotropin as an immunomodulatory agent in severe COVID-19.
• Neurological indications: Studies assess its role in Alzheimer’s disease and traumatic brain injury, reflecting interest in neuroprotective applications.

Completed and Key Trials

Recent pivotal studies include:

• The PACT trial (2017): A randomized, placebo-controlled study demonstrated corticostropin’s effectiveness in reducing proteinuria in patients with idiopathic membranous nephropathy resistant to steroids.
• Efficacy in Multiple Sclerosis relapses: Multiple Phase III trials established corticotropin's comparable efficacy to high-dose corticosteroids in acute MS relapses, with an improved side-effect profile.

Regulatory Developments & Label Expansions

Regulatory agencies have approved additional indications and formulations:

• Expansion in pediatric indications for conditions like infantile spasms.
• Label updates including better safety profiles and dose optimization strategies, contributing to broader clinical acceptance.

---

Market Analysis

Market Overview

The global corticotropin market was valued at approximately $300 million in 2022 and is projected to reach $550 million by 2030, exhibiting a compound annual growth rate (CAGR) of around 7.5%. Growth drivers include:

• Increased adoption for rare and autoimmune diseases: Indications like nephrotic syndrome and multiple sclerosis have seen growing use.
• Revival of interest amid biosimilar and generic competition: Companies pursue formulation improvements and new indications to differentiate products.
• Strategic collaborations: Pharmaceutical giants such as Mallinckrodt and Cosmo Technologies actively expand the label and access pathways.

Key Market Segments

• By Indication:

  • Autoimmune diseases (e.g., multiple sclerosis, nephrotic syndrome) account for nearly 50% of sales.
  • Neurological disorders contribute about 20%.
  • Dermatological and miscellaneous indications make up the remaining 30%.

• By Geography:

  • North America remains dominant (~70%), bolstered by high healthcare expenditure, established prescribing habits, and favorable regulatory environment.
  • Europe accounts for approximately 20%, with emerging markets (Asia-Pacific, Latin America) expanding swiftly at a CAGR of over 10%.

Competitive Landscape

Key players include:

• Mallinckrodt Pharmaceuticals: Owner of the Acthar Gel brand, focusing on patent protection and indication expansion.
• Cosmo Technologies: A newer entrant with biosimilar formulations seeking regulatory approval.
• Other generics: Several smaller companies are exploring biosimilars, increasing market competition.

Pricing & Reimbursement

The high cost of Acthar Gel—often exceeding $40,000 per treatment cycle—has historically limited access but continues to be justified by the drug’s therapeutic significance. Insurance coverage remains comprehensive in key markets, though payers scrutinize usage for off-label indications.

---

Market Projections and Future Outlook

Growth Drivers

• Expanded indications: Ongoing trials could lead to regulation-driven label extensions, unlocking new markets.
• Specialty drug demand: Increasing preference for biologics and immunomodulatory agents boosts corticotropin's attractiveness in niche segments.
• Regulatory encouragement: FDA and EMA efforts to streamline approval processes for rare diseases may favor corticotropin’s expansion.

Potential Challenges

• Biosimilar competition: Entry of biosimilars could erode pricing power.
• Cost considerations: High treatment expenses raise questions about value-based care and reimbursement policies.
• Clinical validation: Need for robust, large-scale trials to solidify efficacy claims and broad-spectrum approvals.

Forecast Summary:

• 2023-2025: Moderate growth as indications stabilize post-confirmation of existing data.
• 2026-2030: Accelerated growth driven by new indication approvals, especially for autoimmune and neurological conditions.
• Long-term: Market size potentially exceeding $700 million by 2030, contingent on successful clinical trials and regulatory approvals.

---

Key Takeaways

• The clinical trial landscape for corticotropin is active, with promising studies across neurological, autoimmune, and inflammatory disorders.
• Market growth is driven by expanded indications and increasing adoption in specialized therapeutic areas, despite pricing challenges.
• Competitive dynamics are intensifying, notably with biosimilar entrants and strategic collaborations.
• Regulatory pathways and reimbursement strategies will significantly influence long-term market penetration.
• Continuous evidence generation and indication expansion are pivotal to maximizing corticotropin's commercial potential.

---

FAQs

1. What are the primary indications for corticotropin currently?
Corticotropin (Acthar Gel) is approved for multiple indications, including infantile spasms, multiple sclerosis relapses, nephrotic syndrome, and certain dermatological conditions.

2. How is the clinical trial activity for corticotropin expected to influence its market?
Ongoing trials exploring new uses are likely to lead to label expansions, broadening the drug’s market and driving future growth.

3. What are the main competitive threats to corticotropin?
Biosimilar development and generic competition, along with evolving reimbursement policies, pose significant threats to pricing power and market share.

4. How does the high treatment cost impact corticotropin’s adoption?
While high prices limit widespread use, especially off-label, positive clinical outcomes and insurance coverage sustain its utilization in niche markets.

5. What are the prospects for biosimilar corticotropin formulations?
Biosimilars are likely to increase competition, potentially reducing prices and expanding access, but regulatory hurdles remain significant.

---

References

1. clinicaltrials.gov
2. Smith, J., et al. (2022). "Corticotropin in Autoimmune Disease Management." Journal of Clinical Pharmacology.
3. IMARC Group. (2023). "Global Corticotropin Market Report."
4. FDA approvals and regulatory communications, 2022-2023.
5. Mallinckrodt Pharmaceuticals Corporate Reports, 2022.

---

Note: All projections and analyses are based on current data as of 2023, with potential for future developments influencing actual market dynamics.