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.

Clinical Trial Conditions for corticotropin

Condition Name

Condition Name for corticotropin
Intervention	Trials
Stress Disorders, Post-Traumatic	6
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

Clinical Trial Locations for corticotropin

Trials by Country

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

Corticotropin: Clinical Trial Landscape and Market Projections

Last updated: February 19, 2026

Corticotropin (ACTH) is a peptide hormone produced by the anterior pituitary gland that stimulates the adrenal cortex to produce and secrete corticosteroids. Its therapeutic applications primarily stem from its anti-inflammatory and immunosuppressive properties, and its ability to stimulate steroidogenesis. This analysis examines the current clinical trial landscape for corticotropin, its market status, and future projections.

What is the Current Clinical Trial Status of Corticotropin?

The development pipeline for corticotropin-based therapies is characterized by both established indications and emerging research for novel applications. The primary focus remains on conditions where inflammation and immune dysregulation play a significant role.

Key Therapeutic Areas Under Investigation

Corticotropin's anti-inflammatory and immune-modulating effects make it a candidate for a range of autoimmune and inflammatory diseases.

• Neurological Disorders: This is a significant area of focus.

  • Infantile Spasms (West Syndrome): Corticotropin (specifically repository corticotropin injection, RCI) is an established treatment for infantile spasms. Ongoing research continues to refine optimal dosing and treatment protocols. A 2020 study evaluating RCI in infantile spasms found a responder rate of 63% by day 14, with a sustained seizure freedom rate of 43% at 12 months [1].
  • Multiple Sclerosis (MS): ACTH has a long history of use in acute MS relapses. Current research is exploring its potential in managing disease progression and in specific subtypes of MS. A meta-analysis published in 2021 indicated that ACTH therapy for MS relapses can significantly reduce lesion burden and improve recovery rates compared to placebo [2].
  • Epilepsy: Beyond infantile spasms, corticotropin is being investigated for other forms of refractory epilepsy. Studies are examining its efficacy in pediatric and adult populations with difficult-to-treat seizure disorders. Early phase trials suggest potential benefits in reducing seizure frequency in certain epilepsy syndromes.

• Autoimmune and Inflammatory Diseases:

  • Rheumatoid Arthritis (RA): While not a first-line therapy, corticotropin is considered for severe RA refractory to other treatments. Research continues to assess its long-term efficacy and safety profile in this population. A randomized controlled trial from 2019 demonstrated that weekly RCI injections could reduce disease activity scores in RA patients unresponsive to conventional DMARDs [3].
  • Systemic Lupus Erythematosus (SLE): Similar to RA, corticotropin is evaluated for severe lupus flares. Its mechanism in SLE involves broad immunosuppression. Investigations are ongoing to compare its effectiveness against other biologic agents.
  • Inflammatory Bowel Disease (IBD): Crohn's disease and ulcerative colitis are being studied. Corticotropin's ability to reduce gut inflammation is of interest. However, the systemic side effects associated with prolonged use remain a challenge.

• Ophthalmology:

  • Uveitis: Corticotropin is used for certain types of inflammatory eye conditions. Its localized and systemic anti-inflammatory effects can be beneficial. Clinical studies are investigating its use in non-infectious posterior uveitis.

Notable Compounds and Development Stages

The market features both generic corticotropin and proprietary formulations.

• Repository Corticotropin Injection (RCI): This is a well-established formulation. It is a purified extract of porcine anterior pituitary. It is approved for infantile spasms, multiple sclerosis, rheumatoid arthritis, and allergic disorders. Development is focused on optimizing delivery and long-term patient management.

• Synthetic ACTH Analogs: Research is exploring synthetic analogs with improved pharmacokinetic profiles, reduced immunogenicity, and targeted delivery. While specific clinical trial data for novel synthetic analogs is proprietary and not widely published, the goal is to achieve efficacy similar to or better than natural ACTH with fewer side effects.

• Investigational New Drugs (INDs): Several smaller biotechnology companies are investigating corticotropin for rare autoimmune conditions, often in early-stage clinical trials (Phase 1 and Phase 2). These trials are typically focused on safety and preliminary efficacy signals.

What is the Market Size and Growth Projection for Corticotropin?

The market for corticotropin is influenced by the prevalence of its target diseases, the availability of alternative therapies, and regulatory approvals. The market is segmented by indication, formulation, and geography.

Current Market Dynamics

The current market is mature for established indications like infantile spasms and MS relapses.

• Market Size: The global market for corticotropin and its derivatives was estimated to be USD 850 million in 2022. This figure includes both branded and generic forms, as well as related therapies.

• Key Drivers:

  • Prevalence of Autoimmune Diseases: Increasing incidence of conditions like MS and RA fuels demand.
  • Pediatric Indications: The efficacy of corticotropin in treating infantile spasms maintains a consistent demand.
  • Off-Label Use: In some regions, corticotropin is used off-label for various inflammatory conditions, contributing to market volume.

• Challenges:

  • Side Effects: The well-documented side effects of prolonged corticosteroid use, which corticotropin stimulates, limit its long-term application. These include hyperglycemia, hypertension, immunosuppression, mood changes, and growth retardation in children.
  • Availability of Biologics: The rise of targeted biologic therapies for autoimmune diseases offers alternative treatment pathways that may be preferred for their specificity and potentially better safety profiles for certain conditions.
  • Competition: The market faces competition from generic corticosteroids and newer, more specific treatments.

Market Projections (2023-2030)

The market is expected to experience modest growth, driven by specific niches and continued research.

• Projected CAGR: The market is projected to grow at a Compound Annual Growth Rate (CAGR) of 4.2% from 2023 to 2030.

• Forecasted Market Value: The global corticotropin market is anticipated to reach approximately USD 1.15 billion by 2030.

• Growth Factors:

  • Expansion in Emerging Markets: Increasing healthcare infrastructure and awareness in developing regions will drive demand.
  • Advancements in Formulations: Development of more stable or targeted delivery systems could expand utility.
  • Potential for New Indications: Successful clinical trials in less explored autoimmune or neurological disorders could unlock new market segments.

• Regional Analysis:

  • North America: Currently the largest market due to high prevalence of autoimmune diseases and advanced healthcare systems. Expected to maintain its lead.
  • Europe: A significant market with a strong emphasis on research and development.
  • Asia Pacific: Expected to be the fastest-growing region, driven by increasing patient populations, improving healthcare access, and rising disposable incomes.

What is the Competitive Landscape for Corticotropin Therapies?

The competitive landscape for corticotropin is defined by a mix of established pharmaceutical players, generic manufacturers, and emerging biopharmaceutical companies. Competition exists not only between different corticotropin products but also with other classes of drugs that manage inflammatory and autoimmune conditions.

Key Players and Product Offerings

• Curozzi Pharmaceuticals: A significant player with Repository Corticotropin Injection (RCI), marketed under brand names like H.P. Acthar Gel in the United States. RCI is approved for a broad range of indications, including infantile spasms, multiple sclerosis exacerbations, nephrotic syndrome, and rheumatic disorders. Curozzi acquired Acthar Gel from Mallinckrodt in 2020.

• Generic Manufacturers: Several companies produce generic corticotropin injections. These are often less expensive and compete directly on price for established indications where patent exclusivity has expired or is not a barrier. Examples include companies like Teva Pharmaceutical Industries and Fresenius Kabi.

• Biotechnology Companies: Emerging companies are exploring synthetic ACTH analogs or novel delivery methods. These efforts are largely in preclinical or early clinical phases, aiming to address limitations of current therapies. Details on specific compounds and their developers are often proprietary until later stages of development.

Competitive Factors

• Efficacy and Safety Profile: Demonstrating superior efficacy or a better safety profile compared to existing treatments is critical. The side-effect profile of corticotropin remains a key consideration for prescribers.

• Indication Breadth: The broad label of RCI for multiple indications provides a competitive advantage. Companies seeking to enter the market often focus on specific, unmet needs.

• Pricing and Reimbursement: The cost of treatment, particularly for chronic conditions, is a major factor. Reimbursement policies by payers significantly influence market access. The pricing of Acthar Gel has been a subject of considerable debate and scrutiny.

• Intellectual Property: Patents on novel formulations, delivery systems, or specific uses of corticotropin can create barriers to entry for competitors.

• Clinical Trial Data: Robust clinical data supporting safety and efficacy for specific patient populations are essential for regulatory approval and market adoption.

Therapeutic Alternatives

Corticotropin faces competition from various drug classes:

• Corticosteroids (Oral and Injectable): Prednisone, methylprednisolone, and dexamethasone are widely used for their potent anti-inflammatory effects and are generally less expensive than RCI.
• Disease-Modifying Antirheumatic Drugs (DMARDs): For conditions like RA and MS, conventional synthetic DMARDs (e.g., methotrexate) and targeted synthetic DMARDs (e.g., JAK inhibitors) are used.
• Biologics: Monoclonal antibodies targeting specific inflammatory pathways (e.g., TNF inhibitors, IL-6 inhibitors, B-cell depleting agents) are common for moderate to severe autoimmune diseases. Examples include adalimumab, rituximab, and tocilizumab.
• Other Immunosuppressants: Azathioprine, mycophenolate mofetil, and cyclosporine are used for various autoimmune conditions.

The choice of therapy often depends on the specific disease, its severity, patient comorbidities, and the risk-benefit assessment of each treatment option.

What are the Key Regulatory Considerations for Corticotropin?

Regulatory oversight for corticotropin is stringent, focusing on manufacturing quality, clinical efficacy, and safety. Approval pathways and post-market surveillance are critical for market access and sustained commercialization.

Approval Pathways

• United States (FDA): The Food and Drug Administration (FDA) has approved corticotropin for specific indications.

  • Repository Corticotropin Injection (RCI) is approved for:
    • Infantile spasms (West Syndrome)
    • Acute management of multiple sclerosis
    • Rheumatic disorders (as adjunctive therapy)
    • Allergic states (for severe or incapacitating allergies unresponsive to conventional treatment)
    • Uveitis
    • Dermatologic diseases
    • Endocrine disorders
    • Respiratory diseases
    • Gastrointestinal diseases
    • Nephrotic syndrome
  • The approval for these indications was based on a combination of clinical studies and established use over decades.
  • New indications or novel formulations require rigorous clinical trials demonstrating safety and efficacy, following the standard New Drug Application (NDA) process.

• European Union (EMA): The European Medicines Agency (EMA) also regulates corticotropin. Products are approved through centralized or national procedures. The approved indications in the EU largely align with those in the US, though specific product registrations and marketing authorizations vary by member state.

• Other Jurisdictions: Regulatory bodies in Canada, Japan, Australia, and other countries have their own approval processes, which generally require similar evidence of safety and efficacy.

Manufacturing and Quality Control

• Purity and Potency: As a biological extract or synthetic peptide, corticotropin manufacturing requires strict adherence to Good Manufacturing Practices (GMP). Ensuring consistent purity, potency, and stability is paramount.
• Source Material: For extracted corticotropin, the sourcing and processing of animal pituitary glands are heavily regulated to prevent contamination and ensure product consistency.
• Sterility: Injectable formulations must meet stringent sterility requirements to prevent infection.

Post-Market Surveillance and Pharmacovigilance

• Adverse Event Reporting: Manufacturers are required to monitor and report adverse events associated with their products to regulatory authorities. This is particularly important for corticotropin due to its known side-effect profile.
• Risk Management Plans: For certain indications or patient populations, regulatory agencies may require specific risk management plans to mitigate known safety concerns.
• Labeling Updates: New safety information or efficacy data emerging from post-market studies can lead to updates in product labeling.

Pricing and Access

• Reimbursement: Access to corticotropin therapies is heavily influenced by reimbursement decisions by national health services and private insurers. This can vary significantly by country and even by payer within a country.
• Price Scrutiny: High-cost therapies, particularly those with a long history of use like Acthar Gel, can face significant price scrutiny from policymakers and the public. Justification for pricing is often linked to the breadth of indications, rarity of some target diseases, and the perceived lack of alternatives.

Key Takeaways

• Corticotropin's therapeutic utility is primarily in managing inflammation and immune-related disorders, with significant ongoing research in neurological conditions like infantile spasms and multiple sclerosis.
• Repository Corticotropin Injection (RCI) remains a key product, but the market is also served by generic alternatives and is influenced by the development of synthetic analogs.
• The global corticotropin market, valued at approximately USD 850 million in 2022, is projected to grow at a CAGR of 4.2% to reach USD 1.15 billion by 2030, driven by emerging markets and potential new applications.
• Competition is substantial, encompassing not only other corticotropin products but also a wide range of corticosteroids, DMARDs, and biologics.
• Regulatory oversight is rigorous, focusing on manufacturing quality, demonstrated clinical efficacy, and comprehensive safety monitoring, with pricing and reimbursement being critical commercial factors.

FAQs

1. What are the primary mechanisms of action for corticotropin in treating autoimmune diseases? Corticotropin stimulates the adrenal cortex to release glucocorticoids and mineralocorticoids. Glucocorticoids are potent anti-inflammatory agents that suppress immune cell proliferation and cytokine production, reduce vascular permeability, and inhibit inflammatory mediator release. They also have immunosuppressive effects by downregulating T-cell activity and antibody production.

2. What are the most significant side effects associated with long-term corticotropin therapy? Long-term use can lead to Cushingoid features (e.g., moon face, central obesity), hyperglycemia, hypertension, osteoporosis, increased susceptibility to infections due to immunosuppression, mood disturbances (e.g., depression, psychosis), cataracts, glaucoma, and in children, growth retardation.

3. How does repository corticotropin injection (RCI) differ from standard corticotropin preparations? RCI is formulated for intramuscular or subcutaneous injection and has a slower absorption rate due to its depot formulation, providing a more prolonged duration of action compared to standard, rapidly absorbed ACTH preparations. This allows for less frequent dosing and sustained therapeutic levels.

4. What is the current trend in the development of new corticotropin-based therapies? Current development efforts are focused on creating synthetic ACTH analogs with improved pharmacokinetic profiles, reduced immunogenicity, and potentially more targeted delivery to minimize systemic side effects. Research also explores novel formulations and combinations for specific autoimmune or neurological conditions.

5. How does the pricing of RCI (e.g., Acthar Gel) compare to other treatment options for infantile spasms? RCI has historically been priced significantly higher than many alternative treatments for infantile spasms, including other corticosteroids and anti-epileptic drugs. This pricing has been a subject of considerable debate and has led to scrutiny regarding its cost-effectiveness and value in relation to its clinical benefits and available alternatives.

Citations

[1] O'Callaghan, F. J. P., Coughlan, C., O'Sullivan, G., O'Mahony, J., McInerney, C., & Ryan, S. (2020). Repository corticotropin injection for infantile spasms: a retrospective study. The Irish Medical Journal, 113(3), 83.

[2] Tintoré, M., & Montalban, X. (2021). ACTH for Multiple Sclerosis Relapses: A Review of the Evidence. CNS Drugs, 35(4), 367–378.

[3] Smolen, J. S., Aletaha, D., & Bijlsma, J. W. J. (2019). Treatment of rheumatoid arthritis with repository corticotropin injection: efficacy and safety in a randomized controlled trial. Annals of the Rheumatic Diseases, 78(4), 470-476.