ACTHAR Therapy for Central Nervous System Sarcoidosis

NCT02920710 | Withdrawn Phase 4 FDA Drug

• 1 other identifier: ACTHAR CNS
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

There is a need for a more reliable, expeditious therapy that can be used as an alternative to glucocorticoids in severe Central Nervous System (CNS) sarcoidosis. This study aims to provide evidence for effectiveness of ACTHAR gel in CNS sarcoidosis, and provide information about its safety and tolerability

Conditions

Sarcoidosis

Outcome Measures

Primary Outcomes (1)

• Proportion of patients with clinically significant improvement - successful glucocorticoid tapering.

  12 Weeks

Secondary Outcomes (1)

• Proportion of patients with clinically significant improvement - no need for escalation of other therapy.

  12 Weeks

Study Arms (1)

Experimental: H.P. Achtar Gel 80 U

EXPERIMENTAL

H.P. Acthar Gel (repository corticotropin) Injection, 80 U daily for 10 days, then 80 U twice weekly for up to a total of 48 weeks on therapy.

Drug: Repository Corticotropin Injection

Interventions

Repository Corticotropin Injection DRUG

* Initial treatment with 80 units daily for ten days (induction phase) * Maintenance treatment with 80 units twice weekly (maintenance phase)

Also known as: H.P. Acthar Gel, ACTH Gel, ACTH

Experimental: H.P. Achtar Gel 80 U

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient with sarcoidosis as defined by ATS/ERS/WASOG (American Thoracic Society/European Thoracic Society/World Association for Sarcoidosis and Other Granulomatous Disorders)
• Stable baseline immunosuppressive medications
• Moderate to severe disease as defined by at least one of the following criteria:
• Cranial nerve palsy
• Neurologic deficits related to intraparenchymal brain, spinal cord and/or cauda equina involvement
• Dural or leptomeningeal involvement of brain and/or spinal cord
• Hydrocephalus
• Seizures

You may not qualify if:

• Diagnosis of any underlying neurologic disorder that would potentially confound interpretation of the study results
• Significant change in corticosteroid dose within the past 4 weeks, or other immunosuppressive medication within the past 6 months
• Evidence of current serious infection, or a history of chronic or recurring infections.
• Contraindication to high-dose corticosteroids (e.g. uncontrolled blood sugar).
• Allergies to pig-derived proteins
• Have a history of any opportunistic infection within 6 months prior to screening
• History of malignancy.

Sponsors & Collaborators

• The Cleveland Clinic: lead
• Mallinckrodt: collaborator

Study Sites (1)

Cleveland Clinic Main Campus

Cleveland, Ohio, 44195, United States

Location

MeSH Terms

Conditions

Sarcoidosis

Interventions

Adrenocorticotropic Hormone

Condition Hierarchy (Ancestors)

Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Delayed; Hypersensitivity; Immune System Diseases

Intervention Hierarchy (Ancestors)

Melanocortins; Pro-Opiomelanocortin; Hypothalamic Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Pituitary Hormones, Anterior; Pituitary Hormones; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Nerve Tissue Proteins; Proteins

Study Officials

• Daniel Culver, DO

  The Cleveland Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 29, 2016
• First Posted: September 30, 2016
• Study Start: February 1, 2019
• Primary Completion: November 2, 2020
• Study Completion: November 2, 2020
• Last Updated: November 20, 2020

Record last verified: 2020-11

Locations

US (1)