ACTH in Progressive Forms of MS

NCT01950234 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 110271
• Study Type: interventional
• Target: 59
• Locations: 1 country
• Sites: 3

Brief Summary

This is a phase II, randomized, double-blind, placebo-controlled, multi-center study to evaluate the safety, tolerability, and efficacy of adrenocorticotropic hormone (ACTH, Acthar gel) administered as a pulsed regimen consisting of injections on three consecutive days per month in patients with progressive forms of Multiple Sclerosis (MS). Patients will be randomly assigned to either an ACTH arm or a placebo arm. The main hypotheses are that 1) pulsed ACTH will be safe and well-tolerated, and 2) pulsed ACTH will slow progression of clinical and paraclinical measures of MS progression compared to placebo.

Conditions

Secondary Progressive Multiple Sclerosis; Primary Progressive Multiple Sclerosis; Progressive Relapsing Multiple Sclerosis

Outcome Measures

Primary Outcomes (1)

• Proportion of Patients Exhibiting a 20% Worsening in T25FW at 36 Months

  Month 36

Secondary Outcomes (21)

• Safety and Tolerability of ACTH: Menstrual Changes [Female]

  Month 36

• Safety and Tolerability of ACTH: DEXA Scans

  Month 36

• Safety and Tolerability of ACTH: Bruising

  Month 36

• Safety and Tolerability of ACTH: Swelling Ankles

  Month 36

• Safety and Tolerability of ACTH: Hair Loss

  Month 36

Study Arms (2)

ACTH

EXPERIMENTAL

ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month

Drug: ACTH

Placebo

PLACEBO COMPARATOR

Placebo subcutaneous injections administered on 3 consecutive days per month

Drug: Placebo

Interventions

ACTH DRUG

Acthar gel

Also known as: Acthar gel

ACTH

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

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

You may qualify if:

• Male or female patients with a confirmed diagnosis of MS by McDonald criteria
• Age \>/= 18 years
• SPMS, PPMS, or PRMS phenotype, according to Lublin and Reingold criteria
• EDSS 2.0 - 6.0, inclusive
• Able to understand the consent process

You may not qualify if:

• Known intolerance of ACTH or corticosteroids
• Diabetes mellitus, defined as pre-existing diagnosis, fasting blood glucose \> 125 mg/dl, or glycosylated hemoglobin \>/= 6.5%
• Osteoporosis, defined as pre-existing diagnosis or T-score on dual-energy x-ray absorptiometry (DEXA) scan of \</= -2.5.
• Current serious medical condition which may interfere with subject's ability to complete the study, or for which pulsed ACTH therapy is contraindicated or might complicate current therapy (e.g., cancer, severe psychiatric illness, chronic infections, autoimmune disorders)
• Treatment with cytotoxic agents (including but not necessarily limited to mitoxantrone, cyclophosphamide, alemtuzumab, or rituximab) within 3 years prior to randomization
• Treatment with non-cytotoxic immunosuppressive agents (including but not necessarily limited to corticosteroids, ACTH, azathioprine, mycophenolate mofetil, methotrexate or natalizumab) within 3 months prior to randomization
• Treatment with FDA-approved first-line MS disease-modifying therapies (B-interferon, glatiramer acetate, fingolimod, teriflunomide or dimethyl fumarate) will be permitted, as long as treatment has been ongoing and stable for at least 3 months prior to randomization
• Treatment with dalfampridine or compounded 4-aminopyridine (4-AP) will be permitted as long as treatment has been ongoing and stable for at least 3 months prior to randomization
• Stimulant medications for fatigue (such as methylphenidate, modafinil, armodafinil, amantadine or dextroamphetamine) will be permitted, but subjects will be asked to not take these medications on study visit days until all study procedures/assessments are completed.

Sponsors & Collaborators

• University of Minnesota: lead
• Mallinckrodt: collaborator

Study Sites (3)

Clinical Neuroscience Research Unit, University of Minnesota

Minneapolis, Minnesota, 55414, United States

Location

Sanford Clinic Neuroscience

Fargo, North Dakota, 58103, United States

Location

Wheaton Franciscan Healthcare - St Francis Center for Neurological Disorders

Milwaukee, Wisconsin, 53215, United States

Location

MeSH Terms

Conditions

Multiple Sclerosis, Chronic Progressive

Interventions

Adrenocorticotropic Hormone

Condition Hierarchy (Ancestors)

Multiple Sclerosis; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

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

Results Point of Contact

• Title: Sarah Hikbert
• Organization: University of Minnesota

hilbe010@umn.edu

612-624-7745

Study Officials

• Adam F Carpenter, MD

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 30, 2013
• First Posted: September 25, 2013
• Study Start: April 17, 2014
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2022
• Last Updated: February 11, 2025
• Results First Posted: February 11, 2025

Record last verified: 2025-02

Locations

US (3)