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ACTH in Progressive Forms of MS

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2016 by University of Minnesota - Clinical and Translational Science Institute
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute Identifier:
First received: August 30, 2013
Last updated: May 13, 2016
Last verified: May 2016
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.

Condition Intervention Phase
Secondary Progressive Multiple Sclerosis
Primary Progressive Multiple Sclerosis
Progressive Relapsing Multiple Sclerosis
Drug: ACTH
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Treatment of Progressive Forms of Multiple Sclerosis With Pulsed ACTH (Acthar Gel)

Resource links provided by NLM:

Further study details as provided by University of Minnesota - Clinical and Translational Science Institute:

Primary Outcome Measures:
  • Proportion of patients exhibiting a 20% worsening in T25FW at 36 months [ Time Frame: Month 36 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Safety and tolerability of ACTH [ Time Frame: Month 36 ] [ Designated as safety issue: Yes ]
    Safety and tolerability will be assessed via safety lab tests, skin and edema assessments, DEXA scans, symptom questionnaires and adverse event assessments.

Other Outcome Measures:
  • Slowed progression of sustained cognitive disability [ Time Frame: Month 36 ] [ Designated as safety issue: No ]
    Brief Repeatable Battery of Neuropsychological Tests (BRB-N)

  • Retinal nerve fiber layer thickness [ Time Frame: Month 36 ] [ Designated as safety issue: No ]
    Decline in retinal nerve fiber layer thickness as measured by optical coherence tomography (OCT)

Estimated Enrollment: 100
Study Start Date: October 2013
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ACTH
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
Drug: ACTH
Acthar gel
Other Name: Acthar gel
Placebo Comparator: Placebo
Placebo subcutaneous injections administered on 3 consecutive days per month
Drug: Placebo


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01950234

Contact: Susan Rolandelli, RN 612-624-7745

United States, Minnesota
Clinical Neuroscience Research Unit, University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55414
Contact: Susan Rolandelli, RN    612-624-7745   
Principal Investigator: Adam F Carpenter, MD         
United States, North Dakota
Sanford Clinic Neuroscience Recruiting
Fargo, North Dakota, United States, 58103
Contact: Tish Skarloken    701-234-4091   
Principal Investigator: Susan Scarberry, MD         
United States, Oklahoma
Oklahoma Medical Research Foundation Multiple Sclerosis Center of Excellence Recruiting
Oklahoma City, Oklahoma, United States, 73104
Contact: Micki Moore    405-271-6241   
Principal Investigator: Gabriel Pardo, MD         
United States, Wisconsin
Wheaton Franciscan Healthcare - St Francis Center for Neurological Disorders Recruiting
Milwaukee, Wisconsin, United States, 53215
Contact: Terry Cannestra, RN    414-769-4013   
Principal Investigator: Bhupendra Khatri, MD         
Sponsors and Collaborators
University of Minnesota - Clinical and Translational Science Institute
Principal Investigator: Adam F Carpenter, MD University of Minnesota - Clinical and Translational Science Institute
  More Information

Responsible Party: University of Minnesota - Clinical and Translational Science Institute Identifier: NCT01950234     History of Changes
Other Study ID Numbers: ACTH-20712 
Study First Received: August 30, 2013
Last Updated: May 13, 2016
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Multiple Sclerosis
Multiple Sclerosis, Chronic Progressive
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Adrenocorticotropic Hormone
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs processed this record on December 06, 2016