Pulse ACTH vs. MP for MS

• ClinicalTrials.gov Identifier: NCT01049451
• Recruitment Status: Completed
• First Posted: January 14, 2010
• Last Update Posted: April 19, 2017
• Sponsor: University of Southern California
• Information provided by (Responsible Party): Regina Berkovich, University of Southern California

Tracking Information

• First Submitted Date: January 13, 2010
• First Posted Date: January 14, 2010
• Last Update Posted Date: April 19, 2017
• Study Start Date: November 2009
• Actual Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: January 13, 2010): Annualized Relapse Rate [ Time Frame: 15 months study: 12 months treatment and 3 months follow up ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures (submitted: January 13, 2010): Multiple Sclerosis Functional Composite (MSFC), Expanded Disability Status Scale score (EDSS) and Multiple Sclerosis Quality of Life (MSQOL), peripheral blood lymphocytes measure for regulatory immune activities. [ Time Frame: 15 months: 12 months treatmen and 3 months follow up ]
• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Pulse ACTH vs. MP for MS
• Official Title: Comparison of Monthly Pulse ACTH (Acthar Gel) Therapy With Methylprednisolone (MP, Solumedrol) for Long-Term Treatment of Multiple Sclerosis (MS) as an Add on Therapy to Beta-interferons (Avonex, Betaseron or Rebif)

Brief Summary

We hypothesize that corticotropin or adrenocorticotrophic hormone (ACTH), administered as Acthar Gel® (MANUFACTURER NAME) is effective in the control of clinical disease activity as a pulse therapy for relapsing-remitting MS when added to standard treatment with beta-interferon.

We wish to determine whether ACTH, when administered as clustered monthly intramuscular injections (monthly pulse therapy) as add-on to beta-interferons, may be a safe and effective alternative to monthly pulse therapy with MP. In addition, we hypothesize that pulse therapy with ACTH alters immune function to favor a regulatory, rather than a pro-inflammatory T cell environment.

• Detailed Description: Please see the final manuscript
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Single (Outcomes Assessor); Primary Purpose: Treatment
• Condition: Multiple Sclerosis

Intervention

• Drug: ACTH
  Subjects assigned to the ACTH arm will receive ACTH (Acthar gel) as intramuscular (IM) injections once a day for 3 consecutive days on a monthly basis, for 12 consecutive months. The dosage of ACTH will be 80 units per injection, for a total of 240 units over the three day period.
  Other Name: Acthar Gel
• Drug: Methylprednisolone
  Subjects assigned to the MP arm will receive intravenous (IV) infusions of 1 gram of MP once a month for 12 months
  Other Name: Solumedrol

Study Arms

• Experimental: ACTH IM monthly
  Subjects assigned to the ACTH arm will receive ACTH (Acthar gel) as intramuscular (IM) injections once a day for 3 consecutive days on a monthly basis, for 12 consecutive months. The dosage of ACTH will be 80 units per injection, for a total of 240 units over the three day period.
  Intervention: Drug: ACTH
• Active Comparator: MP IV monthly
  Subjects assigned to the MP arm will receive intravenous (IV) infusions of 1 gram of MP once a month for 12 months.
  Intervention: Drug: Methylprednisolone

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 17, 2017): 23
• Original Estimated Enrollment (submitted: January 13, 2010): 24
• Actual Study Completion Date: July 2013
• Actual Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

1. Clinically definite multiple sclerosis defined by McDonald Criteria.
2. Between 18-65 years of age.
3. Subject must able to understand and sign the IRB- approved informed consent form prior to the performance of any study-specific procedures and is willing to comply with the required scheduling and assessments of the protocol, including self-administration of study drug (if assigned to the ACTH arm).
4. Subjects who are women of childbearing potential, must have a negative serum pregnancy test at the screening visit, and must be willing to practice a reliable birth-control method.
5. Subjects must have at least one MS relapse within last year and/or at least one new T2 or Gadolinium-enhanced lesion on MRI while on stable interferon therapy.
6. EDSS (Expanded Disability Status Scale) score of 3.0 - 6.5
7. Currently on one of the approved beta-interferon drugs (Avonex, Betaseron, or Rebif) for a minimum of 6 months.

Exclusion Criteria:

1. Women who are either pregnant or breastfeeding, and women of child-bearing potential (defined as not surgically sterile or at least two years post menopausal) who are not using one of the following birth control methods: tubal ligation, implantable contraception device, oral, patch, injectable or transdermal contraceptive, barrier method or sexual activity restricted to vasectomized partner.
2. Uncontrolled hypertension, clinically significant cardiac arrhythmias, gastrointestinal ulcer, uncontrolled diabetes mellitus, osteoporosis, any stage of renal failure, psychiatric disorders or any other clinically significant general health conditions that may interfere with the trial participation.
3. Subject has a history of drug or alcohol abuse within the past year.
4. Subject had corticosteroid treatment within last 90 days.
5. Subject started new medication within last 30 days.
6. Subject is on Tysabri treatment presently or within 6 months of screening.
7. Subject is on Novantrone, Cellcept, Rituxan or other chemotherapeutic treatment presently or within 6 months of screening.
8. Subject is a participant in another research project.
9. Subject has contraindications for either ACTH or MP administration.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01049451
• Other Study ID Numbers: ACTH-001; IND 104,973 ( Registry Identifier: IND 104,973 )
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: Regina Berkovich, University of Southern California
• Original Responsible Party: Questcor Pharmaceuticals, Inc., Questcor Pharmaceutical, Inc.
• Current Study Sponsor: University of Southern California
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Regina R Berkovich, MD, PhD; University of Southern California

• PRS Account: University of Southern California
• Verification Date: April 2017