Clinical trial NCT01888354: Pilot Clinical Trial of ACTHar Gel 14 Days Subcutaneous (SQ)Versus ACTHar Gel Five Days SQ for the Treatment of MS Exacerbations
Official title: Pilot Clinical Trial of ACTHar Gel 14 Days Subcutaneous (SQ) Versus ACTHar Gel Five Days SQ for the Treatment of MS Exacerbations
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Multiple Sclerosis (MS)
Keywords: MS
Multiple Sclerosis
Lead sponsor: The University of Texas Health Science Center, Houston
Sponsor class: OTHER
First posted: 2013-06-27
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This pilot study is designed as a prospective cohort study to determine whether standard subcutaneous (SQ) Highly-Purified (HP) Acthar Gel 14 days is superior to SQ HP Acthar Gel 5 days in the treatment of relapses or attacks in multiple sclerosis (MS).

Detailed description: Evaluations and treatment will be administered as an outpatient in the Neurology Clinic. Each subject will be seen for MS relapse or exacerbation in the Neurology Clinic during a routine or semi-emergent visit. Patients will be offered an FDA approved treatment (ACTHar Gel) for MS attacks under a standard 14 day SQ protocol (standard 14 day SQ protocol (80 IU x 14 days) or a 5 day SQ protocol - (5 day subcutaneous protocol (80 IU x 5 days). Both protocols are within package insert guidelines. The subjects will be evaluated for Extended Disability Status Scales (EDSS), a standardized measure of clinical status in MS and the initial visit and 28 days later (and 90 days later). We will compare the change in EDSS outcomes between day 0 and day 28 (day 90) to determine if the 14 days is superior to 5 day protocol may be clinically equivalent. Patients will also be evaluated for walking, upper extremity function (9 hole peg test), cognition and vision.

Eligibility criteria: Inclusion Criteria:

1. Adult male or female subjects with MS having a relapse (attack) or exacerbation of MS. Acute symptomatic exacerbation of MS present for \> 24 hours and \< 14 days at screening with new or worsening symptoms, and with signs referable to the symptoms in the absence of a fever or active infection.
2. Diagnosis of a relapsing forms of multiple sclerosis before randomization as determined by Poser or McDonald Criteria (standard MS diagnostic criteria).
3. Expanded disability status scale (EDDS) between 2 and 6.5, inclusive at entry.
4. Episodes include study neurologist or neuro-ophthalmologist diagnosed: acute optic neuritis, cerebellar, brainstem dysfunction, myelitis, focal cerebral, and/or definitive focal sensory dysfunction.
5. New objective clinical finding other than the sensory exacerbation or the bowel/bladder signs alone. Sensory deficits alone will not qualify except for optic neuritis.
6. Subjects may continue on their current immunomodulation therapy such as interferons, glatiramer acetate, gilenya or natalizumab.
7. Identified patients must be between the ages of 18 and 55 years, inclusive.
8. Ability to understand and the willingness to sign a written informed consent document.

Exclusion Criteria:

1. Subjects who are pregnant, or nursing.
2. Any patients treated with systemic corticosteroid use within one month of the index episode at screening.
3. Prior use of immunosuppressive treatments within 90 days of index episode (mitoxantrone, azathioprine, Cellcept, IVIg) or plasmapheresis.
4. Unable to perform timed 25 foot walk (ambulation Index), 9 HPT (9 hole peg test), PASAT (Paced Auditory Serial Addition Test) 3.
5. Peripheral or cranial neuropathy as sole problem of acute episode.
6. Subjects with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, or psychiatric illness/social situations that would limit compliance with study requirements.
7. History of any significant cardiac, gastrointestinal, hepatic, pulmonary, or renal disease; immune deficiency; or other medical conditions that would preclude corticosteroid therapy.
8. Subjects with clinical diagnosis of scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of a peptic ulcer, congestive heart failure, hypertension, or sensitivity to proteins of porcine origin.
9. Primary Progressive Multiple Sclerosis (PPMS) (MS without attacks). -

Primary outcomes:
measure: Comparison of EDSS Mean Recovery between 5-day vs 14-day SQ 80 IU ACTHar Gel therapy groups
timeFrame: 28 Days (with 90 day follow-up)
Secondary outcomes:
measure: Comparison of ambulation between the 5-day vs.14-day SQ 80 IU ACTHar Gel therapy groups
timeFrame: 28 Days (with 90 day follow-up)
measure: Comparison of upper extremity function between the 5-day vs.14-day SQ 80 IU ACTHar Gel therapy groups
timeFrame: 28 day (with 90 follow up)
measure: Comparison of subjective impressions of effectiveness between the 5-day vs 14-day SQ 80 IU ACTHar Gel therapy groups
timeFrame: 28 day (with 90 day follow up)
measure: Compare visual function between 5-day vs 14-day SQ 80 IU ACTHar Gel therapy groups
timeFrame: 28 day (with 90 day follow up)
measure: Comparison of cognitive function between the 5-day vs 14-day SQ 80 IU ACTHar Gel therapy groups
timeFrame: 28 day (with 90 day follow up)
Other outcomes:
measure: Comparison of MRI brain activity before and after ACTH treatment between the two treatment groups
timeFrame: 28 Days (with 90 day follow-up)
measure: Comparison of immune changes before and after ACTH treatment between the two treatment groups
timeFrame: 28 Days (with 90 day follow-up)
Locations:
city: Oceanside
country: United States
facility: The MS Center at the Neurology Center in Southern California
state: California
city: Houston
country: United States
facility: The University of Texas-Houston Neurology Clinic
state: Texas
Study officials:
affiliation: The Universtiy of Texas-Houston
name: Staley A. Brod, MD
role: PRINCIPAL_INVESTIGATOR
References: