Clinical Trial: Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses

Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional

Official Title: A Single Center Open Label Pilot Study of Alpha1-Antitrypsin: A Novel Treatment to Mitigate Neuromyelitis Optica Attacks

Brief Summary: Neuromyelitis Optica (NMO) is a rare, devastating demyelinating disease of the central nervous system (CNS) that has different causes and treatments from the more common demyelinating disease multiple sclerosis (MS). Current NMO therapies are nonspecific and have varying and often suboptimal benefit. The investigators will evaluate whether use of alpha1-antitrypsin (A1AT, an FDA-approved medication for patients with congenital deficiency of A1AT associated with emphysema) can benefit acute attacks of NMO, improving patient disability and quality of life.

Detailed Summary:
Sponsor: Stanford University

Current Primary Outcome: Mean change in disability from baseline/nadir to week 24 as assessed by Opticospinal Impairment Score (OSIS) subscale. [ Time Frame: Baseline, Week 1: Day 2, Week 2, 3, 4, 8, 16, and 24. ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Mean change in disability from baseline/nadir to week 24 as assessed by Expanded Disability Status Scale (EDSS). [ Time Frame: Baseline, Week 1: Day 2, Week 2, 3, 4, 8, 16, and 24. ]
  • For patients experiencing optic neuritis, mean change in visual acuity from baseline/nadir to week 24 as assessed by Sloan 2.5% low contrast visual acuity chart. [ Time Frame: Baseline, Week 1: Day 2, Week 2, 3, 4, 8, 16, and 24. ]
  • Mean change in retinal nerve fiber layer from baseline/nadir to week 24 as assessed by optical coherence tomography (OCT). [ Time Frame: Baseline and Week 24 ]
  • Mean change in length of spinal cord lesion from baseline/nadir to week 24 as assessed by magnetic resonance imaging (MRI) T2 sequences. [ Time Frame: Baseline, Week 24 ]


Original Secondary Outcome: Same as current

Information By: Stanford University

Dates:
Date Received: March 11, 2014
Date Started: March 2014
Date Completion:
Last Updated: March 12, 2014
Last Verified: March 2014