Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses

Inclusion Criteria:

   - Provide written informed consent.

   - Age ≥18 and ≤ 75 years.

   - Diagnosis of NMO or NMO spectrum disorder (NMOSD). The diagnosis of NMO will conform
   to the 2006 Wingerchuk criteria.1, 2 The diagnosis of NMOSD will include patients with
   relapsing optic neuritis and aquaporin-4 antibody (AQP4) seropositivity or patients
   with relapsing longitudinally extensive transverse myelitis and AQP4
   seropositivity.2-5 NMO and NMOSD will be collectively referred to as NMO.

   - AQP4-antibody positivity.

   - Present with an acute NMO attack (see definition below).

   - Patients must not have a history of clinically significant infusion reactions with
   administration of biologic agents.

   - If on chronic treatment for NMO, treatment was initiated at least 3 months earlier and
   medication dose is stable. Additional restrictions will be placed on changes in
   concomitant symptomatic medications.

   - A female subject of childbearing potential must have a negative serum pregnancy test
   at the screening visit and agree to use a medically reliable method of contraception
   (e.g., barrier with either spermicide or hormonal contraception) until study
   completion.

   - Agree to answer the questions on the Columbia Suicide Severity Rating Scale at each
   specified visit.

Exclusion Criteria:

   - A woman who is pregnant, breastfeeding, or planning pregnancy.

   - If the patient is enrolled in any other experimental trial or on other experimental
   therapy.

   - If the patient has a known IgA deficiency with IgA-antibodies.

   - Any medical condition or clinically significant laboratory abnormality that in the
   Investigator's judgment may affect the patient's ability to safely complete the study.

Acute attack:

   - The occurrence of new or worsening neurological symptoms consistent with optic
   neuritis, transverse myelitis, or a brain lesion that develop acutely (i.e., patients
   must present within 7 days of symptom onset).

   - The symptoms must persist for at least 48 hours, are not attributable to confounding
   clinical factors (e.g., fever, infection, injury, adverse reactions to concomitant
   medications).

   - The symptoms must be accompanied by sensory, motor or visual acuity objective
   deficits, which must be verified by the examining physician.

   - A single episode of a paroxysmal symptom (e.g., tonic spasm) is not a relapse;
   however, the new onset of multiple occurrences of a paroxysmal symptom over at least
   48 hours can be a relapse if accompanied by a new, corresponding objective deficit.

   - Sensory symptoms with no change on clinical examination, fatigue, mood change, or
   bladder or bowel urgency or incontinence will not be sufficient to establish a
   relapse.