drugset / Trial / NCT02087813

Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses

NCT02087813

Phase 1 Withdrawn Stanford University
Non-randomizedParallel-groupOpen-labelTreatment

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.

Timeline

Start
2014-03
Primary completion
2016-03
Completion

Drugs

EvaluationDrugModalityDoseRoute
Subject Alpha 1-Antitrypsin Protein / enzyme biologic 120 mg/kg Intravenous
Background Methylprednisolone Other / unclassified 1000 mg Intravenous

Indications