Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses
| Status: | Recruiting |
|---|---|
| Healthy: | No |
| Age Range: | 18 - 75 |
| Updated: | 4/2/2016 |
| Start Date: | March 2014 |
| Contact: | Angela Campbell |
| Email: | ajcampbell@stanford.edu |
| Phone: | 650-721-6188 |
A Single Center Open Label Pilot Study of Alpha1-Antitrypsin: A Novel Treatment to Mitigate Neuromyelitis Optica Attacks
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.
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.
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.
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