Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02087813
• Date of registration: 11/03/2014
• Prospective Registration: No
• Primary sponsor: Stanford University
• Public title: Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses A1AT for NMO
• Scientific title: A Single Center Open Label Pilot Study of Alpha1-Antitrypsin: A Novel Treatment to Mitigate Neuromyelitis Optica Attacks
• Date of first enrolment: March 2014
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT02087813
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Alexandra L Goodyear, MD, MS
• Affiliation: Stanford University

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Neuromyelitis Optica

Intervention(s)

Drug: methylprednisolone

Drug: Alpha1-antitrypsin

Primary Outcome(s)

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.]

Secondary Outcome(s)

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]

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 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.]

Secondary ID(s)

IRB-27176

Source(s) of Monetary Support

Please refer to primary and secondary sponsors