UPLIZNA was studied in the largest prospective, placebo-controlled clinical trial in neuromyelitis optica spectrum disorder (NMOSD) to date1a
STUDY DESIGN
N-MOmentum was a phase 2/3, multicentre, double-blind, randomised, placebo-controlled trial with an open-label period (OLP).1a
Randomised controlled period studied 213 AQP4-IgG+ patients and 17 AQP4-IgG– patients for 6 months.1 Only
AQP4-IgG– patients were randomly allocated to placebo, and no attack occurred in this group; efficacy could not be interpreted in the AQP4-IgG– cohort.1
- Inclusion criteria: Adults diagnosed with NMOSD, EDSS score ≤8.0, and a history of ≥1 attack requiring rescue therapy during the year before screening or ≥2 attacks requiring rescue therapy in the 2 years before screening1 (Exclusion Criteria)
- Well-defined primary endpoint: Time to onset of an NMOSD attack by day 1971
- Key secondary endpoints: Worsening of EDSS scoreb from baseline, cumulative number of active magnetic resonance imaging (MRI) lesions,c number of NMOSD-related inpatient hospitalisations (longer than an overnight stay), change from baseline in low-contrast visual acuity binocular score (by low-contrast Landolt C broken ring chart)1
- Robust attack adjudication: All attacks were verified by an independent relapse-adjudication committee using 18 predefined attack criteria1,2
- Dosing: Patients were randomised to receive UPLIZNA 300 mg IV or placebo1
- Patients who had an attack or completed the randomised controlled period could enter the open-label period1
BASELINE CHARACTERISTICS IN THE RANDOMISED CONTROLLED PERIOD1,2
| Mean age | 43 years |
| Gender | 94% female |
| ≥2 attacks (2 years prior) | 83% |
| Median disease duration | 1.1 years (UPLIZNA group) |
| Median baseline EDSS score | 3.5 (range 0 to 8) |
N-MOmentum was developed to reflect the real-world NMOSD patient population and recruited 213 AQP4-IgG+ patients from 25 countries at 99 sites.1 The open-label period was set to continue for a minimum of 1 year and a maximum of 3 years (after the last subject enters), or until regulatory approval for UPLIZNA in the participating country.3
95% of UPLIZNA patients opted to continue treatment in the open-label period.4
PATIENTS WERE GIVEN THE OPTION TO ENTER THE OPEN-LABEL PERIOD IF THEY:
- completed 197 days of the randomised controlled period (RCP), or experienced an adjudication committee-determined NMO/NMOSD attack during the RCP,3
- were in the RCP at the time when 67 NMOSD attacks had occurred,3 or
- were in the RCP when enrolment was discontinued upon recommendation of the DMC based on evidence of efficacy and safety.3
Patients who discontinued the RCP for reasons other than one of these three mentioned above were not eligible for the OLP.3
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aRandomised controlled period studied 213 AQP4-IgG+ patients and 17 AQP4-IgG–patients for 6 months.1
bIncrease of ≥2 (from baseline of 0), ≥1 (from baseline of 1–5), or ≥0.5 (from baseline of ≥5.5).1
cNew Gd-enhancing lesions, or new or enlarging T2 lesions, in the optic nerve, brain, brainstem, and spinal cord.1
AQP4-IgG+: aquaporin-4 immunoglobulin G seropositive; AQP4-IgG–: aquaporin-4 immunoglobulin G seronegative; EDSS: expanded disability status scale; Gd: gadolinium; MRI: magnetic resonance imaging; NMOSD: neuromyelitis optica spectrum disorder; OLP: open-label period; RCP: randomised controlled period; T2: transverse relaxation time; IV: intravenous; DMC: data-monitoring committee.