Trial Design
ULTOMIRIS® was studied in CHAMPION-MG, a randomized, double-blind, placebo-controlled trial with an open-label extension1,2
Patients were randomized to receive either ULTOMIRIS (n=86) or placebo (n=89) for 26 weeks and were subsequently allowed to enter the open-label extension (OLE) period for up to 4 years.3
CHAMPION-MG baseline characteristics1,2
The majority of symptomatic patients were already being treated with an IST4
In the randomized, double-blind, placebo-controlled CHAMPION-MG trial, approximately 90% of patients were taking an IST at baseline.1,2,4
- 47% of patients were taking 2 or more ISTs2
43% of patients received IVIg in the 2 years prior to trial screening.2
More than 90% of patients had MGFA class II or III generalized myasthenia gravis (gMG) with mild or moderate weakness at baseline.2,4
- 7% of patients in the ULTOMIRIS subgroup had class IV gMG (severe weakness)4
CHAMPION-MG studied multiple measures of generalized myasthenia gravis (gMG)1,2
Primary endpoint1:
- Change from baseline to Week 26 in the Myasthenia Gravis Activities of Daily Living (MG-ADL) total scorea
Secondary endpointsb,c:
- Change from baseline to Week 26 in the Quantitative Myasthenia Gravis (QMG) total score1,d
- The proportion of patients with improvements of at least 5 points in their QMG total score1
- Change in Myasthenia Gravis Quality of Life 15-item scale (MG-QoL15r)2
- Change in Neurological Quality of Life (Neuro-QoL) Fatigue assessment2
- The proportion of patients with improvements of at least 3 points in their MG-ADL total score1
aThe MG-ADL is a categorical scale that assesses the impact on daily function of 8 signs or symptoms that are typically affected in gMG. Each item is assessed on a 4-point scale where a score of 0 represents normal function and a score of 3 represents loss of ability to perform that function. The total score ranges from 0 to 24, with the higher scores indicating more impairment.1
bHierarchical testing proceeded from the first to the fifth endpoint, and if statistical significance was not achieved (P-value >0.05), then subsequent endpoints were not considered statistically significant.2
cAll secondary endpoints are at Week 26.1
dThe QMG is a 13-item categorical scale assessing muscle weakness. Each item is assessed on a 4-point scale where a score of 0 represents no weakness and a score of 3 represents severe weakness. The total score ranges from 0 to 39, where higher scores indicate more severe impairment.1
Key inclusion and exclusion criteria
Key inclusion criteria2:
Patients enrolled in this trial had to have an MGFA clinical classification of class II through IV; gMG (diagnosed for at least 6 months) with a positive serologic test for anti-AChR antibodies; MG-ADL total score ≥6; and vaccinations against meningococcal infections. Patients on concomitant ISTs were required to be on stable doses.
Key exclusion criteria2:
Patients were excluded from this trial if they had any active or untreated thymoma or history of thymic carcinoma or thymic malignancy; history of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to screening; clinical features that are consistent with myasthenia gravis crisis/exacerbation or clinical deterioration at the time of the screening visit or at any time prior to randomization.
Patients were excluded if the following therapies were used within timeframes listed:
- IVIg or PE within the 4 weeks prior to randomization (Day 1)
- Rituximab within the 6 months prior to screening
- Any previous treatment with complement inhibitors
AChR, acetylcholine receptor; IST, immunosuppressant therapy; IVIg, intravenous immunoglobulin; MGFA, Myasthenia Gravis Foundation of America; PE, plasma exchange; SOC, standard of care.