Generalized Myasthenia Gravis
Conditions
Keywords
ALXN1720, anti-acetylcholine receptor antibody-positive, acetylcholine receptor, AChR, generalized myasthenia gravis, myasthenia gravis, MG, gMG, complement component 5, C5, VHH antibody
Brief summary
The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of generalized MG (gMG) in adults with autoantibodies against acetylcholine receptor (AChR).
Interventions
COMBINATION_PRODUCTALXN1720
Combination product consisting of syringe prefilled with ALXN1720.
COMBINATION_PRODUCTPlacebo
Combination product consisting of syringe prefilled with placebo.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Intervention model description
This is a treatment study with 2 parallel intervention groups. The study is blinded for sponsor staff, sponsor designees, investigative site personnel, other staff directly associated with the conduct of the study, and study participants.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV * Positive serological test for autoantibodies against AChR
Exclusion criteria
* History of thymectomy, or any other thymic surgery within 12 months prior to Screening * Untreated thymic malignancy, carcinoma, or thymoma * History of Neisseria meningitidis infection * Pregnancy, breastfeeding, or intention to conceive during the course of the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26 | Baseline, Week 26 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Week 26 | Baseline, Week 26 | — |
| Percentage of Responders Based on Reduction of the MG-ADL Total Score at Week 26 | Baseline up to Week 26 | Response is defined as reduction of the MG-ADL total score by \>= 3 points from baseline at Week 26. |
| Percentage of Responders based on Reduction of the QMG Total Score at Week 26 | Baseline up to Week 26 | Response is defined as reduction of the QMG total score by \>= 5 points from baseline at Week 26 |
| Change From Baseline in Myasthenia Gravis Composite (MGC) Total Score at Week 26 | Baseline, Week 26 | — |
| Change from Baseline in the QMG total score at Week 4 | Baseline, Week 4 | — |
Countries
Argentina, Austria, Brazil, Canada, China, Denmark, France, Germany, Israel, Italy, Japan, Netherlands, Poland, Portugal, Serbia, South Korea, Spain, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom, United States
Outcome results
None listed