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).
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
Primary outcome:
1. Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26 (Time Frame - Baseline, Week 26)
Secondary outcome:
1. Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Week 26 (Time Frame - Baseline, Week 26)
2. Percentage of Responders Based on Reduction of the MG-ADL Total Score at Week 26 (Time Frame - Baseline up to Week 26):
Response is defined as reduction of the MG-ADL total score by >= 3 points from baseline
at Week 26.
3. Percentage of Responders based on Reduction of the QMG Total Score at Week 26 (Time Frame - Baseline up to Week 26):
Response is defined as reduction of the QMG total score by >= 5 points from baseline at
Week 26
4. Change From Baseline in Myasthenia Gravis Composite (MGC) Total Score at Week 26 (Time Frame - Baseline, Week 26)
- Experimental: ALXN1720
Participants will receive a weight-based initial (loading) dose of ALXN1720 on Day 1,
followed by weight-based maintenance treatment with ALXN1720 on Day 8 and once every week
(Q1W) thereafter for a total of 26 weeks. Following this randomized controlled treatment
(RCT) period, all participants will receive ALXN1720 in an open-label extension (OLE)
period of 105 weeks. - Placebo Comparator: Placebo
Participants will receive placebo during the 26-week RCT period, after which they will
enter the OLE period of the study and receive ALXN1720.
- ALXN1720:
Combination product consisting of syringe prefilled with ALXN1720. - Placebo:
Combination product consisting of syringe prefilled with placebo.
Quelle: ClinicalTrials.gov
