Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis

Phase 3

Active, not recruiting

• Conditions: Generalized Myasthenia Gravis
• Interventions: Combination Product: ALXN1720; Combination Product: Placebo

• Registration Number: NCT05556096
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-09-23
• Study First Submitted QC: 2022-09-23
• Study First Posted: 2022-09-27
• Study Start: 2022-11-21
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-07
• Last Update Posted: 2025-05-11
• Primary Completion: 2025-05-29
• Study Completion: 2027-08-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 260

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ALXN1720	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	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.

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26	Baseline, Week 26

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Research Site; 🇬🇧 Sheffield, United Kingdom