Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis

Sponsor

Alexion Pharmaceuticals (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05556096

Collaborator

(none)

254

Enrollment

Locations

Arms

55.3

Anticipated Duration (Months)

31.8

Patients Per Site

0.6

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Generalized Myasthenia Gravis	Combination Product: ALXN1720 Combination Product: Placebo	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

254 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

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.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.

Masking:

Double (Participant, Investigator)

Primary Purpose:

Treatment

Official Title:

A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel, Multicenter Study to Evaluate the Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis

Actual Study Start Date :

Nov 21, 2022

Anticipated Primary Completion Date :

Jul 1, 2025

Anticipated Study Completion Date :

Jun 30, 2027

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ALXN1720 Participants will receive a weight-based maintenance treatment with ALXN1720 on Day 1, followed by weight-based maintenance treatment of ALXN1720 on Week 1 (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 96 weeks.	Combination Product: ALXN1720 Combination product consisting of syringe prefilled with ALXN1720.
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.	Combination Product: Placebo Combination product consisting of syringe prefilled with placebo.

Arm

Intervention/Treatment

Experimental: ALXN1720

Participants will receive a weight-based maintenance treatment with ALXN1720 on Day 1, followed by weight-based maintenance treatment of ALXN1720 on Week 1 (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 96 weeks.

Combination Product: ALXN1720

Combination product consisting of syringe prefilled with ALXN1720.

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.

Combination Product: Placebo

Combination product consisting of syringe prefilled with placebo.

Outcome Measures

Primary Outcome Measures

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

Secondary Outcome Measures

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]
Responders based on reduction by >= 3 points in the change from baseline in the MG-ADL total score at Week 26
Percentage of Responders based on Reduction of the QMG Total Score at Week 26 [Baseline up to Week 26]
Responders based on reduction by >= 5 points in the change from baseline in the QMG total score at Week 26
Change From Baseline in Myasthenia Gravis Composite (MGC) Total Score at Week 26 [Baseline, Week 26]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Research Site	Gainesville	Georgia	United States	30501
2	Research Site	Lexington	Kentucky	United States	40503
3	Research Site	Las Vegas	Nevada	United States	89145
4	Research Site	North Charleston	South Carolina	United States	29406
5	Research Site	Madrid		Spain	28034
6	Research Site	Madrid		Spain	28046
7	Research Site	Pamplona		Spain	31008
8	Research Site	San Sebastián de los Reyes		Spain	28702