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Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis

Primary Purpose

Generalized Myasthenia Gravis

Status

Withdrawn

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

ALXN1830

Placebo

About this trial

This is an interventional treatment trial for Generalized Myasthenia Gravis focused on measuring Generalized Myasthenia Gravis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

Diagnosis of myasthenia gravis.
Positive serologic test for anti-acetylcholine receptor antibodies.
Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at Screening.
MG-ADL profile must be ≥ 5.
Participants receiving stable treatment with azathioprine; other immunosuppressive therapies.
Total IgG level at Screening ≥ 600 milligrams/deciliter.

Key Exclusion Criteria:

History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to Screening.
Any untreated thymic malignancy, carcinoma, or thymoma.
Intravenous immunoglobulin within the 6 weeks, and/or use of plasmapheresis/plasma exchange prior to randomization (Day 1).
Use of rituximab within the 3 months (90 days) prior to Screening.
Participants who have received previous treatment with any biological agent or other anti-neonatal fragment crystallizable receptor therapy within 5 half-lives or 90 days after last dose (whichever is longer).
Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the participant's full participation in the study, pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study.

Sites / Locations

Clinical Study Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

ALXN1830 Dosing Arm 1

ALXN1830 Dosing Arm 2

ALXN1830 Dosing Arm 3

Arm Description

Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks.

Participants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks.

Outcomes

Primary Outcome Measures

Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24

AEs And SAEs Up To Week 82

Change From Baseline In Serum Total Immunoglobulin G (IgG)

Secondary Outcome Measures

Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score

Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score

Number Of Participants With At Least A 2-point Improvement In The MG-ADL Score Over 4 Consecutive Weeks

Number Of Participants With At Least A 3-point Improvement In The QMG Score Over 4 Consecutive Weeks

Change From Baseline In Neurological Disorders Fatigue Questionnaire (Neuro-Qol) Fatigue Score

Serum Trough Concentrations Of ALXN1830

Change From Baseline In IgG Subtypes

Incidence Of Anti-drug Antibodies (ADA) And Neutralizing Antibodies (Nab) Against ALXN1830

Titers Of ADA And Nab Against ALXN1830

Full Information

NCT ID

NCT04982289

First Posted

June 21, 2021

Last Updated

February 2, 2022

Sponsor

Alexion

1. Study Identification

Unique Protocol Identification Number

NCT04982289

Brief Title

Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis

Official Title

A Phase 2a Randomized, Double-blind, Placebo-controlled, Parallel Groups, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALXN1830 Administered Subcutaneously in Adult Patients With Generalized Myasthenia Gravis

Study Type

Interventional

2. Study Status

Record Verification Date

February 2022

Overall Recruitment Status

Withdrawn

Why Stopped

Sponsor decision to terminate program

Study Start Date

January 1, 2022 (Anticipated)

Primary Completion Date

January 31, 2023 (Anticipated)

Study Completion Date

January 31, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Alexion

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

This study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics (PD), immunogenicity, and efficacy of subcutaneous (SC) ALXN1830 in adults with generalized myasthenia gravis (gMG).

Detailed Description

Participants have the option to enroll in an Open-label Extension (OLE) Period to receive ALXN1830 up to 58 weeks (including follow-up period).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Generalized Myasthenia Gravis

Keywords

Generalized Myasthenia Gravis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ALXN1830 Dosing Arm 1

Arm Type

Experimental

Arm Description

Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks.

Arm Title

ALXN1830 Dosing Arm 2

Arm Type

Experimental

Arm Description

Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks.

Arm Title

ALXN1830 Dosing Arm 3

Arm Type

Experimental

Arm Description

Participants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks.

Intervention Type

Drug

Intervention Name(s)

ALXN1830

Intervention Description

Administered as an SC infusion.

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

Administered as an SC infusion.

Primary Outcome Measure Information:

Title

Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24

Time Frame

Up to Week 24

Title

AEs And SAEs Up To Week 82

Time Frame

Up to Week 82 (OLE)

Title

Change From Baseline In Serum Total Immunoglobulin G (IgG)

Time Frame

Up to Week 24

Secondary Outcome Measure Information:

Title

Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score

Time Frame

Up to Week 24

Title

Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score

Time Frame

Up to Week 24

Title

Number Of Participants With At Least A 2-point Improvement In The MG-ADL Score Over 4 Consecutive Weeks

Time Frame

Up to Week 24

Title

Number Of Participants With At Least A 3-point Improvement In The QMG Score Over 4 Consecutive Weeks

Time Frame

Up to Week 8

Title

Change From Baseline In Neurological Disorders Fatigue Questionnaire (Neuro-Qol) Fatigue Score

Time Frame

Up to Week 24

Title

Serum Trough Concentrations Of ALXN1830

Time Frame

Up to Week 24

Title

Change From Baseline In IgG Subtypes

Time Frame

Up to Week 24

Title

Incidence Of Anti-drug Antibodies (ADA) And Neutralizing Antibodies (Nab) Against ALXN1830

Time Frame

Up to Week 24

Title

Titers Of ADA And Nab Against ALXN1830

Time Frame

Up to Week 24

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Key Inclusion Criteria: Diagnosis of myasthenia gravis. Positive serologic test for anti-acetylcholine receptor antibodies. Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at Screening. MG-ADL profile must be ≥ 5. Participants receiving stable treatment with azathioprine; other immunosuppressive therapies. Total IgG level at Screening ≥ 600 milligrams/deciliter. Key Exclusion Criteria: History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to Screening. Any untreated thymic malignancy, carcinoma, or thymoma. Intravenous immunoglobulin within the 6 weeks, and/or use of plasmapheresis/plasma exchange prior to randomization (Day 1). Use of rituximab within the 3 months (90 days) prior to Screening. Participants who have received previous treatment with any biological agent or other anti-neonatal fragment crystallizable receptor therapy within 5 half-lives or 90 days after last dose (whichever is longer). Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the participant's full participation in the study, pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study.

Facility Information:

Facility Name

Clinical Study Site

City

Phoenix

State/Province

Arizona

ZIP/Postal Code

85028

Country

United States

12. IPD Sharing Statement

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Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis

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