Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis

NCT04982289 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: ALXN1830-MG-201
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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).

Conditions

Generalized Myasthenia Gravis

Keywords

Generalized Myasthenia Gravis

Outcome Measures

Primary Outcomes (3)

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

  Up to Week 24

• AEs And SAEs Up To Week 82

  Up to Week 82 (OLE)

• Change From Baseline In Serum Total Immunoglobulin G (IgG)

  Up to Week 24

Secondary Outcomes (9)

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

  Up to Week 24

• Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score

  Up to Week 24

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

  Up to Week 24

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

  Up to Week 8

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

  Up to Week 24

Study Arms (3)

ALXN1830 Dosing Arm 1

EXPERIMENTAL

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

Drug: ALXN1830

ALXN1830 Dosing Arm 2

EXPERIMENTAL

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

Drug: ALXN1830

ALXN1830 Dosing Arm 3

EXPERIMENTAL

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

Drug: ALXN1830; Other: Placebo

Interventions

ALXN1830 DRUG

Administered as an SC infusion.

ALXN1830 Dosing Arm 1; ALXN1830 Dosing Arm 2; ALXN1830 Dosing Arm 3

Placebo OTHER

Administered as an SC infusion.

ALXN1830 Dosing Arm 3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (1)

Clinical Study Site

Phoenix, Arizona, 85028, United States

Location

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2021
• First Posted: July 29, 2021
• Study Start: January 1, 2022
• Primary Completion: January 31, 2023
• Study Completion: January 31, 2024
• Last Updated: February 11, 2022

Record last verified: 2022-02

Locations

US (1)