Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis

Alexion Pharmaceuticals

Status and phase: Withdrawn; Phase 2

Conditions: Generalized Myasthenia Gravis

Treatments: Other: Placebo; Drug: ALXN1830

Study type: Interventional

Funder types: Industry

Identifiers

NCT04982289

ALXN1830-MG-201

Details and patient eligibility

About

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).

Full 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).

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

---

0 participants in 3 patient groups

ALXN1830 Dosing Arm 1

Experimental group

Description:

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

Treatment:

Drug: ALXN1830

ALXN1830 Dosing Arm 2

Experimental group

Description:

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

Treatment:

Drug: ALXN1830

ALXN1830 Dosing Arm 3

Experimental group

Description:

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

Treatment:

Other: Placebo

Drug: ALXN1830