Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis

• Sponsor: Alexion Pharmaceuticals (Industry)
• Overall Status: Withdrawn
• CT.gov ID: NCT04982289
• Collaborator: (none)
• Enrollment: 0
• Location: 1
• Arms: 3
• Anticipated Duration (Months): 25
• Patients Per Site Per Month: 0

Study Details

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).

Condition or Disease	Intervention/Treatment	Phase
Generalized Myasthenia Gravis	Drug: ALXN1830; Other: Placebo	Phase 2

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).

Study Design

• Study Type: Interventional
• Actual Enrollment: 0 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• 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
• Anticipated Study Start Date: Jan 1, 2022
• Anticipated Primary Completion Date: Jan 31, 2023
• Anticipated Study Completion Date: Jan 31, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ALXN1830 Dosing Arm 1 Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks.	Drug: ALXN1830; Administered as an SC infusion.
Experimental: ALXN1830 Dosing Arm 2 Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks.	Drug: ALXN1830; Administered as an SC infusion.
Experimental: ALXN1830 Dosing Arm 3 Participants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks.	Drug: ALXN1830; Administered as an SC infusion.; Other: Placebo; Administered as an SC infusion.

Outcome Measures

Primary Outcome Measures

1. Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24 [Up to Week 24]

2. AEs And SAEs Up To Week 82 [Up to Week 82 (OLE)]

3. Change From Baseline In Serum Total Immunoglobulin G (IgG) [Up to Week 24]

Secondary Outcome Measures

1. Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score [Up to Week 24]

2. Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score [Up to Week 24]

3. Number Of Participants With At Least A 2-point Improvement In The MG-ADL Score Over 4 Consecutive Weeks [Up to Week 24]

4. Number Of Participants With At Least A 3-point Improvement In The QMG Score Over 4 Consecutive Weeks [Up to Week 8]

5. Change From Baseline In Neurological Disorders Fatigue Questionnaire (Neuro-Qol) Fatigue Score [Up to Week 24]

6. Serum Trough Concentrations Of ALXN1830 [Up to Week 24]

7. Change From Baseline In IgG Subtypes [Up to Week 24]

8. Incidence Of Anti-drug Antibodies (ADA) And Neutralizing Antibodies (Nab) Against ALXN1830 [Up to Week 24]

9. Titers Of ADA And Nab Against ALXN1830 [Up to Week 24]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Study Site	Phoenix	Arizona	United States	85028

Sponsors and Collaborators

• Alexion Pharmaceuticals

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Alexion Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT04982289
• Other Study ID Numbers: ALXN1830-MG-201
• First Posted: Jul 29, 2021
• Last Update Posted: Feb 11, 2022
• Last Verified: Feb 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Alexion Pharmaceuticals: Generalized Myasthenia Gravis
• Additional relevant MeSH terms: Myasthenia Gravis; Muscle Weakness

Study Results

No Results Posted as of Feb 11, 2022