A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)

NCT ID: NCT06987539

Last Updated: 2025-12-01

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-04-30
• Study Completion Date: 2030-03-13

Brief Summary

The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG, and to assess the safety and tolerability of inebilizumab administered in pediatric participants with gMG.

Conditions

Generalized Myasthenia Gravis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Inebilizumab

Inebilizumab will be administered intravenously (IV).

Group Type: EXPERIMENTAL

Inebilizumab

Intervention Type: DRUG

Inebilizumab will be administered IV.

Interventions

Inebilizumab

Inebilizumab will be administered IV.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and/or guidelines before any study-specific activities/procedures being initiated.
• Age ≥ 2 to < 18 years of age on the day of enrollment.
• Diagnosis of gMG defined as:

• Positive serologic test for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody (Ab) titers as confirmed at screening (1 retest allowed), and
• At least 1 of the following:

• History of abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; or
• History of positive anticholinesterase test (eg, edrophonium chloride test); or
• Participant demonstrated improvement in gMG signs on oral cholinesterase inhibitors, as assessed by the treating physician; or
• Clinical syndrome consistent with a diagnosis of gMG, and not otherwise explained by another condition.
• Myasthenia Gravis Foundation of America Clinical Classification Class II, III, or IV at the time of screening.
• Quantitative Myasthenia Gravis score of 11 or greater at screening.
• Participants may enter the study on:

• Corticosteroids only, with no dose increase within 4 weeks prior to screening, or
• One allowed non-steroidal immunosuppressive therapies (IST) (azathioprine, mycophenolate mofetil, or mycophenolic acid) with continuous use for at least 6 months prior to screening and no dose increase within 4 months prior to screening, or
• Combination of (1) corticosteroids with no dose increase within 4 weeks prior to screening and (2) one allowed non-steroidal IST with continuous use for at least 6 months prior to screening and no dose increase within 4 months prior to screening.
• Participants may enter the study on a stable dose of acetylcholinesterase inhibitors (pyridostigmine dose). The acetylcholinesterase inhibitor dose must have been stable for at least 2 weeks prior to enrollment.
• Vital signs and laboratory parameters within the normal ranges at screening, or, if outside normal ranges, deemed not clinically significant by the investigator.

Exclusion Criteria

• Thymectomy within 12 months prior to baseline (Day 1) visit or planned thymectomy during the duration of the treatment period.
• Unresected thymoma- Participants with benign thymoma resected > 12 months prior to screening may enroll.
• History of recurrent significant infections.
• Known immunodeficiency disorder, including current infection or positive test for human immunodeficiency virus (HIV).
• Positive test for chronic hepatitis B infection at screening.
• History of untreated hepatitis C infection, or positive antibody test for hepatitis C virus (HCV).
• History of active or latent tuberculosis (TB), or a positive QuantiFERON®-TB Gold test at screening, unless treatment for TB was completed per local guidelines.
• Receipt of any biologic B-cell-depleting therapy (eg, rituximab, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab) or any experimental B-cell-depleting agent in the 6 months prior to screening.
• Receipt of any other monoclonal antibody (mAb) or large molecule biologic, including but not limited to FcRn inhibitors, anti-TNF mAbs, anti-janus kinase (JAK) Stat mAbs, and complement inhibitors within 6 months prior to screening.
• Participants who are pregnant or breastfeeding or planning to get pregnant.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amgen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

MD

Role: STUDY_DIRECTOR

Amgen

Locations

Austin Neuromuscular Center

Austin, Texas, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Amgen Call Center

Role: CONTACT

medinfo@amgen.com

866-572-6436

Related Links

http://www.amgentrials.com

AmgenTrials clinical trials website

Other Identifiers

2025-520993-20

Identifier Type: OTHER

Identifier Source: secondary_id

20240236

Identifier Type: -

Identifier Source: org_study_id