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

NCT06987539 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 202402362025-520993-20
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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

Keywords

Generalized Myasthenia Gravis; Inebilizumab; AMG 335; Uplizna

Outcome Measures

Primary Outcomes (9)

• Maximum Observed Concentration (Cmax) of Inebilizumab

  Up to Week 52

• Area Under the Concentration-time Curve (AUC) of Inebilizumab

  Up to Week 52

• Half-life (t1/2) of Inebilizumab

  Up to Week 52

• Clearance (CL) of Inebilizumab

  Up to Week 52

• Volume of Distribution at Steady State (Vss) of Inebilizumab

  Up to Week 52

• Change from Baseline in Cluster of Differentiation 20 (CD20)+ B-cell Counts

  Baseline and Week 78

• Number of Participants Experiencing Treatment-emergent Adverse Events

  Up to Week 78

• Number of Participants Experiencing Clinically Significant Changes in Laboratory Parameters

  Up to Week 78

• Number of Participants Experiencing Clinically Significant Changes in Vital Signs

  Up to Week 78

Secondary Outcomes (5)

• Change from Baseline in Quantitative Myasthenia Gravis (QMG) Score

  Baseline and Week 78

• Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Score

  Baseline and Week 78

• Change from Baseline in Euro Quality of Life-5 Dimension Youth (EQ-5D-Y) Score

  Baseline and Week 78

• Change from Baseline in Neurological Quality of Life (Neuro-QoL) Paediatric Fatigue Score

  Baseline and Week 78

• Number of Participants with Anti-drug Antibodies (ADAs) Present

  Up to Week 78

Study Arms (1)

Inebilizumab

EXPERIMENTAL

Inebilizumab will be administered intravenously (IV).

Drug: Inebilizumab

Interventions

Inebilizumab DRUG

Inebilizumab will be administered IV.

Inebilizumab

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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 must be 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.

You may not qualify if:

• Employees of the Sponsor, contract research organization (CRO), site staff, and their family members.
• 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.
• History of progressive multifocal leukoencephalopathy.
• Participants diagnosed with congenital myasthenic syndromes.
• 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.
• Receipt of the following medications or treatments at any time prior to randomization: alemtuzumab, total lymphoid irradiation, bone marrow transplant, T-cell vaccination therapy, natalizumab.
• Participants who are pregnant or breastfeeding or planning to get pregnant.

Sponsors & Collaborators

• Amgen: lead

Study Sites (1)

Austin Neuromuscular Center

Austin, Texas, 78759, United States

RECRUITING

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

inebilizumab

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 Officials

• MD

  Amgen

  STUDY DIRECTOR

Central Study Contacts

Amgen Call Center

CONTACT

866-572-6436; medinfo@amgen.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2025
• First Posted: May 23, 2025
• Study Start: April 30, 2026
• Primary Completion (Estimated): March 13, 2030
• Study Completion (Estimated): March 13, 2030
• Last Updated: March 16, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication (or other new use) have been granted marketing authorization in both the US and Europe, or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
• Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.

Locations

US (1)