Recruiting
Phase 2

Inebilizumab

Sponsor:

Amgen

Code:

NCT06987539

Conditions

Generalized Myasthenia Gravis

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

Interventions

Inebilizumab

Study Details

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 ID

NCT06987539

Start date

Jul 21, 2026

Status verified date

Aug, 2026

Completion date

Mar 13, 2030

Anticipated

Primary completion date

Mar 13, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

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.
  • 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.

Note:

The maximum allowed dose of prednisone at the time of enrollment will be 40 mg/day or 80 mg every other day, or equivalent corticosteroid dose.

Tacrolimus is allowed in Japan only, with continued use for ≥ 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

  • 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).
  • Active tuberculosis (TB); latent TB with no documented history of adequate treatment per local standard of care; or a positive QuantiFERON®-TB 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 enrollment: alemtuzumab, total lymphoid irradiation, bone marrow transplant, T-cell vaccination therapy, natalizumab.
  • Participants who are pregnant or breastfeeding or planning to get pregnant.
  • Receipt of intravenous immunoglobulin (IVIg) or subcutaneous immunoglobulin (SCIg) within 4 weeks prior to enrollment.

Study Design

Enrollment

15 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Inebilizumab

Inebilizumab will be administered intravenously (IV).

Interventions

Inebilizumab

Inebilizumab will be administered IV.

Primary outcome measure

  • Maximum Observed Concentration (Cmax) of Inebilizumab [ Time Frame: Up to Week 52 ]
  • Area Under the Concentration-time Curve (AUC) of Inebilizumab [ Time Frame: Up to Week 52 ]
  • Half-life (t1/2) of Inebilizumab [ Time Frame: Up to Week 52 ]
  • Clearance (CL) of Inebilizumab [ Time Frame: Up to Week 52 ]
  • Volume of Distribution at Steady State (Vss) of Inebilizumab [ Time Frame: Up to Week 52 ]
  • Change from Baseline in Cluster of Differentiation 20 (CD20)+ B-cell Counts [ Time Frame: Baseline and Week 78 ]
  • Number of Participants Experiencing Treatment-emergent Adverse Events [ Time Frame: Up to Week 78 ]
  • Number of Participants Experiencing Clinically Significant Changes in Laboratory Parameters [ Time Frame: Up to Week 78 ]
  • Number of Participants Experiencing Clinically Significant Changes in Vital Signs [ Time Frame: Up to Week 78 ]

Central Contacts and Locations

Central contacts

Locations

The Childrens Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Austin Neuromuscular Center

Recruiting

Austin, Texas, United States, 78759

More Information

Sponsor

Amgen

Last update posted

Sep 1, 2026

Last verified

Aug, 2026

Keywords

  • Generalized Myasthenia Gravis
  • Inebilizumab
  • AMG 335
  • Uplizna

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Amgen on 2026-09-01.