Inebilizumab in children with generalized myasthenia gravis: drug handling and tolerability
A Phase 2 Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children From 2 Years to Less Than 18 Years of Age With Generalized Myasthenia Gravis (gMG)
This trial will test inebilizumab in children aged 2 to 17 with generalized myasthenia gravis to see how the drug is processed in the body and whether it is safe and well tolerated.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 2 Years to 17 Years |
| Sex | All |
| Sponsor | Amgen Industry-sponsored |
| Drugs / interventions | rituximab, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab, alemtuzumab, natalizumab |
| Locations | 1 site (Austin, Texas) |
| Trial ID | NCT06987539 on ClinicalTrials.gov |
What this trial studies
This Phase 2 interventional trial enrolls children aged 2 to <18 with seropositive generalized myasthenia gravis to characterize the pharmacokinetics and pharmacodynamics of inebilizumab and to monitor safety and tolerability. Participants will receive inebilizumab and undergo blood sampling for PK/PD analyses, regular safety monitoring, and neuromuscular assessments per protocol. Eligibility requires positive anti‑AChR or anti‑MuSK antibodies and a compatible diagnostic history, with guardian consent and child assent as appropriate. The study is sponsored by Amgen and conducted at the Austin Neuromuscular Center with scheduled clinic visits for dosing and follow-up.
Who should consider this trial
Good fit: Ideal participants are children aged 2–17 with generalized myasthenia gravis who test positive for anti‑AChR or anti‑MuSK antibodies, have supporting diagnostic history, and can attend study visits with guardian consent and child assent.
Not a fit: Patients under 2 years or 18 and older, those who are seronegative for anti‑AChR and anti‑MuSK antibodies, or those with medical issues that make participation unsafe are unlikely to benefit from this trial.
Why it matters
Potential benefit: If successful, the trial could define safe pediatric dosing and enable use of inebilizumab to treat children with gMG.
How similar studies have performed: Inebilizumab and other B‑cell–depleting therapies have shown benefit in related antibody‑mediated disorders in adults, but use in pediatric gMG is largely untested.
Eligibility criteria
Show full inclusion / exclusion 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.
* 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.
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).
* 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.
Where this trial is running
Austin, Texas
- Austin Neuromuscular Center — Austin, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Amgen Call Center
- Email: medinfo@amgen.com
- Phone: 866-572-6436
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.