INEBILIZUMAB for Generalized myasthenia gravis

Inclusion criteria

• {"criterion_text":"- Age ≥ 2 to < 18 years of age on the day of enrollment.\n- \"Diagnosis of gMG defined as: Positive serologic test for anti-AChR or anti-MuSK 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.\"\n- Myasthenia Gravis Foundation of America Clinical Classification Class II, III, or IV at the time of screening.\n- Quantitative Myasthenia Gravis score of 11 or greater at screening.\n- \"Participants may enter the study on: (1) Corticosteroids only, with no dose increase within 4 weeks prior to screening, or (2) 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.\"\n- \"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.\""}

Exclusion criteria

• {"criterion_text":"- Thymectomy within 12 months prior to baseline (day 1) visit or planned thymectomy during the duration of the treatment period.\n- \"Unresected thymoma. Note: Participants with benign thymoma resected > 12 months prior to screening may enroll. Benign is defined as no known metastases and no extension into or beyond the capsule on pathological examination. Imaging to evaluate for thymoma must have been performed prior to screening per standard of care.\"\n- Hospitalization for any reason < 30 days prior to screening.\n- Current or recent gMG exacerbation that has not returned to baseline/resolved within at least 30 days prior to screening.\n- History of recurrent significant infections (eg, requiring hospitalization or IV antibiotics).\n- \"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.\"\n- \"Receipt of any other mAb or large molecule biologic, including but not limited to FcRn inhibitors, anti-TNF mAbs, anti-JAK Stat mAbs, and complement inhibitors within 6 months prior to screening.\"\n- Receipt within the 4 weeks prior to screening: Live attenuated vaccine (administration of inactivated [killed] vaccine is acceptable); Blood transfusion\n- \"Participants of childbearing potential unwilling to use protocol-specified method of contraception see (Section 11.5) during treatment and for an additional 6 months after the last dose of investigational product.\""}

Primary endpoints

• {"endpoint_text":"- \"Pharmacokinetic parameters, including maximum observed concentration (Cmax), area under the concentration-time curve (AUC), halflife (t1/2), clearance (CL) and volume of distribution at steady state (Vss)\"","definition_or_measurement_approach":"Pharmacokinetic parameters measured from concentration‑time data (Cmax, AUC, t1/2, CL, Vss) as listed in the endpoint."}
• {"endpoint_text":"- Change from baseline in cluster of differentiation 20 (CD20)+ B-cell counts","definition_or_measurement_approach":"Change from baseline in CD20+ B-cell counts (as stated)."}
• {"endpoint_text":"- \"Incidence of treatment-emergent adverse events, treatment-emergent serious adverse events, adverse events of interest\"","definition_or_measurement_approach":"Incidence (frequency) of treatment-emergent adverse events, serious adverse events and AEs of interest as captured during treatment."}
• {"endpoint_text":"- Changes in laboratory parameters","definition_or_measurement_approach":"Laboratory parameter changes from baseline as monitored in scheduled assessments."}
• {"endpoint_text":"- Changes in vital signs","definition_or_measurement_approach":"Changes from baseline in vital signs measured during scheduled assessments."}

Secondary endpoints

• {"endpoint_text":"- Change in Quantitative Myasthenia Gravis (QMG) score","definition_or_measurement_approach":"Change from baseline in QMG score as specified."}
• {"endpoint_text":"- Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) score","definition_or_measurement_approach":"Change from baseline in MG-ADL score as specified."}
• {"endpoint_text":"- Presence of anti-drug antibodies (ADAs)","definition_or_measurement_approach":"Assessment of presence of anti-drug antibodies (ADAs) as specified."}

Methods

• Recruitment arrangements documents filed (document titles include 'K1 Recruitment arrangements For Publication' and variants) indicating planned formal recruitment procedures.
• Physician-facing referral letters (document: 'K2_Recruitment material_Referral letter_Physician Facing_For Publication' and 'K2_Recruitment material Dr to Dr Letter_For Publication') — channel: physician-to-physician referral; target audience: treating physicians/clinicians.
• GP/Primary care letter approach (document titles include 'Other subject information material GP letter FP' and 'GP letter' variants) — channel: GP letters; target audience: general practitioners to refer eligible pediatric patients.

Italy

Earliest CTIS Part Ii Submission Date

03-11-2025

Latest Decision Or Authorization Date

10-02-2026

Processing Time Days

99

Number Of Sites

2

Number Of Participants

1

Poland

Earliest CTIS Part Ii Submission Date

09-01-2026

Latest Decision Or Authorization Date

09-02-2026

Processing Time Days

31

Number Of Sites

2

Number Of Participants

1

France

Earliest CTIS Part Ii Submission Date

26-11-2025

Latest Decision Or Authorization Date

10-02-2026

Processing Time Days

76

Number Of Sites

1

Number Of Participants

1

Spain

Earliest CTIS Part Ii Submission Date

13-01-2026

Latest Decision Or Authorization Date

11-02-2026

Processing Time Days

29

Number Of Sites

2

Number Of Participants

1

Primary sponsor

Full Name

Amgen Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

PPD Development LP

Responsibilities

[4]

Name

Almac Clinical Technologies LLC

Responsibilities

[3]

Name

Q Squared Solutions Limited

Responsibilities

[4]

Name

Labcorp Central Laboratory Services SARL

Responsibilities

[4]

Third parties

• {"country":"United States","full_name":"Mayo Collaborative Services LLC","duties_or_roles":"[4]","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Veeva Systems Inc.","duties_or_roles":"[6,7,8]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"France","full_name":"Cerba","duties_or_roles":"[4]","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"[4]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"[4]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"[4]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"[3]","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Kayentis","duties_or_roles":"[7]","organisation_type":"Non-Pharmaceutical company"}