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)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 1
- 试验地点
- 2
- 主要终点
- "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)"
研究概览
简要总结
To characterize the pharmacokinetics (PK) of inebilizumab administered in pediatric participants with generalized Myasthenia Gravis (gMG). To characterize the pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG. To assess the safety and tolerability of inebilizumab administered in pediatric participants with gMG.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Age ≥ 2 to < 18 years of age on the day of enrollment.
- •"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."
- •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: (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."
- •"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."
排除标准
- •Thymectomy within 12 months prior to baseline (day 1) visit or planned thymectomy during the duration of the treatment period.
- •"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."
- •Hospitalization for any reason < 30 days prior to screening.
- •Current or recent gMG exacerbation that has not returned to baseline/resolved within at least 30 days prior to screening.
- •History of recurrent significant infections (eg, requiring hospitalization or IV antibiotics).
- •"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 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."
- •Receipt within the 4 weeks prior to screening: Live attenuated vaccine (administration of inactivated [killed] vaccine is acceptable); Blood transfusion
- •"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."
结局指标
主要结局
"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)"
"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)"
Change from baseline in cluster of differentiation 20 (CD20)+ B-cell counts
Change from baseline in cluster of differentiation 20 (CD20)+ B-cell counts
"Incidence of treatment-emergent adverse events, treatment-emergent serious adverse events, adverse events of interest"
"Incidence of treatment-emergent adverse events, treatment-emergent serious adverse events, adverse events of interest"
Changes in laboratory parameters
Changes in laboratory parameters
Changes in vital signs
Changes in vital signs
次要结局
- Change in Quantitative Myasthenia Gravis (QMG) score
- Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) score
- Presence of anti-drug antibodies (ADAs)
研究者
Medical Information
Scientific
Amgen Inc.