An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

Condition(s) studied

Investigational drug(s) / intervention(s)

Zilucoplan: Zilucoplan will be administered subcutaneously to pediatric study participants

Study summary

The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants

Eligibility

Primary outcome measure(s)

• Occurence of treatment emergent adverse events during the course of the study — Baseline (Day 1) to Safety Follow-up (up to Week 60)
  An adverse event (AE) is any untoward medical occurence in a patient or clinical investigation where the study participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
• Occurence of treatment-emergent serious adverse events (TESAEs) — Baseline (Day 1) to Safety Follow-up (up to Week 60)
  A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: Results in death Is life-threatening Requires inpatient hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical event.
• Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product — Baseline (Day 1) to Safety Follow-up (up to Week 60)
  An adverse event (AE) is any untoward medical occurence in a participant or clinical investigation administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.
• Occurence of treatment-emergent infections — Baseline (Day 1) to Safety Follow-up (up to Week 60)
  Percentage of participants who experienced treatment-emergent infections as adverse events. An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment.

Trial sites (6)