A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis
NCT Number: NCT06471361
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE3
Conditions: Generalized Myasthenia Gravis
Interventions: DRUG: Zilucoplan
Sponsor: UCB Biopharma SRL
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 25
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-07-15
Completion Date: 2024-12-23

Summary:
The purpose of this study is to evaluate the effectiveness, safety and tolerability of zilucoplan auto-injector (ZLP-AI) self-administration.

Primary Outcome Measures: Percentage of effective self-administrations of zilucoplan using the zilucoplan-auto-injector (ZLP-AI) from Visit 1 to Visit 8, Effective self-administration is defined as: Complete dose delivery: completeness of the delivery as confirmed by the Investigator. The entire dose of investigational medicinal product (IMP) should be completely delivered (that is, the yellow plunger that can be seen through the device window should be completely depressed)., Visit 1 (Day 1) to Visit 8 (Day 14)
Secondary Outcome Measures: Percentage of effective self administrations of zilucoplan using ZLP-AI at Visit 8, Effective self-administration is defined as: Complete dose delivery: completeness of the delivery as confirmed by the Investigator. The entire dose of IMP should be completely delivered (that is, the yellow plunger that can be seen through the device window should be completely depressed)., Visit 8 (Day 14)|Percentage of effective self administration of zilucoplan using ZLP-AI at Visit 1, Effective self-administration is defined as: Complete dose delivery: completeness of the delivery as confirmed by the Investigator. The entire dose of IMP should be completely delivered (that is, the yellow plunger that can be seen through the device window should be completely depressed)., Visit 1 (Day 1)|Occurrence of serious adverse events (SAEs) during the course of the study, A SAE is defined as any untoward medical occurrence that, at any dose: a. Results in death; b. Is life-threatening; c. Requires inpatient hospitalization or prolongation of existing hospitalization; d. Results in persistent disability/incapacity; e. Is a congenital anomaly/birth defect; f. Important medical events., From Visit 1 (Day 1) up to the Safety Follow-up Visit (up to Day 21)|Occurrence of treatment-emergent adverse events (TEAEs) during the course of the study, Treatment-emergent adverse events (TEAEs) are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment., From Visit 1 (Day 1) up to the Safety Follow-up Visit (up to Day 21)|Occurrence of nonserious adverse device effects (ADE) during the course of the study, An ADE is defined as an adverse event related to the use of an investigational medical device. This definition includes any adverse events resulting from insufficient or inadequate instructions for use, deployment, implantation, installation, or operation, or any malfunction of the investigational medical device as well as any event resulting from use error or from intentional misuse of the investigational medical device., From Visit 1 (Day 1) up to the Safety Follow-up Visit (up to Day 21)|Occurrence of serious adverse device effects (SADE) during the course of the study, A SADE is defined as an adverse device effect that has resulted in any of the consequences characteristic of a serious adverse event. A SAE is defined as any untoward medical occurrence that, at any dose: a. Results in death; b. Is life-threatening; c. Requires inpatient hospitalization or prolongation of existing hospitalization; d. Results in persistent disability/incapacity; e. Is a congenital anomaly/birth defect; f. Important medical events., From Visit 1 (Day 1) up to the Safety Follow-up Visit (up to Day 21)