Trial Outcomes & Findings for A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis (NCT NCT06471361)
NCT ID: NCT06471361
Last Updated: 2026-03-27
Results Overview
Effective SA of ZLP was defined as completeness of the delivery as confirmed by the Investigator. The entire dose of investigational medicinal product (IMP) was completely delivered (ie, the yellow plunger that was seen through the device window was completely depressed). The percentage of effective SA overall was summarized based on the number of ZLP-AIs used and returned, within participants in the Safety Set (SS). Complete Dose Delivery = Total number (no.) of ZLP-AIs with complete dose delivery/Total no. of ZLP-AIs used and returned.
COMPLETED
PHASE3
31 participants
From Visit 1 (Day 1) to Visit 8 (Day 14)
2026-03-27
Participant Flow
The study started to enroll participants in August 2024 and concluded in February 2025.
The Participant Flow refers to the Enrolled Set.
Participant milestones
| Measure |
Zilucoplan
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
Overall Study
STARTED
|
31
|
|
Overall Study
COMPLETED
|
31
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis
Baseline characteristics by cohort
| Measure |
Zilucoplan
n=31 Participants
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
Age, Continuous
|
61.4 years
STANDARD_DEVIATION 11.7 • n=56 Participants
|
|
Age, Customized
18 - <65 years
|
15 Participants
n=56 Participants
|
|
Age, Customized
65 - <85
|
16 Participants
n=56 Participants
|
|
Age, Customized
>=85 years
|
0 Participants
n=56 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=56 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=56 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=56 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
3 Participants
n=56 Participants
|
|
Race/Ethnicity, Customized
White
|
25 Participants
n=56 Participants
|
|
Race/Ethnicity, Customized
Other or Mixed
|
2 Participants
n=56 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
1 Participants
n=56 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
30 Participants
n=56 Participants
|
PRIMARY outcome
Timeframe: From Visit 1 (Day 1) to Visit 8 (Day 14)Population: The SS was consisted of all study participants in the study who received at least 1 dose of ZLP with the ZLP-AI combination product. Number of units analyzed were defined as total no. of ZLP-AIs used and returned in clinic.
Effective SA of ZLP was defined as completeness of the delivery as confirmed by the Investigator. The entire dose of investigational medicinal product (IMP) was completely delivered (ie, the yellow plunger that was seen through the device window was completely depressed). The percentage of effective SA overall was summarized based on the number of ZLP-AIs used and returned, within participants in the Safety Set (SS). Complete Dose Delivery = Total number (no.) of ZLP-AIs with complete dose delivery/Total no. of ZLP-AIs used and returned.
Outcome measures
| Measure |
Zilucoplan
n=449 ZLP-AI used and returned in clinic
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
Percentage of Effective Self-administrations (SA) of Zilucoplan (ZLP) Using the Zilucoplan-auto-injector (ZLP-AI) From Visit 1 to Visit 8
|
99.8 Percentage of ZLP-AI devices
Interval 98.8 to 100.0
|
SECONDARY outcome
Timeframe: Visit 1 (Day 1)Population: The SS was consisted of all study participants in the study who received at least 1 dose of ZLP with the ZLP-AI combination product. Number of units analyzed were defined as total no. of ZLP-AIs used and returned in clinic.
Effective SA of ZLP was defined as completeness of the delivery as confirmed by the Investigator. The entire dose of IMP was completely delivered (ie, the yellow plunger that was seen through the device window was completely depressed). The percentage of effective SA overall was summarized based on the number of ZLP-AIs used and returned, within participants in the SS. Complete Dose Delivery = Total number (no.) of ZLP-AIs with complete dose delivery/Total no. of ZLP-AIs used and returned.
Outcome measures
| Measure |
Zilucoplan
n=32 ZLP-AI used and returned in clinic
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
Percentage of Effective Self-administration of Zilucoplan Using ZLP-AI at Visit 1
|
96.9 Percentage of ZLP-AI devices
Interval 83.8 to 99.9
|
SECONDARY outcome
Timeframe: Visit 8 (Day 14)Population: The SS was consisted of all study participants in the study who received at least 1 dose of ZLP with the ZLP-AI combination product. Number of units analyzed were defined as total no. of ZLP-AIs used and returned in clinic.
Effective SA of ZLP was defined as completeness of the delivery as confirmed by the Investigator. The entire dose of IMP was completely delivered (ie, the yellow plunger that was seen through the device window was completely depressed). The percentage of effective SA overall was summarized based on the number of ZLP-AIs used and returned, within participants in the SS. Complete Dose Delivery = Total number (no.) of ZLP-AIs with complete dose delivery/Total no. of ZLP-AIs used and returned.
Outcome measures
| Measure |
Zilucoplan
n=28 ZLP-AI used and returned in clinic
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
Percentage of Effective Self-administrations of Zilucoplan Using ZLP-AI at Visit 8
|
100.0 Percentage of ZLP-AI devices
Interval 87.7 to 100.0
|
SECONDARY outcome
Timeframe: From Visit 1 (Day 1) up to 40 days after last administration of self-injection of ZLP-AI (up to 54 days after Visit 1)Population: The SS was consisted of all study participants in the study who received at least 1 dose of ZLP with the ZLP-AI combination product.
An SAE was defined as any untoward medical occurrence that, at any dose, met 1 or more of the criteria listed: Led to death; Led to a life-threatening illness or injury; Led to a permanent impairment of a body structure or a body function; Led to a inpatient or prolonged hospitalization; Led to a medical or surgical intervention to prevent life-threatening illness or injury or permanent impairment to a body structure or a body function; Led to fetal distress, fetal death, or a congenital abnormality or birth defect.
Outcome measures
| Measure |
Zilucoplan
n=31 Participants
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
Percentage of Participants With Serious Adverse Events (SAEs) During the Course of the Study
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: From Visit 1 (Day 1) up to 40 days after last administration of self-injection of ZLP-AI (up to 54 days after Visit 1)Population: The SS was consisted of all study participants in the study who received at least 1 dose of ZLP with the ZLP-AI combination product.
An AE was defined as any untoward medical occurrence, unintended disease or injury, or untoward clinical signs (including abnormal laboratory finding) in study participants, users, or other persons, whether or not related to the investigational medical device. A TEAE was defined as an AE starting on or after the date/time of the first self-injection of ZLP-AI and up to and including 40 days after the final self-injection of ZLP-AI (or last contact depending on which occurs first).
Outcome measures
| Measure |
Zilucoplan
n=31 Participants
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Course of the Study
|
22.6 percentage of participants
|
SECONDARY outcome
Timeframe: From Visit 1 (Day 1) up to 40 days after last administration of self-injection of ZLP-AI (up to 54 days after Visit 1)Population: The SS was consisted of all study participants in the study who received at least 1 dose of ZLP with the ZLP-AI combination product.
An ADE was defined as an AE related to the use of an investigational medical device included any AEs 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.
Outcome measures
| Measure |
Zilucoplan
n=31 Participants
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
Percentage of Participants With Non-serious Adverse Device Effects (ADE) During the Course of the Study
|
3.2 percentage of participants
|
SECONDARY outcome
Timeframe: From Visit 1 (Day 1) up to 40 days after last administration of self-injection of ZLP-AI (up to 54 days after Visit 1)Population: The SS was consisted of all study participants in the study who received at least 1 dose of ZLP with the ZLP-AI combination product.
An SAE was defined as any untoward medical occurrence that, at any dose, met 1 or more of the criteria listed: Led to death; Led to a life-threatening illness or injury; Led to a permanent impairment of a body structure or a body function; Led to a inpatient or prolonged hospitalization; Led to a medical or surgical intervention to prevent life-threatening illness or injury or permanent impairment to a body structure or a body function; Led to fetal distress, fetal death, or a congenital abnormality or birth defect. A SADE was defined as an adverse device effect that had resulted in any of the consequences characteristic of a SAE.
Outcome measures
| Measure |
Zilucoplan
n=31 Participants
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
Percentage of Participants With Serious Adverse Device Effects (SADE) During the Course of the Study
|
0 percentage of participants
|
Adverse Events
Zilucoplan
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Zilucoplan
n=31 participants at risk
Participants who had Zilucoplan (ZLP) in MG0011 (NCT04225871) or were administering commercial ZLP on a stable dosing regimen were self-administering ZLP subcutaneously using Auto-Injector (AI) once daily as per their body weight (Dose: 16.6 milligram (mg) ZLP for less than 56 kgs (kilograms); 23.0 mg ZLP for 56 to 77 kgs; 32.4 mg for greater than 77 kgs) from Day 1 to 14 during treatment period.
|
|---|---|
|
General disorders
Injection site pain
|
9.7%
3/31 • Number of events 7 • From Visit 1 (Day 1) up to 40 days after last administration of self-injection of ZLP-AI (up to 54 days after Visit 1)
A TEAE was defined as an AE starting on or after the date/time of the first self-injection of ZLP-AI and up to and including 40 days after the final self-injection of ZLP-AI (or last contact depending on which occurs first).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60