Generalized Myasthenia Gravis
Conditions
Keywords
gMG
Brief summary
The purpose of this study is to evaluate the effectiveness, safety and tolerability of zilucoplan auto-injector (ZLP-AI) self-administration.
Interventions
DRUGZilucoplan
Zilucoplan will be self-administered subcutaneously by study participants at pre-specified time points.
Sponsors
UCB Biopharma SRL
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Study participant is male or female and must be at least 18 years of age at the time of signing the informed consent form (ICF). * Study participant must have a documented diagnosis of gMG, based on study participant's history and supported by previous evaluations. * Study participant is currently participating in ZLP (zilucoplan) study RA101495-02.302 (NCT04225871) or is administering commercial ZLP on a stable dosing regimen for at least 1 month prior to Screening. * Study participants on commercial ZLP need to receive ZLP per the approved local labeling. * Study participant is considered reliable and capable of adhering to the study protocol (eg, able to understand and complete questionnaires and able to adhere to the visit schedule) according to the judgement of the Investigator. * Study participant is willing and capable of self-administering ZLP using the zilucoplan-auto-injector (ZLP AI) according to the instructions for use (IFU), ie, does not have any visual, physical, or other disability or impairment that interferes with his/her capacity to self-administer; if the participant has a caregiver, he/she may assist the participant with the injection. * Vaccination with a quadrivalent meningococcal vaccine and, where available, meningococcal serotype B vaccine at least 14 days prior to investigational medicinal product (IMP) administration, if not vaccinated within 3 years prior to the start of treatment. Booster vaccination(s) should also be administered as clinically indicated, according to the local standard of care, for participants who have been previously vaccinated against Neisseria meningitidis. * Female participants of childbearing potential must have a negative urine pregnancy test prior to the first dose of study drug. * Male and/or female study participants 1. A male participant must agree to use contraception during the Treatment Period and for 40 days after the last dose of study medication, and refrain from donating sperm during this period. 2. A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least 1 of the following conditions applies: * Not a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance the Treatment Period and for 40 days after the last dose of study medication. * Capable of giving signed informed which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
Exclusion criteria
* Study participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the study participant's ability to participate in this study. * Female participants who are breastfeeding, pregnant, or plan to become pregnant during the study. * Study participant has a known hypersensitivity to any components of the study medication (and/or an investigational device) as stated in this protocol. * Study participant has a clinically relevant active infection or a history of serious infection (resulting in hospitalization or requiring intravenous antibiotic treatment) within 6 weeks before Visit 1. * Study participant has a history of meningococcal disease. * Participant has previously participated in this study or participant has previously been assigned to treatment in a study of the medication under investigation in this study (except studies RA101495-02.201 (NCT03315130), RA101495-02.301 (NCT04115293), or RA101495-02.302 (NCT04225871), which are not excluded, unless the participant was required to withdraw from said studies for a safety reason which could reasonably recur). * Participant has participated in another study of an IMP (and/or an investigational device) different from ZLP within the previous 3 months or 5 half-lives, whichever is longer, or is currently participating in another study of an IMP (and/or an investigational device). * Current unstable liver or biliary disease at Screening (Visit 1), per Investigator assessment, defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. NOTE: with exception of stable hepatobiliary conditions (including Gilbert's syndrome, asymptomatic gallstones).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Effective Self-administrations (SA) of Zilucoplan (ZLP) Using the Zilucoplan-auto-injector (ZLP-AI) From Visit 1 to Visit 8 | From Visit 1 (Day 1) to Visit 8 (Day 14) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Effective Self-administration of Zilucoplan Using ZLP-AI at Visit 1 | Visit 1 (Day 1) | 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. |
| Percentage of Effective Self-administrations of Zilucoplan Using ZLP-AI at Visit 8 | Visit 8 (Day 14) | 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. |
| Percentage of Participants With Serious Adverse Events (SAEs) During the Course of the Study | 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) | 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. |
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Course of the Study | 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) | 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). |
| Percentage of Participants With Non-serious Adverse Device Effects (ADE) During the Course of the Study | 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) | 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. |
| Percentage of Participants With Serious Adverse Device Effects (SADE) During the Course of the Study | 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) | 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. |
Countries
Poland, United Kingdom, United States
Contacts
STUDY_DIRECTORUCB Cares
001 844 599 2273
Participant flow
Recruitment details
The study started to enroll participants in August 2024 and concluded in February 2025.
Pre-assignment details
The Participant Flow refers to the Enrolled Set.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 61.4 years STANDARD_DEVIATION 11.7 |
| Age, Customized 18 - <65 years | 15 Participants |
| Age, Customized 65 - <85 | 16 Participants |
| Age, Customized >=85 years | 0 Participants |
| Race/Ethnicity, Customized Asian | 1 Participants |
| Race/Ethnicity, Customized Black or African American | 3 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 1 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 30 Participants |
| Race/Ethnicity, Customized Other or Mixed | 2 Participants |
| Race/Ethnicity, Customized White | 25 Participants |
| Sex: Female, Male Female | 12 Participants |
| Sex: Female, Male Male | 19 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 31 |
| other Total, other adverse events | 3 / 31 |
| serious Total, serious adverse events | 0 / 31 |
Outcome results
None listed