A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis

A Multicenter, Open-Label, Outpatient Study to Evaluate the Safe And Effective Use of a Zilucoplan Auto-Injector Combination Product for Subcutaneous Self-Administration by Study Participants With Generalized Myasthenia Gravis

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

Study Overview

• Status: Completed
• Conditions: Generalized Myasthenia Gravis
• Intervention / Treatment: Drug: Zilucoplan

• Study Type: Interventional
• Enrollment (Actual): 31
• Phase: Phase 3

Contacts and Locations

Study Locations

• Poland
  • Katowice, Poland
    • Dv0013 40609
  • Krakow, Poland
    • Dv0013 40759
  • Poznan, Poland
    • Dv0013 40605
• United Kingdom
  • Oxford, United Kingdom
    • Dv0013 40760
• United States
  • Connecticut
    • New Haven, Connecticut, United States, 06511
      • Dv0013 50628
  • Florida
    • Tampa, Florida, United States, 33612
      • Dv0013 50634
  • Missouri
    • Columbia, Missouri, United States, 64212
      • Dv0013 50648
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • Dv0013 50556
  • Ohio
    • Columbus, Ohio, United States, 43221
      • Dv0013 50635
  • Texas
    • Austin, Texas, United States, 78759
      • Dv0013 50555
  • Wisconsin
    • Greenfield, Wisconsin, United States, 53228
      • Dv0013 50636

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Zilucoplan-auto-injector (ZLP-AI); Study participants will self-administer zilucoplan (ZLP) based on their body weight using auto-injector (AI).	Drug: Zilucoplan; Zilucoplan will be self-administered subcutaneously by study participants at pre-specified time points.; Other Names: RA101495

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Effective Self-administrations (SA) of Zilucoplan (ZLP) Using the Zilucoplan-auto-injector (ZLP-AI) From Visit 1 to Visit 8	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.	From Visit 1 (Day 1) to Visit 8 (Day 14)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Effective Self-administration of Zilucoplan Using ZLP-AI at Visit 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.	Visit 1 (Day 1)
Percentage of Effective Self-administrations of Zilucoplan Using ZLP-AI at Visit 8	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.	Visit 8 (Day 14)
Percentage of Participants With Serious Adverse Events (SAEs) During the Course of the Study	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.	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)
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Course of the Study	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).	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)
Percentage of Participants With Non-serious Adverse Device Effects (ADE) During the Course of the Study	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.	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)
Percentage of Participants With Serious Adverse Device Effects (SADE) During the Course of the Study	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.	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)

Collaborators and Investigators

• Sponsor: UCB Biopharma SRL
• Investigators: Study Director: UCB Cares, 001 844 599 2273

Study record dates

Study Major Dates

• Study Start (Actual): August 27, 2024
• Primary Completion (Actual): January 28, 2025
• Study Completion (Actual): February 3, 2025

Study Registration Dates

• First Submitted: June 18, 2024
• First Submitted That Met QC Criteria: June 18, 2024
• First Posted (Actual): June 24, 2024

Study Record Updates

• Last Update Posted (Actual): March 27, 2026
• Last Update Submitted That Met QC Criteria: March 25, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: gMG
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis; zilucoplan
• Other Study ID Numbers: DV0013; U1111-1302-4369 (Other Identifier: World Health Organization (WHO)); 2023-508287-30 (Registry Identifier: EU Clinical Trials)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
• IPD Sharing Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• IPD Sharing Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed.All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No