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

NCT06471361 | Completed Phase 3 Results FDA Drug

• 3 other identifiers: DV0013U1111-1302-43692023-508287-30
• Study Type: interventional
• Target: 31
• Locations: 3 countries
• Sites: 11

Brief Summary

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

Conditions

Generalized Myasthenia Gravis

Keywords

gMG

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• Percentage of Effective Self-administration of Zilucoplan Using ZLP-AI at Visit 1

  Visit 1 (Day 1)

• Percentage of Effective Self-administrations of Zilucoplan Using ZLP-AI at Visit 8

  Visit 8 (Day 14)

• 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)

• 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)

• 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)

Study Arms (1)

Zilucoplan-auto-injector (ZLP-AI)

EXPERIMENTAL

Study participants will self-administer zilucoplan (ZLP) based on their body weight using auto-injector (AI).

Drug: Zilucoplan

Interventions

Zilucoplan DRUG

Zilucoplan will be self-administered subcutaneously by study participants at pre-specified time points.

Also known as: RA101495

Zilucoplan-auto-injector (ZLP-AI)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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.
• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• UCB Biopharma SRL: lead

Study Sites (11)

Dv0013 50628

New Haven, Connecticut, 06511, United States

Location

Dv0013 50634

Tampa, Florida, 33612, United States

Location

Dv0013 50648

Columbia, Missouri, 64212, United States

Location

Dv0013 50556

Chapel Hill, North Carolina, 27599, United States

Location

Dv0013 50635

Columbus, Ohio, 43221, United States

Location

Dv0013 50555

Austin, Texas, 78759, United States

Location

Dv0013 50636

Greenfield, Wisconsin, 53228, United States

Location

Dv0013 40609

Katowice, Poland

Location

Dv0013 40759

Krakow, Poland

Location

Dv0013 40605

Poznan, Poland

Location

Dv0013 40760

Oxford, United Kingdom

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

zilucoplan

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: UCB
• Organization: Cares

UCBCares@ucb.com

001 844 599 2273

Study Officials

• UCB Cares

  001 844 599 2273

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 18, 2024
• First Posted: June 24, 2024
• Study Start: August 27, 2024
• Primary Completion: January 28, 2025
• Study Completion: February 3, 2025
• Last Updated: March 27, 2026
• Results First Posted: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• 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.
• 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.

Locations

PL (3); GB (1); US (7)