A study to learn if a Zilucoplan auto-injector is safe and works in adults with myasthenia gravis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

UCB Biopharma

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

29 Aug 2024 → 3 Feb 2025

Decision date (initial)

2024-07-15

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2023-508287-30-00

WHO UTN

U1111-1302-4369

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Efficacy, Pharmacokinetic, Safety

To evaluate the effectiveness of zilucoplan-auto-injector (ZLP AI) self administration.

Secondary objectives 1

1. To evaluate the effectiveness of zilucoplan-auto-injector (ZLP AI) self administration using additional effectiveness endpoints. Evaluate the safety and tolerability of the ZLP AI self administrations.

Conditions and MedDRA coding

Generalized Myasthenia Gravis

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share 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.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. 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).
2. Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG), based on study participant's history and supported by previous evaluations.
3. Study participant is currently participating in zilucoplan (ZLP) study MG0011 or is administering commercial ZLP on a stable dosing regimen for at least 1month prior to Screening.
4. Study participants on commercial ZLP need to receive ZLP per the approved local labeling.
5. 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.
6. 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.
7. 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.
8. Female participants of childbearing potential must have a negative urine pregnancy test prior to the first dose of study drug.
9. 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.
10. Capable of giving signed informed which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.

Exclusion criteria 8

1. 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.
2. Female participants who are breastfeeding, pregnant, or plan to become pregnant during the study.
3. Study participant has a known hypersensitivity to any components of the study medication (and/or an investigational device) as stated in this protocol.
4. 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.
5. Study participant has a history of meningococcal disease.
6. 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 MG0009, MG0010, or MG0011, which are not excluded, unless the participant was required to withdraw from said studies for a safety reason which could reasonably recur).
7. 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).
8. 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).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Effective self administrations of zilucoplan using the zilucoplan-auto-injector (ZLP-AI) from Visit 1 to Visit 8.

Secondary endpoints 1

1. - Effective self administration of zilucoplan using ZLP-AI at Visit 8 - Effective self administration of zilucoplan using ZLP-AI at Visit 1 - Occurrence of serious adverse events (SAEs) during the course of the study - Occurrence of treatment-emergent adverse events (TEAEs) during the course of the study - Occurrence of nonserious adverse device effects (ADE) during the course of the study - Occurrence of serious adverse device effects (SADE) during the course of the study

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

UCB Biopharma

Sponsor organisation

UCB Biopharma

Address

Researchdreef 60

City

Anderlecht

Postcode

1070

Country

Belgium

Scientific contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Public contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Third parties 9

Locations

1 EU/EEA country · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 4 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications