## Protocol Section
### Identification Module
**NCT ID:** NCT06435312

**Acronym:** ziMyG+

**Brief Title:** An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

**Official Title:** An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis

#### Organization Study ID Info
**ID:** MG0015
#### Organization
**Class:** INDUSTRY
**Full Name:** UCB Pharma
#### Secondary ID Infos
**Domain:** EU CT Number
**ID:** 2022-502073-42-00
**Type:** OTHER
### Status Module
#### Completion Date
**Date:** 2027-12-07

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-30

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-23

**Overall Status:** NOT_YET_RECRUITING

#### Primary Completion Date
**Date:** 2027-10-26

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-08-13

**Type:** ESTIMATED

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-30
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-23
**Study First Submit QC Date:** 2024-05-23
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** UCB Biopharma SRL

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Is US Export:** False

**Oversight Has DMC:** False

### Description Module

**Brief Summary:** The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants

### Conditions Module

**Conditions:**

- Generalized Myasthenia Gravis

**Keywords:**

- generalized Myasthenia Gravis

- gMG

- RA101495

- Zilucoplan

- pediatric

- MG0015

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 8

**Type:** ESTIMATED

**Phases:**

- PHASE3

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Study participants will receive Zilucoplan at a pre-defined dose based on their weight.

**Intervention Names:**

- Drug: Zilucoplan

**Label:** Zilucoplan Arm

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Zilucoplan Arm

**Description:** Zilucoplan will be administered subcutaneously to pediatric study participants

**Name:** Zilucoplan

**Other Names:**

- RA101495

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** An adverse event (AE) is any untoward medical occurence in a patient or clinical investigation where the study participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

**Measure:** Occurence of treatment emergent adverse events during the course of the study

**Time Frame:** Baseline (Day 1) to Safety Follow-up (up to Week 60)

**Description:** A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose:

Results in death Is life-threatening Requires inpatient hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical event.

**Measure:** Occurence of treatment-emergent serious adverse events (TESAEs)

**Time Frame:** Baseline (Day 1) to Safety Follow-up (up to Week 60)

**Description:** An adverse event (AE) is any untoward medical occurence in a participant or clinical investigation administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.

**Measure:** Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product

**Time Frame:** Baseline (Day 1) to Safety Follow-up (up to Week 60)

**Description:** Percentage of participants who experienced treatment-emergent infections as adverse events.

An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment.

**Measure:** Occurence of treatment-emergent infections

**Time Frame:** Baseline (Day 1) to Safety Follow-up (up to Week 60)

#### Secondary Outcomes

**Description:** Blood samples for the measurement of plasma concentrations of Zilucoplan will be collected at Week 52.

**Measure:** Plasma concentration of Zilucoplan at Week 52

**Time Frame:** Week 52

**Description:** Blood samples for measurement of sRBC lysis will be collected at Week 52.

**Measure:** Sheep red blood cell (sRBC) lysis activity at Week 52

**Time Frame:** Week 52

**Description:** Blood samples for measurement of C5 will be collected at Week 52.

**Measure:** Blood complement component 5 (C5) levels at Week 52

**Time Frame:** Week 52

**Description:** The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability.

**Measure:** Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52

**Time Frame:** Week 52

**Description:** QMG score is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The QMG total score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity.

**Measure:** Quantitative Myasthenia Gravis (QMG) score at Week 52

**Time Frame:** Week 52

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

United States of America (USA) specific inclusion criterion:

- Participant must be ≥ 12 years of age at the time of signing the Informed Consent/Assent according to local regulation.

Rest of World (ROW) specific inclusion criterion:

- Participant must be ≥ 2 years of age at the time of signing the Informed Consent/Assent according to local regulation.

Global specific inclusion criteria:

* Participant has completed the MG0014 according to the protocol, and further treatment with zilucoplan is in the interest of the participant in the investigator´s opinion
* Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care

Exclusion Criteria:

* Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP
* Participant has known positive serology for muscle-specific kinase
* Participant has known hypersensitivity to any components of the IMP
* Participant has a prior history of meningococcal disease

**Minimum Age:** 12 Years

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** ucbcares@ucb.com

**Name:** UCB Cares

**Phone:** +18445992273

**Phone Ext:** (USA)

**Role:** CONTACT

**Contact 2:**

**Name:** UCB Cares

**Phone:** 001 844 599 2273

**Role:** CONTACT

#### Overall Officials

**Official 1:**

**Affiliation:** 001 844 599 2273 (UCB)

**Name:** UCB Cares

**Role:** STUDY_DIRECTOR

### IPD Sharing Statement Module

**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.

**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.

**Info Types:**

- STUDY_PROTOCOL

- SAP

- CSR

**IPD Sharing:** YES

**Time Frame:** Data from this study 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.

**URL:** https://www.Vivli.org

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000009135

- Term: Muscular Diseases

- ID: D000009140

- Term: Musculoskeletal Diseases

- ID: D000020879

- Term: Neuromuscular Manifestations

- ID: D000009461

- Term: Neurologic Manifestations

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000010335

- Term: Pathologic Processes

- ID: D000020361

- Term: Paraneoplastic Syndromes, Nervous System

- ID: D000009423

- Term: Nervous System Neoplasms

- ID: D000009371

- Term: Neoplasms by Site

- ID: D000009369

- Term: Neoplasms

- ID: D000010257

- Term: Paraneoplastic Syndromes

- ID: D000020274

- Term: Autoimmune Diseases of the Nervous System

- ID: D000019636

- Term: Neurodegenerative Diseases

- ID: D000020511

- Term: Neuromuscular Junction Diseases

- ID: D000009468

- Term: Neuromuscular Diseases

- ID: D000001327

- Term: Autoimmune Diseases

- ID: D000007154

- Term: Immune System Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC04

- Name: Neoplasms

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC20

- Name: Immune System Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC05

- Name: Musculoskeletal Diseases

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M12112

- Name: Myasthenia Gravis

- Relevance: HIGH

- As Found: Myasthenia Gravis

- ID: M20944

- Name: Muscle Weakness

- Relevance: HIGH

- As Found: Myasthenia

- ID: M13204

- Name: Paresis

- Relevance: LOW

- As Found: Unknown

- ID: M4554

- Name: Asthenia

- Relevance: LOW

- As Found: Unknown

- ID: M12092

- Name: Muscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12097

- Name: Musculoskeletal Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22619

- Name: Neuromuscular Manifestations

- Relevance: LOW

- As Found: Unknown

- ID: M12404

- Name: Neurologic Manifestations

- Relevance: LOW

- As Found: Unknown

- ID: M16355

- Name: Syndrome

- Relevance: LOW

- As Found: Unknown

- ID: M13170

- Name: Paraneoplastic Syndromes

- Relevance: LOW

- As Found: Unknown

- ID: M22160

- Name: Paraneoplastic Syndromes, Nervous System

- Relevance: LOW

- As Found: Unknown

- ID: M12367

- Name: Nervous System Neoplasms

- Relevance: LOW

- As Found: Unknown

- ID: M4629

- Name: Autoimmune Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22094

- Name: Autoimmune Diseases of the Nervous System

- Relevance: LOW

- As Found: Unknown

- ID: M21558

- Name: Neurodegenerative Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22297

- Name: Neuromuscular Junction Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12411

- Name: Neuromuscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M10200

- Name: Immune System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T3973

- Name: Myasthenia Gravis

- Relevance: HIGH

- As Found: Myasthenia Gravis

### Condition Browse Module - Meshes

- ID: D000009157

- Term: Myasthenia Gravis

- ID: D000018908

- Term: Muscle Weakness

### Intervention Browse Module - Browse Branches

- Abbrev: All

- Name: All Drugs and Chemicals

- Abbrev: VaDiAg

- Name: Vasodilator Agents

### Intervention Browse Module - Browse Leaves

- ID: M4225

- Name: Antibodies

- Relevance: LOW

- As Found: Unknown

- ID: M10184

- Name: Immunoglobulins

- Relevance: LOW

- As Found: Unknown

- ID: M3473

- Name: Acetylcholine

- Relevance: LOW

- As Found: Unknown

### Misc Info Module

- Version Holder: 2024-05-31
