Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruiting
Participants planned
10
Countries
2
Sites
3
Generalized Myasthenia Gravis
Assess the long-term safety and tolerability of an additional 52 weeks (±7 days) of treatment with zilucoplan (ZLP) administered by subcutaneous (sc) injection once daily in pediatric study participants
Key facts
- Sponsor
- UCB Biopharma
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 18 Aug 2025 → ongoing
- Decision date (initial)
- 2023-12-14
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2022-502073-42-00
- WHO UTN
- U1111-1290-3806
- ClinicalTrials.gov
- NCT06435312
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Others, Safety, Pharmacokinetic, Pharmacodynamic
Assess the long-term safety and tolerability of an additional 52 weeks (±7 days) of treatment with zilucoplan (ZLP) administered by subcutaneous (sc) injection once daily in pediatric study participants
Secondary objectives 1
- Assess the pharmacokinetics (PK) of an additional 52 weeks (±7 days) of treatment with zilucoplan (ZLP) administered by subcutaneous (sc) injection once daily in pediatric study participants. Assess the pharmacodynamics (PD) of an additional 52 weeks (±7 days) of treatment with ZLP administered by sc injection once daily in pediatric study participants. Evaluate the activity of an additional 52 weeks (±7 days) of treatment with ZLP administered by sc injection once daily in pediatric study participants.
Conditions and MedDRA coding
Generalized Myasthenia Gravis
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | PT | 10028417 | Myasthenia gravis | 100000004852 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Participant has completed 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 4
- Study participant met any mandatory withdrawal or mandatory permanent IMP discontinuation criteria in MG0014 or permanently discontinued investigational medicinal product (IMP)
- Participant has known positive serology for muscle-specific kinase.
- Participant has a known hypersensitivity to any components of the IMP.
- Participant has a prior history of meningococcal disease
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- • Occurrence of treatment-emergent adverse events (TEAEs) during the course of the study • Occurrence of treatment-emergent serious adverse events (TESAEs) • Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP) • Occurrence of treatment-emergent infections
Secondary endpoints 1
- • Plasma concentration of zilucoplan (ZLP) at Week 52 • Sheep red blood cell (sRBC) lysis activity at Week 52 • Complement component 5 (C5) levels at Week 52 • Myasthenia Gravis Activities of Daily Living (MG ADL) score at Week 52 • Quantitative Myasthenia Gravis (QMG) score at Week 52
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 4
PRD10444104 · Product
- Active substance
- Zilucoplan Sodium
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 00 mg/Kg milligram(s)/kilogram
- Max total dose
- 00 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 53 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- UCB BIOPHARMA SRL
- Paediatric formulation
- No
- Orphan designation
- No
Zilbrysq 32.4 mg solution for injection in pre-filled syringe
PRD10984359 · Product
- Active substance
- Zilucoplan
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 53 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AJ06 — -
- Marketing authorisation
- EU/1/23/1764/005
- MA holder
- UCB PHARMA S.A. (ANDERL BE)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Zilbrysq 16.6 mg solution for injection in pre-filled syringe
PRD10982512 · Product
- Active substance
- Zilucoplan
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 53 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AJ06 — -
- Marketing authorisation
- EU/1/23/1764/001
- MA holder
- UCB PHARMA S.A. (ANDERL BE)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Zilbrysq 23 mg solution for injection in pre-filled syringe
PRD10984301 · Product
- Active substance
- Zilucoplan
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 53 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AJ06 — -
- Marketing authorisation
- EU/1/23/1764/003
- MA holder
- UCB PHARMA S.A. (ANDERL BE)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Auxiliary 1
-
J06BA · Product
- Pharmaceutical form
- PHF00230MIG
- Route of administration
- UNKNOWN USE
- Max daily dose
- 00 DF dosage form
- Max total dose
- 00 DF dosage form
- Max treatment duration
- 1 Week(s)
- Authorisation status
- Authorised
- ATC code
- J06BA — IMMUNOGLOBULINS, NORMAL HUMAN
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
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 14
| Organisation | City, country | Duties |
|---|---|---|
| Labcorp Central Laboratory Services LP ORG-100032236
|
Indianapolis, United States | Laboratory analysis |
| RWS Life Sciences Inc. ORG-100042348
|
East Hartford, United States | Other |
| Parexel International (IRL) Limited ORG-100022780
|
Dublin 2, Ireland | On site monitoring, Code 11, Code 12, Code 13, Other, Code 2, Code 5, Data management, Code 8 |
| U C B ORG-100005041
|
Braine-L'alleud, Belgium | Laboratory analysis |
| 4G Clinical B.V. ORG-100044721
|
Amsterdam, Netherlands | Interactive response technologies (IRT) |
| IQVIA Laboratories LLC ORG-100043195
|
Durham, United States | Laboratory analysis |
| Drug Development Solutions Limited ORG-100045894
|
Ely, United Kingdom | Laboratory analysis |
| CluePoints SA ORL-000000619
|
Belgium | Other |
| Certara USA Inc. ORG-100042611
|
Princeton, United States | Other |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| Azenta Germany GmbH ORG-100022621
|
Griesheim, Germany | Other |
| Advarra Inc. ORG-100045827
|
Columbia, United States | Other |
| Center For Information And Study On Clinical Research Participation Inc. ORG-100044581
|
Boston, United States | Code 11 |
| Syner-G Biopharma Group LLC ORG-100050443
|
Framingham, United States | Code 11 |
Locations
2 EU/EEA countries · 3 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Italy | Authorised, recruitment pending | 1 | 1 |
| Poland | Ongoing, recruiting | 3 | 2 |
| Rest of world
Korea, Republic of, United Kingdom, United States
|
— | 6 | — |
Investigational sites
IRCCS Foundation Istituto Neurologico Carlo Besta
#40144: Dipartimento Neuroscienze PediatricheSC Neuropsichiatria Infantile – Disordini del Movimento, Via Giovanni Celoria 11, 20133, Milan
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Poland | 2025-08-18 | 2025-08-18 | |||
| Italy |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 46 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D2_mg0015-protocol-amend-public-1.0 | N/A |
| Protocol (for publication) | IT-Diary Drug Administration-dayE1-it-IT-public | 1.0 |
| Protocol (for publication) | IT-Diary Drug Administration-week12-it-IT-public | 1.0 |
| Protocol (for publication) | IT-Diary Drug Administration-week24-it-IT-public | 1.0 |
| Protocol (for publication) | IT-Diary Drug Administration-week36-it-IT-public | 1.0 |
| Protocol (for publication) | IT-Diary Drug Administration-week44-it-IT-public | 1.0 |
| Protocol (for publication) | IT-PL-Diary Drug Administration-dayE1-public | 1.0 |
| Protocol (for publication) | IT-PL-Diary Drug Administration-week12-public | 1.0 |
| Protocol (for publication) | IT-PL-Diary Drug Administration-week24-public | 1.0 |
| Protocol (for publication) | IT-PL-Diary Drug Administration-week36-public | 1.0 |
| Protocol (for publication) | IT-PL-Diary Drug Administration-week44-public | 1.0 |
| Protocol (for publication) | IT-PL-quest-C-SSRS-Since-Last-Visit-public | 5.1 |
| Protocol (for publication) | IT-PL-questionnaire-MG-ADL-public | 1.1 |
| Protocol (for publication) | IT-PL-questionnaire-Neuro-QoL-Pediatric-public | 2.1 |
| Protocol (for publication) | IT-PL-questionnaire-PedsQL-Acute-Ages13-18-public | 4.0 |
| Protocol (for publication) | IT-PL-questionnaire-PedsQL-Acute-Ages5-7-public | 4.0 |
| Protocol (for publication) | IT-PL-questionnaire-PedsQL-Acute-Ages8-12-public | 4.0 |
| Protocol (for publication) | IT-quest-C-SSRS-Since-Last-Visit-it-IT-public | 5.1 |
| Protocol (for publication) | IT-quest-Neuro-QoL-Ped-Fatigue-public-it-IT | 2.1 |
| Protocol (for publication) | IT-quest-PedsQL-Acute-Ages13-18-it-IT-public | 4.0 |
| Protocol (for publication) | IT-quest-PedsQL-Acute-Ages5-7-it-IT-public | 4.0 |
| Protocol (for publication) | IT-quest-PedsQL-Acute-Ages8-12-it-IT-public | 4.0 |
| Protocol (for publication) | IT-questionnaire-MG-ADL-public-it-IT | 1.1 |
| Protocol (for publication) | mg0015-protocol-public | n/a |
| Recruitment arrangements (for publication) | ITA Recruitment Flyer Italian MG0015 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Procedure Description English MG0015 Public | 1.0 |
| Recruitment arrangements (for publication) | POL Recruitment Other Polish MG0015 Public | 1.0 |
| Recruitment arrangements (for publication) | POL Recruitment Procedure Description Polish MG0015 Public | 2.0 |
| Subject information and informed consent form (for publication) | mg0015-it-icf-adult-it-IT-public | 2.0 |
| Subject information and informed consent form (for publication) | mg0015-it-icf-adultlja-it-IT-public | 1.0 |
| Subject information and informed consent form (for publication) | mg0015-it-icf-assent form 12-17-it-public | 1.2 |
| Subject information and informed consent form (for publication) | mg0015-it-icf-assent form 6-11-it-public | 1.1 |
| Subject information and informed consent form (for publication) | mg0015-it-icf-future research-it-public | 1.2 |
| Subject information and informed consent form (for publication) | mg0015-it-icf-handling of pd_confidentiality-it-pub | 1.1 |
| Subject information and informed consent form (for publication) | mg0015-it-icf-par-lar-it-IT-public | 2.0 |
| Subject information and informed consent form (for publication) | mg0015-it-icf-parlja-it-IT-public | 1.0 |
| Subject information and informed consent form (for publication) | mg0015-it-icf-pp-it-public | 1.1 |
| Subject information and informed consent form (for publication) | mg0015-pl-icf-13-17-18lja-pl-PL-public | 1.0 |
| Subject information and informed consent form (for publication) | mg0015-pl-icf-ass13-17-and18-pl-PL-public | 2.0 |
| Subject information and informed consent form (for publication) | mg0015-pl-icf-par-pl-PL-public | 2.0 |
| Subject information and informed consent form (for publication) | mg0015-pl-icf-parlja-pl-PL-public | 1.0 |
| Subject information and informed consent form (for publication) | mg0015-pl-icf-pp-en-public | 1.0 |
| Subject information and informed consent form (for publication) | POL Country ICF Procedure Polish MG0015 Public | 1.0 |
| Synopsis of the protocol (for publication) | mg0015-laypersons-protocol-synopsis-it-IT-public | 2.0 |
| Synopsis of the protocol (for publication) | mg0015-laypersons-protocol-synopsis-pl-PL-public | 2.0 |
| Synopsis of the protocol (for publication) | mg0015-laypersons-protocol-synopsis-public | 2.0 |
Application history
11 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-08-18 | Italy | Acceptable 2023-12-11
|
2023-12-14 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-01-29 | Italy | Acceptable 2023-12-11
|
2024-01-29 |
| 3 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-02-23 | Italy | Acceptable 2024-04-09
|
2024-04-10 |
| 4 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-09-04 | Acceptable | 2024-10-09 | |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2024-12-06 | Italy | Acceptable | 2024-12-06 |
| 6 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-02-11 | Italy | Acceptable 2025-05-05
|
2025-05-12 |
| 7 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-05-19 | Italy | Acceptable 2025-05-05
|
2025-05-19 |
| 8 | NON SUBSTANTIAL MODIFICATION | NSM-4 | 2025-05-19 | Acceptable 2025-05-05
|
2025-05-19 | |
| 9 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-08-13 | Italy | Acceptable 2025-11-19
|
2025-11-21 |
| 10 | NON SUBSTANTIAL MODIFICATION | NSM-5 | 2025-12-17 | Italy | Acceptable 2025-11-19
|
2025-12-17 |
| 11 | SUBSTANTIAL MODIFICATION | SM-5 | 2026-02-26 | Italy | Acceptable 2026-04-13
|
2026-04-13 |