ZILUCOPLAN for Generalized myasthenia gravis

Inclusion criteria

• {"criterion_text":"- Completion of a qualifying zilucoplan study\n- Able to provide informed consent, including signing and dating the informed consent form (ICF)\n- Vaccination with a quadrivalent meningococcal vaccine and, where available, meningococcal serotype B vaccine. A booster vaccination should also be administered as clinically indicated, according to the local SOC, for subjects who have been previously vaccinated against Neisseria meningitidis\n- Female subjects of childbearing potential must have a negative urine pregnancy test within 24 hours prior to the first dose of study drug\n- Sexually active female subjects of childbearing potential (i.e., women who are not postmenopausal or who have not had a hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) and all male subjects (who have not been surgically sterilized by vasectomy) must agree to use effective contraception during the study and during the safety follow-up period of 40 days after the last dose of study drug. Postmenopausal women are, for the purposes of this protocol, defined as women who have not had menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a postmenopausal state in women not using hormonal contraception or hormonal replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient."}

Exclusion criteria

• {"criterion_text":"- Pregnant, planning to become pregnant, or nursing female subjects\n- With the exception of a prior zilucoplan trial, participation in another concurrent clinical trial involving an experimental therapeutic intervention (participation in observational studies and/or registry studies is permitted)\n- Unable or unwilling to comply with the requirements of the study\n- Commenced any disallowed medication per the exclusion criteria from the qualifying zilucoplan study or alter the dose of any other concomitant medication, unless medically indicated\n- Any new or worsening medical condition (since entry into the qualifying zilucoplan study) including active malignancy (except curatively resected squamous or basal cell carcinoma of the skin); significant medical disorder; psychiatric disorder; laboratory abnormality or any other reason that, in the opinion of the investigator or Sponsor, would disqualify the subject from participation in this study\n- Hypersensitivity to zilucoplan, placebo or any of its excipients"}

Primary endpoints

• {"endpoint_text":"- •\tIncidence of TEAEs","definition_or_measurement_approach":"Not specified in the provided record (incidence of treatment-emergent adverse events is listed as the primary endpoint)."}

Secondary endpoints

• {"endpoint_text":"- •\tCFB to Week E12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score","definition_or_measurement_approach":"Change from baseline (CFB) to Week E12 in MG-ADL score."}
• {"endpoint_text":"- •\tCFB to Week E12 in the Quantitative Myasthenia Gravis (QMG) Score","definition_or_measurement_approach":"Change from baseline (CFB) to Week E12 in QMG score."}
• {"endpoint_text":"- •\tCFB to Week E12 in the Myasthenia Gravis Composite (MGC) Score","definition_or_measurement_approach":"Change from baseline (CFB) to Week E12 in MGC score."}
• {"endpoint_text":"- •\tCFB to Week E12 in the Myasthenia Gravis – Quality of Life revised (MG-QOL15r) Score","definition_or_measurement_approach":"Change from baseline (CFB) to Week E12 in MG-QOL15r score."}

Spain

Earliest CTIS Part Ii Submission Date

16-07-2024

Latest Decision Or Authorization Date

11-03-2025

Processing Time Days

238

Number Of Sites

3

Number Of Participants

5

Italy

Earliest CTIS Part Ii Submission Date

16-07-2024

Latest Decision Or Authorization Date

17-03-2025

Processing Time Days

244

Number Of Sites

1

Number Of Participants

4

Norway

Earliest CTIS Part Ii Submission Date

16-07-2024

Latest Decision Or Authorization Date

13-03-2025

Processing Time Days

240

Number Of Sites

1

Number Of Participants

6

Poland

Earliest CTIS Part Ii Submission Date

16-07-2024

Latest Decision Or Authorization Date

02-12-2025

Processing Time Days

504

Number Of Sites

11

Number Of Participants

22

Primary sponsor

Full Name

UCB Biopharma

Organisation Type

Pharmaceutical company

Country Of Registered Address

Belgium

Contract research organisations

Name

Icon Clinical Research LLC

Responsibilities

Clinical Operations, Medical Monitoring, Regulatory, Clinical Logistics (sponsorDuties codes: 1,15,5,8)

Name

Parexel International (IRL) Limited

Responsibilities

Regulatory (sponsorDuties code: 8)

Name

A2 Healthcare Corp.

Responsibilities

Clinical Operations and other operational support (sponsorDuties codes include 1,12,15,5; 'Clinical Operations' specified)

Third parties

• {"country":"United States","full_name":"Eclinical Solutions LLC","duties_or_roles":"[{\"code\":\"10\"},{\"code\":\"6\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Exco Intouch Limited","duties_or_roles":"[{\"code\":\"7\"}]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"[{\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Center For Information And Study On Clinical Research Participation Inc.","duties_or_roles":"[{\"code\":\"11\"}]","organisation_type":"Patient organisation/association"}
• {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"[{\"code\":\"15\",\"value\":\"Scales & Linguistic validation services\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"[{\"code\":\"3\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Trilogy Writing & Consulting GmbH","duties_or_roles":"[{\"code\":\"11\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Denmark","full_name":"Nordic Bioscience A/S","duties_or_roles":"[{\"code\":\"15\",\"value\":\"Biomarker testing\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Blueprint Genetics Inc.","duties_or_roles":"[{\"code\":\"15\",\"value\":\"Auto-antibody testing\"}]","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Belgium","full_name":"UCB Pharma","duties_or_roles":"[{\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Japan","full_name":"Labcorp Development Japan K.K.","duties_or_roles":"[{\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Japan","full_name":"A2 Healthcare Corp.","duties_or_roles":"[{\"code\":\"1\"},{\"code\":\"12\"},{\"code\":\"15\",\"value\":\"Clinical Operations\"},{\"code\":\"5\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Icon Clinical Research LLC","duties_or_roles":"[{\"code\":\"1\"},{\"code\":\"15\",\"value\":\"Clinical Operations, Medical Monitoring, Regulatory, Clinical Logistics\"},{\"code\":\"5\"},{\"code\":\"8\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"[{\"code\":\"8\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Drug Development Solutions Limited","duties_or_roles":"[{\"code\":\"15\",\"value\":\"Pharmacokinetic sample analysis and parameter calculations\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"[{\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Japan","full_name":"A2 Healthcare Corp.","duties_or_roles":"[{\"code\":\"1\"},{\"code\":\"12\"},{\"code\":\"15\",\"value\":\"Clinical Operations\"},{\"code\":\"5\"}]","organisation_type":"Pharmaceutical company"}