An extension study to learn if Zilucoplan is safe and works over a long period in adult participants with generalized 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 Dec 2020 → ongoing

Decision date (initial)

2024-08-05

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-512399-37-00

EudraCT number

2019-001565-33

WHO UTN

U1111-1306-6625

ClinicalTrials.gov

NCT04225871

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Pharmacokinetic, Others, Safety, Efficacy

To evaluate the long-term safety and tolerability of zilucoplan in subjects with gMG who have completed a qualifying zilucoplan clinical study

Secondary objectives 2

1. -To evaluate the long-term efficacy of zilucoplan in subjects with gMG who have completed a qualifying zilucoplan clinical study
2. -To confirm the long term safety and tolerability of zilucoplan in subjects with gMG in other safety measures

Conditions and MedDRA coding

Generalized Myasthenia Gravis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Completion of a qualifying zilucoplan study
2. Able to provide informed consent, including signing and dating the informed consent form (ICF)
3. 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
4. Female subjects of childbearing potential must have a negative urine pregnancy test within 24 hours prior to the first dose of study drug
5. 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 6

1. Pregnant, planning to become pregnant, or nursing female subjects
2. 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)
3. Unable or unwilling to comply with the requirements of the study
4. 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
5. 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
6. Hypersensitivity to zilucoplan, placebo or any of its excipients

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. • Incidence of TEAEs

Secondary endpoints 4

1. • CFB to Week E12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score
2. • CFB to Week E12 in the Quantitative Myasthenia Gravis (QMG) Score
3. • CFB to Week E12 in the Myasthenia Gravis Composite (MGC) Score
4. • CFB to Week E12 in the Myasthenia Gravis – Quality of Life revised (MG-QOL15r) Score

Investigational products

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

Test 3

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 16

Locations

4 EU/EEA countries · 16 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

Documents 30 files

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

Application history

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