A study to test efficacy and safety of rozanolixizumab in adult patients with generalized myasthenia gravis

• Register: EUCTR
• Last refreshed on: 13 January 2025
• Main ID: EUCTR2019-000968-18-GB
• Date of registration: 28/06/2019
• Prospective Registration: Yes
• Primary sponsor: UCB Biopharma SPRL
• Public title: A study to test efficacy and safety of rozanolixizumab in adult patients with generalized myasthenia gravis
• Scientific title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Patients with Generalized Myasthenia Gravis. - MycarinGstudy
• Date of first enrolment: 14/11/2019
• Target sample size: 240
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-000968-18
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Canada, Czech Republic, Denmark, France, Germany, Hong Kong, Hungary, Italy, Poland, Russian Federation, Spain, Taiwan, United Kingdom, United States

Contacts

• Name: Clin Trial Reg & Results Disclosure
• Affiliation: UCB BIOSCIENCES GmbH

• Name: Clin Trial Reg & Results Disclosure
• Affiliation: UCB BIOSCIENCES GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
- Study participant must be =18 years of age, at the time of signing the informed consent
- Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at Visit 1, based on study participant’s history and supported by previous evaluations
- Study participant has a confirmed positive record of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) at screening (Visit 1). The presence of autoantibodies may be confirmed with repeat testing at Visit 1
- Study participant has Myasthenia Gravis Foundation of America (MGFA) Class II to IVa at Visit 1
- Study participant with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of at least 3 AND a quantitative myasthenia gravis (QMG) score of at least 11 at Visit 1 and at Baseline (Visit 2)
- Study participant is considered for additional treatment such as intravenous immunoglobulin g (IVIg) or plasma exchange (PEX) by the Investigator
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 216
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 24

Exclusion criteria:
- Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP)
- Study participant has experienced hypersensitivity reaction after exposure to other antineonatal Fc receptor (FcRn) drugs
- Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis a Visit 1 or Visit 2


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Generalized myasthenia gravis
MedDRA version: 21.1 Level: PT Classification code 10028417 Term: Myasthenia gravis System Organ Class: 10029205 - Nervous system disorders

Intervention(s)

Product Name: Rozanolixizumab
Product Code: UCB7665
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: rozanolixizumab
CAS Number: 1584645-37-3
Current Sponsor code: UCB7665
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 140-
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Secondary Objective: Assess safety and tolerability of rozanolixizumab in myasthenia gravis (MG) patients

Timepoint(s) of evaluation of this end point: Baseline and Visit 10 (Day 43)

Main Objective: Demonstrate the clinical efficacy of rozanolixizumab in patients with generalized myasthenia gravis (MG)

Primary end point(s): Change from Baseline to Day 43 (Visit 10) in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1. Visit 10 (Day 43)
2.- 6. Baseline and Visit 10 (Day 43)
7. and 8. From Baseline until End of Study Visit (up to Week 14)

Secondary end point(s): 1. Percentage of participants achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) response at Visit 10
2. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis-Composite score
3. Change from Baseline to (Day 43) Visit 10 in Quantitative Myasthenia Gravis (QMG) score to Visit 10
4. Change from Baseline to Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability’ score
5. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) ‘Physical Fatigue score
6. Change from Baseline to Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) ‘Bulbar Symptoms' score
7. Occurrence of treatment-emergent adverse events (TEAEs)
8. Treatment-emergent adverse events (TEAEs) leading to withdrawal of investigational medicinal product (IMP)

Secondary ID(s)

NCT03971422

MG0003

2019-000968-18-DK

Source(s) of Monetary Support

UCB Biopharma SPRL

Ethics review

Status: Approved
Approval date: 14/11/2019
Contact:

Results

• Results available: Yes
• Date Posted: 06/11/2022
• Date Completed: 26/10/2021
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2019-000968-18/results