A study to evaluate rozanolixizumab in study participants with generalized myasthenia gravis

• Register: EUCTR
• Last refreshed on: 7 December 2020
• Main ID: EUCTR2020-003230-20-GB
• Date of registration: 02/09/2020
• Prospective Registration: Yes
• Primary sponsor: UCB Biopharma SRL
• Public title: A study to evaluate rozanolixizumab in study participants with generalized myasthenia gravis
• Scientific title: An Open-Label Extension Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis
• Date of first enrolment: 30/11/2020
• Target sample size: 230
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-003230-20
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: yes; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 2
• 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, China, Czech Republic, Denmark, France, Georgia, Germany, Hungary, Italy, Japan, Poland, Russian Federation, Serbia, Spain, 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 meet one of the following:
a) completed MG0003 [NCT03971422] or MGC003,
b) required rescue therapy during the Observation Period in MG0003
or
c) completed at least 6 visits in MG0004 [NCT04124965]
- Body weight =35 kg at Baseline (Day 1)
- Study participants may be male or female

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 207
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 23

Exclusion criteria:
- Study participant has a known hypersensitivity to any components of the study medication
- Evidence of active or latent tuberculosis (TB) as documented by medical history and examination, if applicable, chest x-rays (posterior anterior and lateral), and TB testing by a positive (not indeterminate) QuantiFERON®-TB Gold Plus
- Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria in MG0003, MGC003, or MG0004, or permanently discontinued study drug in either study
- Study participant intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of rozanolixizumab
- 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


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-

Primary Outcome(s)

Secondary Objective: Assess the efficacy of 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG)

Primary end point(s): 1. Percentage of participants with treatment-emergent adverse events (TEAEs)
2. Percentage of participants with (TEAEs) leading to permanent withdrawal of investigational medicinal product (IMP)

Timepoint(s) of evaluation of this end point: 1.; 2. From Baseline (Day 1) to End of Study (average of 20 months)

Main Objective: Assess the safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG)

Secondary Outcome(s)

Secondary end point(s): 1. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score within one treatment cycle
2. Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle
3. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle
4. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) ‘Muscle Weakness Fatigability’ score within one treatment cycle
5. Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO ‘Physical Fatigue’ score within one treatment cycle
6. Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO ‘Bulbar symptoms’ score within one treatment cycle
7. MG-ADL responder (=2.0-point improvement from Baseline [Day 1] to end of Day 43) within one treatment cycle
8. Time to MG-ADL response (=2.0-point improvement from Baseline [Day 1]) within one treatment cycle
9. Time between consecutive treatment cycles

Timepoint(s) of evaluation of this end point: 1. - 8. From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
9. From end of the previous treatment cycle (Day 43) to the next treatment cycle (Day 1)

Secondary ID(s)

MG0007

Source(s) of Monetary Support

UCB Biopharma SRL

Ethics review

Status: Approved
Approval date: 30/11/2020
Contact: