Clinical trial NCT03052751: Study to Test the Safety, Tolerability and Efficacy of UCB7665 in Subjects With Moderate to Severe Myasthenia Gravis
Official title: A Multicenter, Randomized, Investigator- and Subject-Blind, Placebo-Controlled, Treatment Sequence Study Evaluating the Safety, Tolerability, and Efficacy of UCB7665 in Subjects With Moderate to Severe Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myasthenia Gravis
Keywords: UCB7665
Myasthenia Gravis
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2017-02-14
Enrollment: 43
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to evaluate the clinical efficacy of UCB7665 as a chronic-intermittent treatment in subjects with generalized myasthenia gravis (MG) who are classified as moderate to severe.


Eligibility criteria: Inclusion Criteria:

* Subject has a well-documented diagnosis of myasthenia gravis (MG) at Visit 1 (Screening), based on subject history and supported by previous evaluations
* Subject would currently be considered for treatment with immunological therapy (immunoglobulin/plasma exchange (IVIG/PLEX)) by the investigator
* Subject has a well-documented record of autoantibodies against anti-acetylcholine receptor (Anti-AChR) or anti-muscle specific kinase (Anti-MuSK) prior to Screening
* Female subjects must either be: postmenopausal, permanently sterilized or if childbearing potential applicable will use a highly effective method of birth control
* Male subjects must be willing to use a method of contraception

Exclusion Criteria:

* Subject has previously received treatment in this study or subject has previously been exposed to UCB7665
* Subject has participated in another study of an investigational medicinal product (IMP; or a medical device) within the previous 30 days of Screening or is currently participating in another study of an investigational medicinal product (IMP; or a medical device)
* Subject has a known hypersensitivity to any components of the IMP
* Subject has a history of hyperprolinemia, since L-proline is a constituent of the UCB7665 IMP
* Subjects with Myasthenia Gravis (MG) only affecting the ocular muscles
* Subjects with severe weakness affecting oropharyngeal or respiratory muscles, or who have myasthenic crisis at Screening or impending crisis
* Subject has quantitative myasthenia gravis (QMG) score of \<11 at Baseline
* Subject has a serum total immunoglobulin G (IgG) level \<= 6g/L at Screening
* Absolute neutrophil count \<1500 cells/mm\^3
* Subject has any medical condition (acute or chronic illness) or psychiatric condition that, in the opinion of the investigator, could jeopardize or would compromise the subject's ability to participate in this study
* Subject has any laboratory abnormality that, in the opinion of the investigator, is clinically significant, has not resolved at randomization, and could jeopardize or would compromise the subject's ability to participate in this study
* Subject has received a live vaccination within 8 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 7 weeks following the final dose of IMP
* Subject has received any experimental biological agent within or outside of a clinical study in the past 3 months or within 5 half-lives prior to Baseline (whichever is longer)

Primary outcomes:
measure: Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score to Visit 9
timeFrame: From Baseline to Visit 9 (up to Day 29)
Secondary outcomes:
measure: Change From Baseline in Myasthenia Gravis-Composite Score to Visit 9
timeFrame: From Baseline to Visit 9 (up to Day 29)
measure: Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MGADL) Score to Visit 9
timeFrame: From Baseline to Visit 9 (up to Day 29)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Mg0002 712
state: California
city: Orange
country: United States
facility: Mg0002 701
state: California
city: Miami
country: United States
facility: Mg0002 713
state: Florida
city: Tampa
country: United States
facility: Mg0002 708
state: Florida
city: Augusta
country: United States
facility: Mg0002 707
state: Georgia
city: Columbus
country: United States
facility: Mg0002 704
state: Ohio
city: Bruxelles
country: Belgium
facility: Mg0002 102
city: Gent
country: Belgium
facility: Mg0002 103
city: Leuven
country: Belgium
facility: Mg0002 101
city: London
country: Canada
facility: Mg0002 203
city: Montréal
country: Canada
facility: Mg0002 202
city: Toronto
country: Canada
facility: Mg0002 201
city: Ostrava-Poruba
country: Czechia
facility: Mg0002 302
city: Aarhus
country: Denmark
facility: Mg0002 401
city: Copenhagen
country: Denmark
facility: Mg0002 402
city: Düsseldorf
country: Germany
facility: Mg0002 505
city: Gummersbach
country: Germany
facility: Mg0002 502
city: Jena
country: Germany
facility: Mg0002 501
city: Barcelona
country: Spain
facility: Mg0002 601
city: Barcelona
country: Spain
facility: Mg0002 602
Study officials:
affiliation: +1 877 822 9493 (UCB)
name: UCB Cares
role: STUDY_DIRECTOR
References:
citation: Bril V, Benatar M, Andersen H, Vissing J, Brock M, Greve B, Kiessling P, Woltering F, Griffin L, Van den Bergh P; MG0002 Investigators. Efficacy and Safety of Rozanolixizumab in Moderate to Severe Generalized Myasthenia Gravis: A Phase 2 Randomized Control Trial. Neurology. 2021 Feb 9;96(6):e853-e865. doi: 10.1212/WNL.0000000000011108. Epub 2020 Nov 20.
citation: Smith B, Kiessling A, Lledo-Garcia R, Dixon KL, Christodoulou L, Catley MC, Atherfold P, D'Hooghe LE, Finney H, Greenslade K, Hailu H, Kevorkian L, Lightwood D, Meier C, Munro R, Qureshi O, Sarkar K, Shaw SP, Tewari R, Turner A, Tyson K, West S, Shaw S, Brennan FR. Generation and characterization of a high affinity anti-human FcRn antibody, rozanolixizumab, and the effects of different molecular formats on the reduction of plasma IgG concentration. MAbs. 2018 Oct;10(7):1111-1130. doi: 10.1080/19420862.2018.1505464. Epub 2018 Sep 12.