Study to Test the Safety, Tolerability and Efficacy of UCB7665 in Subjects With Moderate to Severe Myasthenia Gravis

Phase 2

Completed

Conditions: Myasthenia Gravis

Interventions: Drug: UCB7665
Other: Placebo

Registration Number: NCT03052751

Lead Sponsor: UCB Biopharma S.P.R.L.

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.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 43

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)

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Dosage Regimen 2	Placebo	Subjects randomized in dosage regimen 2 will receive 3 doses of placebo in dosing period 1 and will then be re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
Dosage Regimen 1	UCB7665	Subjects randomized in dosage regimen 1 will receive 3 doses of UCB7655 (dose 1) in dosing period 1 and will then be re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
Dosage Regimen 2	UCB7665	Subjects randomized in dosage regimen 2 will receive 3 doses of placebo in dosing period 1 and will then be re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score to Visit 9	From Baseline to Visit 9 (up to Day 29)	The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity. The change from Baseline is calculated, a negative value indicating improvement and a positive value worsening.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Myasthenia Gravis-Composite Score to Visit 9	From Baseline to Visit 9 (up to Day 29)	The total Myasthenia Gravis (MG)-composite score was obtained by summing the responses to each individual item (10 items; Grade: 0-9 depending on item). The score ranges from 0 to 50, with lower scores indicating lower disease activity. The change from Baseline is calculated, a negative value indicating improvement and a positive value worsening.
Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MGADL) Score to Visit 9	From Baseline to Visit 9 (up to Day 29)	The total MGDAL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with lower scores indicating lower disease activity. The change from Baseline is calculated, a negative value indicating improvement and a positive value worsening.

Trial Locations

Locations (20): Mg0002 102
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Bruxelles, Belgium
Mg0002 103
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Gent, Belgium
Mg0002 101
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Leuven, Belgium
Mg0002 602
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Barcelona, Spain
Mg0002 505
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Düsseldorf, Germany
Mg0002 502
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Gummersbach, Germany
Mg0002 707
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Augusta, Georgia, United States
Mg0002 704
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Columbus, Ohio, United States
Mg0002 203
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London, Canada
Mg0002 202
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Montréal, Canada
Mg0002 401
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Aarhus, Denmark
Mg0002 708
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Tampa, Florida, United States
Mg0002 601
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Barcelona, Spain
Mg0002 302
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Ostrava-Poruba, Czechia
Mg0002 701
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Orange, California, United States
Mg0002 713
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Miami, Florida, United States
Mg0002 201
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Toronto, Canada
Mg0002 501
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Jena, Germany
Mg0002 402
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Copenhagen, Denmark
Mg0002 712
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Los Angeles, California, United States