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

• ClinicalTrials.gov Identifier: NCT03052751
• Recruitment Status: Completed
• First Posted: February 14, 2017
• Last Update Posted: June 4, 2019
• Sponsor: UCB Biopharma S.P.R.L.
• Information provided by (Responsible Party): UCB Pharma ( UCB Biopharma S.P.R.L. )

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Myasthenia Gravis	Drug: UCB7665; Other: Placebo	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 43 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: May 15, 2017
• Actual Primary Completion Date: May 31, 2018
• Actual Study Completion Date: August 6, 2018

Arms and Interventions

Arm	Intervention/treatment
Experimental: Dosage Regimen 1; 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).	Drug: UCB7665; UCB7665 will be administered in 2 different dosages (dose 1 and dose 2) in dosage regimen 1 and 2. UCB7665 (INN: Rozanolixizumab) is a humanized monoclonal antibody that is being developed for treatment of IgG autoantibody-mediated conditions such as myasthenia gravis (MG); Other Name: Rozanolixizumab
Experimental: Dosage Regimen 2; 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).	Drug: UCB7665; UCB7665 will be administered in 2 different dosages (dose 1 and dose 2) in dosage regimen 1 and 2. UCB7665 (INN: Rozanolixizumab) is a humanized monoclonal antibody that is being developed for treatment of IgG autoantibody-mediated conditions such as myasthenia gravis (MG); Other Name: Rozanolixizumab; Other: Placebo; Placebo will be administered in period 1 of dosage regimen 2.

Outcome Measures

Primary Outcome Measures :

1. Change from Baseline in Quantitative Myasthenia Gravis (QMG) score to Visit 9 [ Time Frame: Baseline and Visit 9 (Day 29) ]
   The total QMG score is 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.

Secondary Outcome Measures :

1. Change from Baseline in Myasthenia Gravis-Composite score to Visit 9 [ Time Frame: Baseline and Visit 9 (Day 29) ]
   The total Myasthenia Gravis(MG)-composite score is 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.
2. Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MGADL) score to Visit 9 [ Time Frame: Baseline and Visit 9 (Day 29) ]
   The total MGDAL score is 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.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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)

Contacts and Locations

Locations

United States, California

Mg0002 712

Los Angeles, California, United States, 90033

Mg0002 701

Orange, California, United States, 92868

United States, Florida

Mg0002 713

Miami, Florida, United States, 33136

Mg0002 708

Tampa, Florida, United States, 33612

United States, Georgia

Mg0002 707

Augusta, Georgia, United States, 30912

United States, Ohio

Mg0002 704

Columbus, Ohio, United States, 43210

Belgium

Mg0002 102

Bruxelles, Belgium

Mg0002 103

Gent, Belgium

Mg0002 101

Leuven, Belgium

Canada

Mg0002 203

London, Canada

Mg0002 202

Montréal, Canada

Mg0002 201

Toronto, Canada

Czechia

Mg0002 302

Ostrava-Poruba, Czechia

Denmark

Mg0002 401

Aarhus, Denmark

Mg0002 402

Copenhagen, Denmark

Germany

Mg0002 505

Düsseldorf, Germany

Mg0002 502

Gummersbach, Germany

Mg0002 501

Jena, Germany

Spain

Mg0002 601

Barcelona, Spain

Mg0002 602

Barcelona, Spain

Sponsors and Collaborators

UCB Biopharma S.P.R.L.

Investigators

• Study Director: UCB Cares; +1 877 822 9493 (UCB)

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

• Responsible Party: UCB Biopharma S.P.R.L.
• ClinicalTrials.gov Identifier: NCT03052751 History of Changes
• Other Study ID Numbers: MG0002; 2016-002698-36 ( EudraCT Number )
• First Posted: February 14, 2017
• Last Update Posted: June 4, 2019
• Last Verified: May 2019

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by UCB Pharma ( UCB Biopharma S.P.R.L. ):

UCB7665; Myasthenia Gravis

Additional relevant MeSH terms:

Muscle Weakness; Myasthenia Gravis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Pathologic Processes; Signs and Symptoms; Autoimmune Diseases of the Nervous System; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases