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

NCT ID: NCT03052751

Last Updated: 2021-08-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 43 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-05-15
• Study Completion Date: 2018-08-06

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.

Conditions

Myasthenia Gravis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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).

Group Type: EXPERIMENTAL

UCB7665

Intervention Type: DRUG

UCB7665 will be administered in 2 different dosages (dose 1 and dose 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)

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).

Group Type: EXPERIMENTAL

UCB7665

Intervention Type: DRUG

UCB7665 will be administered in 2 different dosages (dose 1 and dose 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)

Placebo

Intervention Type: OTHER

Placebo will be administered in period 1 of dosage regimen 2.

Interventions

UCB7665

UCB7665 will be administered in 2 different dosages (dose 1 and dose 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)

Intervention Type: DRUG

Placebo

Placebo will be administered in period 1 of dosage regimen 2.

Intervention Type: OTHER

Other Intervention Names

Rozanolixizumab

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)

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

UCB Biopharma S.P.R.L.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

UCB Cares

Role: STUDY_DIRECTOR

+1 877 822 9493 (UCB)

Locations

Mg0002 712

Los Angeles, California, United States

Mg0002 701

Orange, California, United States

Mg0002 713

Miami, Florida, United States

Mg0002 708

Tampa, Florida, United States

Mg0002 707

Augusta, Georgia, United States

Mg0002 704

Columbus, Ohio, United States

Mg0002 102

Brussels, , Belgium

Mg0002 103

Ghent, , Belgium

Mg0002 101

Leuven, , Belgium

Mg0002 203

London, , Canada

Mg0002 202

Montreal, , Canada

Mg0002 201

Toronto, , Canada

Mg0002 302

Ostrava-Poruba, , Czechia

Mg0002 401

Aarhus, , Denmark

Mg0002 402

Copenhagen, , Denmark

Mg0002 505

Düsseldorf, , Germany

Mg0002 502

Gummersbach, , Germany

Mg0002 501

Jena, , Germany

Mg0002 601

Barcelona, , Spain

Mg0002 602

Barcelona, , Spain

Countries

United States; Belgium; Canada; Czechia; Denmark; Germany; Spain

References

Matic A, Alfaidi N, Bril V. An evaluation of rozanolixizumab-noli for the treatment of anti-AChR and anti-MuSK antibody-positive generalized myasthenia gravis. Expert Opin Biol Ther. 2023 Jul-Dec;23(12):1163-1171. doi: 10.1080/14712598.2023.2296126. Epub 2023 Dec 28.

Reference Type: DERIVED

PMID: 38099334 (View on PubMed)

Regnault A, Morel T, de la Loge C, Mazerolle F, Kaminski HJ, Habib AA. Measuring Overall Severity of Myasthenia Gravis (MG): Evidence for the Added Value of the MG Symptoms PRO. Neurol Ther. 2023 Oct;12(5):1573-1590. doi: 10.1007/s40120-023-00464-x. Epub 2023 May 11.

Reference Type: DERIVED

PMID: 37166675 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 33219142 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 30130439 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2016-002698-36

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MG0002

Identifier Type: -

Identifier Source: org_study_id