search
Back to results

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

Primary Purpose

Myasthenia Gravis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
UCB7665
Placebo
Sponsored by
UCB Biopharma S.P.R.L.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myasthenia Gravis focused on measuring UCB7665, Myasthenia Gravis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • Mg0002 712
  • Mg0002 701
  • Mg0002 713
  • Mg0002 708
  • Mg0002 707
  • Mg0002 704
  • Mg0002 102
  • Mg0002 103
  • Mg0002 101
  • Mg0002 203
  • Mg0002 202
  • Mg0002 201
  • Mg0002 302
  • Mg0002 401
  • Mg0002 402
  • Mg0002 505
  • Mg0002 502
  • Mg0002 501
  • Mg0002 601
  • Mg0002 602

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Dosage Regimen 1

Dosage Regimen 2

Arm Description

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).

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).

Outcomes

Primary Outcome Measures

Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score to Visit 9
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

Change From Baseline in Myasthenia Gravis-Composite Score to Visit 9
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
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.

Full Information

First Posted
February 10, 2017
Last Updated
July 12, 2021
Sponsor
UCB Biopharma S.P.R.L.
search

1. Study Identification

Unique Protocol Identification Number
NCT03052751
Brief Title
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

2. Study Status

Record Verification Date
July 2021
Overall Recruitment Status
Completed
Study Start Date
May 15, 2017 (Actual)
Primary Completion Date
May 31, 2018 (Actual)
Study Completion Date
August 6, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
UCB Biopharma S.P.R.L.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myasthenia Gravis
Keywords
UCB7665, Myasthenia Gravis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
This is an Investigator- and Subject-Blind study.
Allocation
Randomized
Enrollment
43 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Dosage Regimen 1
Arm Type
Experimental
Arm Description
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).
Arm Title
Dosage Regimen 2
Arm Type
Experimental
Arm Description
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).
Intervention Type
Drug
Intervention Name(s)
UCB7665
Other Intervention Name(s)
Rozanolixizumab
Intervention Description
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
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered in period 1 of dosage regimen 2.
Primary Outcome Measure Information:
Title
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score to Visit 9
Description
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.
Time Frame
From Baseline to Visit 9 (up to Day 29)
Secondary Outcome Measure Information:
Title
Change From Baseline in Myasthenia Gravis-Composite Score to Visit 9
Description
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.
Time Frame
From Baseline to Visit 9 (up to Day 29)
Title
Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MGADL) Score to Visit 9
Description
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.
Time Frame
From Baseline to Visit 9 (up to Day 29)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
UCB Cares
Organizational Affiliation
+1 877 822 9493 (UCB)
Official's Role
Study Director
Facility Information:
Facility Name
Mg0002 712
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Mg0002 701
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Mg0002 713
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Mg0002 708
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Mg0002 707
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Mg0002 704
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Mg0002 102
City
Bruxelles
Country
Belgium
Facility Name
Mg0002 103
City
Gent
Country
Belgium
Facility Name
Mg0002 101
City
Leuven
Country
Belgium
Facility Name
Mg0002 203
City
London
Country
Canada
Facility Name
Mg0002 202
City
Montréal
Country
Canada
Facility Name
Mg0002 201
City
Toronto
Country
Canada
Facility Name
Mg0002 302
City
Ostrava-Poruba
Country
Czechia
Facility Name
Mg0002 401
City
Aarhus
Country
Denmark
Facility Name
Mg0002 402
City
Copenhagen
Country
Denmark
Facility Name
Mg0002 505
City
Düsseldorf
Country
Germany
Facility Name
Mg0002 502
City
Gummersbach
Country
Germany
Facility Name
Mg0002 501
City
Jena
Country
Germany
Facility Name
Mg0002 601
City
Barcelona
Country
Spain
Facility Name
Mg0002 602
City
Barcelona
Country
Spain

12. IPD Sharing Statement

Citations:
PubMed Identifier
33219142
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.
Results Reference
derived
PubMed Identifier
30130439
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.
Results Reference
derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs