A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis

Generalized Myasthenia Gravis Clinical Trial

Official title:

An Open-Label Extension Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04650854
• Other study ID #: MG0007
• Secondary ID: 2020-003230-20
• Status: Completed
• Phase: Phase 3
• Start date: February 3, 2021
• Est. completion date: January 25, 2024

Study information

• Verified date: February 2024
• Source: UCB Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to assess the safety, tolerability and efficacy of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 165
• Est. completion date: January 25, 2024
• Est. primary completion date: January 25, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Study participant must meet one of the following:

1. completed MG0003 [NCT03971422]

2. required rescue therapy during the Observation Period in MG0003 or

3. completed at least 6 visits in MG0004 [NCT04124965]

• Body weight =35 kg at Baseline (Day 1)

• Study participants may be male or female

Exclusion Criteria:

• Study participant has a known hypersensitivity to any components of the study medication or other anti-neonatal Fc receptor (FcRn) medications

• Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)

• Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria in MG0003, or MG0004, or permanently discontinued study drug in either study

• Study participant intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of rozanolixizumab

• Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis

Related Conditions & MeSH terms

• Generalized Myasthenia Gravis
• Muscle Weakness
• Myasthenia Gravis

Intervention

Drug:
• Rozanolixizumab
Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.

Locations

Country	Name	City	State
Canada	Mg0007 50071	Edmonton
Canada	Mg0007 50066	Montreal
Canada	Mg0007 50124	Montreal
Canada	Mg0007 50070	Quebec
Canada	Mg0007 50069	Toronto
Czechia	Mg0007 40125	Ostrava - Poruba
Czechia	Mg0007 40124	Praha 2
Denmark	Mg0007 40128	Aalborg
Denmark	Mg0007 40127	Aarhus
Denmark	Mg0007 40126	Copenhagen
France	Mg0007 40129	Bordeaux
France	Mg0007 40360	Limoges
France	Mg0007 40132	Nice Cedex 1
France	Mg0007 40133	Paris
France	Mg0007 40131	Strasbourg
Georgia	Mg0007 20160	Tbilisi
Georgia	Mg0007 20161	Tbilisi
Georgia	Mg0007 20163	Tbilisi
Georgia	Mg0007 20164	Tbilisi
Georgia	Mg0007 20165	Tbilisi
Germany	Mg0007 40134	Essen
Germany	Mg0007 40140	Göttingen
Germany	Mg0007 40139	Jena
Germany	Mg0007 40078	Leipzig
Germany	Mg0007 40177	Münster
Italy	Mg0007 40283	Bologna
Italy	Mg0007 40144	Milano
Italy	Mg0007 40307	Napoli
Italy	Mg0007 40146	Pavia
Italy	Mg0007 40148	Roma
Italy	Mg0007 40150	Roma
Japan	Mg0007 20035	Bunkyo-ku
Japan	Mg0007 20068	Chiba-shi
Japan	Mg0007 20078	Hanamaki-shi
Japan	Mg0007 20079	Hiroshima
Japan	Mg0007 20075	Kobe
Japan	Mg0007 20071	Nagasaki-shi
Japan	Mg0007 20077	Sendai
Japan	Mg0007 20070	Shinjuku-ku
Japan	Mg0007 20076	Shinjuku-ku
Japan	Mg0007 20032	Suita
Poland	Mg0007 40155	Gdansk
Poland	Mg0007 40154	Lodz
Poland	Mg0007 40151	Lublin
Poland	Mg0007 40153	Poznan
Russian Federation	Mg0007 20169	Novosibirsk
Russian Federation	Mg0007 20001	Saint-petersburg
Russian Federation	Mg0007 20028	Saint-petersburg
Russian Federation	Mg0007 20055	Saint-petersburg
Serbia	Mg0007 40467	NIS
Spain	Mg0007 40160	Barcelona
Spain	Mg0007 40157	Hospitalet de Llobregat
Spain	Mg0007 40350	Murcia
Spain	Mg0007 40308	San Sebastián de Los Reyes
Taiwan	Mg0007 20081	Taipei City
Taiwan	Mg0007 20086	Taipei City
United States	Mg0007 50075	Augusta	Georgia
United States	Mg0007 50323	Honolulu	Hawaii
United States	Mg0007 50113	Houston	Texas
United States	Mg0007 50114	Indianapolis	Indiana
United States	Mg0007 50121	Lexington	Kentucky
United States	Mg0007 50120	Miami	Florida
United States	Mg0007 50122	Miami	Florida
United States	Mg0007 50077	New York	New York
United States	Mg0007 50092	Orange	California
United States	Mg0007 50096	Philadelphia	Pennsylvania
United States	Mg0007 50099	San Francisco	California
United States	Mg0007 50073	Tampa	Florida
United States	Mg0007 50090	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
UCB Biopharma SRL

Countries where clinical trial is conducted

United States,  Canada,  Czechia,  Denmark,  France,  Georgia,  Germany,  Italy,  Japan,  Poland,  Russian Federation,  Serbia,  Spain,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of participants with treatment-emergent adverse events (TEAEs)	An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.	From Baseline (Day 1) to End of Study (average of 20 months)
Primary	Percentage of participants with TEAEs leading to withdrawal of investigational medicinal product (IMP)	An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.	From Baseline (Day 1) to End of Study (average of 20 months)
Secondary	Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score within one treatment cycle	The Outcome Measure is applicable for the first 3 treatment cycles. The total MG-ADL 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 a higher score indicating more disability.; A positive change indicates worsening and a negative change indicates improvement.	From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
Secondary	Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle	The Outcome Measure is applicable for the first 3 treatment cycles. 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.	From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
Secondary	Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle	The Outcome Measure is applicable for the first 3 treatment cycles. The total MG-C 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.	From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
Secondary	Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score within one treatment cycle	The Outcome Measure is applicable for the first 3 treatment cycles. The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).; The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.	From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
Secondary	Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' score within one treatment cycle	The Outcome Measure is applicable for the first 3 treatment cycles. The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).; The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.	From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
Secondary	Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar symptoms' score within one treatment cycle	The Outcome Measure is applicable for the first 3 treatment cycles. The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).; The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.	From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
Secondary	MG-ADL responder (=2.0-point improvement from Baseline [Day 1] to end of Day 43) within one treatment cycle	The Outcome Measure is applicable for the first 3 treatment cycles. A MG-ADL responder is defined as achieving at least 2.0-point improvement in the MG-ADL score from Baseline.	From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
Secondary	Time to MG-ADL response (=2.0-point improvement from Baseline [Day 1]) within one treatment cycle	The Outcome Measure is applicable for the first 3 treatment cycles. Time to achieve MG-ADL response, defined as at least 2.0-point improvement from Baseline.	From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
Secondary	Time between consecutive treatment cycles	Time between consecutive treatment cycles: Study participants will be assessed for MG worsening prior to repeated cycles. In case of symptom worsening (eg, an increase of 2.0 points on the MG-ADL or 3.0 points on the QMG scale) between assessments, resulting in a need for additional treatment, study participants will undergo another 6-week treatment cycle followed by an Observation Period, based on the Investigator's discretion.	From end of the 6-week treatment cycle (Day 43) to the next 6-week treatment cycle (Day 1), assessed up to 2.5 years