MedPath

Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Phase 3
Active, not recruiting
Conditions
Generalized Myasthenia Gravis
Interventions
Registration Number
NCT04225871
Lead Sponsor
UCB Biopharma SRL
Brief Summary

The RAISE-XT study is an open-label extension study to evaluate the long-term efficacy, safety, and tolerability of zilucoplan in subjects with gMG who have previously participated in a qualifying Ra Pharmaceuticals sponsored zilucoplan study.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
200
Inclusion Criteria
  • Completion of a qualifying zilucoplan study
Exclusion Criteria
  • With the exception of a prior zilucoplan trial, participation in another concurrent clinical trial involving an experimental therapeutic intervention (participation in observational studies and/or registry studies is permitted)

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
0.3 mg/kg zilucoplan (RA101495)zilucoplan (RA101495)-
Primary Outcome Measures
NameTimeMethod
Incidence of treatment-emergent adverse events (TEAEs)From Baseline (Day 1) to Safety Follow-Up Visit (up to 36 months)

A treatment-emergent adverse event is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication.

Secondary Outcome Measures
NameTimeMethod
Change from Baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) ScoreFrom Baseline (Day 1) to Week 12

The MG-ADL profile provides an assessment of MG symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 8 items represents the ADL score. The ADL score can range from 0 (least severe) to 24 (most severe).

Change from Baseline to Week 12 in the the Quantitative Myasthenia Gravis (QMG) ScoreFrom Baseline (Day 1) to Week 12

The QMG test is a standardized quantitative strength scoring system and measures 13 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the QMG score. The QMG score can range from 0 (least severe) to 39 (most severe).

Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) ScoreFrom Baseline (Day 1) to Week 12

The MGC is a 10-item scale that has been used to measure the clinical status of patients with Myasthenia Gravis (MG) in order to evaluate treatment response. The MGC has 4-point Likerttype Scale response options ranging from 0 to 2, 3, 4, 5, 6 or 9 according to the item (weighted response options). The total score is the sum of all items (range 0-50) where higher scores indicate more severe impairment due to the disease.

Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) ScoreFrom Baseline (Day 1) to Week 12

The MG-QOL15r is a 15-item survey that was designed to assess quality of life in patients with MG. The MG-QoL has 3-point Likert Scale response options ranging from 0 to 2. The MGQoL15r score can range from 0 to 30, where higher scores indicate more severe impact of the disease on aspects of the patient's life.

Trial Locations

Locations (70)

Mg0011 41

🇺🇸

Mobile, Alabama, United States

Mg0011 116

🇺🇸

Phoenix, Arizona, United States

Mg0011 31

🇺🇸

Irvine, California, United States

MG0011 4

🇺🇸

Los Angeles, California, United States

Mg0011 220

🇺🇸

Pasadena, California, United States

Mg0011 160

🇺🇸

San Francisco, California, United States

Mg0011 24

🇺🇸

New Haven, Connecticut, United States

Mg0011 135

🇺🇸

Augusta, Georgia, United States

Mg0011 188

🇺🇸

Glenview, Illinois, United States

Mg0011 156

🇺🇸

Indianapolis, Indiana, United States

Mg0011 32

🇺🇸

Kansas City, Kansas, United States

Mg0011 33

🇺🇸

Detroit, Michigan, United States

Mg0011 49

🇺🇸

East Lansing, Michigan, United States

Mg0011 134

🇺🇸

Columbia, Missouri, United States

Mg0011 117

🇺🇸

Las Vegas, Nevada, United States

Mg0011 30

🇺🇸

Buffalo, New York, United States

Mg0011 123

🇺🇸

Great Neck, New York, United States

Mg0011 23

🇺🇸

New York, New York, United States

Mg0011 47

🇺🇸

New York, New York, United States

Mg0011 22

🇺🇸

Chapel Hill, North Carolina, United States

Mg0011 122

🇺🇸

Cleveland, Ohio, United States

Mg0011 38

🇺🇸

Columbus, Ohio, United States

Mg0011 40

🇺🇸

Pittsburgh, Pennsylvania, United States

Mg0011 128

🇺🇸

Charleston, South Carolina, United States

Mg0011 28

🇺🇸

Cordova, Tennessee, United States

Mg0011 131

🇺🇸

Austin, Texas, United States

Mg0011 19

🇺🇸

Dallas, Texas, United States

Mg0011 39

🇺🇸

Salt Lake City, Utah, United States

Mg0011 164

🇺🇸

Charlottesville, Virginia, United States

Mg0011 154

🇺🇸

Seattle, Washington, United States

Mg0011 45

🇺🇸

Milwaukee, Wisconsin, United States

Mg0011 11

🇨🇦

Montreal, Canada

Mg0011 204

🇫🇷

Lille, France

Mg0011 118

🇫🇷

Nice, France

Mg0011 105

🇫🇷

Paris, France

Mg0011 137

🇫🇷

Strasbourg, France

Mg0011 150

🇩🇪

Göttingen, Germany

Mg0011 129

🇩🇪

Tübingen, Germany

Mg0011 132

🇮🇹

Milano, Italy

Mg0011 126

🇮🇹

Roma, Italy

Mg0011 151

🇯🇵

Chiba, Japan

Mg0011 136

🇯🇵

Hanamaki-shi, Japan

Mg0011 179

🇯🇵

Kita-gun, Japan

Mg0011 153

🇯🇵

Meguro-ku, Japan

Mg0011 146

🇯🇵

Nagasaki-shi, Japan

Mg0011 169

🇯🇵

Narita, Japan

Mg0011 152

🇯🇵

Sapporo, Japan

Mg0011 144

🇯🇵

Sendai, Japan

Mg0011 163

🇯🇵

Shinjuku-ku, Japan

Mg0011 141

🇯🇵

Tokyo, Japan

Mg0011 140

🇳🇴

Bergen, Norway

Mg0011 143

🇳🇴

Oslo, Norway

Mg0011 195

🇵🇱

Katowice, Poland

Mg0011 213

🇵🇱

Katowice, Poland

Mg0011 192

🇵🇱

Krakow, Poland

Mg0011 193

🇵🇱

Krakow, Poland

Mg0011 211

🇵🇱

Kraków, Poland

Mg0011 205

🇵🇱

Lublin, Poland

Mg0011 194

🇵🇱

Nowa Sól, Poland

Mg0011 209

🇵🇱

Poznań, Poland

Mg0011 201

🇵🇱

Warszawa, Poland

Mg0011 210

🇵🇱

Zabrze, Poland

Mg0011 214

🇵🇱

Łódź, Poland

Mg0011 133

🇪🇸

Barcelona, Spain

Mg0011 168

🇪🇸

Barcelona, Spain

Mg0011 138

🇪🇸

Bilbao, Spain

Mg0011 119

🇬🇧

Oxford, United Kingdom

Mg0011 130

🇬🇧

Sheffield, United Kingdom

Mg0011 27

🇺🇸

Washington, District of Columbia, United States

Mg0011 25

🇺🇸

Tampa, Florida, United States

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