Back to resultsSafety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE)
Primary Purpose
Myasthenia Gravis, Generalized
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
zilucoplan (RA101495)
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Myasthenia Gravis, Generalized
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
- Positive serology for acetylcholine receptor (AChR) autoantibodies
- MG-ADL Score of ≥ 6 at Screening and Baseline
- QMG score ≥ 12 at Screening and Baseline
- No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
- No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
Exclusion Criteria:
- Thymectomy within 12 months prior to Baseline or scheduled to occur during the 12 week Treatment Period
- History of meningococcal disease
- Current or recent systemic infection within 2 weeks prior to Baseline or injection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline
Sites / Locations
- Site 41: Diagnostic and Medical Clinic
- Site 116: Neuromuscular Clinic and Research Center
- Site 4: University of Southern California
- Site 31: University of California Irvine
- Site 220: Investigator Site
- Site 160: Forbes Norris MDA/ALS Research and Treatment Center
- Site 24: Yale University
- Site 27: George Washington University
- Site 182: Gelasio Baras Neurology
- Site 25: University of South Florida
- Site 135: Augusta University Medical Center
- Site 176: Hawaii Pacific Neuroscience
- Site 188: North Shore Medical Group - Glenview
- Site 156: Indiana University Health Neuroscience Center
- Site 156: University of Kansas Medical Center
- Site 32: Kansas University Medical Center Research Institute
- Site 221: Neurology Center of New England
- Site 33: Detroit medical Center - University Health Center
- Site 49: Michigan State University
- Site 127: University of Minnesota
- Site 134: Neurology and Sleep Disorders Clinic
- Site 117: Las Vegas Clinic
- Site 123: Northwell Health Neuroscience Institute
- Site 23: Hospital for Special Surgery
- Site 47: Mount Sinai Hospital
- Site 22: University of North Carolina
- Site 15: Duke University
- Site 122: Cleveland Clinic
- Site 38: Ohio State University
- Site 40: Allegheny Neurological Associates
- Site 128: Medical University of South Carolina
- Site 185: Neurology Clinic Cordova
- Site 131: Austin Neuromuscular Center
- Site 19: University of Texas Southwestern
- Site 39: University of Utah
- Site 164: University of Virginia Health System
- Site 154: University of Washington
- Site 45: Center for Neurological Disorders
- Site 44: London Health Sciences Centre University Hospital
- Site 11: Montreal Neurological Institute and Hospital (McGill University)
- Site 191: Centre Hosptitalier Universitaire d'Angers
- Site 204: Centre Hospitalier Régional Universitaire de Lille
- Site 118: Hôpital Pasteur
- Site 105: Pitié-Salpêtrière University Hospital
- Site 137: Les Hôpitaux Universitaires de Strasbourg
- Site 150: Universitätsmedizin Göttingen
- Site 129: Universitätsklinikum Tübingen
- Site 126: Fondazione IRCCS Istituto Neurologico Carlo Besta
- Site 132: Università Cattolica del Sacro Cuore - Campus di Milano
- Site 169: International University of Health and Welfare Narita Hospital
- Site 151: Chiba University Hospital
- Site 136: General Hanamaki Hospital
- Site 179: Kagawa University Hospital - Collagen disease/Rheumatic int
- Site 146: Nagasaki University Hospital
- Site 152: Hokkaido Medical Center
- Site 144: Sendai Medical Center
- Site 153: Toho University Ohashi Medical Center
- Site 163: Tokyo Medical University Hospital
- Site 141: Keio University Hospital
- Site 165: Osaka University Hospital
- Site 140: Haukeland University Hospital / Health Bergen
- Site 143: Oslo Universitetssykehus
- Site 205: Prywatny Gabinet Lekarski Urszula Chyrchel-Paszkiewicz
- Site 194: Twoja Przychodnia - Centrum Medyczne Nowa Sól
- Site 214: AmiCare Centrum Medyczne
- Site 192: Krakowski Szpital Specjalistyczny im. Jana Pawła II
- Site 201: Centrum Medyczne Pratia - Warszawa
- Site 195: Wielospecjalistyczna Poradnia Lekarska Synapsis
- Site 213: Niepubliczny Zakład Opieki Zdrowotnej NOVO-MED
- Site 209: Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD
- Site 193: Krakowska Akademia Neurologii - Centrum Neurologii Kliniczne
- Site 211: Specjalistyczne Gabinety Sp. z o.o.
- Site 210: Clinhouse Centrum Medyczne
- Site 133: Hospital Universitari Vall d'Hebrón
- Site 168: Hospital de la Santa Creu i Sant Pau
- Site 138: Hospital Universitario de Basurto
- Site 119: Oxford University Hospitals NHS Foundation Trust
- Site 130: Royal Hallamshire Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
0.3 mg/kg zilucoplan (RA101495)
Placebo
Arm Description
Outcomes
Primary Outcome Measures
Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score
The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement.
Secondary Outcome Measures
Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score
The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement.
Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score
The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing [0 to 6], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement.
Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score
The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicated improvement.
Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period
Time to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1.
Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy
Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the missing at Random (MAR) assumption.
Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy
Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.
Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12
Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04115293
Brief Title
Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Acronym
RAISE
Official Title
A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Study Type
Interventional
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
September 17, 2019 (Actual)
Primary Completion Date
December 30, 2021 (Actual)
Study Completion Date
December 30, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ra Pharmaceuticals, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myasthenia Gravis, Generalized
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
174 (Actual)
8. Arms, Groups, and Interventions
Arm Title
0.3 mg/kg zilucoplan (RA101495)
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
zilucoplan (RA101495)
Intervention Description
Daily subcutaneous (SC) injection
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Daily subcutaneous (SC) injection
Primary Outcome Measure Information:
Title
Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score
Description
The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement.
Time Frame
From Baseline to End of Treatment (Week 12)
Secondary Outcome Measure Information:
Title
Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score
Description
The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement.
Time Frame
From Baseline to End of Treatment (Week 12)
Title
Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score
Description
The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing [0 to 6], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement.
Time Frame
From Baseline to End of Treatment (Week 12)
Title
Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score
Description
The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicated improvement.
Time Frame
From Baseline to End of Treatment (Week 12)
Title
Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period
Description
Time to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1.
Time Frame
From Baseline to End of Treatment (Week 12)
Title
Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy
Description
Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the missing at Random (MAR) assumption.
Time Frame
End of Treatment (Week 12)
Title
Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy
Description
Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.
Time Frame
End of Treatment (Week 12)
Title
Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12
Description
Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.
Time Frame
End of Treatment (Week 12)
Title
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
Description
A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Time Frame
From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up])
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
74 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
Positive serology for acetylcholine receptor (AChR) autoantibodies
MG-ADL Score of ≥ 6 at Screening and Baseline
QMG score ≥ 12 at Screening and Baseline
No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
Exclusion Criteria:
Thymectomy within 12 months prior to Baseline or scheduled to occur during the 12 week Treatment Period
History of meningococcal disease
Current or recent systemic infection within 2 weeks prior to Baseline or injection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
UCB Cares
Organizational Affiliation
0018445992273 (UCB)
Official's Role
Study Director
Facility Information:
Facility Name
Site 41: Diagnostic and Medical Clinic
City
Mobile
State/Province
Alabama
ZIP/Postal Code
36604
Country
United States
Facility Name
Site 116: Neuromuscular Clinic and Research Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85028
Country
United States
Facility Name
Site 4: University of Southern California
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Site 31: University of California Irvine
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Site 220: Investigator Site
City
Pasadena
State/Province
California
ZIP/Postal Code
91101
Country
United States
Facility Name
Site 160: Forbes Norris MDA/ALS Research and Treatment Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94115
Country
United States
Facility Name
Site 24: Yale University
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Facility Name
Site 27: George Washington University
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20037
Country
United States
Facility Name
Site 182: Gelasio Baras Neurology
City
Miami
State/Province
Florida
ZIP/Postal Code
33175
Country
United States
Facility Name
Site 25: University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Site 135: Augusta University Medical Center
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Site 176: Hawaii Pacific Neuroscience
City
Honolulu
State/Province
Hawaii
ZIP/Postal Code
96817
Country
United States
Facility Name
Site 188: North Shore Medical Group - Glenview
City
Glenview
State/Province
Illinois
ZIP/Postal Code
60026-1339
Country
United States
Facility Name
Site 156: Indiana University Health Neuroscience Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Site 156: University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Site 32: Kansas University Medical Center Research Institute
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Site 221: Neurology Center of New England
City
Foxboro
State/Province
Massachusetts
ZIP/Postal Code
02035
Country
United States
Facility Name
Site 33: Detroit medical Center - University Health Center
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Site 49: Michigan State University
City
East Lansing
State/Province
Michigan
ZIP/Postal Code
48824
Country
United States
Facility Name
Site 127: University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Site 134: Neurology and Sleep Disorders Clinic
City
Columbia
State/Province
Missouri
ZIP/Postal Code
65212
Country
United States
Facility Name
Site 117: Las Vegas Clinic
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89145
Country
United States
Facility Name
Site 123: Northwell Health Neuroscience Institute
City
Great Neck
State/Province
New York
ZIP/Postal Code
11021
Country
United States
Facility Name
Site 23: Hospital for Special Surgery
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Site 47: Mount Sinai Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Site 22: University of North Carolina
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Site 15: Duke University
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Site 122: Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Site 38: Ohio State University
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Site 40: Allegheny Neurological Associates
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15212
Country
United States
Facility Name
Site 128: Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Site 185: Neurology Clinic Cordova
City
Cordova
State/Province
Tennessee
ZIP/Postal Code
38018
Country
United States
Facility Name
Site 131: Austin Neuromuscular Center
City
Austin
State/Province
Texas
ZIP/Postal Code
78756
Country
United States
Facility Name
Site 19: University of Texas Southwestern
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Site 39: University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Site 164: University of Virginia Health System
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Site 154: University of Washington
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
Site 45: Center for Neurological Disorders
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53215
Country
United States
Facility Name
Site 44: London Health Sciences Centre University Hospital
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5A5
Country
Canada
Facility Name
Site 11: Montreal Neurological Institute and Hospital (McGill University)
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H3A 2B4
Country
Canada
Facility Name
Site 191: Centre Hosptitalier Universitaire d'Angers
City
Angers Cedex 9
Country
France
Facility Name
Site 204: Centre Hospitalier Régional Universitaire de Lille
City
Lille
Country
France
Facility Name
Site 118: Hôpital Pasteur
City
Nice
ZIP/Postal Code
06000
Country
France
Facility Name
Site 105: Pitié-Salpêtrière University Hospital
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
Site 137: Les Hôpitaux Universitaires de Strasbourg
City
Strasbourg
ZIP/Postal Code
67091
Country
France
Facility Name
Site 150: Universitätsmedizin Göttingen
City
Göttingen
ZIP/Postal Code
37075
Country
Germany
Facility Name
Site 129: Universitätsklinikum Tübingen
City
Tübingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
Site 126: Fondazione IRCCS Istituto Neurologico Carlo Besta
City
Milan
ZIP/Postal Code
20133
Country
Italy
Facility Name
Site 132: Università Cattolica del Sacro Cuore - Campus di Milano
City
Roma
ZIP/Postal Code
20123
Country
Italy
Facility Name
Site 169: International University of Health and Welfare Narita Hospital
City
Narita
State/Province
Chiba
ZIP/Postal Code
286-8520
Country
Japan
Facility Name
Site 151: Chiba University Hospital
City
Chiba
ZIP/Postal Code
260-8677
Country
Japan
Facility Name
Site 136: General Hanamaki Hospital
City
Iwata
ZIP/Postal Code
025-0075
Country
Japan
Facility Name
Site 179: Kagawa University Hospital - Collagen disease/Rheumatic int
City
Kita-gun
Country
Japan
Facility Name
Site 146: Nagasaki University Hospital
City
Nagasaki
ZIP/Postal Code
852-8501
Country
Japan
Facility Name
Site 152: Hokkaido Medical Center
City
Sapporo
ZIP/Postal Code
063-0005
Country
Japan
Facility Name
Site 144: Sendai Medical Center
City
Sendai
ZIP/Postal Code
983-8520
Country
Japan
Facility Name
Site 153: Toho University Ohashi Medical Center
City
Tokyo
ZIP/Postal Code
153-8515
Country
Japan
Facility Name
Site 163: Tokyo Medical University Hospital
City
Tokyo
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
Site 141: Keio University Hospital
City
Tokyo
ZIP/Postal Code
160-8582
Country
Japan
Facility Name
Site 165: Osaka University Hospital
City
Tokyo
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Site 140: Haukeland University Hospital / Health Bergen
City
Bergen
ZIP/Postal Code
5021
Country
Norway
Facility Name
Site 143: Oslo Universitetssykehus
City
Oslo
ZIP/Postal Code
0450
Country
Norway
Facility Name
Site 205: Prywatny Gabinet Lekarski Urszula Chyrchel-Paszkiewicz
City
Lublin
State/Province
Lubelskie
ZIP/Postal Code
20-093
Country
Poland
Facility Name
Site 194: Twoja Przychodnia - Centrum Medyczne Nowa Sól
City
Nowa Sól
State/Province
Lubuskie
ZIP/Postal Code
67-100
Country
Poland
Facility Name
Site 214: AmiCare Centrum Medyczne
City
Łódź
State/Province
Lódzkie
ZIP/Postal Code
90-644
Country
Poland
Facility Name
Site 192: Krakowski Szpital Specjalistyczny im. Jana Pawła II
City
Kraków
State/Province
Malopolskie
ZIP/Postal Code
31-202
Country
Poland
Facility Name
Site 201: Centrum Medyczne Pratia - Warszawa
City
Warszawa
State/Province
Mazowieckie
ZIP/Postal Code
01-868
Country
Poland
Facility Name
Site 195: Wielospecjalistyczna Poradnia Lekarska Synapsis
City
Katowice
State/Province
Slaskie
ZIP/Postal Code
40-123
Country
Poland
Facility Name
Site 213: Niepubliczny Zakład Opieki Zdrowotnej NOVO-MED
City
Katowice
State/Province
Slaskie
ZIP/Postal Code
40-650
Country
Poland
Facility Name
Site 209: Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD
City
Poznań
State/Province
Wielkopolskie
ZIP/Postal Code
61-853
Country
Poland
Facility Name
Site 193: Krakowska Akademia Neurologii - Centrum Neurologii Kliniczne
City
Kraków
Country
Poland
Facility Name
Site 211: Specjalistyczne Gabinety Sp. z o.o.
City
Kraków
Country
Poland
Facility Name
Site 210: Clinhouse Centrum Medyczne
City
Lublin
ZIP/Postal Code
20-093
Country
Poland
Facility Name
Site 133: Hospital Universitari Vall d'Hebrón
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Site 168: Hospital de la Santa Creu i Sant Pau
City
Barcelona
Country
Spain
Facility Name
Site 138: Hospital Universitario de Basurto
City
Bilbao
ZIP/Postal Code
48013
Country
Spain
Facility Name
Site 119: Oxford University Hospitals NHS Foundation Trust
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Facility Name
Site 130: Royal Hallamshire Hospital
City
Sheffield
ZIP/Postal Code
S10 2JF
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed;in this case and to protect participants, individual patient-level data would not be made available.
IPD Sharing Time Frame
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
IPD Sharing Access Criteria
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
IPD Sharing URL
http://www.Vivli.org
Citations:
PubMed Identifier
37059508
Citation
Howard JF Jr, Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Sivakumar K, Smilowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Lowcock R, Vanderkelen M, Leite MI; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023 May;22(5):395-406. doi: 10.1016/S1474-4422(23)00080-7.
Results Reference
result
Learn more about this trial
Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
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