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CompletedNCT04115293RAISEUpdated Jul 16, 2025Results posted

Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis

A Phase 3 interventional study of zilucoplan (RA101495) and Placebo in Myasthenia Gravis, Generalized, sponsored by Ra Pharmaceuticals, Inc.. Completed at 77 sites in 10 countries. Open to participants aged 18 Years to 74 Years. Per ClinicalTrials.gov, last updated 2025-07-16.

Sponsored by Ra Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
174
Allocation
Randomized
Ages
18 Years to 74 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Myasthenia Gravis, Generalized

Browse trials for

03

Who can participate

Ages eligible
18 Years to 74 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
174 participants (actual)

Study arms

  • Experimental
    0.3 mg/kg zilucoplan (RA101495)

    Drug: zilucoplan (RA101495)

  • Placebo comparator
    Placebo

    Drug: Placebo

Interventions

  • Drugzilucoplan (RA101495)

    Daily subcutaneous (SC) injection

  • DrugPlacebo

    Daily subcutaneous (SC) injection

05

What researchers measure

Primary outcomes

  1. 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.

    Time frame: From Baseline to End of Treatment (Week 12)

Secondary outcomes

  1. 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.

    Time frame: From Baseline to End of Treatment (Week 12)

  2. 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.

    Time frame: From Baseline to End of Treatment (Week 12)

  3. 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 frame: From Baseline to End of Treatment (Week 12)

  4. 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.

    Time frame: From Baseline to End of Treatment (Week 12)

  5. 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.

    Time frame: End of Treatment (Week 12)

  6. 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.

    Time frame: End of Treatment (Week 12)

  7. 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.

    Time frame: End of Treatment (Week 12)

  8. 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.

    Time frame: From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up])

06

Results

Posted Jan 17, 2023

Participant flow

The study started to enroll participants in September 2019 and concluded in December 2021.

Participant flow — Overall Study
MilestonePlaceboZilucoplan 0.3 mg/kg
Started8886
Completed8482
Not completed44
Withdrew: Adverse event02
Withdrew: Withdrawal by subject21
Withdrew: Physician decision10
Withdrew: Death11

Outcome measures

PrimaryChange 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.

Time frame:
From Baseline to End of Treatment (Week 12)
Reported as:
Least squares mean · score on a scale
Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score-2.30 (-3.17 to -1.43)-4.39 (-5.28 to -3.50)
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · MMRM ANCOVA · p = <0.001 · Ls mean difference: -2.09 · 95% CI -3.24 to -0.95
SecondaryChange 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.

Time frame:
From Baseline to End of Treatment (Week 12)
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score-3.25 (-4.32 to -2.17)-6.19 (-7.29 to -5.08)
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · MMRM ANCOVA · p = <0.001 · Ls mean difference: -2.94 · 95% CI -4.39 to -1.49
SecondaryChange 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.

Time frame:
From Baseline to End of Treatment (Week 12)
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score-5.42 (-6.98 to -3.86)-8.62 (-10.22 to -7.01)
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · MMRM ANCOVA · p = 0.0023 · Ls mean difference: -3.20 · 95% CI -5.24 to -1.16
SecondaryChange 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 frame:
From Baseline to End of Treatment (Week 12)
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score-3.16 (-4.65 to -1.67)-5.65 (-7.17 to -4.12)
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · MMRM ANCOVA · p = 0.0128 · Ls mean difference: -2.49 · 95% CI -4.45 to -0.54
SecondaryTime 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.

Time frame:
From Baseline to End of Treatment (Week 12)
Reported as:
Median · Days
Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period
DaysPlaceboZilucoplan 0.3 mg/kg
Time to First Receipt of Rescue Therapy Over the 12-week Treatment PeriodNA (NA to NA)NA (NA to NA)
SecondaryPercentage 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.

Time frame:
End of Treatment (Week 12)
Reported as:
Number · percentage of participants
Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy
percentage of participantsPlaceboZilucoplan 0.3 mg/kg
Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy5.814.0
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Regression, Logistic · p = 0.0885 · Odds ratio (or): 2.608 · 95% CI 0.866 to 7.860
SecondaryPercentage 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.

Time frame:
End of Treatment (Week 12)
Reported as:
Number · percentage of participants
Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy
percentage of participantsPlaceboZilucoplan 0.3 mg/kg
Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy46.173.1
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Regression, Logistic · p = <0.001 · Odds ratio (or): 3.184 · 95% CI 1.662 to 6.101
SecondaryPercentage 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.

Time frame:
End of Treatment (Week 12)
Reported as:
Number · percentage of participants
Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12
percentage of participantsPlaceboZilucoplan 0.3 mg/kg
Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 1233.058.0
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Regression, Logistic · p = 0.0012 · Odds ratio (or): 2.865 · 95% CI 1.518 to 5.409
SecondaryPercentage 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.

Time frame:
From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up])
Reported as:
Number · percentage of participants
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
percentage of participantsPlaceboZilucoplan 0.3 mg/kg
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)70.576.7

Adverse events

Collected over From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Placebo1/88 (1.1%)13/88 (14.8%)34/88 (38.6%)
Zilucoplan 0.3 mg/kg1/86 (1.2%)11/86 (12.8%)41/86 (47.7%)
Most frequent serious events
Showing 10 of 20
Most frequent serious events
EventPlaceboZilucoplan 0.3 mg/kg
Myasthenia gravisNervous system disorders5/882/86
COVID-19Infections and infestations2/881/86
COVID-19 pneumoniaInfections and infestations2/881/86
AnaemiaBlood and lymphatic system disorders0/881/86
Aphthous ulcerGastrointestinal disorders0/881/86
Oesophageal candidiasisInfections and infestations0/881/86
Oral candidiasisInfections and infestations0/881/86
PneumoniaInfections and infestations0/881/86
SepsisInfections and infestations0/881/86
Lipase increasedInvestigations0/881/86
Most frequent other events
Showing 10 of 12
Most frequent other events
EventPlaceboZilucoplan 0.3 mg/kg
Injection site bruisingGeneral disorders8/8814/86
HeadacheNervous system disorders14/8813/86
DiarrhoeaGastrointestinal disorders2/889/86
Injection site painGeneral disorders3/888/86
Myasthenia gravisNervous system disorders5/888/86
Urinary tract infectionInfections and infestations4/887/86
ContusionInjury, poisoning and procedural complications3/887/86
Lipase increasedInvestigations1/886/86
NasopharyngitisInfections and infestations3/885/86
Amylase increasedInvestigations2/885/86

Baseline characteristics

The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

Age, Categorical
Age, Categorical(Participants)PlaceboZilucoplan 0.3 mg/kgTotal
<=18 years000
Between 18 and 65 years6264126
>=65 years262248
Age, Continuous
Age, Continuous(years)PlaceboZilucoplan 0.3 mg/kgTotal
Mean53.3 ± 15.752.6 ± 14.653.0 ± 15.1
Sex: Female, Male
Sex: Female, Male(Participants)PlaceboZilucoplan 0.3 mg/kgTotal
Female475299
Male413475
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)PlaceboZilucoplan 0.3 mg/kgTotal
American Indian/Alaska native101
Asian14721
Black7613
Native Hawaiian or other Pacific Islander000
White6266128
Other/Mixed000
Missing4711
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)PlaceboZilucoplan 0.3 mg/kgTotal
Hispanic or Latino5712
Not Hispanic or Latino7972151
Missing4711
07

Study locations

77 sites
  • Site 41: Diagnostic and Medical Clinic
    Mobile, Alabama 36604, United States
  • Site 116: Neuromuscular Clinic and Research Center
    Phoenix, Arizona 85028, United States
  • Site 4: University of Southern California
    Los Angeles, California 90033, United States
  • Site 31: University of California Irvine
    Orange, California 92868, United States
  • Site 220: Investigator Site
    Pasadena, California 91101, United States
  • Site 160: Forbes Norris MDA/ALS Research and Treatment Center
    San Francisco, California 94115, United States
  • Site 24: Yale University
    New Haven, Connecticut 06510, United States
  • Site 27: George Washington University
    Washington D.C., District of Columbia 20037, United States
  • Site 182: Gelasio Baras Neurology
    Miami, Florida 33175, United States
  • Site 25: University of South Florida
    Tampa, Florida 33612, United States
  • Site 135: Augusta University Medical Center
    Augusta, Georgia 30912, United States
  • Site 176: Hawaii Pacific Neuroscience
    Honolulu, Hawaii 96817, United States
  • Site 188: North Shore Medical Group - Glenview
    Glenview, Illinois 60026-1339, United States
  • Site 156: Indiana University Health Neuroscience Center
    Indianapolis, Indiana 46202, United States
  • Site 32: Kansas University Medical Center Research Institute
    Kansas City, Kansas 66160, United States
  • Site 221: Neurology Center of New England
    Foxborough, Massachusetts 02035, United States
  • Site 33: Detroit medical Center - University Health Center
    Detroit, Michigan 48202, United States
  • Site 49: Michigan State University
    East Lansing, Michigan 48824, United States
  • Site 127: University of Minnesota
    Minneapolis, Minnesota 55455, United States
  • Site 134: Neurology and Sleep Disorders Clinic
    Columbia, Missouri 65212, United States
  • Site 117: Las Vegas Clinic
    Las Vegas, Nevada 89145, United States
  • Site 123: Northwell Health Neuroscience Institute
    Great Neck, New York 11021, United States
  • Site 23: Hospital for Special Surgery
    New York, New York 10021, United States
  • Site 47: Mount Sinai Hospital
    New York, New York 10029, United States
  • Site 22: University of North Carolina
    Chapel Hill, North Carolina 27599, United States
  • Site 15: Duke University
    Durham, North Carolina 27710, United States
  • Site 122: Cleveland Clinic
    Cleveland, Ohio 44195, United States
  • Site 38: Ohio State University
    Columbus, Ohio 43210, United States
  • Site 40: Allegheny Neurological Associates
    Pittsburgh, Pennsylvania 15212, United States
  • Site 128: Medical University of South Carolina
    Charleston, South Carolina 29425, United States
  • Site 185: Neurology Clinic Cordova
    Cordova, Tennessee 38018, United States
  • Site 131: Austin Neuromuscular Center
    Austin, Texas 78756, United States
  • Site 19: University of Texas Southwestern
    Dallas, Texas 75390, United States
  • Site 39: University of Utah
    Salt Lake City, Utah 84132, United States
  • Site 164: University of Virginia Health System
    Charlottesville, Virginia 22908, United States
  • Site 154: University of Washington
    Seattle, Washington 98195, United States
  • Site 45: Center for Neurological Disorders
    Milwaukee, Wisconsin 53215, United States
  • Site 44: London Health Sciences Centre University Hospital
    London, Ontario N6A 5A5, Canada
  • Site 11: Montreal Neurological Institute and Hospital (McGill University)
    Montreal, Quebec H3A 2B4, Canada
  • Site 191: Centre Hosptitalier Universitaire d'Angers
    Angers, France
  • Site 204: Centre Hospitalier Régional Universitaire de Lille
    Lille, France
  • Site 118: Hôpital Pasteur
    Nice, 06000, France
  • Site 105: Pitié-Salpêtrière University Hospital
    Paris, 75013, France
  • Site 137: Les Hôpitaux Universitaires de Strasbourg
    Strasbourg, 67091, France
  • Site 150: Universitätsmedizin Göttingen
    Göttingen, 37075, Germany
  • Site 129: Universitätsklinikum Tübingen
    Tübingen, 72076, Germany
  • Site 126: Fondazione IRCCS Istituto Neurologico Carlo Besta
    Milan, 20133, Italy
  • Site 132: Università Cattolica del Sacro Cuore - Campus di Milano
    Roma, 20123, Italy
  • Site 169: International University of Health and Welfare Narita Hospital
    Narita, Chiba 286-8520, Japan
  • Site 151: Chiba University Hospital
    Chiba, 260-8677, Japan
  • Site 136: General Hanamaki Hospital
    Iwata, 025-0075, Japan
  • Site 179: Kagawa University Hospital - Collagen disease/Rheumatic int
    Kita-gun, Japan
  • Site 146: Nagasaki University Hospital
    Nagasaki, 852-8501, Japan
  • Site 152: Hokkaido Medical Center
    Sapporo, 063-0005, Japan
  • Site 144: Sendai Medical Center
    Sendai, 983-8520, Japan
  • Site 153: Toho University Ohashi Medical Center
    Tokyo, 153-8515, Japan
  • Site 163: Tokyo Medical University Hospital
    Tokyo, 160-0023, Japan
  • Site 141: Keio University Hospital
    Tokyo, 160-8582, Japan
  • Site 165: Osaka University Hospital
    Tokyo, 565-0871, Japan
  • Site 140: Haukeland University Hospital / Health Bergen
    Bergen, 5021, Norway
  • Site 143: Oslo Universitetssykehus
    Oslo, 0450, Norway
  • Site 209: Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD
    Poznan, Greater Poland Voivodeship 61-853, Poland
  • Site 192: Krakowski Szpital Specjalistyczny im. Jana Pawła II
    Krakow, Lesser Poland Voivodeship 31-202, Poland
  • Site 205: Prywatny Gabinet Lekarski Urszula Chyrchel-Paszkiewicz
    Lublin, Lublin Voivodeship 20-093, Poland
  • Site 194: Twoja Przychodnia - Centrum Medyczne Nowa Sól
    Nowa Sól, Lubusz Voivodeship 67-100, Poland
  • Site 214: AmiCare Centrum Medyczne
    Lodz, Lódzkie 90-644, Poland
  • Site 201: Centrum Medyczne Pratia - Warszawa
    Warsaw, Masovian Voivodeship 01-868, Poland
  • Site 195: Wielospecjalistyczna Poradnia Lekarska Synapsis
    Katowice, Silesian Voivodeship 40-123, Poland
  • Site 213: Niepubliczny Zakład Opieki Zdrowotnej NOVO-MED
    Katowice, Silesian Voivodeship 40-650, Poland
  • Site 193: Krakowska Akademia Neurologii - Centrum Neurologii Kliniczne
    Krakow, Poland
  • Site 211: Specjalistyczne Gabinety Sp. z o.o.
    Krakow, Poland
  • Site 210: Clinhouse Centrum Medyczne
    Lublin, 20-093, Poland
  • Site 133: Hospital Universitari Vall d'Hebrón
    Barcelona, 08035, Spain
  • Site 168: Hospital de la Santa Creu i Sant Pau
    Barcelona, Spain
  • Site 138: Hospital Universitario de Basurto
    Bilbao, 48013, Spain
  • Site 119: Oxford University Hospitals NHS Foundation Trust
    Oxford, OX3 9DU, United Kingdom
  • Site 130: Royal Hallamshire Hospital
    Sheffield, S10 2JF, United Kingdom
08

References and documents

Publications

  • 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. PubMed 37059508 ↗
  • Weiss MD, Freimer M, Leite MI, Maniaol A, Utsugisawa K, Bloemers J, Boroojerdi B, Howard E, Savic N, Howard JF Jr. Improvement of fatigue in generalised myasthenia gravis with zilucoplan. J Neurol. 2024 May;271(5):2758-2767. doi: 10.1007/s00415-024-12209-3. Epub 2024 Feb 24. PubMed 38400914 ↗
  • de la Borderie G, Chimits D, Boroojerdi B, Brock M, Duda PW, Grimson F, Mahoney P, Strimenopoulou F, Cutter G, Aban I, Brauner S, Petersson M, Howard JF Jr, Bennett N. Maintenance of zilucoplan efficacy in patients with generalised myasthenia gravis up to 24 weeks: a model-informed analysis. Ther Adv Neurol Disord. 2024 Sep 21;17:17562864241279125. doi: 10.1177/17562864241279125. eCollection 2024. PubMed 39314260 ↗
  • Hewamadduma C, Freimer M, Genge A, Leite MI, Utsugisawa K, Vu T, Boroojerdi B, Grimson F, Savic N, Vanderkelen M, Howard JF Jr; RAISE-XT study team. Changes in corticosteroid and non-steroidal immunosuppressive therapy with long-term zilucoplan treatment in generalized myasthenia gravis. J Neurol. 2025 Jun 12;272(7):457. doi: 10.1007/s00415-025-13113-0. PubMed 40504283 ↗
  • Utsugisawa K, Masuda M, Boroojerdi B, Grimson F, Howard JF Jr; RAISE Study Team. Efficacy of zilucoplan in patients with generalised myasthenia gravis who have not previously received immunoglobulin or plasma exchange: A subgroup analysis from the Phase 3 RAISE study. J Neurol Sci. 2025 Jul 15;474:123550. doi: 10.1016/j.jns.2025.123550. Epub 2025 May 16. PubMed 40450840 ↗

Study documents

  • Study protocol · Dec 18, 2020
  • Statistical analysis plan · Dec 6, 2021

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — 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.

Supporting information: Study protocol, Sap, Csr

09

Registry details

Key details

Study ID
NCT04115293
Lead sponsor
Ra Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Oct 4, 2019
Start date
Sep 17, 2019
Primary completion
Dec 30, 2021
Completion
Dec 30, 2021
Results posted
Jan 17, 2023
Last update
Jul 16, 2025

Study contacts

UCB Cares
study director · 0018445992273 (UCB)

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jul 2025. You cannot join it, but the record below documents what was studied.

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