Myasthenia Gravis, GeneralizedPhase 3RAISENCT04115293
Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
A Phase 3 study testing zilucoplan (RA101495) in people with Myasthenia Gravis, Generalized, run by Ra Pharmaceuticals, Inc. at 77 sites in 10 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 74 years
- Conditions the study is for
- Myasthenia Gravis, Generalized
- What is being tested?
- zilucoplan (RA101495)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 174 participants
- Where
- 77 sitesin 10 countries
- Study dates
- 17 Sep 2019to 30 Dec 2021
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 74 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
77 sites in 10 countries.
77 sites in 10 countries
United States37 sites
Site 135: Augusta University Medical Center
Augusta, Georgia, 30912
Site 131: Austin Neuromuscular Center
Austin, Texas, 78756
Site 22: University of North Carolina
Chapel Hill, North Carolina, 27599
Site 128: Medical University of South Carolina
Charleston, South Carolina, 29425
Site 164: University of Virginia Health System
Charlottesville, Virginia, 22908
Site 122: Cleveland Clinic
Cleveland, Ohio, 44195
Site 134: Neurology and Sleep Disorders Clinic
Columbia, Missouri, 65212
Site 38: Ohio State University
Columbus, Ohio, 43210
Site 185: Neurology Clinic Cordova
Cordova, Tennessee, 38018
Site 19: University of Texas Southwestern
Dallas, Texas, 75390
Site 33: Detroit medical Center - University Health Center
Detroit, Michigan, 48202
Site 15: Duke University
Durham, North Carolina, 27710
Site 49: Michigan State University
East Lansing, Michigan, 48824
Site 221: Neurology Center of New England
Foxborough, Massachusetts, 02035
Site 188: North Shore Medical Group - Glenview
Glenview, Illinois, 60026-1339
Site 123: Northwell Health Neuroscience Institute
Great Neck, New York, 11021
Site 176: Hawaii Pacific Neuroscience
Honolulu, Hawaii, 96817
Site 156: Indiana University Health Neuroscience Center
Indianapolis, Indiana, 46202
Site 32: Kansas University Medical Center Research Institute
Kansas City, Kansas, 66160
Site 117: Las Vegas Clinic
Las Vegas, Nevada, 89145
Site 4: University of Southern California
Los Angeles, California, 90033
Site 182: Gelasio Baras Neurology
Miami, Florida, 33175
Site 45: Center for Neurological Disorders
Milwaukee, Wisconsin, 53215
Site 127: University of Minnesota
Minneapolis, Minnesota, 55455
Site 41: Diagnostic and Medical Clinic
Mobile, Alabama, 36604
Site 24: Yale University
New Haven, Connecticut, 06510
Site 23: Hospital for Special Surgery
New York, New York, 10021
Site 47: Mount Sinai Hospital
New York, New York, 10029
Site 31: University of California Irvine
Orange, California, 92868
Site 220: Investigator Site
Pasadena, California, 91101
Site 116: Neuromuscular Clinic and Research Center
Phoenix, Arizona, 85028
Site 40: Allegheny Neurological Associates
Pittsburgh, Pennsylvania, 15212
Site 39: University of Utah
Salt Lake City, Utah, 84132
Site 160: Forbes Norris MDA/ALS Research and Treatment Center
San Francisco, California, 94115
Site 154: University of Washington
Seattle, Washington, 98195
Site 25: University of South Florida
Tampa, Florida, 33612
Site 27: George Washington University
Washington D.C., District of Columbia, 20037
Japan11 sites
Site 151: Chiba University Hospital
Chiba, 260-8677
Site 136: General Hanamaki Hospital
Iwata, 025-0075
Site 179: Kagawa University Hospital - Collagen disease/Rheumatic int
Kita-gun
Site 146: Nagasaki University Hospital
Nagasaki, 852-8501
Site 169: International University of Health and Welfare Narita Hospital
Narita, Chiba, 286-8520
Site 152: Hokkaido Medical Center
Sapporo, 063-0005
Site 144: Sendai Medical Center
Sendai, 983-8520
Site 153: Toho University Ohashi Medical Center
Tokyo, 153-8515
Site 163: Tokyo Medical University Hospital
Tokyo, 160-0023
Site 141: Keio University Hospital
Tokyo, 160-8582
Site 165: Osaka University Hospital
Tokyo, 565-0871
Poland11 sites
Site 195: Wielospecjalistyczna Poradnia Lekarska Synapsis
Katowice, Silesian Voivodeship, 40-123
Site 213: Niepubliczny Zakład Opieki Zdrowotnej NOVO-MED
Katowice, Silesian Voivodeship, 40-650
Site 192: Krakowski Szpital Specjalistyczny im. Jana Pawła II
Krakow, Lesser Poland Voivodeship, 31-202
Site 193: Krakowska Akademia Neurologii - Centrum Neurologii Kliniczne
Krakow
Site 211: Specjalistyczne Gabinety Sp. z o.o.
Krakow
Site 214: AmiCare Centrum Medyczne
Lodz, Lódzkie, 90-644
Site 205: Prywatny Gabinet Lekarski Urszula Chyrchel-Paszkiewicz
Lublin, Lublin Voivodeship, 20-093
Site 210: Clinhouse Centrum Medyczne
Lublin, 20-093
Site 194: Twoja Przychodnia - Centrum Medyczne Nowa Sól
Nowa Sól, Lubusz Voivodeship, 67-100
Site 209: Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD
Poznan, Greater Poland Voivodeship, 61-853
Site 201: Centrum Medyczne Pratia - Warszawa
Warsaw, Masovian Voivodeship, 01-868
France5 sites
Site 191: Centre Hosptitalier Universitaire d'Angers
Angers
Site 204: Centre Hospitalier Régional Universitaire de Lille
Lille
Site 118: Hôpital Pasteur
Nice, 06000
Site 105: Pitié-Salpêtrière University Hospital
Paris, 75013
Site 137: Les Hôpitaux Universitaires de Strasbourg
Strasbourg, 67091
Spain3 sites
Site 133: Hospital Universitari Vall d'Hebrón
Barcelona, 08035
Site 168: Hospital de la Santa Creu i Sant Pau
Barcelona
Site 138: Hospital Universitario de Basurto
Bilbao, 48013
Canada2 sites
Site 44: London Health Sciences Centre University Hospital
London, Ontario, N6A 5A5
Site 11: Montreal Neurological Institute and Hospital (McGill University)
Montreal, Quebec, H3A 2B4
Germany2 sites
Site 150: Universitätsmedizin Göttingen
Göttingen, 37075
Site 129: Universitätsklinikum Tübingen
Tübingen, 72076
Italy2 sites
Site 126: Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133
Site 132: Università Cattolica del Sacro Cuore - Campus di Milano
Roma, 20123
Norway2 sites
Site 140: Haukeland University Hospital / Health Bergen
Bergen, 5021
Site 143: Oslo Universitetssykehus
Oslo, 0450
United Kingdom2 sites
Site 119: Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 9DU
Site 130: Royal Hallamshire Hospital
Sheffield, S10 2JF
About this study
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
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.
- Conditions
- Myasthenia Gravis, Generalized
- Being tested
- zilucoplan (RA101495), Placebo
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
zilucoplan (RA101495)Drug
Daily subcutaneous (SC) injection
PlaceboDrug
Daily subcutaneous (SC) injection
| Study group | Role | What they receive |
|---|---|---|
| 0.3 mg/kg zilucoplan (RA101495) | Experimental | Not described Receives: Drug: zilucoplan (RA101495) |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From Baseline to End of Treatment (Week 12)
Secondary outcomes (8)
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.
Measured: From Baseline to End of Treatment (Week 12)
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.
Measured: From Baseline to End of Treatment (Week 12)
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.
Measured: From Baseline to End of Treatment (Week 12)
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.
Measured: From Baseline to End of Treatment (Week 12)
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.
Measured: End of Treatment (Week 12)
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.
Measured: End of Treatment (Week 12)
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.
Measured: End of Treatment (Week 12)
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.
Measured: From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up])
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2019
- Study start17 Sep 2019
- Primary completion30 Dec 2021
- Study completion30 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Myasthenia Gravis, GeneralizedStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 174 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ra Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 0018445992273 (UCB)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04115293
- Sponsor's protocol ID
- RA101495-02.301
- Acronym
- RAISE
- Status last verified
- Jul 2025
- First submitted
- 2 Oct 2019
- Last updated
- 16 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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.
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 37059508Weiss 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 38400914de 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 39314260Hewamadduma 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 40504283Utsugisawa 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 at a glance
From the registry record, last updated by the sponsor on 16 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04115293
- Acronym / sponsor ID
- RAISE / RA101495-02.301
- Conditions
- Myasthenia Gravis, Generalized
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 174 participants (actual)
- Lead sponsor
- Ra Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Sep 2019 (actual)
- Primary completion
- 30 Dec 2021 (actual)
- Study completion
- 30 Dec 2021 (actual)
- First posted
- 4 Oct 2019 (actual)
- Results
- Posted 17 Jan 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 0.3 mg/kg zilucoplan (RA101495) | Experimental | Drug: zilucoplan (RA101495) |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| zilucoplan (RA101495)Daily subcutaneous (SC) injection | Drug | 0.3 mg/kg zilucoplan (RA101495) |
| PlaceboDaily subcutaneous (SC) injection | Drug | Placebo |
Primary outcome
Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score
Time frame: From Baseline to End of Treatment (Week 12)
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.
8 secondary outcomes
Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score
Time frame: From Baseline to End of Treatment (Week 12)
Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score
Time frame: From Baseline to End of Treatment (Week 12)
Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score
Time frame: From Baseline to End of Treatment (Week 12)
Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period
Time frame: From Baseline to End of Treatment (Week 12)
Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy
Time frame: End of Treatment (Week 12)
Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy
Time frame: End of Treatment (Week 12)
Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12
Time frame: End of Treatment (Week 12)
Percentage of Participants With Treatment-emergent Adverse Events (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])
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 37059508Howard 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 publication: PubMed 38400914Weiss 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.
- Results publication: PubMed 39314260de 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.
- Results publication: PubMed 40504283Hewamadduma 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.
- Derived from this study: PubMed 40450840Utsugisawa 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.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Cares study director
0018445992273 (UCB)
77 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing zilucoplan (RA101495) in people with Myasthenia Gravis, Generalized, run by Ra Pharmaceuticals, Inc. at 77 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 77 sites in United States, Japan, Poland, France, Spain and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Jan 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score (time frame: From Baseline to End of Treatment (Week 12)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04115293, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2025.