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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04115293
Recruitment Status : Completed
First Posted : October 4, 2019
Results First Posted : January 17, 2023
Last Update Posted : May 2, 2024
Sponsor:
Information provided by (Responsible Party):
UCB Pharma ( Ra Pharmaceuticals, Inc. )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Myasthenia Gravis, Generalized
Interventions Drug: zilucoplan (RA101495)
Drug: Placebo
Enrollment 174
Recruitment Details The study started to enroll participants in September 2019 and concluded in December 2021.
Pre-assignment Details The Participant flow refers to the Randomized Set.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period. Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Period Title: Overall Study
Started 88 86
Completed 84 82
Not Completed 4 4
Reason Not Completed
Adverse Event             0             2
Withdrawal by Subject             2             1
Physician Decision             1             0
Death             1             1
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg Total
Hide Arm/Group Description Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period. Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period. Total of all reporting groups
Overall Number of Baseline Participants 88 86 174
Hide Baseline Analysis Population Description
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  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 88 participants 86 participants 174 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
62
  70.5%
64
  74.4%
126
  72.4%
>=65 years
26
  29.5%
22
  25.6%
48
  27.6%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 88 participants 86 participants 174 participants
53.3  (15.7) 52.6  (14.6) 53.0  (15.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 88 participants 86 participants 174 participants
Female
47
  53.4%
52
  60.5%
99
  56.9%
Male
41
  46.6%
34
  39.5%
75
  43.1%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
 Number Analyzed 88 participants 86 participants 174 participants
American Indian/Alaska native
1
   1.1%
0
   0.0%
1
   0.6%
Asian
14
  15.9%
7
   8.1%
21
  12.1%
Black
7
   8.0%
6
   7.0%
13
   7.5%
Native Hawaiian or other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
White
62
  70.5%
66
  76.7%
128
  73.6%
Other/Mixed
0
   0.0%
0
   0.0%
0
   0.0%
Missing
4
   4.5%
7
   8.1%
11
   6.3%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
 Number Analyzed 88 participants 86 participants 174 participants
Hispanic or Latino
5
   5.7%
7
   8.1%
12
   6.9%
Not Hispanic or Latino
79
  89.8%
72
  83.7%
151
  86.8%
Missing
4
   4.5%
7
   8.1%
11
   6.3%
1.Primary Outcome
Title Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description:
Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Number of Participants Analyzed 88 86
Least Squares Mean (95% Confidence Interval)
Unit of Measure: score on a scale
-2.30
(-3.17 to -1.43)
-4.39
(-5.28 to -3.50)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Zilucoplan 0.3 mg/kg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method MMRM ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -2.09
Confidence Interval (2-Sided) 95%
-3.24 to -0.95
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description:
Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Number of Participants Analyzed 88 86
Least Squares Mean (95% Confidence Interval)
Unit of Measure: score on a scale
-3.25
(-4.32 to -2.17)
-6.19
(-7.29 to -5.08)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Zilucoplan 0.3 mg/kg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method MMRM ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -2.94
Confidence Interval (2-Sided) 95%
-4.39 to -1.49
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description:
Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Number of Participants Analyzed 88 86
Least Squares Mean (95% Confidence Interval)
Unit of Measure: score on a scale
-5.42
(-6.98 to -3.86)
-8.62
(-10.22 to -7.01)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Zilucoplan 0.3 mg/kg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0023
Comments [Not Specified]
Method MMRM ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -3.20
Confidence Interval (2-Sided) 95%
-5.24 to -1.16
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description:
Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Number of Participants Analyzed 88 86
Least Squares Mean (95% Confidence Interval)
Unit of Measure: score on a scale
-3.16
(-4.65 to -1.67)
-5.65
(-7.17 to -4.12)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Zilucoplan 0.3 mg/kg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0128
Comments [Not Specified]
Method MMRM ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -2.49
Confidence Interval (2-Sided) 95%
-4.45 to -0.54
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description:
Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Number of Participants Analyzed 88 86
Median (Full Range)
Unit of Measure: Days
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
[1]
The median time to first receipt of rescue therapy was not estimated due to the less number of events.
6.Secondary Outcome
Title Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description:
Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Number of Participants Analyzed 88 86
Measure Type: Number
Unit of Measure: percentage of participants
5.8 14.0
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Zilucoplan 0.3 mg/kg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0885
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 2.608
Confidence Interval (2-Sided) 95%
0.866 to 7.860
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description:
Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Number of Participants Analyzed 88 86
Measure Type: Number
Unit of Measure: percentage of participants
46.1 73.1
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Zilucoplan 0.3 mg/kg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 3.184
Confidence Interval (2-Sided) 95%
1.662 to 6.101
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description:
Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Number of Participants Analyzed 88 86
Measure Type: Number
Unit of Measure: percentage of participants
33.0 58.0
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Zilucoplan 0.3 mg/kg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0012
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 2.865
Confidence Interval (2-Sided) 95%
1.518 to 5.409
Estimation Comments [Not Specified]
9.Secondary Outcome
Title Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
Hide 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])
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set (SS) included all participants who received at least 1 dose of study drug based on the actual study treatment received.
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description:
Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Number of Participants Analyzed 88 86
Measure Type: Number
Unit of Measure: percentage of participants
70.5 76.7
Time Frame From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up])
Adverse Event Reporting 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.
 
Arm/Group Title Placebo Zilucoplan 0.3 mg/kg
Hide Arm/Group Description Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period. Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
All-Cause Mortality
Placebo Zilucoplan 0.3 mg/kg
Affected / at Risk (%) Affected / at Risk (%)
Total   1/88 (1.14%)      1/86 (1.16%)    
Hide Serious Adverse Events
Placebo Zilucoplan 0.3 mg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   13/88 (14.77%)      11/86 (12.79%)    
Blood and lymphatic system disorders     
Anaemia * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
Gastrointestinal disorders     
Vomiting * 1  1/88 (1.14%)  1 0/86 (0.00%)  0
Aphthous ulcer * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
Infections and infestations     
Oesophageal candidiasis * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
Oral candidiasis * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
COVID-19 * 1  2/88 (2.27%)  2 1/86 (1.16%)  1
COVID-19 pneumonia * 1  2/88 (2.27%)  2 1/86 (1.16%)  1
Herpes simplex meningoencephalitis * 1  1/88 (1.14%)  2 0/86 (0.00%)  0
Pneumonia * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
Sepsis * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
Investigations     
Lipase increased * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Metastases to meninges * 1  1/88 (1.14%)  1 0/86 (0.00%)  0
Basal cell carcinoma * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
Nervous system disorders     
Cerebral haemorrhage * 1  1/88 (1.14%)  1 0/86 (0.00%)  0
Cerebrovascular accident * 1  1/88 (1.14%)  1 0/86 (0.00%)  0
Myasthenia gravis * 1  5/88 (5.68%)  6 2/86 (2.33%)  3
Pregnancy, puerperium and perinatal conditions     
Hyperemesis gravidarum * 1  1/88 (1.14%)  1 0/86 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Chronic obstructive pulmonary disease * 1  1/88 (1.14%)  1 0/86 (0.00%)  0
Pulmonary embolism * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
Skin and subcutaneous tissue disorders     
Angioedema * 1  0/88 (0.00%)  0 1/86 (1.16%)  1
1
Term from vocabulary, MedDRA 24
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Zilucoplan 0.3 mg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   34/88 (38.64%)      41/86 (47.67%)    
Gastrointestinal disorders     
Diarrhoea * 1  2/88 (2.27%)  2 9/86 (10.47%)  9
Vomiting * 1  5/88 (5.68%)  5 3/86 (3.49%)  3
General disorders     
Injection site bruising * 1  8/88 (9.09%)  11 14/86 (16.28%)  18
Injection site pain * 1  3/88 (3.41%)  3 8/86 (9.30%)  9
Infections and infestations     
Urinary tract infection * 1  4/88 (4.55%)  4 7/86 (8.14%)  7
Nasopharyngitis * 1  3/88 (3.41%)  3 5/86 (5.81%)  5
Injury, poisoning and procedural complications     
Contusion * 1  3/88 (3.41%)  3 7/86 (8.14%)  8
Investigations     
Lipase increased * 1  1/88 (1.14%)  1 6/86 (6.98%)  6
Amylase increased * 1  2/88 (2.27%)  2 5/86 (5.81%)  5
Nervous system disorders     
Headache * 1  14/88 (15.91%)  19 13/86 (15.12%)  16
Myasthenia gravis * 1  5/88 (5.68%)  6 8/86 (9.30%)  10
Skin and subcutaneous tissue disorders     
Rash * 1  5/88 (5.68%)  9 3/86 (3.49%)  3
1
Term from vocabulary, MedDRA 24
*
Indicates events were collected by non-systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: UCB
Organization: Cares
Phone: 001 844 599 2273
EMail: UCBCares@ucb.com
Publications of Results:
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.
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.
Layout table for additonal information
Responsible Party: UCB Pharma ( Ra Pharmaceuticals, Inc. )
ClinicalTrials.gov Identifier: NCT04115293    
Other Study ID Numbers: RA101495-02.301
First Submitted: October 2, 2019
First Posted: October 4, 2019
Results First Submitted: December 19, 2022
Results First Posted: January 17, 2023
Last Update Posted: May 2, 2024