The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
45
Daily subcutaneous (SC) injection
Daily subcutaneous (SC) injection
Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score
The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for myasthenia gravis (MG). The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.
Time frame: From Baseline to Week 12
Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale
The MG-ADL is a brief 8-item survey designed to evaluate MG symptom severity. The scale consists of 8 items each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the 8 individual scores with range of 0-24. Higher scores are associated with more severe symptoms of MG.
Time frame: From Baseline to Week 12
Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life 15r (MG-QOL15r) Survey
The MG-QOL15r is a 15-item survey that was designed to assess quality of life in participants with MG. The survey consisted of 15 questions and the corresponding responses were each scored on a 0-2 point scale (0=Not much at all, 1=Somewhat, 2=Very Much). The total score is the sum of the 15 individual item scores with a range of 0-30. Higher scores indicate more severe impact of the disease on aspects of the participant's life.
Time frame: From Baseline to Week 12
Main Portion: Change From Baseline to Week 12 in the MG Composite Scale Total Score
The MG Composite is a 10-item scale that has been used to measure the clinical status of participants with MG, in order to evaluate treatment response. It consists of 10 items which included ptosis (score range=0 to 3), double vision on lateral gaze left/right/both (score range=0 to 4), eye closure (score range=0 to 2), talking (score range=0 to 6), chewing (score range=0 to 6), swallowing (score range=0 to 6), breathing (score range=0 to 9), neck flexion or extension (score range=0 to 4), shoulder abduction (score range=0 to 5) and hip flexion (score range= 0 to 5). The total score is the sum of the 10 individual scores with a range of 0-50. Higher scores in the MG Composite indicate more severe impairment due to the disease.
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...and 20 more locations
Time frame: From Baseline to Week 12
Main Portion: Percentage of Participants With >= 3-point Reduction in QMG Total Score at Week 12
The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.
Time frame: Week 12
Main Portion: Percentage of Participants Who Required Rescue Therapy Over the 12-week Treatment Period
Percentage of participants who used at least 1 dose of rescue medication were reported.
Time frame: Up to Week 12
Main Portion: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A serious adverse event (SAE) is any untoward medical occurrence that at any dose resulted in death, life-threatening, significant or persistent disability/incapacity, congenital anomaly/birth defect, important medical event, initial inpatient hospitalization or prolongation of hospitalization.
Time frame: From Baseline to Week 12
Main Portion: Change From Baseline in Sheep Red Blood Cell (sRBC) Lysis Assay at Week 12 (Pre-dose)
A BioTek ELx800 automated microplate reader is used to measure the optical density at 415 nanometer (nm) of human plasma samples to calculate the percent lysis of sheep erythrocytes that has occurred as a result of total complement activity. The measure of complement activity is determined by the degree of hemolysis of the erythrocytes.
Time frame: Baseline and Week 12 (Pre-dose)
Main Portion: Change From Baseline in C5 Levels at Week 12 (Pre-dose)
Blood samples were collected from participants to assess Complement Component 5C levels.
Time frame: Baseline and Week 12 (Pre-dose)
Main Portion: Plasma Concentration of RA101495 and Its Major Metabolites
RA102758 and RA103488 are the metabolites of RA101495.
Time frame: 1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12
Main Portion: Maximum Plasma Concentration (Cmax) on Day 1
Cmax is defined as the maximum observed plasma concentration.
Time frame: Pre-dose, 1, 3 and 6 hours postdose on Day 1
Main Portion: Time Corresponding to Cmax (Tmax) on Day 1
Tmax is defined as the time to observe maximum plasma concentration.
Time frame: Pre-dose, 1, 3 and 6 hours postdose on Day 1
Main Portion: Metabolites (RA102758 and RA103488) to Parent Ratio
RA102758 and RA103488 are the metabolites of RA101495.
Time frame: Pre-dose, 1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12