Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of =6 Eosinophils Per High-power Field (Eos/Hpf) in All Three Regions at Week 24 |
Esophageal intraepithelial eosinophil count obtained by esophageal endoscopy with biopsies (all 3 esophageal regions: proximal, mid, and distal). |
At week 24 |
|
| Primary |
Absolute Change From Baseline in Dysphagia Symptom Questionnaire (DSQ) Total Score at Week 24 |
The DSQ is used to measure the frequency and intensity of dysphagia. DSQ scores can range from 0 to 84, with a lower score indicating less-frequent or less-severe dysphagia. |
Baseline and week 24 |
|
| Secondary |
Percent Change From Baseline in Peak Esophageal Intraepithelial Eosinophil Count (Eos/Hpf) in All Three Regions at Week 24 |
Esophageal intraepithelial eosinophil count obtained by esophageal endoscopy with biopsies (all 3 esophageal regions: proximal, mid, and distal). A greater esophageal intraepithelial eosinophil count from baseline indicates worsening disease. |
Baseline and week 24 |
|
| Secondary |
Percent Change From Baseline in DSQ Total Score at Week 24 |
The DSQ is used to measure the frequency and intensity of dysphagia. DSQ scores can range from 0 to 84, with a lower score indicating less-frequent or less-severe dysphagia. |
Baseline and week 24 |
|
| Secondary |
Absolute Change From Baseline in Eosinophilic Esophagitis Histology Scoring System (EoEHSS) Mean Grade Score at Week 24 |
Severity (grade) and extent (stage) of esophageal abnormalities were scored by blinded, central pathologists using a 4-point scale (0 normal; 3 maximum change) for eight features: eosinophil density, basal zone hyperplasia, eosinophil abscesses, eosinophil surface layering, dilated intercellular spaces, surface epithelial alteration, dyskeratotic epithelial cells and lamina propria fibrosis (absent/present). Higher score indicates greater severity and extent of histological abnormalities. |
Baseline and week 24 |
|
| Secondary |
Absolute Change From Baseline in EoEHSS Mean Stage Score at Week 24 |
Severity (grade) and extent (stage) of esophageal abnormalities were scored by blinded, central pathologists using a 4-point scale (0 normal; 3 maximum change) for eight features: eosinophil density, basal zone hyperplasia, eosinophil abscesses, eosinophil surface layering, dilated intercellular spaces, surface epithelial alteration, dyskeratotic epithelial cells and lamina propria fibrosis (absent/present). Higher score indicates greater severity and extent of histological abnormalities. |
Baseline and week 24 |
|
| Secondary |
Absolute Change From Baseline in EoE Endoscopic Reference Total Score (EoE-EREFS) at Week 24 |
EoE esophageal characteristics analyzed based on the EoE-EREFS, a scoring system for inflammatory and remodeling features of disease. The overall total score ranges from 0 to 18 with higher number indicating worse disease. |
Baseline and week 24 |
|
| Secondary |
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of <15 Eos/Hpf in All Three Regions at Week 24 |
Esophageal intraepithelial eosinophil count obtained by esophageal endoscopy with biopsies (all 3 esophageal regions: proximal, mid, and distal). |
At week 24 |
|
| Secondary |
Normalized Enrichment Score (NES) for the Relative Change From Baseline in the EoE Diagnostic Panel (EDP) at Week 24 |
NES reflects the degree to which the activity level of a set of disease transcripts is overrepresented at the extremes (top or bottom) of the entire ranked list of transcripts within a sample and is normalized by accounting for the number of transcripts in the set. An NES of 0 indicates no change from baseline, a negative score reflects a reduction in the disease score (more like normal) and a positive score reflects worsening (more active disease). |
Baseline and week 24 |
|
| Secondary |
NES for the Relative Change From Baseline in the Type 2 Inflammation Signature (T2INF) at Week 24 |
NES reflects the degree to which the activity level of a set of disease transcripts is overrepresented at the extremes (top or bottom) of the entire ranked list of transcripts within a sample and is normalized by accounting for the number of transcripts in the set. An NES of 0 indicates no change from baseline, a negative score reflects a reduction in the disease score (more like normal) and a positive score reflects worsening (more active disease). |
Baseline and week 24 |
|
| Secondary |
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of =1 Eos/Hpf in All Three Regions at Week 24 |
Esophageal intraepithelial eosinophil count obtained by esophageal endoscopy with biopsies (all 3 esophageal regions: proximal, mid, and distal). |
At week 24 |
|
| Secondary |
Absolute Change From Baseline in Health-related Quality of Life (QoL) Average Score as Measured by EoE Impact Questionnaire (EoE-IQ) at Week 24 |
The EoE-IQ measures impact of EoE on emotional, social, work & school, & sleep aspects. Participants were asked to respond to 11 questions based on experience living with EoE during past 7 days. Response to each item is on a 5-point scale (1=Not at all [impacted] 2=A little, 3=Somewhat, 4=Quite a bit, 5=Extremely [impacted]). The average score is the sum of non-missing responses divided by the number of items with non-missing responses. The average score can range from 1 to 5; a higher score is indicative of a more negative impact. |
Baseline and week 24 |
|
| Secondary |
Absolute Change From Baseline in Severity of EoE Symptoms Other Than Dysphagia as Measured by EoE Symptom Questionnaire (EoE-SQ) at Week 24 |
The EoE-SQ asks about symptoms that participants with EoE may have (chest pain, stomach pain, burning feeling in chest, food or liquid coming back up into throat, throwing up) during the past 7 days. Response to the severity of each symptom based on the worst experience in the past 7 days is on a scale of 0 to 10 (higher is worse). The EoE-SQ severity score is calculated as the sum of the severity scores from questions 1 to 3 (chest pain, stomach pain, burning feeling in chest), which could range from 0 to 30; a higher score is indicative of more severe symptoms. |
Baseline and week 24 |
|
| Secondary |
Absolute Change From Baseline in Frequency of EoE Symptoms Other Than Dysphagia as Measured by EoE-SQ at Week 24 |
The EoE-SQ asks about symptoms that participants with EoE may have (chest pain, stomach pain, burning feeling in chest, food or liquid coming back up into throat, throwing up) during the past 7 days. Response to the frequency of each symptom is on a 5-point scale (1 = 'Never', 2 = 'One day', 3 = ' 2-6 days', 4 = 'Once a day', 5 = 'More than once a day'). The EoE-SQ frequency score is calculated as the sum of the frequency scores from the 5 items which could range from 5 to 25; a higher score is indicative of higher frequency of symptoms. |
Baseline and week 24 |
|
| Secondary |
Percentage of Participants Who Received Rescue Treatment During the Placebo-controlled, Double-blind Treatment Period at Week 24 |
|
At week 24 |
|
| Secondary |
Absolute Change From Baseline in Esophageal Distensibility Plateau Measured by Functional Lumen Imaging, if Collected, at Week 24 |
|
At week 24 |
|
| Secondary |
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of =6 Eosinophils Per High-power Field (Eos/Hpf) in All Three Regions at Week 52 |
Esophageal intraepithelial eosinophil count obtained by esophageal endoscopy with biopsies (all 3 esophageal regions: proximal, mid, and distal). |
At week 52 |
|
| Secondary |
Absolute Change in Dysphagia Symptom Questionnaire (DSQ) Total Score at Week 52 |
The DSQ is used to measure the frequency and intensity of dysphagia. DSQ scores can range from 0 to 84, with a lower score indicating less-frequent or less-severe dysphagia. |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Percent Change in DSQ Total Score at Week 52 |
The DSQ is used to measure the frequency and intensity of dysphagia. DSQ scores can range from 0 to 84, with a lower score indicating less-frequent or less-severe dysphagia. |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Absolute Change in EoE Endoscopic Reference Total Score (EoE-EREFS) at Week 52 |
EoE esophageal characteristics analyzed based on the EoE-EREFS, a scoring system for inflammatory and remodeling features of disease. The overall total score ranges from 0 to 18 with higher number indicating worse disease. |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Percent Change in Peak Esophageal Intraepithelial Eosinophil Count (Eos/Hpf) in All Three Regions at Week 52 |
Esophageal intraepithelial eosinophil count obtained by esophageal endoscopy with biopsies (all 3 esophageal regions: proximal, mid, and distal). A greater esophageal intraepithelial eosinophil count from baseline indicates worsening disease. |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Absolute Change in EoE Histology Scoring System (EoEHSS) Mean Grade Score at Week 52 |
Severity (grade) and extent (stage) of esophageal abnormalities were scored by blinded, central pathologists using a 4-point scale (0 normal; 3 maximum change) for eight features: eosinophil density, basal zone hyperplasia, eosinophil abscesses, eosinophil surface layering, dilated intercellular spaces, surface epithelial alteration, dyskeratotic epithelial cells and lamina propria fibrosis (absent/present). Higher score indicates greater severity and extent of histological abnormalities. |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Absolute Change in EoEHSS Mean Stage Score at Week 52 |
Severity (grade) and extent (stage) of esophageal abnormalities were scored by blinded, central pathologists using a 4-point scale (0 normal; 3 maximum change) for eight features: eosinophil density, basal zone hyperplasia, eosinophil abscesses, eosinophil surface layering, dilated intercellular spaces, surface epithelial alteration, dyskeratotic epithelial cells and lamina propria fibrosis (absent/present). Higher score indicates greater severity and extent of histological abnormalities. |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of <15 Eos/Hpf in All Three Regions at Week 52 |
Esophageal intraepithelial eosinophil count obtained by esophageal endoscopy with biopsies (all 3 esophageal regions: proximal, mid, and distal). |
At week 52 |
|
| Secondary |
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of =1 Eos/Hpf in All Three Regions at Week 52 |
Esophageal intraepithelial eosinophil count obtained by esophageal endoscopy with biopsies (all 3 esophageal regions: proximal, mid, and distal). |
At week 52 |
|
| Secondary |
Absolute Change in Health-related QOL as Measured by EoE-IQ at Week 52 |
The EoE-IQ measures impact of EoE on emotional, social, work & school, & sleep aspects. Participants were asked to respond to 11 questions based on experience living with EoE during past 7 days. Response to each item is on a 5-point scale (1=Not at all [impacted] 2=A little, 3=Somewhat, 4=Quite a bit, 5=Extremely [impacted]). The average score is the sum of non-missing responses divided by the number of items with non-missing responses. The average score can range from 1 to 5; a higher score is indicative of a more negative impact. |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Absolute Change From Baseline in Severity of EoE Symptoms Other Than Dysphagia as Measured by EoE-SQ at Week 52 |
The EoE-SQ asks about symptoms that participants with EoE may have (chest pain, stomach pain, burning feeling in chest, food or liquid coming back up into throat, throwing up) during the past 7 days. Response to the severity of each symptom based on the worst experience in the past 7 days is on a scale of 0 to 10 (higher is worse). The EoE-SQ severity score is calculated as the sum of the severity scores from questions 1 to 3 (chest pain, stomach pain, burning feeling in chest), which could range from 0 to 30; a higher score is indicative of more severe symptoms. |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Absolute Change From Baseline in Frequency of EoE Symptoms Other Than Dysphagia as Measured by EoE-SQ at Week 52 |
The EoE-SQ asks about symptoms that participants with EoE may have (chest pain, stomach pain, burning feeling in chest, food or liquid coming back up into throat, throwing up) during the past 7 days. Response to the frequency of each symptom is on a 5-point scale (1 = 'Never', 2 = 'One day', 3 = ' 2-6 days', 4 = 'Once a day', 5 = 'More than once a day'). The EoE-SQ frequency score is calculated as the sum of the frequency scores from the 5 items which could range from 5 to 25; a higher score is indicative of higher frequency of symptoms. |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Percentage of Participants Who Received Rescue Medication During the 28-week Extended Active Treatment Period |
|
Baseline (of Part C) to week 28 |
|
| Secondary |
NES for the Relative Change From Baseline in EoE Diagnostic Panel (EDP) at Week 52 |
NES reflects the degree to which the activity level of a set of disease transcripts is overrepresented at the extremes (top or bottom) of the entire ranked list of transcripts within a sample and is normalized by accounting for the number of transcripts in the set. An NES of 0 indicates no change from baseline, a negative score reflects a reduction in the disease score (more like normal) and a positive score reflects worsening (more active disease). |
Baseline (of previous study part) and week 52 |
|
| Secondary |
NES for the Relative Change in the Type 2 Inflammation Signature (T2INF) at Week 52 |
NES reflects the degree to which the activity level of a set of disease transcripts is overrepresented at the extremes (top or bottom) of the entire ranked list of transcripts within a sample and is normalized by accounting for the number of transcripts in the set. An NES of 0 indicates no change from baseline, a negative score reflects a reduction in the disease score (more like normal) and a positive score reflects worsening (more active disease). |
Baseline (of previous study part) and week 52 |
|
| Secondary |
Concentration of Functional Dupilumab in Serum at Week 52 |
|
Baseline (of Part C) up to week 52 |
|
| Secondary |
Incidence of Treatment-emergent Anti-drug Antibody (ADA) Response |
Number of treatment-emergent ADA responses to dupilumab reported. |
Baseline (of previous study part) up to week 52 |
|
