Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Other |
Change from baseline of EHP-101 compared to placebo in Patient-Reported Outcomes Measurement Information system-29 Short |
(PROMIS)-29 Short measures self-reported physical, mental wellbeing. It consists of 29 questions. The questions are ranked on a 5-point Likert Scale. |
Day 85 |
|
| Other |
Change from baseline of EHP-101 compared to placebo in 5 dimensions Itch Score |
5-D Itch Score comprises the following 5 dimensions: degree, duration, direction, disability and distribution. The recall period is two weeks. |
Day 85 |
|
| Other |
Change from baseline of EHP-101 compared to placebo in University of California, Los Angeles Scleroderma Clinical Trial Consortium Gastrointestinal Tract Questionnaire |
The UCLA SCTC GIT 2.0 has 7 multi-item scales: Reflux, Distension/Bloating, Diarrhea, Fecal Soilage, Constipation, Emotional Well-being, and Social Functioning and a total GIT score. All scales are scored from 0 [better health-related quality of life (HRQOL)] to 3 (worse HRQOL) except the diarrhea and constipation scales (ranges 0-2 and 0-2.5, respectively). The Total GIT Score, is developed by averaging 6 of 7 scales (excluding constipation). The recall period is one week. |
Day 85 |
|
| Other |
Change from baseline of EHP-101 compared to placebo in Functional Assessment of Chronic Illness Therapy: Fatigue |
The FACIT Fatigue Scale is a short, 13-item, easy to administer tool that measures an individual's level of fatigue during their usual daily activities over the past week. The composite score of all item will be used. The total score ranges from 0 to 52. The recall period is one week. |
Day 85 |
|
| Other |
Change from baseline of EHP-101 compared to placebo in the European Quality of Life 5 Domain Questionnaire |
The EQ-5D descriptive system comprises the following 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression and as overall health using a "thermometer" visual analog scale with response options ranging from 0 (worst imaginable health) to 100 (best imaginable health). |
Day 85 |
|
| Other |
Quantitation of drug plasma levels at different timepoints to determine trough |
To determine trough level plasma concentrations of drug substance of EHP 101 |
Day 113 |
|
| Primary |
Incidence and severity of Treatment Emergent Adverse Events |
This safety outcome combines the measure of the number of subjects experiencing adverse events (AEs), the nature and severity of those AEs and their relationship to the study treatments. |
Day 113 |
|
| Secondary |
Treatment effect of EHP-101 compared to placebo as measured by the American College of Rheumatology composite response index in diffuse cutaneous Systemic Sclerosis |
The ACR CRISS exponential algorithm determines the predicted probability of improvement from baseline, incorporating change in modified Rodnan Skin Score (mRSS), Forced Vital Capacity (FVC) % predicted, physician and patient global assessments, and Scleroderma Health Assessment Questionnaire Disability Index (S-HAQ-DI). The outcome is a continuous variable between 0.0 and 1.0 (0 - 100%). Subjects are not considered improved (ACR CRISS score = 0) if they develop new: 1) renal crisis; 2) decline in FVC% predicted by 15% (relative) from baseline and confirmed after 1 month; or 3) left ventricular failure (systolic ejection fraction < 45%); or 4) new pulmonary artery hypertension on right heart catheterization requiring treatment. |
Day 85 and Day 113 |
|
| Secondary |
Treatment effect of EHP-101 compared to placebo in modified Rodnan Skin Score |
The mRSS consists of an evaluation of patient's skin thickness rated by clinical palpation using a 0-3 scale (0 = normal skin; 1 = mild thickness; 2 = moderate thickness; 3 = severe thickness with inability to pinch the skin into a fold for each of 17 surface anatomic areas of the body: face, anterior chest, abdomen, and, with right and left sides of the body separately evaluated, the fingers, forearms, upper arms, thighs, lower legs, dorsum of hands and feet. Individual values are summed and defined as the total skin score. Total score is 0 to 51. |
Day 85 and Day 113 |
|
| Secondary |
Treatment effect of EHP-101 compared to placebo in forced vital capacity percent predicted |
Forced vital capacity (FVC) is a standard pulmonary function test used to quantify respiratory muscle weakness . FVC was the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100% |
Day 85 and Day 113 |
|
| Secondary |
Treatment effect of EHP-101 compared to placebo in physician global assessment score |
The Physician Global Assessment of disease activity will be performed using a segmented numerical version of the visual analogue scale in which the physician selects a whole number (0-10 integers) that best reflects the overall disease activity. The numerical rating score is anchored by 2 verbal descriptors, one of "no disease activity" (score of 0) and one of "worse imaginable disease activity" (score of 10), with numbers 1-9 spaced equidistance in between. The physician will select an integer to describe disease activity. The recall period is one week. |
Day 85 and Day 113 |
|
| Secondary |
Treatment effect of EHP-101 compared to placebo in patient global assessments score |
The Patient Global Assessment will be performed with a segmented numerical version of the visual analogue scale in which the subject selects a whole number (0-10 integers) that best reflects the overall disease activity. The numerical rating score is anchored by two verbal descriptors, one of "no disease activity" (score of 0) and one of "worse imaginable disease activity" (score of 10), with numbers 1-9 spaced equidistance in between. The subject will select an integer to describe disease activity. The recall period is one week. |
Day 85 and Day 113 |
|
| Secondary |
Treatment effect of EHP-101 compared to placebo in Scleroderma Health Assessment Questionnaire - Disability Index |
S-HAQ-DI includes 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are two or three questions for each section. Scoring within each section is from 0 (without any difficulty) to 3 (unable to do). The eight scores of the eight sections are summed and divided by 8. If one section is not completed by a subject, the summed score is divided by 7. As such, maximum scores can vary with a min of 0. The result is the DI, the disability index or functional disability index. The recall period is one week. |
Day 85 and Day 113 |
|
