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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04166552




Registration number
NCT04166552
Ethics application status
Date submitted
6/11/2019
Date registered
18/11/2019
Date last updated
12/10/2020

Titles & IDs
Public title
Evaluation of Safety, Tolerability and Preliminary Efficacy of EHP-101 in Diffuse Cutaneous Systemic Sclerosis
Scientific title
A Phase IIa, Double-Blind, Randomised, Intracohort Placebo-Controlled, Multicentre Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of EHP-101 in Patients With Diffuse Cutaneous Systemic Sclerosis
Secondary ID [1] 0 0
EHP-101-SS01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Diffuse Cutaneous Systemic Sclerosis 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Patients will be randomized to receive EHP-101 or Placebo
Treatment: Drugs - Patients will be randomized to receive EHP-101 or Placebo

Experimental: EHP-101 low dose once a day -

Experimental: EHP-101 low dose twice a day -

Experimental: EHP-101 high dose once a day -

Experimental: EHP-101 high dose twice a day -


Treatment: Drugs: Patients will be randomized to receive EHP-101 or Placebo
EHP-101 or placebo will be taken once a day

Treatment: Drugs: Patients will be randomized to receive EHP-101 or Placebo
EHP-101 or placebo will be taken twice a day

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
Day 113
Secondary outcome [1] 0 0
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.
Timepoint [1] 0 0
Day 85 and Day 113
Secondary outcome [2] 0 0
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.
Timepoint [2] 0 0
Day 85 and Day 113
Secondary outcome [3] 0 0
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%
Timepoint [3] 0 0
Day 85 and Day 113
Secondary outcome [4] 0 0
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.
Timepoint [4] 0 0
Day 85 and Day 113
Secondary outcome [5] 0 0
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.
Timepoint [5] 0 0
Day 85 and Day 113
Secondary outcome [6] 0 0
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.
Timepoint [6] 0 0
Day 85 and Day 113

Eligibility
Key inclusion criteria
- Patients male and female =18 years and =70 years at the time of consent;

- American College of Rheumatology/ European League Against Rheumatism 2013 Criteria for
diffuse cutaneous systemic sclerosis (dcSSc);

- Documented SSc for up to 6 years from the first non-Raynaud's phenomenon with a total
mRSS of =15;

- No new or increased doses of immunosuppressants medications within 3 months prior to
Screening;

- Effective method of contraception for participants and their partners.
Minimum age
18 Years
Maximum age
70 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Severe or unstable Systemic Sclerosis (SSc) or SSc with end-stage organ failure;

- Patient with FVC <60%;

- History of clinically significant medical condition or concurrent medical therapies
that would exclude the patient, preclude participation in the clinical trial,
influence response to study product, or interfere with study assessments;

- Any one of the following values for laboratory tests at screening:

- Haemoglobin <9 g/dL;

- Neutrophils <1.0 x 10^9/L;

- Platelets <75 x 10^9/L;

- Creatinine clearance <50 mL/min according to modified Cockcroft-Gault equation;

- Serum transaminases >2.0 x upper normal limit;

- Total bilirubin =1.5 x upper limit of normal.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Griffith University - Gold Coast
Recruitment hospital [3] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
9726 - Gold Coast
Recruitment postcode(s) [3] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
New Zealand
State/province [5] 0 0
Wellington

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Emerald Health Pharmaceuticals Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Iqvia Pty Ltd
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this trial is to evaluate the safety, tolerability, pharmacokinetics, and
preliminary efficacy of EHP-101 in adult subjects with diffuse cutaneous Systemic Sclerosis
(dcSSc).
Trial website
https://clinicaltrials.gov/show/NCT04166552
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Nicolaas Pool
Address 0 0
Country 0 0
Phone 0 0
+61 466 924 638
Fax 0 0
Email 0 0
Nicolaas.Pool@iqvia.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04166552