Trial Review

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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04166552
Ethics application status
Date submitted
6/11/2019
Date registered
18/11/2019
Date last updated
21/09/2023

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
Assessment method [1] 0 0
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
Assessment method [1] 0 0
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
Assessment method [2] 0 0
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
Assessment method [3] 0 0
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
Assessment method [4] 0 0
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
Assessment method [5] 0 0
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
Assessment method [6] 0 0
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 =74 years at the time of consent;
* American College of Rheumatology/ European League Against Rheumatism 2013 Criteria for SSc; dcSSc (skin thickening on upper arms, upper legs, or trunk);
* 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
74 Years
Sex
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;
* History of gastrointestinal dysmotility requiring total parenteral nutrition or hospitalization within 6 months before Visit 1;
* 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;
* Estimated creatinine clearance <50 mL/min according to the 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
Suspended
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
11/06/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2024
Actual
Sample size
Target
36
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [3] 0 0
Footscray Hospital - Footscray
Recruitment hospital [4] 0 0
Griffith University - Gold Coast
Recruitment hospital [5] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment hospital [6] 0 0
Westmead Hospital - Sydney
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
2050 - Camperdown
Recruitment postcode(s) [3] 0 0
3011 - Footscray
Recruitment postcode(s) [4] 0 0
9726 - Gold Coast
Recruitment postcode(s) [5] 0 0
6150 - Murdoch
Recruitment postcode(s) [6] 0 0
2145 - Sydney
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
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
New Jersey
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
New Zealand
State/province [9] 0 0
Wellington
Country [10] 0 0
Puerto Rico
State/province [10] 0 0
Caguas
Country [11] 0 0
Puerto Rico
State/province [11] 0 0
San Juan

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Emerald Health Pharmaceuticals
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT04166552