A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis
- Conditions
- Lamellar Ichthyosis
- Interventions
- Drug: CD5789 Cream VehicleDrug: CD5789 Cream 100 µg/gDrug: CD5789 Cream 200 µg/g
- Registration Number
- NCT03738800
- Lead Sponsor
- Mayne Pharma International Pty Ltd
- Brief Summary
This is a phase 2 randomized, multi-center, double-blind, vehicle controlled, 90 day, safety, efficacy, and systemic exposure study followed by a 90 day open-label extension of trifarotene cream in adults and adolescents with autosomal recessive ichthyosis with lamellar scale.
- Detailed Description
This is a 2-cohort, multicenter study in subjects with moderate to severe LI. Adults (Cohort A) and adults and adolescents (Cohort B) will be randomized in a double-blind fashion to 1 of 2 doses of active or vehicle and treated twice weekly for 90 days. Subjects who complete the randomized, double-blind portion of the study will be eligible to enter a 90 day, open-label extension study.
Approximately 15 adults (≥18 years old) will be randomized into the first cohort of subjects (Cohort A) in a 1:1:1 ratio and treated twice weekly for up to 90 days. If no safety issues are identified, both adults and adolescents (ages 12-17 years, inclusive) will be allowed to enroll in Cohort B. Subjects in Cohort B will be randomized 1:1:1 and treated twice weekly for up to 90 days in the same manner as subjects in Cohort A.
All subjects who complete 90 days of double-blind study treatment will be eligible to enroll in a 90 open-label extension. Subjects in the open-label extension will receive active twice weekly for up to 90 days.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 65
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description CD5789 Cream Vehicle CD5789 Cream Vehicle CD5789 Cream Vehicle, topical, 50g CD5789 Cream 100 µg/g CD5789 Cream 100 µg/g CD5789 100 µg/g, topical, 50g CD5789 Cream 200 µg/g CD5789 Cream 200 µg/g CD5789 200 µg/g, topical, 50g
- Primary Outcome Measures
Name Time Method The Percentage of Subjects in Each Treatment Group Who Experienced Successful Resolution of LI. 90 Days The percentage of subjects in each treatment group who experienced successful resolution of LI where "success" is defined as clear/almost clear on treated areas and at least a 2-grade change from Baseline at Day 90/end-of-treatment (EOT) in the Double-blind Period on the 5-point IGA full body scale.
- Secondary Outcome Measures
Name Time Method The Difference in Mean Scores Using Individual Score for Roughness 90 Days The amount of roughness of the skin will be measured on a 5-point scale. 0 (Clear) Smooth skin
1. (Almost Clear) Hardly palpably roughness
2. (Mild) Mild roughness (fine sand paper-like)
3. (Moderate) Moderate, coarse roughness (coarse sand paper-like)
4. (Severe) Very coarse skin (broken cornflakes-like)The Difference in Mean Scores Using Palm Sole Assessment 90 Days Thickening of the skin on the palms and soles will be measured on a 5-point scale:
0 (Clear) No thickening, no roughness, no fissure
1. (Almost Clear) Only slight thickening, minimal to no roughness, no fissures
2. (Mild) Some thickening, mild roughness on palpation, few fissures may be present
3. (Moderate) Substantial and diffuse thickening, coarse roughness on palpation may be present, fissures may be present
4. (Severe) Very thickened and rough skin, numerous fissuresTotal 16-point Visual Index for Ichthyosis Severity (VIIS) 90 Days 5-point Visual Index for Ichthyosis Severity (VIIS) for scaling (overall 16 points) for scaling, i.e. 0-4 points for 4 body areas: chest/abdomen, back, arms and legs) where minimum is 0 and maximum is 16 (e.g. 4 points for each of the four body parts).
0 (Clear) No scaling
1. (Almost Clear) Very fine, non-coalescent scales
2. (Mild) Small and thin, non-coalescent scales
3. (Moderate) Large and rather thick scales starting to coalesce
4. (Severe) Very large, adherent, coalescent and very thick scalesThe Difference in Proportion of Subjects With Presence of Fissures on Soles Between the Active and Vehicle Groups 90 Days Fissuring will be assessed by recording the presence or absence of fissures, the number of fissures present, and the pain associated with each fissure. The subject will assess pain associated with fissures as ranging from 0-3 (none, mild, moderate, severe) at day 90 between the active trifarotene cream HE1 and vehicle groups
Quality of Life Measurement Per Dermatology Life Quality Index (DLQI) 90 Days The DLQI, or the Dermatology Quality of Life Index, is a dermatology-specific Quality of Life instrument. It is a simple 10-question validated questionnaire with 6 domains (symptoms and feelings, daily activities, leisure, work and school, personal relationships, and treatment); higher scores indicate poorer quality of life. Responses collected are on a scale of 0-3 depending on the question relevance to the subject.
Response (Score) Very much (scored 3) A lot (scored 2) A little (scored 1) Not at all (scored 0) Not relevant (scored 0) A minimum score of 0 and maximum score of 30 is obtained by summing the score of each question. The higher the score, the more quality of life is impaired.
0-1 = no effect at all on patient's life 2-5 = small effect on patient's life 6-10 = moderate effect on patient's life 11-20 = very large effect on patient's life 21-30 = extremely large effect on patient's lifeThe Difference in Proportion of Subjects With Presence of Fissures on Palms Between the Active and Vehicle Groups 90 Days Fissuring will be assessed by recording the presence or absence of fissures, the number of fissures present, and the pain associated with each fissure. The subject will assess pain associated with fissures as ranging from 0-3 (none, mild, moderate, severe) at day 90 between the active trifarotene cream HE1 and vehicle groups
Trial Locations
- Locations (36)
Medical Center of Private Enterprise "Dzerkalo"
🇺🇦Dnipro, Ukraine
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Yale University
🇺🇸New Haven, Connecticut, United States
Dawes Fretzin Clinical Research Group, LLC
🇺🇸Indianapolis, Indiana, United States
DermAssociates, PC
🇺🇸Rockville, Maryland, United States
Texas Dermatology and Laser Specialists
🇺🇸San Antonio, Texas, United States
Eastern Health Monash University
🇦🇺Box Hill, Australia
Veracity Clinical Research
🇦🇺Brisbane, Australia
CHU de Toulouse- Hospital Larrey
🇫🇷Toulouse, France
Universitätsklinkum Frankfurt
🇩🇪Frankfurt, Germany
Dermatologie pédiatrique
🇫🇷Paris, France
Premier Specialists Ptd Ltd
🇦🇺Sydney, Australia
Charite - Universitaetsmedizin Berlin
🇩🇪Berlin, Germany
Kath. Kinderkrankenhaus Wilhelmstift
🇩🇪Hamburg, Germany
Hospital Nino Jesus
🇪🇸Madrid, Spain
Hospital Niño Jesús
🇪🇸Madrid, Spain
Clinica Universidad de Navarra (Madrid)
🇪🇸Madrid, Spain
Clinica Universidad de Navarra
🇪🇸Pamplona, Spain
Medical Center "Family Medicine Clinic"
🇺🇦Dnipro, Ukraine
Ternopil Regional Clinical Dermatovenereological Dispensary
🇺🇦Ternopil', Ukraine
Community Institution "Zaporizhzhya Regional Dermatovenereology Clinical Hospital"
🇺🇦Zaporizhzhya, Ukraine
The Hospital for Sick Children
🇨🇦Toronto, Canada
Ludwig-Maximilians University
🇩🇪Munich, Germany
Universitatsmedizin Rostock
🇩🇪Rostock, Germany
Hospital Clinic de Barcelona
🇪🇸Barcelona, Spain
TCR Medical Corporation
🇺🇸San Diego, California, United States
NorthShore University HealthSystem
🇺🇸Skokie, Illinois, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
CHU Charles Nicolle
🇫🇷Rouen, France
Royal Children's Hospital
🇦🇺Parkville, Australia
Tel Aviv Sourasky Mc
🇮🇱Tel Aviv, Israel
Dnipropetrovsk State Hospital of Dermatovenerology
🇺🇦Dnipro, Ukraine
TDC PE "Asclepius"
🇺🇦Uzhhorod, Ukraine
Royal London Hospital Barts Health Nhs Trust
🇬🇧London, United Kingdom