NCT03738800

A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis

A Phase 2 Randomized, Multicenter, Double-blind, Vehicle Controlled, 90-Day, Safety, Efficacy & Systemic Exposure Study of Trifarotene (CD5789) Cream HE1 in Adults and Adolescents With Autosomal Recessive Ichthyosis With Lamellar Scale

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 65 (actual)
• Sponsor: Mayne Pharma International Pty Ltd · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Lamellar Ichthyosis

Interventions

Type	Name	Description
DRUG	CD5789 Cream 200 µg/g	A fixed dose (determined at Visit 1) of 200 µg/g applied topically twice weekly to up to 90% BSA
DRUG	CD5789 Cream 100 µg/g	A fixed dose (determined at Visit 1) of 100 µg/g applied topically twice weekly to up to 90% BSA
DRUG	CD5789 Cream Vehicle	A fixed dose (determined at Visit 1) applied topically twice weekly, up to 36 g per dose up to 90% BSA

Timeline

• Start date: 2019-05-01
• Primary completion: 2021-09-03
• Completion: 2021-09-03
• First posted: 2018-11-13
• Last updated: 2023-08-14
• Results posted: 2023-08-14

Locations

36 sites across 9 countries: United States, Australia, Canada, France, Germany, Israel, Spain, Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study