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

NCT03738800 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: 18-ICH-001
• Study Type: interventional
• Target: 65
• Locations: 9 countries
• Sites: 36

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.

Conditions

Lamellar Ichthyosis

Outcome Measures

Primary Outcomes (1)

• The Percentage of Subjects in Each Treatment Group Who Experienced Successful Resolution of LI.

  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.

  90 Days

Secondary Outcomes (6)

• Total 16-point Visual Index for Ichthyosis Severity (VIIS)

  90 Days

• The Difference in Mean Scores Using Individual Score for Roughness

  90 Days

• The Difference in Mean Scores Using Palm Sole Assessment

  90 Days

• The Difference in Proportion of Subjects With Presence of Fissures on Palms Between the Active and Vehicle Groups

  90 Days

• Quality of Life Measurement Per Dermatology Life Quality Index (DLQI)

  90 Days

Other Outcomes (10)

• The Difference in Mean Ectropion Scores Between the Active and Vehicle Groups

  180 Days

• Quality of Life Measurement Per EQ-5D-5L

  180 Days

• Incidents of Adverse Events

  180 Days

Study Arms (3)

CD5789 Cream 200 µg/g

EXPERIMENTAL

CD5789 200 µg/g, topical, 50g

Drug: CD5789 Cream 200 µg/g

CD5789 Cream 100 µg/g

EXPERIMENTAL

CD5789 100 µg/g, topical, 50g

Drug: CD5789 Cream 100 µg/g

CD5789 Cream Vehicle

PLACEBO COMPARATOR

CD5789 Cream Vehicle, topical, 50g

Drug: CD5789 Cream Vehicle

Interventions

CD5789 Cream 200 µg/g DRUG

A fixed dose (determined at Visit 1) of 200 µg/g applied topically twice weekly to up to 90% BSA

CD5789 Cream 200 µg/g

CD5789 Cream 100 µg/g DRUG

A fixed dose (determined at Visit 1) of 100 µg/g applied topically twice weekly to up to 90% BSA

CD5789 Cream 100 µg/g

CD5789 Cream Vehicle DRUG

A fixed dose (determined at Visit 1) applied topically twice weekly, up to 36 g per dose up to 90% BSA

CD5789 Cream Vehicle

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may not qualify if:

• Subject has any variant of ichthyosis other than LI or another disorder of keratinization, including syndromic ichthyoses.
• Subject has current moderate or severe stinging/burning at Screening.
• Subject has an ongoing cutaneous infection or any other significant concomitant skin disease (other than the LI) which, in the investigator's opinion, may interfere with the study assessments.
• Subject with fasting triglycerides \>200 mg/dL or \>2.25 mmol/L and/or total cholesterol \>250 mg/dL or \>6.5 mmol/L. Subjects whose triglycerides and/or total cholesterol are within normal limits with a stable dose of lipid-lowering agents for at least 6 months may be included.
• Subject was previously treated with trifarotene/CD5789 in an acne or ichthyosis study.
• Subject has any other significant concomitant disease, or poorly controlled medical condition other than LI that in the investigator's opinion may put him or her at risk if he or she takes part in the study, and/or that may interfere with the study assessments.
• Subject has a medical condition that potentially alters bone metabolism (e.g., osteoporosis, thyroid dysfunction, Cushing syndrome, Crohn's disease, or ulcerative colitis). Subjects with hypothyroidism who are on a stable dose of thyroid hormone replacement therapy and whose thyroid-stimulating hormone (TSH) is normal may be included
• Subject is being treated for major depression disorder and/or has a history of major depression or suicide attempt requiring hospitalization, medications, and close psychiatric surveillance to prevent suicide attempts.
• Subject with positive serology for hepatitis B surface antigen, hepatitis C, or are known to be HIV positive or to have AIDS at Screening.
• Subject with any of the following laboratory values at Screening:
• Aspartate aminotransferase or alanine aminotransferase \>1.5 × upper limit of normal defined by the laboratory
• Total bilirubin \>1.25 × ULN at Screening. Subjects with known Gilbert's syndrome may be included with total bilirubin \>1.25 × ULN
• Hemoglobin \<12.5 g/dL for men and \<11.5 g/dL for women
• Platelets \<150 × 109/L or \>400 × 109/L.
• Subject has any clinically other significant abnormal laboratory value (hematology, chemistry, or urinalysis) at Screening that, in the investigator's opinion, may put the subject at risk if he or she takes part in the study, and/or that may interfere with the study assessments.

Sponsors & Collaborators

• Mayne Pharma International Pty Ltd: lead

Study Sites (36)

TCR Medical Corporation

San Diego, California, 92123, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Yale University

New Haven, Connecticut, 06519, United States

Location

NorthShore University HealthSystem

Skokie, Illinois, 60077, United States

Location

Dawes Fretzin Clinical Research Group, LLC

Indianapolis, Indiana, 46250, United States

Location

DermAssociates, PC

Rockville, Maryland, 20850, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Texas Dermatology and Laser Specialists

San Antonio, Texas, 78218, United States

Location

Eastern Health Monash University

Box Hill, Australia

Location

Veracity Clinical Research

Brisbane, Australia

Location

Royal Children's Hospital

Parkville, Australia

Location

Premier Specialists Ptd Ltd

Sydney, Australia

Location

The Hospital for Sick Children

Toronto, Canada

Location

Dermatologie pédiatrique

Paris, France

Location

CHU Charles Nicolle

Rouen, France

Location

CHU de Toulouse- Hospital Larrey

Toulouse, France

Location

Charite - Universitaetsmedizin Berlin

Berlin, Germany

Location

Universitätsklinkum Frankfurt

Frankfurt, Germany

Location

Kath. Kinderkrankenhaus Wilhelmstift

Hamburg, Germany

Location

Ludwig-Maximilians University

Munich, Germany

Location

Universitatsmedizin Rostock

Rostock, Germany

Location

Tel Aviv Sourasky Mc

Tel Aviv, Israel

Location

Hospital Clinic de Barcelona

Barcelona, Spain

Location

Hospital Nino Jesus

Madrid, 28009, Spain

Location

Clinica Universidad de Navarra (Madrid)

Madrid, Spain

Location

Hospital Niño Jesús

Madrid, Spain

Location

Clinica Universidad de Navarra

Pamplona, Spain

Location

Medical Center of Private Enterprise "Dzerkalo"

Dnipro, 49000, Ukraine

Location

Dnipropetrovsk State Hospital of Dermatovenerology

Dnipro, 49074, Ukraine

Location

Medical Center "Family Medicine Clinic"

Dnipro, 49600, Ukraine

Location

Ternopil Regional Clinical Dermatovenereological Dispensary

Ternopil, 46006, Ukraine

Location

TDC PE "Asclepius"

Uzhhorod, 88000, Ukraine

Location

Community Institution "Zaporizhzhya Regional Dermatovenereology Clinical Hospital"

Zaporizhzhya, 69063, Ukraine

Location

Royal London Hospital Barts Health Nhs Trust

London, United Kingdom

Location

MeSH Terms

Conditions

Ichthyosis, Lamellar

Interventions

trifarotene

Condition Hierarchy (Ancestors)

Ichthyosiform Erythroderma, Congenital; Ichthyosis; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Keratosis; Skin Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Mayne Pharma
• Organization: Mayne Pharma

us.medicalinformation@maynepharma.com

1 252 752 3800

Study Officials

• Keith A. Choate, MD

  Yale University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2018
• First Posted: November 13, 2018
• Study Start: May 1, 2019
• Primary Completion: September 3, 2021
• Study Completion: September 3, 2021
• Last Updated: August 14, 2023
• Results First Posted: August 14, 2023

Record last verified: 2023-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); ES (5); FR (3); US (10); CA (1); IL (1); UA (6); DE (5); AU (4)