Lamellar ichthyosis (LI) Autosomal Recessive Ichthyosis with Lamellar Scale MedDRA version: 20.0 Level: LLT Classification code 10023686 Term: Lamellar ichthyosis System Organ Class: 100000004850
Conditions
Sponsors
Mayne Pharma
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. For Cohort A: subject is =18 years old; for Cohort B: subject is =12 years old. 2. Subject has known diagnosis of LI. 3. Subject has moderate to severe (VIIS 3-4) LI on at least 2 of the 4 body areas assessed(chest/abdomen, back, arms, and legs). 4. Subject has signed an ICF at Screening before any investigational procedures. Subjects =65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: 1. Subject has any variant of ichthyosis other than LI or another disorder of keratinization including syndromic ichthyoses. 2. Subject has a history of or current moderate or severe stinging/burning at Screening. 3. 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. 4. Subject with a known lipid disorder unless well controlled by stable doses of lipid-lowering agents for at least 6 months. 5. Subject was previously treated with trifarotene/CD5789, including the acne formulation, or participated in previous studies for ichthyosis. 6. Subject has known skeletal disease, hypertriglyceridemia, hypercholesterolemia, liver disease, or other poorly controlled medical conditions. 7. Subject has a medical condition that potentially alters bone metabolism (e.g., osteoporosis, thyroid dysfunction, Cushing syndrome), Crohn’s disease, or any other significant concomitant disease 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. 8. Subject is being treated for major depression disorder. 9. Subject with positive serology for hepatitis B surface antigen, hepatitis C, or are known to be HIV positive or to have AIDS at Screening. 10. Subject with any of the following laboratory values at Screening: a. Aspartate aminotransferase or alanine aminotransferase >1.5 × upper limit of normal defined by the laboratory b. Triglycerides >200 mg/dL c. Total cholesterol >250 mg/dL d. Hemoglobin 400 × 109/L. 11. 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. 12. Subject has a history of long QT syndrome or clinically significant electrocardiogram (ECG) abnormalities, including clinically significant conduction disorders or significant arrhythmias, QTcF interval >450 ms, PR interval is not between 120 and 220 ms (inclusive), HR >100 bpm or 110 ms, or QT intervals that cannot be consistently analyzed. 13. Subject has a known allergy or sensitivity to any of the components of the investigational products. 14. Subject has been exposed to excessive ultraviolet (UV) radiations on the treated zones within 1 month before Baseline visit or who is planning intensive UV exposure during the study (e.g., occupational exposure to the sun, sunbathing, phototherapy, etc.). 15. Subject is inherently sensitive to sunlight. 16. Subject is presumed to be abusing drugs or alcohol at Screening or Baseline Visits based on medical history or current clinical symptoms. 17. Subject is participating in another interventional clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the safety and efficacy of 2 concentrations of trifarotene cream HE1 versus vehicle in adults and adolescents with moderate to severe autosomal recessive ichthyosis with lamellar scale, also known as lamellar ichthyosis (LI) after 12 weeks of treatment.;Secondary Objective: • To assess systemic exposure to trifarotene and its major metabolites after topical application of the investigational product (IP) on up to 90% body surface area (BSA) twice weekly. • To assess safety for up to 24 weeks of dosing with open-label trifarotene cream HE1 200 µg/g.;Primary end point(s): The number of subjects in each treatment group who experience successful resolution of LI where “success” is defined as clear/almost clear overall and at least a 50% reduction from Baseline at Week 12/end-of-treatment (EOT) in the Double-blind Period on the overall 16-point VIIS for scaling (i.e., 0-4 points on each of the 4 body areas: chest/abdomen, back, arms, and legs).;Timepoint(s) of evaluation of this end point: Week 12/end-of-treatment (EOT) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • The difference in mean scores between the active trifarotene cream HE1 and vehicle groups in the following assessment indices: - Investigator’s Global Assessment (0-4) for each body area - Palm/sole Assessment (Scale: 0–4) - Individual score for roughness (Scale: 0–4) overall • Quality of life per Dermatology Life Quality Index (DLQI) • The difference in proportion of subjects with presence of fissures (presence/absence, number of fissures, and pain associated with fissures on a 0-3 scale) between the active trifarotene cream HE1 and vehicle groups Exploratory endpoints: • The difference in mean ectropion (Ectropion Severity Score [ESS] of 0–8) scores between the active trifarotene cream HE1 and vehicle groups • Quality of life per EQ-5D-5L Safety endpoints: • Reported serious adverse events (SAEs), treatment-emergent AEs (TEAEs), and changes in clinical laboratory tests, vital signs, physical examinations, and 12-lead ECGs • Local tolerability (Scale: 0–3 [none, mild, moderate, severe], determined by the investigator) on each of the 4 body areas (chest/abdomen, back, legs, arms) Pharmacokinetic endpoints: Plasma concentrations of CD5789 and its major metabolites will be measured.;Timepoint(s) of evaluation of this end point: Secondary and exploratory efficacy endpoints as well as PK endpoints will be presented for all study visits (Day 1,14,30,60,90 and 104,120,150,180,194). Safety endpoints will be monitored throughout the entire study. | — |
Countries
Australia, Canada, France, Germany, Israel, Spain, Ukraine, United Kingdom, United States
Contacts
Public ContactPhoevos Hughes
Mayne Pharma
Outcome results
None listed