A Phase 2, Multicenter, Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pefcalcitol Ointment, 0.005%, Applied Topically Twice Daily (BID) for 8 Weeks in Adolescent Subjects 12 to < 17 Years of Age With Plaque Psoriasis

Study Overview
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Brief Summary
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A multi-center, open-label study that will evaluate the safety/tolerability and pharmacodynamics as well as the pharmacokinetic profile (sub-population analysis), in 50 evaluable adolescents 12 to < 17 years of age.

Official Title
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A Phase 2, Multicenter, Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pefcalcitol Ointment, 0.005%, Applied Topically Twice Daily (BID) for 8 Weeks in Adolescent Subjects 12 to < 17 Years of Age With Plaque Psoriasis

Conditions
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Psoriasis

Intervention / Treatment
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* Drug: pefcalcitol ointment, 0.005%


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: male or females 12 to < 17 years of age Have a confirmed diagnosis of plaque psoriasis Negative pregnancy test Group 1: Have plaque psoriasis with an IGA score of ≥ 2 (mild), with at least one lesion ) and up to 20% Body Surface Area (BSA) involvement not including psoriasis on the face and scalp Group 2: Have plaque psoriasis with a IGA score of ≥ 2 (mild), with plaque psoriasis involving at least 10% and up to 20% Body Surface Area (BSA) not including psoriasis on the face and scalp Exclusion Criteria: known allergy or intolerance to the study drug or other vitamin D3 analogs or any of its components history of or active generalized guttate, pustular or erythrodermic exfoliative psoriasis history or presence of contact dermatitis induced by a topical medicine or other serious skin condition that is not well controlled Use topical treatments known to have beneficial effects on psoriasis Use phototherapy, oral corticosteroids, oral retinoid, oral immunosuppressive/immunomodulate drugs, cytostatics, cyclosporine or methotrexate within 30 days prior to the first dose of study drug Use any approved biologics for psoriasis within 30 days or 5 half-lives of the biologic before the first dose of study drug Are treated with medications known to worsen psoriasis Are taking an oral vitamin D Are taking medications that affect calcium metabolism; Subjects who have an average of three (3) QTcF measurements of > 450 milliseconds as shown on the ECG (Group 2 only); Have clinically significant abnormal calcium homeostasis parameters at Visit 1; Have clinically significant liver or renal dysfunction Have any other clinically significant laboratory abnormalities, co-morbidities or psychiatric conditions which that would place the subject at increased risk or would confound the primary or secondary objectives of the study; Use of any investigational drugs or biologics and/or participated in any clinical trial within the last 60 days before the day of the first dose of study drug or are taking part in a non-medication study which, that would interfere with study compliance or outcome assessments; Are pregnant or lactating females; Have a known history of congenital or acquired immunodeficiency.

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: pefcalcitol<br>pefcalcitol 0.005% BID for 8 weeks | Drug: pefcalcitol ointment, 0.005%<br>* pefcalcitol ointment, 0.005%<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in incidence and severity of application site adverse events |  | screening, weeks 0,2,4 and 8 | 
| Change in incidence and severity of all AEs and their relationship to study drug |  | screening, weeks 0,2,4 and 8 | 
| Changes from baseline (pre-dose, Day 1) in safety laboratory parameters |  | week 8 | 
| Changes from baseline (pre-dose, Day 1) in calcium homeostasis parameters (total serum calcium and albumin-corrected calcium levels, plasma parathyroid hormone (PTH), and serum alkaline phosphatase) |  | week 8 | 
| Determination of plasma concentrations of Pefcalcitol and its metabolites |  | Day 1 and Day 15 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of subjects with an IGA score of absence (0) or very mild (1) with a minimum of a 2-grade improvement from baseline |  | week 8 | 
| Proportion of subjects who achieved an mPASI 75 from baseline (Week 0) to Week 8, or who achieved an mPASI 50 and a 5-point improvement in the CDLQI from baseline |  | week 8 | 
| Proportion of subjects who achieved success in each of the three individual components of the mPASI (scaling, thickness and erythema) from baseline |  | week 8 | 
| Proportion of subjects who achieved a 5-point improvement in the CDLQI from baseline |  | week 8 |