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

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.

Study Overview

• Status: Withdrawn
• Conditions: Psoriasis
• Intervention / Treatment: Drug: pefcalcitol ointment, 0.005%

• Study Type: Interventional
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 16 years (Child)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: pefcalcitol; pefcalcitol 0.005% BID for 8 weeks	Drug: pefcalcitol ointment, 0.005%; pefcalcitol ointment, 0.005%

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Maruho Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Anticipated): June 1, 2019
• Primary Completion (Anticipated): December 1, 2019
• Study Completion (Anticipated): December 1, 2019

Study Registration Dates

• First Submitted: November 16, 2016
• First Submitted That Met QC Criteria: November 17, 2016
• First Posted (Estimate): November 22, 2016

Study Record Updates

• Last Update Posted (Actual): April 18, 2023
• Last Update Submitted That Met QC Criteria: April 13, 2023
• Last Verified: January 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Skin Diseases, Papulosquamous; Psoriasis
• Other Study ID Numbers: CLS008-CO-PR-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No