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

Phase 2

Withdrawn

• Conditions: Psoriasis
• Interventions: Drug: pefcalcitol ointment, 0.005%

• Registration Number: NCT02970331
• Lead Sponsor: Maruho Co., Ltd.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-11-16
• Study First Submitted QC: 2016-11-17
• Study First Posted: 2016-11-22
• Status Verified: 2019-01
• Study Start: 2019-06
• Primary Completion: 2019-12
• Study Completion: 2019-12
• Last Update Submitted: 2023-04-13
• Last Update Posted: 2023-04-18

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
pefcalcitol	pefcalcitol ointment, 0.005%	pefcalcitol 0.005% BID for 8 weeks

Primary Outcome Measures

Name	Time	Method
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
Change in incidence and severity of application site adverse events	screening, weeks 0,2,4 and 8
Changes from baseline (pre-dose, Day 1) in safety laboratory parameters	week 8
Change in incidence and severity of all AEs and their relationship to study drug	screening, weeks 0,2,4 and 8

Secondary Outcome Measures

Name	Time	Method
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 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