Clinical Trial: A Safety and Tolerability Study of Topical PAT-001 in Congenital Ichthyosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Randomized, Bilateral Comparison, Vehicle-Controlled, Safety and Tolerability Study of Topical PAT-001 for the Treatment of Congenital Ichthyosis

Brief Summary: Congenital ichthyosis (CI) is a large, heterogeneous family of inherited skin disorders of cornification resulting from an abnormality of skin keratinization, such as scaling and thickening of the skin. Treatment options include keratolytic agents, which can abruptly lead to extensive shedding or peeling of scales. PAT-001 primarily acts as a keratolytic agent; thus, making it a potential drug candidate for the treatment of skin disorders associated with hyperkeratinization, such as CI. The current study intends to evaluate the safety and tolerability of PAT-001 in patients with CI of either the Lamellar or X-Linked subtypes.

Detailed Summary:

The management of CI is a life-long endeavor, which remains largely symptomatic (i.e., emollients with or without keratolytics agents) and commonly focused on reducing scaling and/or skin lubrication with both systemic and topical treatments. A first-line therapy includes hydration and lubrication accomplished by creams and ointments containing low concentrations of salt, urea, or glycerol, which increase the water-binding capacity of the horny layer. Addition of keratolytics agents are used to decrease corneocyte cohesiveness, to promote desquamation, and to dissolve keratins and lipids (e.g., α-hydroxy acids, salicylic acid, high dose urea, propylene glycol, N-acetylcysteine, and retinoids). Systemic retinoid treatment is reserved for those patients refractory to topical agents because of long-term adverse effects and teratogenicity.

This is a two part, Phase 2, multicenter, proof-of-concept (POC) study of the safety and tolerability of PAT-001 for the treatment of Congenital ichthyosis (CI) in patients ages 12 years of age and older. Part 1 will be a double-blind, randomized, vehicle controlled, bilateral comparison of two treatments (PAT-001 [0.1% or 0.2%] vs. vehicle) for eight (8) weeks.

Part 2 will be a double-blind, active only treatment comparison of the two PAT-001 concentrations (0.1% or 0.2%) for an additional four (4) weeks. Subjects will have the option to participate in the PK portion of the study.


Sponsor: Patagonia Pharmaceuticals, LLC

Current Primary Outcome:

  • Adverse Events (AEs) [ Time Frame: Day 0 through Day 84 ]
    AEs will be assessed by the investigator and the incidence (severity and causality) of any local and systemic AEs will be reported.
  • Incidence of Local Skin Reactions (LSRs) [ Time Frame: Up to Day 84 ]
    LSRs including burning/stinging, pain, and pruritus will be assessed in each Treatment Area using a four-point ordinal scale where 0=none, 1=mild, 2=moderate, and 3=severe at each clinic visit to allow a comparison between Treatment Groups and Test Articles.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Investigator's Global Assessment (IGA) using a five-point scale [ Time Frame: Up to Day 84 ]
    Overall severity of ichthyosis will be graded using a five-point scale (IGA) from 0=clear to 4=severe. This is a static morphological scale that refers to a point in time and not a comparison to Baseline.
  • Individual Clinical Signs/Symptoms using a five-point scale [ Time Frame: Up to Day 84 ]
    Overall severity of erythema, scaling, fissuring, and papulation/lichenification will be graded using a five-point scale from 0=clear to 4=severe. This is a static morphological scale that refers to a point in time and not a comparison to Baseline.


Original Secondary Outcome: Same as current

Information By: Patagonia Pharmaceuticals, LLC

Dates:
Date Received: August 3, 2016
Date Started: January 2017
Date Completion: December 2017
Last Updated: April 19, 2017
Last Verified: April 2017