NCT02864082

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Patagonia Pharmaceuticals, LLC · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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 description

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 pharmacokinetics (PK) portion of the study.

Conditions

• Congenital Ichthyosis

Interventions

Type	Name	Description
DRUG	PAT-001, 0.1%	PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
DRUG	PAT-001, 0.2%	PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
DRUG	Vehicle for PAT-001 0.1%	Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.1%.
DRUG	Vehicle for PAT-001 0.2%	Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.2%.

Timeline

• Start date: 2017-03-08
• Primary completion: 2018-02-13
• Completion: 2018-12-04
• First posted: 2016-08-11
• Last updated: 2021-09-29
• Results posted: 2021-09-02

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study