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

NCT ID: NCT02864082

Last Updated: 2021-09-29

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 19 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-03-08
• Study Completion Date: 2018-12-04

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 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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

PAT-001 0.1%

Part 1: Bilateral comparison. Patients will have two comparable Treatment Areas: PAT-001, 0.1% (e.g., left side) and Vehicle, 0.0% (e.g., right side). This comparison lasts from Weeks 0-8 Part 2: Patients will apply only PAT-001, 0.1% to both Treatment Areas from Weeks 8-12.

Group Type: EXPERIMENTAL

PAT-001, 0.1%

Intervention Type: DRUG

PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.

PAT-001 0.2%

Part 1: Bilateral comparison. Patients will have two comparable Treatment Areas: PAT-001, 0.2% (e.g., left side) and Vehicle, 0.0% (e.g., right side). This comparison lasts from Weeks 0-8 Part 2: Patients will apply PAT-001, 0.2% to both Treatment Areas.

Group Type: EXPERIMENTAL

PAT-001, 0.2%

Intervention Type: DRUG

PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.

Vehicle for PAT-001 0.1% arm

Part 1: Bilateral comparison. Patients will have two comparable Treatment Areas: PAT-001, 0.1% (e.g., left side) and Vehicle, 0.0% (e.g., right side). The application of vehicle only lasts from Weeks 0-8.

Group Type: PLACEBO_COMPARATOR

Vehicle for PAT-001 0.1%

Intervention Type: DRUG

Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.1%.

Vehicle for PAT-001 0.2% arm

Part 1: Bilateral comparison. Patients will have two comparable Treatment Areas: PAT-001, 0.2% (e.g., left side) and Vehicle, 0.0% (e.g., right side). The application of vehicle only lasts from Weeks 0-8.

Group Type: PLACEBO_COMPARATOR

Vehicle for PAT-001 0.2%

Intervention Type: DRUG

Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.2%.

Interventions

PAT-001, 0.1%

PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.

Intervention Type: DRUG

PAT-001, 0.2%

PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.

Intervention Type: 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%.

Intervention Type: 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%.

Intervention Type: DRUG

Other Intervention Names

PAT-001; PAT-001; Vehicle; Vehicle

Eligibility Criteria

Inclusion Criteria

• Patients of either sex aged 12 years or older.
• Females of childbearing potential should use appropriate contraception. Women of childbearing potential must have a negative pregnancy test at screening and baseline visits.
• Patient and legal representative(s), if applicable, has provided written informed consent.
• Patient has congenital ichthyosis of either lamellar or X-Linked subtype.
• Patient has two contralateral comparable Treatment Areas (e.g., each arm is affected and treatments areas can be applied equally).
• Patient is, except for their ichthyosis, in good general health.

Exclusion Criteria

• Patient is pregnant or breast feeding, or is planning to become pregnant during the study.
• Patient has inflammatory skin disease unrelated to ichthyosis.
• Patient is currently using concomitant retinoid therapy, within two weeks (topical) or 12 weeks (oral) of Visit 2/Baseline.
• Patient is currently taking concomitant immunosuppressive drugs, including systemic corticosteroids, within two weeks of Visit 2/Baseline.
• Patient is currently enrolled in an investigational drug or device study.
• Patient has used an investigational drug or investigational device treatment within 30 days prior to Visit 2/Baseline.
• Patient is unable to communicate or cooperate with the investigator due to language problems, impaired cerebral function, or physical limitations.
• Patient is known to be noncompliant or is unlikely to comply with the requirements of the study protocol (e.g., due to alcoholism, drug dependency, mental incapacity) in the opinion of the investigator.

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Patagonia Pharmaceuticals, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Zachary Rome, BS

Role: STUDY_DIRECTOR

Sponsor GmbH

Locations

TCR Medical Corporation

San Diego, California, United States

Yale Center for Clinical Investigation

New Haven, Connecticut, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Paddington Testing Co., Inc

Philadelphia, Pennsylvania, United States

Texas Dermatology and Laser Specialists

San Antonio, Texas, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

205-9051-201

Identifier Type: -

Identifier Source: org_study_id