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

Primary Purpose

Congenital Ichthyosis

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

PAT-001, 0.1%

PAT-001, 0.2%

Vehicle for PAT-001 0.1%

Vehicle for PAT-001 0.2%

About this trial

This is an interventional treatment trial for Congenital Ichthyosis focused on measuring X-linked, lamellar

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

TCR Medical Corporation
Yale Center for Clinical Investigation
Ann & Robert H. Lurie Children's Hospital of Chicago
Paddington Testing Co., Inc
Texas Dermatology and Laser Specialists

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Placebo Comparator

Arm Label

PAT-001 0.1%

PAT-001 0.2%

Vehicle for PAT-001 0.1% arm

Vehicle for PAT-001 0.2% arm

Arm Description

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.

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.

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.

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.

Outcomes

Primary Outcome Measures

Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)

The number of participants with 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) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle

LSRs including burning/stinging, pain, and pruritus (itch) will be assessed in each Treatment Area using a four-point ordinal scale where 0=none, 1=mild, 2=moderate, and 3=severe (based on the investigator's evaluation of the skin reaction) at each clinic visit to allow a comparison between Treatment Groups and Test Articles. Only LSRs that require medical intervention (e.g., prescription medication) or require withholding or reduction in dosing frequency of the test articles will be documented in this LSR Table. Any LSRs that are not listed here will be recorded as AEs.

Secondary Outcome Measures

Number of Participants Achieving Improvement to State of Clear, Almost Clear or Mild in the Investigator's Global Assessment (IGA) Using a Five-point Scale at Day 57 (Part 1)

Overall severity of ichthyosis will be graded using a five-point scale Investigator Global Assessment (IGA) based upon a 5 point scale going from 0=clear., 1=almost clear, 2=mild, 3=moderate to 4=severe. Scoring is based upon investigator evaluation. This is a static morphological scale that refers to a point in time and not a comparison to Baseline.

Number of Participants Achieving an Improvement of at Least 1 Point Score in the Individual Clinical Signs/Symptoms of Erythema, Scaling, Fissuring and Papulation/Lichenification Using a Five-point Scale

Overall severity of erythema (redness), scaling , fissuring (cracks in skin), and papulation/lichenification (skin thickening, increased pigmentation and/or exaggerated skin lines, formation of papules) will be graded using a five-point scale from 0=clear, 1=almost clear, 2=mild, 3=moderate to 4=severe. This is a static morphological scale that refers to a point in time and not a comparison to Baseline. This scoring is based upon investigator discretion.

Full Information

NCT ID

NCT02864082

First Posted

August 3, 2016

Last Updated

September 2, 2021

Sponsor

Patagonia Pharmaceuticals, LLC

1. Study Identification

Unique Protocol Identification Number

NCT02864082

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2021

Overall Recruitment Status

Completed

Study Start Date

March 8, 2017 (Actual)

Primary Completion Date

February 13, 2018 (Actual)

Study Completion Date

December 4, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Patagonia Pharmaceuticals, LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Congenital Ichthyosis

Keywords

X-linked, lamellar

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

19 (Actual)

8. Arms, Groups, and Interventions

Arm Title

PAT-001 0.1%

Arm Type

Experimental

Arm Description

Arm Title

PAT-001 0.2%

Arm Type

Experimental

Arm Description

Arm Title

Vehicle for PAT-001 0.1% arm

Arm Type

Placebo Comparator

Arm Description

Arm Title

Vehicle for PAT-001 0.2% arm

Arm Type

Placebo Comparator

Arm Description

Intervention Type

Drug

Intervention Name(s)

PAT-001, 0.1%

Other Intervention Name(s)

PAT-001

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

PAT-001, 0.2%

Other Intervention Name(s)

PAT-001

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

Vehicle for PAT-001 0.1%

Other Intervention Name(s)

Vehicle

Intervention Description

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

Intervention Name(s)

Vehicle for PAT-001 0.2%

Other Intervention Name(s)

Vehicle

Intervention Description

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

Primary Outcome Measure Information:

Title

Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8)

Description

The number of participants with AEs will be assessed by the investigator and the incidence (severity and causality) of any local and systemic AEs will be reported.

Time Frame

Day 0 through Day 57 (Weeks 0-8)

Title

Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle

Description

Time Frame

Up to Day 84 (Weeks 0-12)

Secondary Outcome Measure Information:

Title

Number of Participants Achieving Improvement to State of Clear, Almost Clear or Mild in the Investigator's Global Assessment (IGA) Using a Five-point Scale at Day 57 (Part 1)

Description

Time Frame

Up to Day 57

Title

Description

Time Frame

Up to Day 57 (Weeks 0-8)

Other Pre-specified Outcome Measures:

Title

Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints

Description

Serum concentrations for PAT-001 0.1% and PAT-001 0.2% looking at blood levels obtained at timepoints outlined

Time Frame

Day 1 (0,1, 2, 3, and 4 hours post Dose)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Zachary Rome, BS

Organizational Affiliation

Sponsor GmbH

Official's Role

Study Director

Facility Information:

Facility Name

TCR Medical Corporation

City

San Diego

State/Province

California

ZIP/Postal Code

92123

Country

United States

Facility Name

Yale Center for Clinical Investigation

City

New Haven

State/Province

Connecticut

ZIP/Postal Code

06519

Country

United States

Facility Name

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

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60611

Country

United States

Facility Name

Paddington Testing Co., Inc

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19103

Country

United States

Facility Name

Texas Dermatology and Laser Specialists

City

San Antonio

State/Province

Texas

ZIP/Postal Code

78218

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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