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

NCT02864082 | Completed Phase 2 Results FDA Drug

• 1 other identifier: 205-9051-201
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 5

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.

Conditions

Congenital Ichthyosis

Keywords

X-linked, lamellar

Outcome Measures

Primary Outcomes (2)

• 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.

  Day 0 through Day 57 (Weeks 0-8)

• 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.

  Up to Day 84 (Weeks 0-12)

Secondary Outcomes (2)

• 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)

  Up to Day 57

• 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

  Up to Day 57 (Weeks 0-8)

Other Outcomes (1)

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

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

Study Arms (4)

PAT-001 0.1%

EXPERIMENTAL

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.

Drug: PAT-001, 0.1%

PAT-001 0.2%

EXPERIMENTAL

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.

Drug: PAT-001, 0.2%

Vehicle for PAT-001 0.1% arm

PLACEBO COMPARATOR

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.

Drug: Vehicle for PAT-001 0.1%

Vehicle for PAT-001 0.2% arm

PLACEBO COMPARATOR

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.

Drug: Vehicle for PAT-001 0.2%

Interventions

PAT-001, 0.1% DRUG

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

Also known as: PAT-001

PAT-001 0.1%

PAT-001, 0.2% DRUG

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

Also known as: PAT-001

PAT-001 0.2%

Vehicle for PAT-001 0.1% DRUG

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

Also known as: Vehicle

Vehicle for PAT-001 0.1% arm

Vehicle for PAT-001 0.2% DRUG

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

Also known as: Vehicle

Vehicle for PAT-001 0.2% arm

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Patagonia Pharmaceuticals, LLC: lead

Study Sites (5)

TCR Medical Corporation

San Diego, California, 92123, United States

Location

Yale Center for Clinical Investigation

New Haven, Connecticut, 06519, United States

Location

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

Chicago, Illinois, 60611, United States

Location

Paddington Testing Co., Inc

Philadelphia, Pennsylvania, 19103, United States

Location

Texas Dermatology and Laser Specialists

San Antonio, Texas, 78218, United States

Location

MeSH Terms

Conditions

Ichthyosis Vulgaris

Condition Hierarchy (Ancestors)

Ichthyosis; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Keratosis; Skin Diseases; Skin and Connective Tissue Diseases

Limitations and Caveats

Data should be interpreted with caution due to small sample sizes

Results Point of Contact

• Title: Alan Mendelsohn, MD
• Organization: Timber Pharmaceuticals

amendelsohn@timberpharma.com

2673004249

Study Officials

• Zachary Rome, BS

  Sponsor GmbH

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 3, 2016
• First Posted: August 11, 2016
• Study Start: March 8, 2017
• Primary Completion: February 13, 2018
• Study Completion: December 4, 2018
• Last Updated: September 29, 2021
• Results First Posted: September 2, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)