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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02864082
Recruitment Status : Completed
First Posted : August 11, 2016
Last Update Posted : June 19, 2019
Sponsor:
Information provided by (Responsible Party):
Patagonia Pharmaceuticals, LLC

Tracking Information
First Submitted Date  ICMJE August 3, 2016
First Posted Date  ICMJE August 11, 2016
Last Update Posted Date June 19, 2019
Actual Study Start Date  ICMJE March 8, 2017
Actual Primary Completion Date February 13, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 8, 2016)
  • 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 Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 8, 2016)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: August 8, 2016)
Pharmacokinetics (AUC) [ Time Frame: Day 1 (0,1, 2, 3, and 4 hours post Dose) ]
Area under the plasma concentration versus time curve (AUC)
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE A Safety and Tolerability Study of Topical PAT-001 in Congenital Ichthyosis
Official Title  ICMJE 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 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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Congenital Ichthyosis
Intervention  ICMJE
  • Drug: PAT-001, 0.1%
    PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
    Other Name: PAT-001
  • Drug: PAT-001, 0.2%
    PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
    Other Name: PAT-001
  • Drug: Vehicle
    Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001.
Study Arms  ICMJE
  • Group 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).

    Part 2: Patients will apply PAT-001, 0.1% to both Treatment Areas.

    Interventions:
    • Drug: PAT-001, 0.1%
    • Drug: Vehicle
  • Group 2 (Part 1)

    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).

    Part 2: Patients will apply PAT-001, 0.2% to both Treatment Areas.

    Interventions:
    • Drug: PAT-001, 0.2%
    • Drug: Vehicle
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 20, 2017)
19
Original Estimated Enrollment  ICMJE
 (submitted: August 8, 2016)
18
Actual Study Completion Date  ICMJE December 4, 2018
Actual Primary Completion Date February 13, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02864082
Other Study ID Numbers  ICMJE 205-9051-201
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Patagonia Pharmaceuticals, LLC
Study Sponsor  ICMJE Patagonia Pharmaceuticals, LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Zachary Rome, BS Sponsor GmbH
PRS Account Patagonia Pharmaceuticals, LLC
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP