A Safety and Tolerability Study of Topical PAT-001 in Congenital Ichthyosis
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|ClinicalTrials.gov Identifier: NCT02864082|
Recruitment Status : Completed
First Posted : August 11, 2016
Results First Posted : September 2, 2021
Last Update Posted : September 29, 2021
|Condition or disease||Intervention/treatment||Phase|
|Congenital Ichthyosis||Drug: PAT-001, 0.1% Drug: PAT-001, 0.2% Drug: Vehicle for PAT-001 0.1% Drug: Vehicle for PAT-001 0.2%||Phase 2|
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 :||Interventional (Clinical Trial)|
|Actual Enrollment :||19 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Randomized, Bilateral Comparison, Vehicle-Controlled, Safety and Tolerability Study of Topical PAT-001 for the Treatment of Congenital Ichthyosis|
|Actual Study Start Date :||March 8, 2017|
|Actual Primary Completion Date :||February 13, 2018|
|Actual Study Completion Date :||December 4, 2018|
Experimental: 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.
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
Experimental: 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.
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
Placebo Comparator: 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.
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%.
Other Name: Vehicle
Placebo Comparator: 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.
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%.
Other Name: Vehicle
- Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) [ Time Frame: Day 0 through Day 57 (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 [ Time Frame: Up to Day 84 (Weeks 0-12) ]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.
- 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) [ Time Frame: Up to Day 57 ]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 [ Time Frame: Up to Day 57 (Weeks 0-8) ]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.
- Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints [ Time Frame: Day 1 (0,1, 2, 3, and 4 hours post Dose) ]Serum concentrations for PAT-001 0.1% and PAT-001 0.2% looking at blood levels obtained at timepoints outlined
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02864082
|United States, California|
|TCR Medical Corporation|
|San Diego, California, United States, 92123|
|United States, Connecticut|
|Yale Center for Clinical Investigation|
|New Haven, Connecticut, United States, 06519|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60611|
|United States, Pennsylvania|
|Paddington Testing Co., Inc|
|Philadelphia, Pennsylvania, United States, 19103|
|United States, Texas|
|Texas Dermatology and Laser Specialists|
|San Antonio, Texas, United States, 78218|
|Study Director:||Zachary Rome, BS||Sponsor GmbH|