Congenital Ichthyosis
Conditions
Keywords
X-linked, lamellar
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.
Interventions
DRUGPAT-001, 0.1%
PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
DRUGPAT-001, 0.2%
PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
DRUGVehicle 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%.
DRUGVehicle 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%.
Sponsors
Patagonia Pharmaceuticals, LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | 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 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | 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 | 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. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | 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 |
Countries
United States
Participant flow
Recruitment details
Recruitment: March 8, 2017-February 13, 2018. Recruited at 5 clinics in United States associated with US Universities by practicing dermatologists
Pre-assignment details
Each patient received both vehicle application and either PAT-001 0.1% or PAT-001 0.2% on 2 different identical matching parts of their bodies (e.g, upper thighs). Since each of the 19 patients were treated with both PAT-001 and vehicle, the number of treatment areas was 38 (19 x 2) but the number of patients was 19.
Participants by arm
| Arm | Count |
|---|---|
| Group 1 PAT-001 0.1% and Vehicle Part 1 (weeks 0-8): 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 (weeks 8-12): Patients will apply PAT-001, 0.1% to both Treatment Areas.
PAT-001, 0.1%: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001. | 10 |
| Group 2 PAT-001 0.2% and Vehicle Part 1 (weeks 0-8): 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: (weeks 8-12) Patients will apply PAT-001, 0.2% to both Treatment Areas.
PAT-001, 0.2%: PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
Vehicle: Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001. | 9 |
| Total | 19 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 1 |
| Overall Study | Lost to Follow-up | 0 | 1 |
| Overall Study | Withdrawal by Subject | 2 | 2 |
Baseline characteristics
| Characteristic | Group 1 PAT-001 0.1% and Vehicle | Group 2 PAT-001 0.2% and Vehicle | Total |
|---|---|---|---|
| Age, Continuous | 37.5 years STANDARD_DEVIATION 20.3 | 47.2 years STANDARD_DEVIATION 18.4 | 42 years STANDARD_DEVIATION 19.3 |
| BMI | 28.8 kg/m^2 | 26.4 kg/m^2 | 27.7 kg/m^2 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 10 Participants | 9 Participants | 19 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment United States | 10 participants | 9 participants | 19 participants |
| Sex: Female, Male Female | 5 Participants | 4 Participants | 9 Participants |
| Sex: Female, Male Male | 5 Participants | 5 Participants | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 10 | 0 / 9 | 0 / 10 | 0 / 9 |
| other Total, other adverse events | 7 / 10 | 7 / 9 | 2 / 10 | 1 / 9 |
| serious Total, serious adverse events | 0 / 10 | 1 / 9 | 0 / 10 | 0 / 9 |
Outcome results
Primary
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.
Time frame: Up to Day 84 (Weeks 0-12)
Population: Participants may have had more than one type of LSR
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Group 1 PAT-001 0.1% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site dermatitis (moderate) | 0 participants |
| Group 1 PAT-001 0.1% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Pruritis (mild) | 1 participants |
| Group 1 PAT-001 0.1% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site pruritis (severe) | 1 participants |
| Group 1 PAT-001 0.1% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site reaction (mild) | 0 participants |
| Group 1 PAT-001 0.1% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site rash (mild) | 1 participants |
| Group 1 PAT-001 0.1% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site irritation (mild) | 3 participants |
| Group 1 PAT-001 0.1% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Dermatitis (mild) | 0 participants |
| Group 1 PAT-001 0.1% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Pruritis (moderate) | 1 participants |
| Group 1 PAT-001 0.1% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site pain (moderate) | 1 participants |
| Group 2 PAT-001 0.2% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site reaction (mild) | 0 participants |
| Group 2 PAT-001 0.2% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site pruritis (severe) | 0 participants |
| Group 2 PAT-001 0.2% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site pain (moderate) | 0 participants |
| Group 2 PAT-001 0.2% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site irritation (mild) | 2 participants |
| Group 2 PAT-001 0.2% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site dermatitis (moderate) | 0 participants |
| Group 2 PAT-001 0.2% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site rash (mild) | 0 participants |
| Group 2 PAT-001 0.2% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Pruritis (moderate) | 1 participants |
| Group 2 PAT-001 0.2% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Pruritis (mild) | 1 participants |
| Group 2 PAT-001 0.2% | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Dermatitis (mild) | 0 participants |
| Group 1 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site reaction (mild) | 1 participants |
| Group 1 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site irritation (mild) | 0 participants |
| Group 1 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Dermatitis (mild) | 1 participants |
| Group 1 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Pruritis (mild) | 0 participants |
| Group 1 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site pain (moderate) | 0 participants |
| Group 1 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site pruritis (severe) | 0 participants |
| Group 1 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site rash (mild) | 0 participants |
| Group 1 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Pruritis (moderate) | 0 participants |
| Group 1 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site dermatitis (moderate) | 1 participants |
| Group 2 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site dermatitis (moderate) | 0 participants |
| Group 2 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site reaction (mild) | 0 participants |
| Group 2 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site irritation (mild) | 0 participants |
| Group 2 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site pain (moderate) | 0 participants |
| Group 2 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Dermatitis (mild) | 2 participants |
| Group 2 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Pruritis (moderate) | 0 participants |
| Group 2 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site pruritis (severe) | 0 participants |
| Group 2 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site Pruritis (mild) | 0 participants |
| Group 2 Vehicle | Incidence of Local Skin Reactions (LSRs) in Participants Treated With PAT-001 0.1%, 0.2% and/or Vehicle | Application site rash (mild) | 1 participants |
Primary
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.
Time frame: Day 0 through Day 57 (Weeks 0-8)
Population: Number of Participants with Adverse events
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients where the TEAE was Considered Probably Related to Treatment | 1 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Not Related to Treatment | 1 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Mild TEAE | 4 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Subjects with TEAE | 7 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Definitely Related to Treatment | 1 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Deaths due to TEAE | 0 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Serious TEAE | 0 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Treatment Emergent Adverse Events (TEAE) within treatment area | 4 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Possibly Related to Treatment | 1 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients who Discontinued in the trial due to TEAE | 1 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Severe TEAE | 1 participants |
| Group 1 PAT-001 0.1% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Moderate TEAE | 3 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Treatment Emergent Adverse Events (TEAE) within treatment area | 5 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Moderate TEAE | 3 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Serious TEAE | 1 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Definitely Related to Treatment | 1 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients where the TEAE was Considered Probably Related to Treatment | 0 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Mild TEAE | 6 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Subjects with TEAE | 7 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Severe TEAE | 1 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients who Discontinued in the trial due to TEAE | 1 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Deaths due to TEAE | 0 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Not Related to Treatment | 5 participants |
| Group 2 PAT-001 0.2% | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Possibly Related to Treatment | 4 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Mild TEAE | 3 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Subjects with TEAE | 5 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Treatment Emergent Adverse Events (TEAE) within treatment area | 3 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Deaths due to TEAE | 0 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Serious TEAE | 0 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients who Discontinued in the trial due to TEAE | 0 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Severe TEAE | 0 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Moderate TEAE | 2 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Definitely Related to Treatment | 0 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients where the TEAE was Considered Probably Related to Treatment | 0 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Possibly Related to Treatment | 1 participants |
| Group 1 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Not Related to Treatment | 4 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Severe TEAE | 0 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients who Discontinued in the trial due to TEAE | 0 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Subjects with TEAE | 5 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients where the TEAE was Considered Probably Related to Treatment | 0 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Serious TEAE | 0 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Deaths due to TEAE | 0 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Not Related to Treatment | 4 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Possibly Related to Treatment | 1 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Mild TEAE | 3 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients with Moderate TEAE | 2 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Treatment Emergent Adverse Events (TEAE) within treatment area | 3 participants |
| Group 2 Vehicle | Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) | Number of Patients Where the TEAE was Considered Definitely Related to Treatment | 0 participants |
Secondary
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.
Time frame: Up to Day 57 (Weeks 0-8)
Population: IGA score improvement of at least 1 point on IGA for scaling, erythema, papulation/lichenification and fissuring at Day 57 in patients receiving continuous 0.1% and 0.2% PAT-001
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Group 1 PAT-001 0.1% | 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 | Scaling | 9 participants |
| Group 1 PAT-001 0.1% | 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 | Fissuring | 6 participants |
| Group 1 PAT-001 0.1% | 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 | Papulation/lichenification | 9 participants |
| Group 1 PAT-001 0.1% | 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 | Erythema | 5 participants |
| Group 2 PAT-001 0.2% | 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 | Fissuring | 6 participants |
| Group 2 PAT-001 0.2% | 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 | Papulation/lichenification | 6 participants |
| Group 2 PAT-001 0.2% | 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 | Erythema | 5 participants |
| Group 2 PAT-001 0.2% | 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 | Scaling | 6 participants |
| Group 1 Vehicle | 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 | Papulation/lichenification | 6 participants |
| Group 1 Vehicle | 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 | Fissuring | 5 participants |
| Group 1 Vehicle | 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 | Erythema | 4 participants |
| Group 1 Vehicle | 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 | Scaling | 7 participants |
| Group 2 Vehicle | 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 | Erythema | 6 participants |
| Group 2 Vehicle | 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 | Fissuring | 6 participants |
| Group 2 Vehicle | 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 | Scaling | 8 participants |
| Group 2 Vehicle | 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 | Papulation/lichenification | 7 participants |
Secondary
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.
Time frame: Up to Day 57
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Group 1 PAT-001 0.1% | 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) | 9 Participants |
| Group 2 PAT-001 0.2% | 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) | 8 Participants |
| Group 1 Vehicle | 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) | 6 Participants |
| Group 2 Vehicle | 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) | 7 Participants |
Other Pre-specified
Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints
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)
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1 PAT-001 0.1% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | 1 Hour | 1.5 ng/mL | Standard Deviation 0.6505 |
| Group 1 PAT-001 0.1% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | 3 Hours | 1.37 ng/mL | Standard Deviation 0.297 |
| Group 1 PAT-001 0.1% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | 2 Hours | 1.425 ng/mL | Standard Deviation 0.5303 |
| Group 1 PAT-001 0.1% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | 4 Hours | 1.375 ng/mL | Standard Deviation 0.0778 |
| Group 1 PAT-001 0.1% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | Pre-Dose | 1.495 ng/mL | Standard Deviation 0.6718 |
| Group 2 PAT-001 0.2% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | 4 Hours | 1.453 ng/mL | Standard Deviation 0.3625 |
| Group 2 PAT-001 0.2% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | Pre-Dose | 2.48 ng/mL | Standard Deviation 1.795 |
| Group 2 PAT-001 0.2% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | 1 Hour | 2.227 ng/mL | Standard Deviation 1.3725 |
| Group 2 PAT-001 0.2% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | 2 Hours | 2.12 ng/mL | Standard Deviation 1.2257 |
| Group 2 PAT-001 0.2% | Pharmacokinetics of PAT-001 0.1% and 0.2% at Different Timepoints | 3 Hours | 2.063 ng/mL | Standard Deviation 1.5332 |