Congenital IchthyosisPhase 2NCT02864082

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

A Phase 2 study testing PAT-001, 0.1% and PAT-001, 0.2% in people with Congenital Ichthyosis, run by Patagonia Pharmaceuticals, LLC at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 12 years and older
Conditions the study is for: Congenital Ichthyosis
What is being tested?: PAT-001, 0.1% and PAT-001, 0.2%
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 19 participants
Where: 5 sitesUnited States
Study dates: 8 Mar 2017to 4 Dec 2018
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 12 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

United States5 sites

Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Yale Center for Clinical Investigation
New Haven, Connecticut, 06519
Paddington Testing Co., Inc
Philadelphia, Pennsylvania, 19103
Texas Dermatology and Laser Specialists
San Antonio, Texas, 78218
TCR Medical Corporation
San Diego, California, 92123

Other trials in United States

About this study

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

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.

Read the 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.

Conditions: Congenital Ichthyosis
Being tested: PAT-001, 0.1%, PAT-001, 0.2%, Vehicle for PAT-001 0.1%, Vehicle for PAT-001 0.2%
Keywords: X-linked, lamellar
Medical subject headings: Ichthyosis Vulgaris

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PAT-001, 0.1%Drug
Also called PAT-001
PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.
PAT-001, 0.2%Drug
Also called PAT-001
PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.
Vehicle for PAT-001 0.1%Drug
Also called Vehicle
Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.1%.
Vehicle for PAT-001 0.2%Drug
Also called Vehicle
Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.2%.

Study groups in NCT02864082
Study group	Role	What they receive
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. Receives: 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. Receives: 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. Receives: 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. Receives: Drug: Vehicle for PAT-001 0.2%

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: 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)
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.
Measured: 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
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.
Measured: Up to Day 57 (Weeks 0-8)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

8 Mar 20174 Dec 2018

First posted11 Aug 2016 (estimated)
Study start8 Mar 2017
Primary completion13 Feb 2018
Study completion4 Dec 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Congenital IchthyosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 19 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02864082
Sponsor's protocol ID: 205-9051-201
Status last verified: Sep 2021
First submitted: 3 Aug 2016
Last updated: 29 Sep 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 29 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Bilateral Comparison, Vehicle-Controlled, Safety and Tolerability Study of Topical PAT-001 for the Treatment of Congenital Ichthyosis
ClinicalTrials.gov ID: NCT02864082
Acronym / sponsor ID: 205-9051-201
Conditions: Congenital Ichthyosis
Keywords: X-linked, lamellar
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 19 participants (actual)
Lead sponsor: Patagonia Pharmaceuticals, LLCIndustry (pharma, biotech, devices)
Study start: 8 Mar 2017 (actual)
Primary completion: 13 Feb 2018 (actual)
Study completion: 4 Dec 2018 (actual)
First posted: 11 Aug 2016 (estimated)
Results: Posted 2 Sep 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 4

Arm	Type	Receives
PAT-001 0.1%	Experimental	Drug: 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.
PAT-001 0.2%	Experimental	Drug: 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.
Vehicle for PAT-001 0.1% arm	Placebo comparator	Drug: Vehicle for 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). The application of vehicle only lasts from Weeks 0-8.
Vehicle for PAT-001 0.2% arm	Placebo comparator	Drug: Vehicle for 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). The application of vehicle only lasts from Weeks 0-8.

Who may qualify

Ages: 12 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (6 listed)

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).

Key exclusion criteria (8 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
PAT-001, 0.1%Also: PAT-001PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.	Drug	PAT-001 0.1%
PAT-001, 0.2%Also: PAT-001PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.	Drug	PAT-001 0.2%
Vehicle for PAT-001 0.1%Also: VehicleVehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.1%.	Drug	Vehicle for PAT-001 0.1% arm
Vehicle for PAT-001 0.2%Also: VehicleVehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.2%.	Drug	Vehicle for PAT-001 0.2% arm

Primary outcome

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.

2 secondary outcomes

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
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)

Results and safety

Results posted to the registry on 2 Sep 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Group 1 PAT-001 0.1% and Vehicle	10	7
Group 2 PAT-001 0.2% and Vehicle	9	5

Primary outcome: 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). Number. Unit: participants

Measure	Group 1 PAT-001 0.1% n = 10	Group 2 PAT-001 0.2% n = 9	Group 1 Vehicle n = 10	Group 2 Vehicle n = 9
Number of Subjects with TEAE	7	7	5	5
Number of Treatment Emergent Adverse Events (TEAE) within treatment area	4	5	3	3
Number of Deaths due to TEAE	0	0	0	0
Number of Serious TEAE	0	1	0	0
Number of Patients who Discontinued in the trial due to TEAE	1	1	0	0
Number of Patients with Severe TEAE	1	1	0	0
Number of Patients with Moderate TEAE	3	3	2	2
Number of Patients with Mild TEAE	4	6	3	3

Population analysed: Number of Participants with Adverse events

Primary outcome: 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). Number. Unit: participants

Measure	Group 1 PAT-001 0.1% n = 10	Group 1 Vehicle n = 10	Group 2 PAT-001 0.2% n = 9	Group 2 Vehicle n = 9
Application site pruritis (severe)	1	0	0	0
Application site irritation (mild)	3	2	0	0
Application site dermatitis (moderate)	0	0	1	0
Application site rash (mild)	1	0	0	1
Application site reaction (mild)	0	0	1	0
Application site pain (moderate)	1	0	0	0
Application site Pruritis (moderate)	1	1	0	0
Application site Pruritis (mild)	1	1	0	0

Population analysed: Participants may have had more than one type of LSR

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: 84 Days. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Group 1 PAT-001 0.1%	0 of 10 (0%)	7 of 10 (70%)	0 of 10
Group 2 PAT-001 0.2%	1 of 9 (11.1%)	7 of 9 (77.8%)	0 of 9
Group 1 Vehicle	0 of 10 (0%)	2 of 10 (20%)	0 of 10
Group 2 Vehicle	0 of 9 (0%)	1 of 9 (11.1%)	0 of 9

Limitations and caveats (from the sponsor): Data should be interpreted with caution due to small sample sizes

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Zachary Rome, BS study director

Sponsor GmbH

5 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PAT-001, 0.1% and PAT-001, 0.2% in people with Congenital Ichthyosis, run by Patagonia Pharmaceuticals, LLC at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 2 Sep 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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 study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02864082.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2021.