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Repurposing Dupilumab for Management of Pruritic Genetic Inflammatory Skin Disorders

Primary Purpose

Skin Diseases

Status
Recruiting
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Dupilumab
Sponsored by
Northwestern University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Skin Diseases focused on measuring Itch, Pruritus, epidermolysis bullosa, ichthyosis, genetic skin disorders

Eligibility Criteria

6 Months - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Male or female > 6 months of age at screening visit Clinical diagnosis of a genetic skin disorder at the screening visit, ideally with genetic or histological confirmation. Must have had the gene with one or more variants identified by genotyping. If the genotype has not been performed or has not been performed at a CLIA-approved laboratory, be willing to provide a sample (saliva, buccal swab, blood) for genetic testing before starting the dupilumab. Average Itch Numerical Rating Scale (NRS) ≥ 4 and Worst Itch NRS of at least 5 during the previous 7 days (self-reported if >8 years old; proxy reported if under 8 years) Must be willing to provide information weekly about Average and Worst Itch/self- or proxy-assessed severity and wear the sensor device to track itch and sleep weekly throughout the first 24 weeks of the trial (Parts A and B). Must be willing and able to adhere to the prohibitions and restrictions specified in this protocol. Subject, parent/caregiver or legal guardians, as appropriate, are able to understand and complete the study requirements and study-related questionnaires Exclusion Criteria: Subjects < 6 months of age at screening visit. Unable to provide informed consent or assent (or who do not have consent from a Legally Authorized Representative if < 18 years). Diagnosis of ichthyosis vulgaris as the sole inherited disorder Used of dupilumab within 5 drug half-lives (105 days) of baseline visit Subjects who have used any of the following treatments within 4 weeks, or within a period equal to 5 times the half-life of the drug, before the baseline visit, whichever is longer: Immunosuppressive/immunomodulating drugs (eg, systemic corticosteroids, cyclosporine, mycophenolate-mofetil, IFN-γ, Janus kinase inhibitors, azathioprine, methotrexate, etc.), systemic anti-inflammatory medication, or phototherapy Other biologics: within 5 half-lives (if known) or 16 weeks, whichever is longer Initiation of topical or systemic retinoids, topical keratolytics, or topical anti-inflammatory agents within 4 weeks before study start/Part A (systemic retinoids and topical medications/emollients can be used during the trial if started at least 4 wks before the observation period and continued throughout Parts A and B). Note: Rescue therapy for disease flares or local infection will be allowed per investigator discretion but must be for no more than a total of 1 week during any 4-week period and, if topical, involve application to less than 10% BSA. Subjects with active infections or recent history of serious infections, malignancies or history of malignancies, or any severe, progressive, or uncontrolled renal, hepatic, hematologic, endocrine, pulmonary, cardiac, neurologic or psychiatric cerebral disease, or signs or symptoms thereof. It is recognized that patients with ichthyosis may have arthritis, while patients with junctional or dystrophic EB may have a variety of associated issues (eg nutritional, anemia, etc). The decision to include will be based on investigator's discretion but must reflect the likelihood for stable disease and lack of anticipated interference with assessment of itch. Treatment with a live (attenuated) vaccine within 4 weeks before the Week 0 visit when dupilumab is initiated; use of vaccination during the study requires consultation with the study investigator and primary care provider. Active acute or chronic infection requiring treatment with systemic antibiotics/ anti-virals/ anti-fungals within 2 weeks before the initiation of dupilumab (start of dupilumab can be delayed). Delay in initiation because of treatment with a topical antimicrobial to a localized superficial site will be determined by the investigator.

Sites / Locations

  • Ann & Robert H. Lurie Children's Hospital of ChicagoRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open-Label Dupilumab

Arm Description

Outcomes

Primary Outcome Measures

Change in Itch
The Primary Objective of this study is to determine if dupilumab changes itch across a range of pruritic genetic disorders of the skin. At least 50% of patients achieving reduction by at least 2 points in Worst Itch NRS (minimal meaningful reduction in Worst Itch is 1-2 points) We will compare the average Worst Itch NRS during an 8-week observational period (not a static single point) with the average Worst Itch NRS during the dupilumab treatment period at Weeks 9-16 on dupilumab (by which time itch is maximally suppressed by dupilumab in atopic dermatitis trials). This will allow for the possibility of fluctuation in itch (which we also hope to capture in the Part A observation period).

Secondary Outcome Measures

Occurrence of Adverse Events during Dupilumab Treatment
Number of adverse events (AEs) and serious AEs (SAEs) through Weeks 16, 52, and 104.

Full Information

First Posted
December 5, 2022
Last Updated
October 1, 2023
Sponsor
Northwestern University
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1. Study Identification

Unique Protocol Identification Number
NCT05649098
Brief Title
Repurposing Dupilumab for Management of Pruritic Genetic Inflammatory Skin Disorders
Official Title
Repurposing Dupilumab for Management of Pruritic Genetic Inflammatory Skin Disorders: a Single-Site Pilot Study
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 12, 2023 (Actual)
Primary Completion Date
June 30, 2025 (Anticipated)
Study Completion Date
June 1, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Northwestern University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
Severe itch is a common symptom of many genetic skin disorders and leads to a negative impact on patient quality of life. The investigators hypothesize that: a) intervention with dupilumab will improve itch in patients with pruritic genetic inflammatory skin disorders, even those not recognized to be Th2-driven; and b) the administration of dupilumab will be well-tolerated, regardless of underlying genetic skin disorder. The total clinical study duration will be 26 months (104 Weeks). The treatment period will include a 16-week open-label phase and a 20-month long-term extension phase for those who qualify and wish to continue.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Skin Diseases
Keywords
Itch, Pruritus, epidermolysis bullosa, ichthyosis, genetic skin disorders

7. Study Design

Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Open-Label Dupilumab
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Dupilumab
Intervention Description
The treatment period will include a 16-week open-label phase and a 20-month long-term extension phase for those who qualify and wish to continue.
Primary Outcome Measure Information:
Title
Change in Itch
Description
The Primary Objective of this study is to determine if dupilumab changes itch across a range of pruritic genetic disorders of the skin. At least 50% of patients achieving reduction by at least 2 points in Worst Itch NRS (minimal meaningful reduction in Worst Itch is 1-2 points) We will compare the average Worst Itch NRS during an 8-week observational period (not a static single point) with the average Worst Itch NRS during the dupilumab treatment period at Weeks 9-16 on dupilumab (by which time itch is maximally suppressed by dupilumab in atopic dermatitis trials). This will allow for the possibility of fluctuation in itch (which we also hope to capture in the Part A observation period).
Time Frame
16 weeks
Secondary Outcome Measure Information:
Title
Occurrence of Adverse Events during Dupilumab Treatment
Description
Number of adverse events (AEs) and serious AEs (SAEs) through Weeks 16, 52, and 104.
Time Frame
104 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female > 6 months of age at screening visit Clinical diagnosis of a genetic skin disorder at the screening visit, ideally with genetic or histological confirmation. Must have had the gene with one or more variants identified by genotyping. If the genotype has not been performed or has not been performed at a CLIA-approved laboratory, be willing to provide a sample (saliva, buccal swab, blood) for genetic testing before starting the dupilumab. Average Itch Numerical Rating Scale (NRS) ≥ 4 and Worst Itch NRS of at least 5 during the previous 7 days (self-reported if >8 years old; proxy reported if under 8 years) Must be willing to provide information weekly about Average and Worst Itch/self- or proxy-assessed severity and wear the sensor device to track itch and sleep weekly throughout the first 24 weeks of the trial (Parts A and B). Must be willing and able to adhere to the prohibitions and restrictions specified in this protocol. Subject, parent/caregiver or legal guardians, as appropriate, are able to understand and complete the study requirements and study-related questionnaires Exclusion Criteria: Subjects < 6 months of age at screening visit. Unable to provide informed consent or assent (or who do not have consent from a Legally Authorized Representative if < 18 years). Diagnosis of ichthyosis vulgaris as the sole inherited disorder Used of dupilumab within 5 drug half-lives (105 days) of baseline visit Subjects who have used any of the following treatments within 4 weeks, or within a period equal to 5 times the half-life of the drug, before the baseline visit, whichever is longer: Immunosuppressive/immunomodulating drugs (eg, systemic corticosteroids, cyclosporine, mycophenolate-mofetil, IFN-γ, Janus kinase inhibitors, azathioprine, methotrexate, etc.), systemic anti-inflammatory medication, or phototherapy Other biologics: within 5 half-lives (if known) or 16 weeks, whichever is longer Initiation of topical or systemic retinoids, topical keratolytics, or topical anti-inflammatory agents within 4 weeks before study start/Part A (systemic retinoids and topical medications/emollients can be used during the trial if started at least 4 wks before the observation period and continued throughout Parts A and B). Note: Rescue therapy for disease flares or local infection will be allowed per investigator discretion but must be for no more than a total of 1 week during any 4-week period and, if topical, involve application to less than 10% BSA. Subjects with active infections or recent history of serious infections, malignancies or history of malignancies, or any severe, progressive, or uncontrolled renal, hepatic, hematologic, endocrine, pulmonary, cardiac, neurologic or psychiatric cerebral disease, or signs or symptoms thereof. It is recognized that patients with ichthyosis may have arthritis, while patients with junctional or dystrophic EB may have a variety of associated issues (eg nutritional, anemia, etc). The decision to include will be based on investigator's discretion but must reflect the likelihood for stable disease and lack of anticipated interference with assessment of itch. Treatment with a live (attenuated) vaccine within 4 weeks before the Week 0 visit when dupilumab is initiated; use of vaccination during the study requires consultation with the study investigator and primary care provider. Active acute or chronic infection requiring treatment with systemic antibiotics/ anti-virals/ anti-fungals within 2 weeks before the initiation of dupilumab (start of dupilumab can be delayed). Delay in initiation because of treatment with a topical antimicrobial to a localized superficial site will be determined by the investigator.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Northwestern Dermatology CTU
Phone
312-227-6817
Email
NUderm-research@northwestern.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Amy Paller
Organizational Affiliation
Northwestern University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dermatology CTU
Email
NUderm-research@northwestern.edu
First Name & Middle Initial & Last Name & Degree
Amy Paller

12. IPD Sharing Statement

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Repurposing Dupilumab for Management of Pruritic Genetic Inflammatory Skin Disorders

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