Repurposing Dupilumab for Management of Pruritic Genetic Inflammatory Skin Disorders

ConditionSkin Diseases

Trial statusRecruiting

Trial phaseEarly Phase 1

Trial typeInterventional

Biological sexAll

Age6+

SponsorNorthwestern University

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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)

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

Dupilumab
Drug
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.

Treatment groups

30 Participants

are divided into 1 treatment group

Group A: Open-Label DupilumabExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

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).

Time frame

16 weeks

Secondary outcomes

Occurrence of Adverse Events during Dupilumab Treatment

Number of adverse events (AEs) and serious AEs (SAEs) through Weeks 16, 52, and 104.

Time frame

104 weeks

Sponsors and contacts

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Northwestern University

Lead sponsor

Source ClinicalTrials.gov

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