An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in the Treatment of Patients With Ichthyoses

Part of paid clinical trials in Chicago, Illinois.

Sponsor
Northwestern University
Study ID
NCT04549792
Phase
EARLY_PHASE1
Status
Completed

Conditions

  • Ichthyosis

Eligibility Criteria

Sex
ALL
Age
6 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Ustekinumab — DRUG
    Each subject will receive ustekinumab at Baseline (Day 0) and Months 1, 3, 5, 7, 9, and 11. During the LTE, subjects will receive injections every 8 weeks for one year: Month 13, Month 15, Month 17, Month 19, Month 21, and Month 23. Subjects will come back in for a follow-up visit at Month 25 for an end of study visit (no drug administration).

Study Details

The ichthyoses are a group of lifelong genetic disorders that share characteristics of generalized skin thickening, scaling and underlying cutaneous inflammation. The vast majority are orphan disorders and are associated with extremely poor quality of life related to social ostracism from altered appearance, associated itchiness and discomfort, and functional limitations from the skin disease. Among the more common "orphan" forms of ichthyosis are autosomal recessive congenital ichthyosis (ARCI; includes lamellar ichthyosis/LI and congenital ichthyosiform erythroderma/CIE), Netherton syndrome (NS) and epidermolytic ichthyosis (EI). However, there are dozens of other syndromic and non-syndromic ichthyotic disorders as well. Therapy is time-consuming for patients or parents and is supportive, focusing on clearance of the scaling. There are no therapies based on our growing understanding of what causes the disease. We have recently found marked elevations in Th17/IL-23 pathway cytokines and chemokines in the skin of individuals with ichthyosis, most similar to the inflammatory pattern of psoriasis. While the significance of the high expression of Th17/IL-23 pathway genes across all forms of ichthyosis studied to date is unknown, the high expression of genes of the Th17/IL-23 pathway in psoriasis is thought to be causative for the disease manifestations. We propose that IL-12/IL-23 -targeting therapeutics will safely suppress the inflammation and possibly the other features of ichthyosis, improving quality of life. As a proof-of-concept study, we propose to treat children (6 years of age and higher) and adults with ichthyotic disorders with ustekinumab in an open-label trial to serially assess clinical response to and safety of ustekinumab for this group of disorders.

Key Dates

Start date
Apr 1, 2021
Status verified
Jan 2025
Primary completion
Jan 12, 2024
Completion
Jun 26, 2024

Study Design

Enrollment
13 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Open Label

Primary Outcome Measure

Reduction in Total Ichthyosis Severity Score [ Time Frame: 7 months after initiation of study drug ]

Locations (1)

FacilityCityStateZIPSite coordinators
Northwestern University/Lurie Children's HospitalChicagoIllinois60611-

Find similar trials in Chicago, IL

By research site
Northwestern University/Lurie Children's Hospital· Chicago, IL

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