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NCT04549792 · ClinicalTrials.gov registry record · Early Phase 1
An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in the Treatment of Patients With Ichthyoses
A Early Phase 1 study, sponsored by Northwestern University.
- Completed
- Registry status
- Early Phase 1
- Development phase
- 13
- Enrollment target
NCT04549792: Completed Early Phase 1 study, sponsored by Northwestern University.
NCT04549792 is a Early Phase 1 study that has completed, run by Northwestern University. The registered enrollment target is 13 participants, below the 53-participant average among 2,764 other Early Phase 1 trials with a reported enrollment target (75% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04549792, a Early Phase 1 study, has completed, sponsored by Northwestern University.
- COMPLETED
- Registry status
- Early Phase 1
- Development phase
- 13 participants
- Enrollment target
Study Summary
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.
Primary Outcome
To evaluate the efficacy of ustekinumab for ichthyosis, as measured by an at least 50% reduction in severity using the Ichthyosis Severity Score (ISS) measure. The ISS measures from 0-32, with 0 being the least clinically severe and 32 being the most clinically severe.
Interventions
- DRUG Ustekinumab
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2021-04-01 |
| Est. Completion | 2024-06-26 |
| Phase | Early Phase 1 |
What the finished NCT04549792 record still lists
NCT04549792 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 13 participants, a relatively small participant target, below the 53-participant average among 2,764 other Early Phase 1 trials with a reported enrollment target (75% lower).
The record links to 0 conditions, and to 1 intervention - of which Ustekinumab is the first listed.
NCT04549792 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04549792 about?
NCT04549792 is a clinical study titled "An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in the Treatment of Patients With Ichthyoses". 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 ap...
What is the current status of trial NCT04549792?
This trial is currently completed. It is a Early Phase 1 study. The enrollment target is 13 participants. The study started on 2021-04-01. Estimated completion is 2024-06-26.
What interventions are being tested in trial NCT04549792?
The interventions under investigation include: Ustekinumab (DRUG).
Who is sponsoring clinical trial NCT04549792?
This trial is sponsored by Northwestern University, which has 1,209 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04549792's enrollment target sits among peer trials
13 1504th of 2000 higher than 472 of 2,000 other Early Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Early Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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