Trial Outcomes & Findings for An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in the Treatment of Patients With Ichthyoses (NCT NCT04549792)
NCT ID: NCT04549792
Last Updated: 2025-02-12
Results Overview
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.
Recruitment status
COMPLETED
Study phase
EARLY_PHASE1
Target enrollment
13 participants
Primary outcome timeframe
7 months after initiation of study drug
Results posted on
2025-02-12
Participant Flow
Participant milestones
| Measure |
Open Label
Ustekinumab: 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).
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|---|---|
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Overall Study
STARTED
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13
|
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Overall Study
COMPLETED
|
13
|
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in the Treatment of Patients With Ichthyoses
Baseline characteristics by cohort
| Measure |
Open Label
n=13 Participants
Ustekinumab: 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).
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|---|---|
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Subtype
NIPAL4-CIE
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1 Participants
n=5 Participants
|
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Subtype
GJA-1-Erythrokeratodermia variabilis et progressiva
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1 Participants
n=5 Participants
|
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Age, Continuous
|
11.8 years
STANDARD_DEVIATION 7.8 • n=5 Participants
|
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Sex: Female, Male
Female
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7 Participants
n=5 Participants
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Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Hispanic or Latino
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2 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Black
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1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · White
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9 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
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1 Participants
n=5 Participants
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Race/Ethnicity, Customized
Race · Mixed
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2 Participants
n=5 Participants
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Region of Enrollment
United States
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13 participants
n=5 Participants
|
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Subtype
Harlequin Ichthyosis (HI)
|
6 Participants
n=5 Participants
|
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Subtype
Netherton Syndrome (NS)
|
3 Participants
n=5 Participants
|
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Subtype
KRT10-EI/KRT1-EI
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 7 months after initiation of study drugTo 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.
Outcome measures
| Measure |
Open Label
n=13 Participants
Ustekinumab: 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).
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|---|---|
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Reduction in Total Ichthyosis Severity Score
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-12.2 units on a scale
Standard Deviation 29.9
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PRIMARY outcome
Timeframe: 7 months after initiation of study drugTo evaluate the safety of ustekinumab for ichthyosis, based on occurrence of bacterial and fungal infections
Outcome measures
| Measure |
Open Label
n=13 Participants
Ustekinumab: 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).
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|---|---|
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Occurence of Bacterial and Fungal Infections
Superficial skin infections
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3 infections
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Occurence of Bacterial and Fungal Infections
Streptococcal throat infection
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2 infections
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Occurence of Bacterial and Fungal Infections
Onychomycosis
|
1 infections
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Adverse Events
Open Label
Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Open Label
n=13 participants at risk
Ustekinumab: 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).
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|---|---|
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Infections and infestations
Hospitalization secondary to sepsis
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15.4%
2/13 • Number of events 7 • 100 weeks
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Other adverse events
| Measure |
Open Label
n=13 participants at risk
Ustekinumab: 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).
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|---|---|
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General disorders
Headache
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23.1%
3/13 • Number of events 3 • 100 weeks
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Gastrointestinal disorders
Abdominal pain
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Reproductive system and breast disorders
Amenorrhea
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Musculoskeletal and connective tissue disorders
Broken wrist
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Infections and infestations
COVID-19 Infection
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Eye disorders
Dry eyes
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Skin and subcutaneous tissue disorders
Pustular psoriasis like eruption
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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General disorders
Heat exhaustion
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7.7%
1/13 • Number of events 2 • 100 weeks
|
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Skin and subcutaneous tissue disorders
Increased pain associated with ichthyosis
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7.7%
1/13 • Number of events 1 • 100 weeks
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Skin and subcutaneous tissue disorders
Miliaria
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Infections and infestations
Onychomycosis
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Surgical and medical procedures
Tooth extraction
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Skin and subcutaneous tissue disorders
Strep Throat Infection
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Skin and subcutaneous tissue disorders
Skin pustules
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Infections and infestations
Viral exanthem
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Infections and infestations
Viral gastroenteritis
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7.7%
1/13 • Number of events 1 • 100 weeks
|
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Skin and subcutaneous tissue disorders
Viral upper respiratory infection
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23.1%
3/13 • Number of events 3 • 100 weeks
|
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Skin and subcutaneous tissue disorders
Skin Infection
|
30.8%
4/13 • Number of events 4 • 100 weeks
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place