Netherton syndrome (NS)
Conditions
Brief summary
• Primary endpoints: • Safety assessment includes adverse events (nature and incidence), physical examination, vital signs, laboratory safety, and local tolerability. • Primary efficacy endpoint: o The change in Investigator Global Assessment (IGA) score 0-4 (see table in Appendix B) at EOT compared to baseline.
Detailed description
• Secondary efficacy endpoints: • The skin condition will be characterized primarily by Ichthyosis Area Severity Index (IASI), which integrates erythema (IASI-E) and scaling (IASI-S); this score has been recently developed and validated by applying it to patients affected with NS (Paller et al., 2017). • Skin itching will be assessed by VAS and a multidimensional 5-D pruritus scale (Phan et al., 2012). • Skin surface pH and transepidermal water loss (TEWL) will be measured on treated areas.
Interventions
None listed
Sponsors
Sixera Pharma AB
Eligibility
Sex/Gender
All
Age
18 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| • Primary endpoints: • Safety assessment includes adverse events (nature and incidence), physical examination, vital signs, laboratory safety, and local tolerability. • Primary efficacy endpoint: o The change in Investigator Global Assessment (IGA) score 0-4 (see table in Appendix B) at EOT compared to baseline. | — |
Secondary
| Measure | Time frame |
|---|---|
| • Secondary efficacy endpoints: • The skin condition will be characterized primarily by Ichthyosis Area Severity Index (IASI), which integrates erythema (IASI-E) and scaling (IASI-S); this score has been recently developed and validated by applying it to patients affected with NS (Paller et al., 2017). • Skin itching will be assessed by VAS and a multidimensional 5-D pruritus scale (Phan et al., 2012). • Skin surface pH and transepidermal water loss (TEWL) will be measured on treated areas. | — |
Countries
Austria, France, Germany, Sweden
Outcome results
None listed