The aim of this study is to evaluate the effect of Gladskin treatment on the occurrence of skin symptoms in patients with NS. Secondary we want to retrieve information about the effect of Gladskin on quality of life, safety, the load of S. aureus…
ID
Source
Brief title
Condition
- Bacterial infectious disorders
- Skin and subcutaneous tissue disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
1. Patient reported disease severity score using the Patient Global Assessment
(PGA)
Secondary outcome
1. Patient reported disease severity scores
- Pruritus Numerical Rating Scale (Pruritus NRS)
- Pain Numerical Rating Scale (Pain NRS)
- Desquamation Numerical Rating Scale (Desquamation NRS)
- Erythema Numerical Rating Scale (Erythema NRS)
- Sleep Numerical Rating Scale (Sleep NRS)
2. Doctor reported disease severity scores:
- Skin infection defined by the use of topical *or systemic antibiotics
- Visual index for Ichthyosis severity (VIIS)
4. S. aureus load and other microorganisms on skin and nose mucosa measured by
DNA based methods and culture.
5. Quality of life using Skindex-29 questionnaires
6. Safety and tolerability of Gladskin:
- Adverse device effect, therapy cessation for adverse device effects
- Serious Adverse Device Effect (SADE)
- Blood tests including infection parameters (leukocytes, total and
differentiation), renal function (creatinine, eGFR), liver function tests
(ASAT, ALAT, bilirubin).
Background summary
Colonization of the skin and mucosa with Staphylococcus (S.) aureus has been
suggested to be associated with the multifactorial pathogenesis of NS. It might
influence the skin symptoms of NS and causes recurrent skin infections in these
patients. Increasing multidrug resistance of S. aureus points out the need for
development of alternative treatment options next to antibiotics that are often
used to treat skin symptoms and infections in NS patients. Gladskin is a
product for topical use, the proprietary enzyme in the Gladskin products is
called Staphefekt SA.100 (Staphefekt). Staphefekt is an endolysin that
specifically lyses the cell membrane of S. aureus. In vitro results showed that
Staphefekt kills S. aureus, leaving the commensal flora intact. Treatment with
Gladskin might decrease S. aureus colonization of the skin and consequently
decrease symptoms and severity of S. aureus related disease, such as NS.
Study objective
The aim of this study is to evaluate the effect of Gladskin treatment on the
occurrence of skin symptoms in patients with NS. Secondary we want to retrieve
information about the effect of Gladskin on quality of life, safety, the load
of S. aureus and the further microbiome.
Study design
A descriptive case series evaluating the effects of Gladskin usage in NS
patients.
Intervention
- Run in phase to monitor current treatment using medication diary (week 0-2)
- Apply Gladskin on the skin twice daily (intervention phase: week 3-10)
- Follow up phase to monitor treatment and severity scoring using a patient
diary (week 11-12)
- 3 visits of 30-60 minutes. Visit 1 is ahome visit (if patients prefer). Visit
2 and 3 are at the research department of the Erasmus MC
- Visit 1: questionnaire, medical check-up, skinswab, skinscrub, nose swab,
photograph
- Visit 2 and 3: questionnaire, medical check-up, skinswab, skinscrub, nose
swab, photograph, blood sampling
- Weekly short questionnaire in patient diary: medication usage, patient
global assessment, sleep, pruritus, pain, erythema and desquamation NRS
Study burden and risks
The risk of using Gladskin is low. Since endolysins are proteins, they are
inherently non-toxic. Theoretically toxicity can occur when in the purification
process of the lysins a toxic substance of the host bacteria is co-purified.
Staphefekt is tested on purity (host cell contamination and endotoxin levels)
according to European regulations. Staphefekt is a large size protein molecule
(>50kDa) and will not penetrate or pass intact skin. Therefore an immunogenetic
reaction with the formation of antibodies against Staphefect is highly inlekely
when applied on the intact skin. Literature shows that possible formed
antibodies are non-neutralizing and in animal testing no adverse effects of
other endolysins are seen. There is a possibility of an allergic reaction to
other compnents of the product or the sampling solution (Tween 80). Because of
the skin barrier impairment in NS patients, penetration of the Staphefekt
protein in the skin can not be excluded. With this in mind extra safety
measures will be taken:
- Patients can contact us by phone 24h a day if any problems occur
- A progressive lubrication application schedule
- Blood samples at visit 3 and 4 measuring leukocytes, creatinine, eGFR, ASAT
and ALAT.
Dr.Molewaterplein 40
Rotterdam 3015 GD
NL
Dr.Molewaterplein 40
Rotterdam 3015 GD
NL
Listed location countries
Age
Inclusion criteria
- 18 years of age or above
- Having a diagnosis of Netherton syndrome
- Able to read patient information, fill out online questionnaires and provide informed consent
Exclusion criteria
- Systemic antibiotics within the previous 4 weeks
- Topical antibiotics within the previous 7 days
- Gladskin within the previous 7 days
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL63546.078.18 |