- Assess whether early markers (in blood and/or skin) can predict the clinical response to treatment (i.e. surface area of repigmented skin after 3 and 6 months of standard of care treatment).- Assess the relation between markers in blood, skin…
ID
Source
Brief title
Condition
- Pigmentation disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
1. Clinical non-invasive parameters
Surface area of depigmented skin will be assessed at Baseline, and after 3
months and 6 months of treatment from a target lesion (copy sheet method) and
from the total body (VES score and VASI score) to determine the extent of the
disease.
2. Laboratory parameters
-Biomarkers will be assessed from blood, suction blister fluid and punch
biopsies at Baseline and after 3 months of treatment.
3. Imaging
-Confocal reflectance microscopy imaging will be used for non-invasive imaging
of melanocytes and inflammatory cells in vitiligo.
-Digital imaging of the target lesion.
4. Patient Reported Outcomes (PROs)
-The DLQI questionnaire will be used to collect Quality of Life data at
Baseline, 3 months and 6 months after treatment.
-The PHQ-9 will be used to collect data on the impact of vitiligo on the
presence and severity of depression.
Secondary outcome
-
Background summary
Non-segmental vitiligo (NSV) is a chronic autoimmune disorder leading to
destruction of melanocytes. The depigmentation in vitiligo is caused by
autoimmune reactions against melanocytes, including specific T cell and
antibody responses. Several factors including biochemical, neuronal and
environmental may contribute to the development of the autoimmunity in vitiligo.
Vitiligo is sometimes considered to be merely a cosmetic problem. However, its
psychosocial impact and social stigmatization in various societies must not be
neglected. Multiple studies show a significant negative effect of vitiligo on
the quality of life. The importance of treatment is often underestimated due to
the lack of recognition of the impact of the disease for patients.
So far, the treatment of vitiligo still remains a challenge. There is no
curative treatment available for NSV. Current treatments include topical
agents, phototherapy and surgical techniques which aim to improve melanocyte
proliferation and stimulate repigmentation. However, these interventions are
not satisfactory in all patients as complete repigmentation is usually not
achieved.
Due to the unsatisfactory and limited treatment options there is a crucial need
to identify biomarkers of disease activity to support clinical trials for
vitiligo. Different biomarkers have been reported to be linked to the disease
activity and autoimmune process in NSV. However, comprehensive studies
comparing these biomarkers in different tissues are not available. Moreover,
the relation between these biomarkers and repigmentation resulting from
different treatments is unclear. We hypothesize that some biomarkers may
predict the response to treatment.
Our research depends on the availability of bodily material (blood, skin and
suction blister fluid) of vitiligo patients. Furthermore, we will assess the
quality of life and psychosocial changes during treatment using
patient-reported outcome questionnaires.
Study objective
- Assess whether early markers (in blood and/or skin) can predict the clinical
response to treatment (i.e. surface area of repigmented skin after 3 and 6
months of standard of care treatment).
- Assess the relation between markers in blood, skin biopsy and suction blister
fluid.
- Investigate the use of non-invasive confocal reflectance microscopy imaging
as well as imaging based on digital photography to assess early clinical
response.
- Assess psychosocial changes during treatment using patient-reported outcomes
by assessing the impact of vitiligo on quality of life factors (using the
Dermatology Quality of Life Questionnaire [DLQI]) and depression (using the
Patient Health Questionnaire * 9 [PHQ-9]).
Study design
Prospective cohort study in patients with non-segmental vitiligo who receive
standard of care treatment.
Study burden and risks
The standard of care treatment will not be changed or influenced by
participation in the study. Standard of care treatment will be prescribed to
the patients prior to enrolment into the study, based on the prescribing
physician*s medical assessment.
The time investment for the patient will be approximately 120 minutes for each
of the first two visits. The last visit could take place on the same day of the
routine control in patients receiving treatment for their vitiligo and will be
approximately 30 minutes.
Risks for the patients due to sampling procedures are minimal. Blood sampling
will occur at Baseline and at 3 months of treatment, at which a maximum volume
of 50 ml of blood will be collected at each visit. Vena puncture to draw blood
can result in a hematoma at the site of puncture.
Suction blisters are extremely superficial (150 µL µm) and usually heal without
a scar. However, pigmentation changes may occur that generally resolve within
12 months. Fluid from suction blisters will be collected at Baseline (2 suction
blisters) and at 3 months (1 suction blister).
Skin punch biopsies are routine diagnostic procedures in dermatology and leave
mild textural changes, which can be minimized when a suture is applied after
harvesting. The risk of infections due to these procedures is minimal (< 1 %).
Punch biopsies (4 mm diameter) will be collected at Baseline and at 3 months.
There is no benefit expected for patients participating in this study. This
study may help in identifying early markers for treatment response and could
therefore be important to identify non-responders at an early stage. Moreover,
this study will contribute to the knowledge on the pathogenesis of vitiligo. *
Meibergdreef 9
Amsterdam 1100 DD
NL
Meibergdreef 9
Amsterdam 1100 DD
NL
Listed location countries
Age
Inclusion criteria
- Patients with non-segmental vitiligo commencing standard of care treatment,
consisting of a potent topical corticosteroid, such as FP cream, alone or in
combination with NB-UVB phototherapy for at least 6 months.
- Patients with new lesions and/or signs of disease progression (active
disease) within the past 6 months prior to Screening.
- Males and females aged *18
- Patients able to communicate well with the investigator, to understand the
requirements of the study, as well as understand and sign the written informed
consent.
Exclusion criteria
- Patients that have received phototherapy or systemic immunosuppressive
treatment during the last 6 weeks prior to Screening.
- Patients that have received topical anti-inflammatory treatment (topical
corticosteroids, calcineurin inhibitors) during the last 4 weeks prior to
Screening.
- Patients currently receiving treatments other than potent topical
corticosteroids, such as FP 0.05% cream, calcineurin inhibitors (tacrolimus,
pimecrolimus) or NB-UVB phototherapy for non-segmental vitiligo.
- Recurrent HSV skin infections.
- Patients with a history of hypertrophic scars or keloid.
- Patients with a history of hypersensitivity to (UVB or UVA) light and/or
allergy to local anaesthesia.
- Pregnant or nursing (lactating) women, where pregnancy is defined as the
state of a female after conception and until the termination of gestation,
confirmed by a positive hCG laboratory test (unless female is confirmed
post-menopausal).
- Patients with haemophilia or other clotting disorders
- Patients previously diagnosed with depression according to the DSM5 by a
qualifying physician.
- Patients without lesions located at suitable areas for biopsies / suction
blisters sampling (e.g. the face)
Design
Recruitment
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 | NL66309.018.18 |