Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT06218082 |
| Other study ID # |
2022DER127 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
May 15, 2025 |
| Est. completion date |
March 2029 |
Study information
| Verified date |
June 2024 |
| Source |
The Royal Wolverhampton Hospitals NHS Trust |
| Contact |
Viktoria Eleftheriadou |
| Phone |
01902307999 |
| Email |
viktoria.eleftheriadou[@]nhs.net |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational [Patient Registry]
|
Clinical Trial Summary
Vitiligo is the most common depigmentation disorder affecting around 1% of the population
worldwide. Fifty two percent of patients develop vitiligo before the age of 20 and around 80%
develop vitiligo before the age of 30 years old.1 Vitiligo often presents in childhood and
tends to be a lifelong disease, requiring prolonged courses of phototherapy.
Currently no national or international registry for patients with vitiligo exists. Individual
dermatologists maintain a database of such patients, however no coordinated efforts have been
made to combine these individual registries into a broader national registry. Finally,
recently published British Association of Dermatologists (BAD) guideline for the management
of vitiligo, recommended the development of a national registry for people with vitiligo
undergoing systemic or light therapy to identify outcomes and safety.
Description:
Vitiligo is the most common depigmentation disorder affecting around 1% of the population
worldwide. Fifty two percent of patients develop vitiligo before the age of 20 and around 80%
develop vitiligo before the age of 30 years old. Vitiligo often presents in childhood and
tends to be a lifelong disease, requiring prolonged courses of phototherapy.
Vitiligo is an autoimmune skin disorder and is often associated with multiple other
autoimmune disorders such as thyroid disorders, diabetes, IBS, pernicious anemia, alopecia
areata amongst others. Current treatments include topical corticosteroids and calcineurin
inhibitors, phototherapy, combination of phototherapy with oral corticosteroids, laser and
surgical interventions. Other treatment modalities such as Methotrexate have also been
trialed. Currently available treatments are being used off license with uncertain therapeutic
success.
Previously, it has been shown that vitiligo has an inverse relationship with melanoma, which
means that people with vitiligo are less likely to develop melanoma. A recent systematic
review and meta-analysis looking into the risk of skin cancer in people with vitiligo showed
that compared with people without vitiligo, people with vitiligo had a significantly lower
risk of nonmelanoma skin cancer. The same pattern occurred for melanoma (but was not
statistically significant). However, this review was limited by the small number of included
studies and high heterogeneity due to methodological and clinical differences between the
included studies. In addition, there is lack of data on the skin cancer risk for vitiligo
patients who receive prolonged phototherapy courses, especially regarding high cumulative
exposures in children with less deeply pigmented skin. This is especially important when
NB-UVB is combined with topical tacrolimus treatment, due to theoretical increased risk of
skin cancer.
In addition, there is an urgent need for new, effective and licensed treatment for vitiligo.
In the dawn on new therapeutic era of new emerging treatments such as topical and oral JAK
inhibitors, evidence will be needed on the effectiveness, safety and cost effectiveness of
such therapies compared to other treatments. In order to gather evidence on clinical
effectiveness of vitiligo treatments and to assist with updating of clinical guidance
pathway, as well as to gather information on possible risk factors, adverse events,
maintenance regime and assist with conduction of post marketing studies, prospective
multicentre registry is needed for vitiligo patients who are treated with phototherapy,
combination treatments (with phototherapy), oral agents (such as oral corticosteroids)
including new emerging JAK inhibitors (once there are available in the UK).
Oral corticosteroids combined with phototherapy are recommended by the recently published BAD
guidelines for cases of rapidly progressive vitiligo. Case reports were published on
initiation of methotrexate and oral JAK inhibitors for the treatment of vitiligo.
In addition, there is no firm data on disease progression and especially predictors of
rapidly progressive vitiligo are largely unknown, which is a challenging scenario for the
patient and their clinician.
Currently no national or international registry for patients with vitiligo exists. Individual
dermatologists maintain a database of such patients, however no coordinated efforts have been
made to combine these individual registries into a broader national registry. Finally,
recently published British Association of Dermatologists (BAD) guideline for the management
of vitiligo, recommended the development of a national registry for people with vitiligo
undergoing systemic or light therapy to identify outcomes and safety.2
As an initial, preliminary step for the development of the online UK registry for vitiligo
patients, development of a pilot multi-centred registry in the UK is needed. In addition,
such registry will be co-ordinated with a pilot multi-centred registry in France and the USA.
Each registry will be developed separately (country-specific) and there will be no data
sharing between the registries, however there will be co-ordination between the CIs of the
registries with regards to the outcomes collected.
Another challenge in vitiligo has been consistency in the use of core outcome domains and
measures instruments to define treatment success or failure. The international Initiative for
Outcomes for vitiligo (INFO) has previously addressed this heterogeneity and defined 3
outcome domains as being essential: repigmentation, side effects/harms and maintenance of
gained repigmentation. Four items were further recommended for inclusion but were not
considered to be essential: cosmetic acceptability of the results, quality of life, cessation
of spreading of vitiligo and tolerability or burden of treatment.5 INFO has subsequently
published recommendations on how to measure repigmentation in target lesion. Percentage of
repigmentation in quartiles (0-25%, 26-50%, 51-79%, 80-100%) should be measured as well as
cosmetic acceptability of results should be assessed using the Vitiligo Noticeability Scale.6
There are also several other established instruments exist (such as VASI, VIPs, VITs) to
measure these domains and these are suitable for use in a registry.
A long-term safety data will be vital to provide pharmacovigilance, particularly regarding
rare or delayed adverse effects of treatment. Hence a prospective multicentre registry study
is greatly needed to evaluate the real-world use of phototherapy (including combination
treatments with phototherapy), topical JAK inhibitors, systemic immuno-modulatory therapies
in adults and children undergoing treatment for vitiligo to provide effectiveness and safety
data beyond the confines of short-term randomised controlled trials (RCTs). Such a registry
will ultimately inform treatment guidelines, provide pharmacogenetic and pharmacodynamic
research data, and help to identify those patients likely to experience rapid disease
progression, ultimately improving and personalising patient care. With novel topical and oral
immunomodulatory therapies having just entering vitiligo clinical practice, the timing is
ideal to harness the high-quality data collection offered by the UK NHS.
