Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT02837965 |
| Other study ID # |
PR12042* |
| Secondary ID |
|
| Status |
Completed |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
September 26, 2013 |
| Est. completion date |
December 31, 2018 |
Study information
| Verified date |
February 2024 |
| Source |
CHU de Reims |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
The study is an observational, multi-center, prospective, non-interventional and open-label
data collection study assessing outcomes, treatment patterns, adverse events and costs in
patients diagnosed with bullous pemphigoid. The patient enrollment period will be 1 year with
a follow-up (observation period) of 1 year for each patient. Four dermatology centres in
France will participate. The hypothesis to be answered by the study is that superpotent
topical corticosteroid therapy is properly used to treat bullous pemphigoid in real-world
life as recommended by French guidelines and whether this treatment influences the medical
costs by comparison with systemic therapies (e.g. methotrexate or prednisone).
Description:
Bullous pemphigoid is the most common autoimmune blistering disease of the skin, usually
affecting the elderly. It is a chronic disease characterized by spontaneous exacerbations and
remissions. Bullous pemphigoid is mediated by IgG autoantibodies directed against
hemidesmosomal proteins (BP180, BP230), which are involved in dermal-epidermal adhesion in
the skin. The diagnosis of bullous pemphigoid is based on clinical and immunopathological
findings, including skin direct immunofluorescence. The disease usually requires on average a
1-year duration of treatment although a long-term maintenance therapy may be necessary some
cases. High doses oral corticosteroids have been considered as the mainstay of treatment for
many years, but are deleterious, with a high rate of treatment side effects, including
mortality. Topical superpotent corticosteroids have been demonstrated to be as effective but
safer than high doses of oral corticosteroids, reducing side effects and mortality rate,
while controlling the disease in between 95% to 100% of cases. Despite its high efficacy,
topical superpotent corticosteroid therapy is often considered as poorly convenient. In
addition, immunosuppressant drugs and methotrexate are poorly tolerated or frequently
contra-indicated in those elderly patients with bullous pemphigoid. To date, no study has
evaluated the real-world use and costs of the different treatments in this disease.
Purpose The project is an observational, multi-center, prospective, non-interventional and
open-label data collection study assessing outcomes, adverse events, treatment patterns and
related costs in patients diagnosed with bullous pemphigoid. The patient enrollment period
will be 1 year with a follow-up (observation period) of 1 year for each patient. Four
dermatology centres in France will participate.
Primary objective:
The specific goal is to describe in each patient with bullous pemphigoid the therapeutic
management, including: i) all topical and systemic medications and nursing cares; ii)
clinical and biological monitoring and iii) clinical outcome during the first year of
treatment. The hypothesis to be answered by the study is that topical superpotent
corticosteroid therapy is properly used to treat bullous pemphigoid in real-world life as
recommended by French guidelines and whether this treatment influences the medical costs by
comparison with systemic therapies (e.g. methotrexate or prednisone).
Secondary objectives:
- To determine the factors influencing the first-line therapy : topical superpotent
corticosteroid therapy (treatment scheme 1 or TS1) versus systemic therapy (treatment
scheme 2 or TS2).
- To determine the factors influencing disease outcome during the first year of treatment,
especially TS1 versus TS2.
- To determine total, medical and non-medical costs, during the first year of treatment
and to compare these costs between patients treated according TS1 versus TS2.
Exploratory objectives
- To sequentially evaluate several blood biomarkers, including anti-BP180 and anti-BP230 IgG
autoantibodies, IL-17, IL-23, IL-12 or other biologic makers and to compare them according
the treatment scheme (TS1 versus TS2).
Included patients with bullous pemphigoid will be followed for 1 year. Baseline and 6
follow-up visits are planned to record disease activity, treatment and laboratory monitoring
modalities and to collect blood samples for ancillary studies. Clinical data recorded at
baseline are gender, age and associated medical conditions (neurological disorders: dementia,
stroke, Parkinson's disease, multiple sclerosis; malignancy; other diseases). Baseline
clinical evaluation includes the number of daily new blisters for 3 consecutive days,
localization of skin or mucous membrane blisters and erosions and BPDAI (bullous pemphigoid
disease area index). Extensive bullous pemphigoid is defined as the occurrence of at least 10
new daily blisters. The first-line treatment used will be recorded (superpotent topical CS,
i.e. clobetasol propionate cream, 20-30g per day; methotrexate; other systemic therapy). At
each visit, the presence of pruritus, erythematous or eczematous or urticarial plaques, and
the number of new daily blisters are recorded and a BPDAI (bullous pemphigoid disease area
index) is calculated. Topical and systemic treatments for bullous pemphigoid are recorded at
each visit, as the occurrence and date of relapse, which eventually occurred. Relapse is
defined as the reappearance of ≥3 new daily blisters along with pruritus and/or erythematous,
eczematous or urticarial plaques.
