Pilot Efficacy and Safety Study of Oral DF2156A in Patients With Active Bullous Pemphigoid
Status:
Terminated
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
The study will be a phase 2, multicentre, single arm, pilot study. It has been designed to
determine if DF2156A has sufficient activity to warrant its further development.
A total of twelve (12) BP patients will be involved, who will be administered DF2156A orally
at the dose of 150 mg twice a day for a maximum of 14 days.
Recruitment will be competitive among the study sites, until the planned number of patients
is enrolled. Competitive recruitment has been chosen to increase the speed of recruitment and
to account for any unexpected occurrence at a site that negatively impact enrolment rate.
The single arm design has been chosen as an appropriate tool for this pilot phase 2 study,
considering that BP is a rare disease where a placebo control is not acceptable. Moreover, as
there is no spontaneous acute recovery from the active blistering condition, any improvement
in patient outcome can be attributed to a positive effect of the Investigational Product.
Each patient will be involved in the study for a screening period, for 14 days of treatment,
for all required measurements up to hospital discharge (planned on day 8+1 of treatment) and
for one assessment occasion on day 15+1, either during hospital stay or after hospital
discharge (out-patient visit). An optional post-treatment visit might be scheduled at day
30+3.
The objective of this clinical trial is to evaluate whether DF2156A has a potential in
improving the clinical outcome in patients with active blistering BP to warrant its further
development. The safety of DF2156A in the specific clinical setting will be also evaluated.