Short-term safety, efficacy and mode of action of apremilast in mild to moderate cutaneous pemphigoid: a phase IIa open label single arm study.

Phase 2

Completed

Conditions: parapemphigus
10014982

Registration Number: NL-OMON49116

Lead Sponsor: niversitair Medisch Centrum Groningen

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 10

Inclusion Criteria

adult (>= 18 years of age) male or female patients with recently diagnosed mild
to moderate localized or generalized cutaneous pemphigoid, or patients that
were in complete remission without treatment that have a mild to moderate
flare-up of the disease.

Exclusion Criteria

Women of childbearing potential without contraception; women who are pregnant
or planning to become pregnant or who are lactating; patients that use systemic
immunosuppressive medication provided the treatment cannot be stopped before
Visit 2; any condition which would make the patient unsuitable for treatment,
or requires steroid use. Patients with PHQ-9 (Patients Health Questionnaire-9)
score >= 10. Contradiction or known allergy for PDE4 inhibitors;

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Partial remission will be analysed in week sixteen (t=16). Partial remission is<br /><br>defined as presence of transient new lesions that heal within one week OR >=30%<br /><br>decrease of VAS. At week six (t= 6), patients that showed no disease control on<br /><br>apremilast combined with doxycycline will be excluded from the pilot study.<br /><br>Disease control is defined as the time that new lesions cease to form and<br /><br>established lesions begin to heal OR pruritic symptoms start to abate (minimal<br /><br>1-point decrease of VAS).</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>• Achievement of complete remission at week sixteen. Complete remission is<br /><br>defined as absence of new or established lesions or pruritus.30<br /><br>• Proportion of patient with drug survival at t=16.<br /><br>• Proportion of patients with disease control at t=6.<br /><br>• Mean decrease in periphery blood eosinophil count.<br /><br>• Mean decrease in BP180 Nc16a titers by ELISA.<br /><br>• Change in genexpression by RNA sequencing measured at t=0 and t=16.<br /><br><br /><br>The following outcomes will be used for measuring clinical efficacy:<br /><br>• mean reduction in Visual Analogue Scale (VAS)<br /><br>• mean decrease in Dermatology Quality of Life index (DLQI), autoimmune<br /><br>blistering disease quality of life (AIBDQOL) and treatment autoimmune bullous<br /><br>diseases quality of life (TABQOL);<br /><br>• mean reduction of Bullous Pemphigoid Disease Area Index (BPDAI). </p><br>