Pilot Efficacy and Safety Study of Oral DF2156A in Patients With Active Bullous Pemphigoid
Bullous Pemphigoid
About this trial
This is an interventional treatment trial for Bullous Pemphigoid focused on measuring Autoimmune inflammatory blistering disorder
Eligibility Criteria
Inclusion Criteria:
- Male and female patients aged >50 years.
- Patients with newly diagnosed or relapsing bullous pemphigoid based on clinical diagnosis to be confirmed by direct immunofluorescence and indirect immunofluorescence on salt-spit skin (or BP180 and/or BP230 ELISA). Confirmation by laboratory tests will be obtained ideally before or anyway within one week after enrolment.
For the purpose of this study, clinical relapses are defined as re-appearance of clinical symptoms after the patient had attained remission lasting for more than 3 months without immunosuppressive treatment. In patients with relapsing BP, clinical diagnosis will be confirmed by indirect immunofluorescence or BP180 and/or BP230 ELISA only.
- Patients with mild to moderate active blistering disease (total number of blisters between 1 and 30) whether associated or not with urticarial/eczematous lesions.
- Patients with modified ABSIS score ≤50
Patients free from any systemic treatments that may affect the course of the disease with the following off-period prior to enrolment:
- 3 weeks: steroids, dapsone, tetracyclines, nicotinamide,
- 3 months: azathioprine, mycofenolate mofetil, cyclophosphamide, methotrexate, intravenous immunoglobulins, immunoadsorption, TNF antagonists
- 12 months: rituximab, leflunomide
- Patients free from any topical treatments other than topical antibiotics and antiseptics in the 4 days prior to enrolment.
- Patients able to comply with the protocol procedures for the duration of the study, including scheduled follow-up visits and examinations.
- Patients able to provide informed consent.
Exclusion Criteria:
- Patients with a Karnofsky rating score <40%.
- Patients with mucosal involvement.
- Patients with moderate to severe renal impairment as per calculated creatinine clearance (CLcr) < 50 mL/min according to the Cockcroft-Gault formula (Cockcroft-Gault , 1976).
- Patients with hepatic dysfunction defined by increased ALT/AST > 3 x upper limit of normal (ULN) and increased total bilirubin > 3 mg/dL [>51.3 μmol/L].
- Patients with hypoalbuminemia defined as serum albumin < 3 g/dL.
- Patients with a baseline (day 0/1, pre-dose) QTcF > 470 msec.
- Patients who had a myocardial infarction in the 6 months prior to enrolment.
- Patients on treatment with phenytoin, warfarin, sulphonylurea hypoglycemics (e.g. tolbutamide, glipizide, glibenclamide/glyburide, glimepiride, nateglinide) and high dose of amitriptyline (> 50 mg/day).
- Patients with known hypersensitivity to non-steroidal antiinflammatory drugs.
- Patients using any investigational agent within 12 months prior to enrolment.
- Pregnant or breast feeding women. Unwillingness to use effective contraceptive measures up to 2 months after the end of study drug administration (females and males).
Additional Exclusion Criteria for Germany only:
- Patients with hypokalemia defined as serum potassium < 3.5 mmol/L.
- Patients with clinically relevant bradycardia (heart rate < 50 beats/min)
- Patients with a complete left bundle branch block.
- Patients with a history of uncontrolled or labile hypertension
- Patients with a history of congestive heart failure.
- Patients with a history of cardiomyopathy.
- Patients with unstable angina pectoris.
- Patients with a personal or family history of congenital or documented acquired QT interval prolongation.
- Patients with a significant atrial or ventricular arrhythmia or symptomatic arrhythmia in the past.
Sites / Locations
- Department of Dermatology - Universitäts-Hautklinik; Hauptstraße 7
- Klinik für Dermatologie, Allergologie und Venerologie - Universitätsklinikum Schleswig-Holstein, Campus Lübeck; Ratzeburger Allee 160
- Klinik für Dermatologie und Allergologie - Philips Universität; 35037
- I Divisione di Dermatologia, Istituto Dermopatico dell'Immacolata, IRCCS;
Arms of the Study
Arm 1
Experimental
DF2156A 150 mg
150 mg capsule twice a day (every 12 h) for a maximum of 14 days