Pilot phase 2 clinical trial performed in some hospitals to evaluate the efficacy and the adverse events in a group of patients with active bullous pemphigoid that will receive DF2156A at the dose of 150 mg, oral route, twice a day.

Conditions: active bullous pemphigoid
MedDRA version: 14.0Level: LLTClassification code 10006567Term: Bullous pemphigoidSystem Organ Class: 10040785 - Skin and subcutaneous tissue disorders
Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Registration Number: EUCTR2011-000756-42-DE

Lead Sponsor: Dompé s.p.a.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 12

Inclusion Criteria

To be eligible for inclusion into this study, each patient must fulfil the following 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

- Patients with a Karnofsky rating score <40%.
- Patients with mucosal involvementPatients 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, sulphanylurea 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).
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 cardiomiopathy.
- 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.