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.

• Register: EUCTR
• Last refreshed on: 15 July 2013
• Main ID: EUCTR2011-000756-42-DE
• Date of registration: 08/06/2011
• Prospective Registration: Yes
• Primary sponsor: Dompé s.p.a.
• Public title: 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.
• Scientific title: A phase 2, multicentre, single arm, pilot study to assess the efficacy and the safety of 150 mg twice a day oral DF2156A in patients with active bullous pemphigoid. - DF2156A in patients with active bullous pemphigoid
• Target sample size: 12
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-000756-42
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Germany, Italy

Contacts

• Name: Development Project Management
• Affiliation: Dompé s.p.a.

• Name: Development Project Management
• Affiliation: Dompé s.p.a.

Key inclusion & exclusion criteria

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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

active bullous pemphigoid
MedDRA version: 14.0 Level: LLT Classification code 10006567 Term: Bullous pemphigoid System Organ Class: 10040785 - Skin and subcutaneous tissue disorders

Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Intervention(s)

Product Name: not applicable
Product Code: DF2156A
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: ladarixin
CAS Number: 865625-56-5
Current Sponsor code: DF2156A
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 75-

Primary Outcome(s)

Main Objective: 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.

Timepoint(s) of evaluation of this end point: time frame: day 0/1 (predose), 8 and 15; time frame: day 0/1 (predose), 8 and 15; time frame: day 0/1 (predose), 8 and 15; time frame: screenitime frame: day 8ng and day 15; time frame: day 8; time frame: day 30

Primary end point(s): Total number of blisters and percent change from baseline; Modified ABSIS score and percent change from baseline; Physician Global Assessment (PGA) score measured on a 0-10 scale; Absolute value and % change from baseline; Pruritus measured on a 10 cm visual analogue scale. Absolute value and percent change from baseline; Eosinophil blood count. Absolute number and percent change from baseline; Number and percentage of patients with treatment failure (drug discontinuation due to disease worsening); Number and percentage of patients completely free from blisters; Number of patients who are still free from blisters without requiring any systemic or topical rescue treatment - Optional

Secondary Objective: Not applicable

Secondary Outcome(s)

Secondary end point(s): Not applicable

Timepoint(s) of evaluation of this end point: Not applicable

Secondary ID(s)

MEX0111

Source(s) of Monetary Support

Dompé s.p.a.