Mild to Moderate Bullous Pemphigoid MedDRA version: 21.1 Level: LLT Classification code 10006567 Term: Bullous pemphigoid System Organ Class: 100000004858
Conditions
Sponsors
Alkahest, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age 60-95 years, inclusive at screening. 2. Clinical diagnosis of mild or moderate BP at screening: o Mild BP is defined as BPDAI = 10 OR =65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: 1. Severe BP (BPDAI score = 56 OR > 30 new blisters per day OR > 30% affected body surface). 2. Initiation of gliptins and other treatments (e.g., etanercept, sulfasalazine, furosemide, penicillin) that can trigger BP if this treatment was started within 4 weeks prior to screening and is considered possibly related to the onset of BP. 3. Initiation of any concomitant medication in the last 3 months prior to screening and assessed by the investigator as possibly related to the development of BP. 4. Planned use of intravenous immunoglobulin or other concomitant treatments for BP (i.e., doxycycline, dapsone) during the study period. 5. Life expectancy of 10 mg prednisone or equivalent/day) within 14 days of first dose of study agent or known diseases (other than BP) that could require the use of systemic steroids within the study period. Subjects who have received a single high-dose of steroids (intravenous or oral) 14 days or more before the administration of the first dose of study drug are eligible for enrollment. 14. Clinically relevant abnormal laboratory value at screening, including hematology, blood chemistry, or urinalysis (laboratory testing may be repeated once during the screening phase). 15. Significant alcohol or drug abuse within past 2 years. 16. Based on ECG reading, subjects with a risk of QT prolongation including: • A baseline prolongation of QTc (using Fridericia’s formula: = 450 ms in men and = 470 ms in women) with confirmation on a repeat ECG. • A history of additional risk factors for Torsades de pointes arrhythmia (e.g., heart failure, hypokalemia, family history of Long QT Syndrome, etc.). 17. The use of concomitant medications known to prolong the QT/QTc interval. 18. Significant medical conditions (as determined by medical history, examination, and clinical investigations at screening) that may, in the opinion of the investigator, result in the any of the following: • Put the subject at risk because of participation in the study. • Influence the results of the study. • Cause concern regarding the subject’s ability to participate in the study. 19. Malignancy for which the subject is currently undergoing resection, radiation, or chemotherapy. 20. Participation in studies of i
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: To assess treatment safety of the proposed dosing regimen. Additional secondary endpoints include assessment of time to disease control; time to rescue therapy; change in BP Disease Area Index (BPDAI) score by treatment week and at disease control; and change in pruritis as assessed by the BPDAI-Visual Analog Scale (BPDAI-VAS) by treatment week and at disease control. In addition, change in skin (biopsy) eosinophil counts and overall steroid use required to achieve disease control will be assessed.;Main Objective: To investigate the proportion of subjects who achieve disease control (defined as = 3 new blisters/day and healing of existing blisters) following topical steroid treatment with adjunctive AKST4290 without receiving rescue therapy.;Primary end point(s): The proportion of subjects who achieve disease control (= 3 new blisters/day and healing of existing blisters) without requiring rescue therapy.;Timepoint(s) of evaluation of this end point: At 1-3 weeks depending on when subjects reach disease control. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Safety as assessed by the incidence, seriousness, and severity of adverse events (AEs). • Time to disease control by treatment Day/Week. • Time to rescue therapy by treatment Day/Week. • Change from baseline in BPDAI score by treatment Week and at disease control. • Change from baseline in pruritus as evaluated by the BPDAI-VAS by treatment week and at disease control. • Change from baseline in skin biopsy eosinophil levels at disease control. • Evaluation of total cumulative steroid exposure at baseline, by treatment Week and at disease control. • Evaluation of maximum daily steroid dose at baseline, by treatment Week and at disease control.;Timepoint(s) of evaluation of this end point: At 1-3 weeks depending on when subjects reach disease control. | — |
Countries
Bulgaria, Germany
Contacts
Public ContactJonas Hannestadt, MD, PhD
Alkahest, Inc.
Outcome results
None listed