A Study to Assess the Therapeutic Effect and Safety of Adjunctive AKST4290 in Subjects With Bullous Pemphigoid
Study Details
Study Description
Brief Summary
This study will evaluate the therapeutic effect and safety of adjunctive AKST4290 in subjects with bullous pemphigoid (BP).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
This is a randomized, double-blind, placebo-controlled study to assess the therapeutic effect and safety of adjunctive AKST4290 in subjects with bullous pemphigoid (BP). Subjects will receive topical mometasone furoate cream (MFC) therapy concurrently with study agent (placebo or AKST4290) in an inpatient setting until disease control is reached (duration of inpatient stay is dependent upon individual disease course, but is estimated between 1-3 weeks).
Subjects will receive rescue therapy at any time if their clinical condition worsens or if their clinical condition fails to improve by the completion of Week 1 on study treatment, as assessed by the investigator. Rescue therapy will consist of whole-body clobetasol propionate cream (CPC) (15-50g) and/or oral prednisone (0.5 mg/kg per day), as determined by the investigator. Subjects who receive rescue therapy will remain in the study until disease control, unless they are withdrawn or withdraw from participation.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Mometasone furoate + AKST4290 Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached. |
Drug: Mometasone furoate
Topical mometasone furoate
Drug: AKST4290
Oral AKST4290
|
| Placebo Comparator: Mometasone furoate + Placebo Subjects will receive mometasone furoate concurrently with placebo until disease control is reached. |
Drug: Mometasone furoate
Topical mometasone furoate
Drug: Placebo
Oral placebo
|
Outcome Measures
Primary Outcome Measures
- The proportion of subjects who achieve disease control [Baseline to up to 3 weeks (until disease control)]
The proportion of subjects who achieve disease control (≤ 3 new lesions/day and healing of existing lesions) without requiring rescue therapy.
Secondary Outcome Measures
- Safety as assessed by the incidence, seriousness and severity of AEs [Baseline to 5 weeks]
Treatment emergent AEs summarized by MedDRA coding terms; separate tabulations will be produced for incidence, seriousness and severity of AEs.
- Time to disease control [Baseline to 3 weeks]
Time to disease control will be compared between treatment groups by treatment day and week using a Kaplan-Meier curve.
- Time to rescue therapy [Baseline to 3 weeks]
Time to rescue therapy will be compared between treatment groups by treatment day and week using a Kaplan-Meier curve.
- The Bullous Pemphigoid Disease Area Index (BPDAI) score [Baseline to 3 weeks]
Change from baseline in BPDAI score by treatment week and at disease control. Scores can range from 0 to 360 for BPDAI total activity and 0 to 12 for BPDAI damage, with higher scores indicating greater disease activity or damage.
- The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS) [Baseline to 3 weeks]
Change from baseline in pruritus as evaluated by the BPDAI-VAS by treatment week and at disease control. Scores can range from 0 to 30, with higher scores indicating a worse condition.
- Total cumulative steroid exposure [Baseline to 3 weeks]
Evaluation of total cumulative steroid exposure at baseline, by treatment week, and at disease control.
- Maximum daily steroid dose [Baseline to 3 weeks]
Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Clinical diagnosis of mild to moderate BP at screening.
-
Treatment naïve or initiation of whole-body high potency topical steroid treatment ≤ 7 days of screening (lesion-only treatment for any amount of time with any topical steroids prior to screening is allowed without restriction).
-
Provide a signed and dated informed consent form in accordance with local regulations and/or IRB/IEC guidelines.
Exclusion Criteria:
-
Severe BP.
-
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.
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Any concomitant medications in the last 3 months prior to screening and assessed by the investigator as possibly related to the development of BP.
-
Planned use of intravenous immunoglobulin or other concomitant treatments for BP (i.e., doxycycline, dapsone) during the study period.
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Use of systemic immunosuppressants (i.e., mycophenolate, azathioprine, methotrexate) within 4 weeks prior to screening.
-
Treatment with rituximab within 1 year prior to screening.
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Subjects taking warfarin.
-
Use of systemic steroids (>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.
-
Clinically relevant abnormal laboratory value at screening, including hematology, blood chemistry, or urinalysis (laboratory testing may be repeated once during the screening phase).
-
Participation in studies of investigational drugs must have been discontinued within 30 days or 5 half lives of the drug (whichever was longer) prior to screening.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie | Dresden | Germany | 01307 | |
| 2 | Universitätsklinikum Düsseldorf Klinik für Dermatologie | Düsseldorf | Germany | 40225 | |
| 3 | Universitätsklinikum Erlangen - Hautklinik | Erlangen | Germany | 91054 | |
| 4 | Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie | Freiburg | Germany | 79104 | |
| 5 | Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin | Lübeck | Germany | 23538 | |
| 6 | Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik | Magdeburg | Germany | 39120 | |
| 7 | Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC) | Mainz | Germany | 55131 | |
| 8 | Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie | Würzburg | Germany | 97080 |
Sponsors and Collaborators
- Alkahest, Inc.
Investigators
- Study Director: Alkahest Medical Monitor, Alkahest, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- AKST4290-221