A Study to Assess the Therapeutic Effect and Safety of Adjunctive AKST4290 in Subjects With Bullous Pemphigoid
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|ClinicalTrials.gov Identifier: NCT04499235|
Recruitment Status : Completed
First Posted : August 5, 2020
Last Update Posted : July 8, 2021
|Condition or disease||Intervention/treatment||Phase|
|Pemphigoid, Bullous||Drug: Mometasone furoate Drug: AKST4290 Drug: Placebo||Phase 2|
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 Subjects who receive rescue therapy will remain in the study until disease control, unless they are withdrawn or withdraw from participation.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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||30 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid|
|Actual Study Start Date :||January 30, 2020|
|Actual Primary Completion Date :||March 29, 2021|
|Actual Study Completion Date :||April 14, 2021|
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
Placebo Comparator: Mometasone furoate + Placebo
Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.
Drug: Mometasone furoate
Topical mometasone furoate
- The proportion of subjects who achieve disease control [ Time Frame: 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.
- Safety as assessed by the incidence, seriousness and severity of AEs [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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) [ Time Frame: 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 [ Time Frame: Baseline to 3 weeks ]Evaluation of total cumulative steroid exposure at baseline, by treatment week, and at disease control.
- Maximum daily steroid dose [ Time Frame: Baseline to 3 weeks ]Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04499235
|Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie|
|Dresden, Germany, 01307|
|Universitätsklinikum Düsseldorf Klinik für Dermatologie|
|Düsseldorf, Germany, 40225|
|Universitätsklinikum Erlangen - Hautklinik|
|Erlangen, Germany, 91054|
|Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie|
|Freiburg, Germany, 79104|
|Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin|
|Lübeck, Germany, 23538|
|Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik|
|Magdeburg, Germany, 39120|
|Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC)|
|Mainz, Germany, 55131|
|Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie|
|Würzburg, Germany, 97080|
|Study Director:||Alkahest Medical Monitor||Alkahest, Inc.|