- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04499235
A Study to Assess the Therapeutic Effect and Safety of Adjunctive AKST4290 in Subjects With Bullous Pemphigoid
Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid
Study Overview
Status
Conditions
Intervention / Treatment
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 Subjects who receive rescue therapy will remain in the study until disease control, unless they are withdrawn or withdraw from participation.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 Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Dresden, Germany, 01307
- Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie
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Düsseldorf, Germany, 40225
- Universitätsklinikum Düsseldorf Klinik für Dermatologie
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Erlangen, Germany, 91054
- Universitätsklinikum Erlangen - Hautklinik
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Freiburg, Germany, 79104
- Universitatsklinikum Freiburg Klinik fur Dermatologie und Venerologie
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Lübeck, Germany, 23538
- Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin
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Magdeburg, Germany, 39120
- Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik
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Mainz, Germany, 55131
- Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC)
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Würzburg, Germany, 97080
- Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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.
- 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.
- Use of systemic immunosuppressants (i.e., mycophenolate, azathioprine, methotrexate) within 4 weeks prior to screening.
- Treatment with rituximab within 1 year prior to screening.
- 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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Double
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Mometasone furoate + AKST4290
Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.
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Oral AKST4290
Topical mometasone furoate
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Placebo Comparator: Mometasone furoate + Placebo
Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.
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Oral placebo
Topical mometasone furoate
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy
Time Frame: Baseline to up to 3 weeks (until disease control)
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Disease control is defined as ≤ 3 new blisters/eczematous lesions/urticarial plaques/day and healing of existing blisters/eczematous lesions/urticarial plaques without requiring rescue therapy.
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Baseline to up to 3 weeks (until disease control)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants With TEAEs, Assessed by Seriousness and Severity
Time Frame: Baseline to 5 weeks
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Treatment-emergent AEs summarized by MedDRA coding terms; separate tabulations produced for incidence, seriousness and severity of AEs
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Baseline to 5 weeks
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Time to Disease Control
Time Frame: Baseline to up to 3 weeks (until disease control)
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Time to disease control by treatment day/week.
The time to disease control is calculated as the date of disease control minus Date of Visit 2 (Baseline (Day 1)) plus 1.
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Baseline to up to 3 weeks (until disease control)
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Time to Rescue Therapy
Time Frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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Time to rescue therapy by treatment day/week.
The time to rescue therapy is calculated as the start date of the first rescue therapy minus Date of Visit 2 (Baseline (Day 1)) plus 1.
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Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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The Bullous Pemphigoid Disease Area Index (BPDAI) Score
Time Frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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Change from baseline in BPDAI score at End of Treatment (EOT).
Subscales for the BPDAI include the skin blister score (range 0-120), skin urticarial score (range 0-120), mucosal activity score (range 0-120), and damage score (range 0-12).
Higher scores indicate greater disease activity or damage.
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Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)
Time Frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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Change from baseline in pruritus as evaluated by the BPDAI-VAS at End of Treatment (EOT).
EOT occurs at disease control (up to 3 weeks) or at Week 3 when the subject is discontinued from treatment due to not reaching disease control.
Scores for the BPDAI-VAS can range from 0 to 30, with higher scores indicating a worse condition.
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Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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Total Cumulative Steroid Exposure
Time Frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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Total cumulative steroid exposure (cortisol equivalent/kg) by treatment group
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Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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Maximum Daily Steroid Dose
Time Frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control.
Study Day 1 is defined as the initiation of study treatment.
1 mg/kg prednisolon(e) = 5 mg/kg cortisone.
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Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Alkahest Medical Monitor, Alkahest, Inc.
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- AKST4290-221
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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