Clinical trial NCT04499235: A Study to Assess the Therapeutic Effect and Safety of Adjunctive AKST4290 in Subjects With Bullous Pemphigoid
Official title: Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Pemphigoid, Bullous
Keywords: Autoimmune disease
Skin disease
Vesiculobullous
Lead sponsor: Alkahest, Inc.
Sponsor class: INDUSTRY
First posted: 2020-08-05
Enrollment: 6
Sex: ALL
Minimum age (years): 60
Maximum age (years): 95
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will evaluate the therapeutic effect and safety of adjunctive AKST4290 in subjects with bullous pemphigoid (BP).

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 Subjects who receive rescue therapy will remain in the study until disease control, unless they are withdrawn or withdraw from participation.

Eligibility 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.
* 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.

Primary outcomes:
measure: The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy
timeFrame: Baseline to up to 3 weeks (until disease control)
Secondary outcomes:
measure: Number of Participants With TEAEs, Assessed by Seriousness and Severity
timeFrame: Baseline to 5 weeks
measure: Time to Disease Control
timeFrame: Baseline to up to 3 weeks (until disease control)
measure: Time to Rescue Therapy
timeFrame: 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.
measure: The Bullous Pemphigoid Disease Area Index (BPDAI) Score
timeFrame: 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.
measure: The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)
timeFrame: 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.
measure: Total Cumulative Steroid Exposure
timeFrame: 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.
measure: Maximum Daily Steroid Dose
timeFrame: 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.
Other outcomes:
Locations:
city: Dresden
country: Germany
facility: Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie
city: Düsseldorf
country: Germany
facility: Universitätsklinikum Düsseldorf Klinik für Dermatologie
city: Erlangen
country: Germany
facility: Universitätsklinikum Erlangen - Hautklinik
city: Freiburg
country: Germany
facility: Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie
city: Lübeck
country: Germany
facility: Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin
city: Magdeburg
country: Germany
facility: Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik
city: Mainz
country: Germany
facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC)
city: Würzburg
country: Germany
facility: Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie
Study officials:
affiliation: Alkahest, Inc.
name: Alkahest Medical Monitor
role: STUDY_DIRECTOR
References: