Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid
Overview
- Phase
- Phase 2
- Intervention
- Mometasone furoate
- Conditions
- Pemphigoid, Bullous
- Sponsor
- Alkahest, Inc.
- Enrollment
- 6
- Locations
- 8
- Primary Endpoint
- The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy
- Status
- Terminated
- Last Updated
- 2 years ago
Overview
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 Subjects who receive rescue therapy will remain in the study until disease control, unless they are withdrawn or withdraw from participation.
Investigators
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.
Arms & Interventions
Mometasone furoate + AKST4290
Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.
Intervention: Mometasone furoate
Mometasone furoate + AKST4290
Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.
Intervention: AKST4290
Mometasone furoate + Placebo
Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.
Intervention: Mometasone furoate
Mometasone furoate + Placebo
Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.
Intervention: Placebo
Outcomes
Primary Outcomes
The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy
Time Frame: Baseline to up to 3 weeks (until disease control)
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.
Secondary Outcomes
- Number of Participants With TEAEs, Assessed by Seriousness and Severity(Baseline to 5 weeks)
- Time to Rescue Therapy(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.)
- The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)(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.)
- Maximum Daily Steroid Dose(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.)
- Time to Disease Control(Baseline to up to 3 weeks (until disease control))
- The Bullous Pemphigoid Disease Area Index (BPDAI) Score(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.)
- Total Cumulative Steroid Exposure(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.)