A Study to Assess the Therapeutic Effect and Safety of Adjunctive AKST4290 in Subjects With Bullous Pemphigoid

NCT ID: NCT04499235

Last Updated: 2023-09-14

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-01-30
• Study Completion Date: 2021-04-14

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 Subjects who receive rescue therapy will remain in the study until disease control, unless they are withdrawn or withdraw from participation.

Conditions

Pemphigoid, Bullous

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Mometasone furoate + AKST4290

Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.

Group Type: EXPERIMENTAL

Mometasone furoate

Intervention Type: DRUG

Topical mometasone furoate

AKST4290

Intervention Type: DRUG

Oral AKST4290

Mometasone furoate + Placebo

Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.

Group Type: PLACEBO_COMPARATOR

Mometasone furoate

Intervention Type: DRUG

Topical mometasone furoate

Placebo

Intervention Type: DRUG

Oral placebo

Interventions

Mometasone furoate

Topical mometasone furoate

Intervention Type: DRUG

AKST4290

Oral AKST4290

Intervention Type: DRUG

Placebo

Oral placebo

Intervention Type: DRUG

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.

• Minimum Eligible Age: 60 Years
• Maximum Eligible Age: 95 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Alkahest, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Alkahest Medical Monitor

Role: STUDY_DIRECTOR

Alkahest, Inc.

Locations

Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie

Dresden, , Germany

Universitätsklinikum Düsseldorf Klinik für Dermatologie

Düsseldorf, , Germany

Universitätsklinikum Erlangen - Hautklinik

Erlangen, , Germany

Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie

Freiburg im Breisgau, , Germany

Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin

Lübeck, , Germany

Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik

Magdeburg, , Germany

Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC)

Mainz, , Germany

Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie

Würzburg, , Germany

Countries

Germany

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

AKST4290-221

Identifier Type: -

Identifier Source: org_study_id