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

NCT04499235 | Terminated Phase 2 Results DMC

• 1 other identifier: AKST4290-221
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 8

Brief Summary

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

Conditions

Pemphigoid, Bullous

Keywords

Autoimmune disease; Skin disease; Vesiculobullous

Outcome Measures

Primary Outcomes (1)

• The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy

  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.

  Baseline to up to 3 weeks (until disease control)

Secondary Outcomes (7)

• Number of Participants With TEAEs, Assessed by Seriousness and Severity

  Baseline to 5 weeks

• Time to Disease Control

  Baseline to up to 3 weeks (until disease control)

• 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 (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.

• 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.

Study Arms (2)

Mometasone furoate + AKST4290

EXPERIMENTAL

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

Drug: Mometasone furoate; Drug: AKST4290

Mometasone furoate + Placebo

PLACEBO COMPARATOR

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

Drug: Mometasone furoate; Drug: Placebo

Interventions

Mometasone furoate DRUG

Topical mometasone furoate

Mometasone furoate + AKST4290; Mometasone furoate + Placebo

AKST4290 DRUG

Oral AKST4290

Mometasone furoate + AKST4290

Placebo DRUG

Oral placebo

Mometasone furoate + Placebo

Eligibility Criteria

• Age: 60 Years - 95 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alkahest, Inc.: lead

Study Sites (8)

Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie

Dresden, 01307, Germany

Location

Universitätsklinikum Düsseldorf Klinik für Dermatologie

Düsseldorf, 40225, Germany

Location

Universitätsklinikum Erlangen - Hautklinik

Erlangen, 91054, Germany

Location

Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie

Freiburg im Breisgau, 79104, Germany

Location

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

Lübeck, 23538, Germany

Location

Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik

Magdeburg, 39120, Germany

Location

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

Mainz, 55131, Germany

Location

Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie

Würzburg, 97080, Germany

Location

MeSH Terms

Conditions

Pemphigoid, Bullous; Autoimmune Diseases; Skin Diseases

Interventions

Mometasone Furoate

Condition Hierarchy (Ancestors)

Skin Diseases, Vesiculobullous; Skin and Connective Tissue Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Results Point of Contact

• Title: Head of Communications
• Organization: Alkahest, Inc.

info@alkahest.com

(650) 801-0474

Study Officials

• Alkahest Medical Monitor

  Alkahest, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 31, 2020
• First Posted: August 5, 2020
• Study Start: January 30, 2020
• Primary Completion: March 29, 2021
• Study Completion: April 14, 2021
• Last Updated: September 14, 2023
• Results First Posted: September 14, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

DE (8)