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

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Primary Purpose

Status

Terminated

Phase

Phase 2

Locations

Germany

Study Type

Interventional

Intervention

Mometasone furoate

AKST4290

Placebo

About this trial

This is an interventional treatment trial for Pemphigoid, Bullous focused on measuring Autoimmune disease, Skin disease, Vesiculobullous

Eligibility Criteria

60 Years - 95 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie
Universitätsklinikum Düsseldorf Klinik für Dermatologie
Universitätsklinikum Erlangen - Hautklinik
Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie
Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin
Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik
Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC)
Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Mometasone furoate + AKST4290

Mometasone furoate + Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Number of Participants With TEAEs, Assessed by Seriousness and Severity

Treatment-emergent AEs summarized by MedDRA coding terms; separate tabulations produced for incidence, seriousness and severity of AEs

Time to Disease Control

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.

Time to Rescue Therapy

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.

The Bullous Pemphigoid Disease Area Index (BPDAI) Score

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.

The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)

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.

Total Cumulative Steroid Exposure

Total cumulative steroid exposure (cortisol equivalent/kg) by treatment group

Maximum Daily Steroid Dose

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.

Full Information

NCT ID

NCT04499235

First Posted

July 31, 2020

Last Updated

September 1, 2023

Sponsor

Alkahest, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04499235

Brief Title

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

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Terminated

Why Stopped

The study was prematurely terminated by the Sponsor due to operational challenges stemming from the coronavirus disease 2019 (COVID-19) pandemic, treatment limitations, rarity of the disease, and drug supply considerations.

Study Start Date

January 30, 2020 (Actual)

Primary Completion Date

March 29, 2021 (Actual)

Study Completion Date

April 14, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Alkahest, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

No

Studies a U.S. FDA-regulated Device Product

No

Product Manufactured in and Exported from the U.S.

Yes

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pemphigoid, Bullous

Keywords

Autoimmune disease, Skin disease, Vesiculobullous

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

6 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Mometasone furoate + AKST4290

Arm Type

Experimental

Arm Description

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

Arm Title

Mometasone furoate + Placebo

Arm Type

Placebo Comparator

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Mometasone furoate

Intervention Description

Topical mometasone furoate

Intervention Type

Drug

Intervention Name(s)

AKST4290

Intervention Description

Oral AKST4290

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Oral placebo

Primary Outcome Measure Information:

Title

The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy

Description

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.

Time Frame

Baseline to up to 3 weeks (until disease control)

Secondary Outcome Measure Information:

Title

Number of Participants With TEAEs, Assessed by Seriousness and Severity

Description

Treatment-emergent AEs summarized by MedDRA coding terms; separate tabulations produced for incidence, seriousness and severity of AEs

Time Frame

Baseline to 5 weeks

Title

Time to Disease Control

Description

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.

Time Frame

Baseline to up to 3 weeks (until disease control)

Title

Time to Rescue Therapy

Description

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.

Time Frame