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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04499235
Recruitment Status : Recruiting
First Posted : August 5, 2020
Last Update Posted : August 12, 2020
Sponsor:
Information provided by (Responsible Party):
Alkahest, Inc.

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

Condition or disease Intervention/treatment Phase
Pemphigoid, Bullous Drug: Mometasone furoate Drug: AKST4290 Drug: Placebo Phase 2

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 Subjects who receive rescue therapy will remain in the study until disease control, unless they are withdrawn or withdraw from participation.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid
Actual Study Start Date : January 30, 2020
Estimated Primary Completion Date : April 2021
Estimated Study Completion Date : May 2021


Arm Intervention/treatment
Experimental: Mometasone furoate + AKST4290
Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.
Drug: Mometasone furoate
Topical mometasone furoate

Drug: AKST4290
Oral AKST4290

Placebo Comparator: Mometasone furoate + Placebo
Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.
Drug: Mometasone furoate
Topical mometasone furoate

Drug: Placebo
Oral placebo




Primary Outcome Measures :
  1. The proportion of subjects who achieve disease control [ Time Frame: Baseline to up to 3 weeks (until disease control) ]
    The proportion of subjects who achieve disease control (≤ 3 new lesions/day and healing of existing lesions) without requiring rescue therapy.


Secondary Outcome Measures :
  1. Safety as assessed by the incidence, seriousness and severity of AEs [ Time Frame: Baseline to 5 weeks ]
    Treatment emergent AEs summarized by MedDRA coding terms; separate tabulations will be produced for incidence, seriousness and severity of AEs.

  2. Time to disease control [ Time Frame: Baseline to 3 weeks ]
    Time to disease control will be compared between treatment groups by treatment day and week using a Kaplan-Meier curve.

  3. Time to rescue therapy [ Time Frame: Baseline to 3 weeks ]
    Time to rescue therapy will be compared between treatment groups by treatment day and week using a Kaplan-Meier curve.

  4. The Bullous Pemphigoid Disease Area Index (BPDAI) score [ Time Frame: Baseline to 3 weeks ]
    Change from baseline in BPDAI score by treatment week and at disease control. Scores can range from 0 to 360 for BPDAI total activity and 0 to 12 for BPDAI damage, with higher scores indicating greater disease activity or damage.

  5. The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS) [ Time Frame: Baseline to 3 weeks ]
    Change from baseline in pruritus as evaluated by the BPDAI-VAS by treatment week and at disease control. Scores can range from 0 to 30, with higher scores indicating a worse condition.

  6. Total cumulative steroid exposure [ Time Frame: Baseline to 3 weeks ]
    Evaluation of total cumulative steroid exposure at baseline, by treatment week, and at disease control.

  7. Maximum daily steroid dose [ Time Frame: Baseline to 3 weeks ]
    Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control.



Information from the National Library of Medicine

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Ages Eligible for Study:   60 Years to 95 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04499235


Contacts
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Contact: Alkahest Clinical Trials 650-801-0474 trials@alkahest.com

Locations
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Germany
Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie Recruiting
Dresden, Germany, 01307
Universitätsklinikum Düsseldorf Klinik für Dermatologie Recruiting
Düsseldorf, Germany, 40225
Universitätsklinikum Erlangen - Hautklinik Recruiting
Erlangen, Germany, 91054
Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie Recruiting
Freiburg, Germany, 79104
Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin Recruiting
Lübeck, Germany, 23538
Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik Recruiting
Magdeburg, Germany, 39120
Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC) Recruiting
Mainz, Germany, 55131
Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie Recruiting
Würzburg, Germany, 97080
Sponsors and Collaborators
Alkahest, Inc.
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Responsible Party: Alkahest, Inc.
ClinicalTrials.gov Identifier: NCT04499235    
Other Study ID Numbers: AKST4290-221
First Posted: August 5, 2020    Key Record Dates
Last Update Posted: August 12, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Keywords provided by Alkahest, Inc.:
Autoimmune disease
Skin disease
Vesiculobullous
Additional relevant MeSH terms:
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Pemphigoid, Bullous
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases
Mometasone Furoate
Anti-Inflammatory Agents
Dermatologic Agents
Anti-Allergic Agents