Pemphigoid, BullousPhase 2NCT04499235
A Study to Assess the Therapeutic Effect and Safety of Adjunctive AKST4290 in Subjects With Bullous Pemphigoid
A Phase 2 study testing Mometasone furoate and AKST4290 in people with Pemphigoid, Bullous, run by Alkahest, Inc. at 8 sites in Germany.
Trial summary
This study will evaluate the therapeutic effect and safety of adjunctive AKST4290 in subjects with bullous pemphigoid (BP). Read more about this study
- Who is it for?
- Ages 60 to 95 years
- Conditions the study is for
- Pemphigoid, Bullous
- What is being tested?
- Mometasone furoate and AKST4290
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 8 sitesGermany
- Study dates
- 30 Jan 2020to 14 Apr 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 60 to 95 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
Germany8 sites
Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie
Dresden, 01307
Universitätsklinikum Düsseldorf Klinik für Dermatologie
Düsseldorf, 40225
Universitätsklinikum Erlangen - Hautklinik
Erlangen, 91054
Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie
Freiburg im Breisgau, 79104
Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin
Lübeck, 23538
Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik
Magdeburg, 39120
Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC)
Mainz, 55131
Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie
Würzburg, 97080
About this study
Official title: Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid
This study will evaluate the therapeutic effect and safety of adjunctive AKST4290 in subjects with bullous pemphigoid (BP).
Read the 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
- Being tested
- Mometasone furoate, AKST4290, Placebo
- Keywords
- Autoimmune disease, Skin disease, Vesiculobullous
- Medical subject headings
- Pemphigoid, Bullous, Autoimmune Diseases, Skin Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mometasone furoateDrug
Topical mometasone furoate
AKST4290Drug
Oral AKST4290
PlaceboDrug
Oral placebo
| Study group | Role | What they receive |
|---|---|---|
| Mometasone furoate + AKST4290 | Experimental | Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached. Receives: Drug: Mometasone furoate; Drug: AKST4290 |
| Mometasone furoate + Placebo | Placebo comparator | Subjects will receive mometasone furoate concurrently with placebo until disease control is reached. Receives: Drug: Mometasone furoate; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to up to 3 weeks (until disease control)
Secondary outcomes (7)
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
Measured: Baseline to 5 weeks
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.
Measured: Baseline to up to 3 weeks (until disease control)
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.
Measured: 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
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.
Measured: 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)
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.
Measured: 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
Total cumulative steroid exposure (cortisol equivalent/kg) by treatment group
Measured: 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
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.
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2020
- Study start30 Jan 2020
- Primary completion29 Mar 2021
- Study completion14 Apr 2021
Why it 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pemphigoid, BullousStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alkahest, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Alkahest Medical Monitor, Study director, Alkahest, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04499235
- Sponsor's protocol ID
- AKST4290-221
- Status last verified
- Sep 2023
- First submitted
- 31 Jul 2020
- Last updated
- 14 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid
- ClinicalTrials.gov ID
- NCT04499235
- Acronym / sponsor ID
- AKST4290-221
- Conditions
- Pemphigoid, Bullous
- Keywords
- Autoimmune disease, Skin disease, Vesiculobullous
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason 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.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Alkahest, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Jan 2020 (actual)
- Primary completion
- 29 Mar 2021 (actual)
- Study completion
- 14 Apr 2021 (actual)
- First posted
- 5 Aug 2020 (actual)
- Results
- Posted 14 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mometasone furoate + AKST4290 | Experimental | Drug: Mometasone furoate, Drug: AKST4290Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached. |
| Mometasone furoate + Placebo | Placebo comparator | Drug: Mometasone furoate, Drug: PlaceboSubjects will receive mometasone furoate concurrently with placebo until disease control is reached. |
Who may qualify
- Ages
- 60 to 95 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mometasone furoateTopical mometasone furoate | Drug | Mometasone furoate + AKST4290, Mometasone furoate + Placebo |
| AKST4290Oral AKST4290 | Drug | Mometasone furoate + AKST4290 |
| PlaceboOral placebo | Drug | Mometasone furoate + Placebo |
Primary outcome
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.
7 secondary outcomes
Number of Participants With TEAEs, Assessed by Seriousness and Severity
Time frame: Baseline to 5 weeks
Time to Disease Control
Time frame: Baseline to up to 3 weeks (until disease control)
Time to Rescue Therapy
Time frame: 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
Time frame: 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)
Time frame: 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
Time frame: 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
Time frame: 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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alkahest Medical Monitor study director
Alkahest, Inc.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mometasone furoate and AKST4290 in people with Pemphigoid, Bullous, run by Alkahest, Inc. at 8 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Sep 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy (time frame: Baseline to up to 3 weeks (until disease control)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2023.