Bullous PemphigoidPhase 3ARREST-BPNCT05061771
Nomacopan Therapy in Adult Patients With Bullous Pemphigoid Receiving Adjunct Oral Corticosteroid Therapy (ARREST-BP)
A Phase 3 study testing nomacopan (rVA576) in people with Bullous Pemphigoid, run by AKARI Therapeutics at 12 sites in 4 countries.
Trial summary
A phase III two-part study of nomacopan, a bifunctional inhibitor of complement component C5 and leukotriene B4 (LTB4), for the treatment of moderate and severe bullous pemphigoid. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Bullous Pemphigoid
- What is being tested?
- nomacopan (rVA576)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 12 sitesin 4 countries
- Study dates
- 6 May 2022to 1 Aug 2022
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female between 18 and 89 years of age inclusive at the time of consent with Karnofsky score of 50% or more at screening
- Male or female ≥90 years of age at the time of consent with Karnofsky score of 70% or more at screening
- Diagnosis of Bullous Pemphigoid either newly diagnosed or relapsing
- Patients with confirmed atypical Bullous Pemphigoid
- Bullous Pemphigoid classified as either moderate or severe on the basis of the Investigator Global Assessment (IGA) at randomisation
- Willing to receive immunisation against Neisseria meningitidis and/or antibiotic prophylaxis
- Provision of voluntary written informed consent
Who cannot join
- Patients with recalcitrant BP that have never achieved CDA or who have never been in complete disease remission despite long term treatment with super potent topical steroid or oral cotricosteroid
- Epidermolysis bullosa acquisita, mucous membrane pemphigoid, or anti p200 pemphigoid
- Mucosal lesions BPDAI score accounts for ≥30% of total BPDAI activity score at randomisation
- BP considered to be drug induced, in particular diagnosis of BP made within two months of starting a drug well known to induce BP
- Treatment with BP-directed biologics including: a) Any cell-depleting agents including, but not limited to, rituximab within 12 months prior to baseline, b) Other biologics within five half-lives (if known) or 16 weeks prior to the baseline, whichever is longer, or c) Intravenous immunoglobulin within 16 weeks prior to the baseline.
- Taking > 0.3 mg/kg/day OCS at screening
Show all 17Show fewer
- Treatment with systemic immunomodulators such as dapsone or doxycycline within four half-lives of the drugs prior to baseline Day 1
- Treatment with immunosuppressants within the last two weeks prior to baseline
- Treatment with an anti-complement therapy or with Zileuton within the last three months prior to baseline
- OCS dose no more than 0.3mg/kg/day in the 7 days before screening visit
- Taking super-potent topical corticosteroids and unable to discontinue them at or before the screening assessment
- Active systemic or organ system bacterial or fungal infection or progressive severe infection
- Known congenital immunodeficiency or a history of acquired immunodeficiency including a positive human immunodeficiency virus (HIV) test
- Active infection with hepatitis B or C
- Positive nasal throat swab for Neisseria species
- Known hypersensitivity to nomacopan and any of its excipients
- Receipt of live attenuated vaccines within 2 weeks of Day 1
Trial locations and contacts
12 sites in 4 countries.
12 sites in 4 countries
United States7 sites
David Fivenson MD PLC
Ann Arbor, Michigan, 48103
Duke Dermatology
Durham, North Carolina, 27710
Wright State Physicians 725 University Blvd.
Fairborn, Ohio, 45324
Dawes Fretzin Clinical Research Group LLC
Indianapolis, Indiana, 46250
Tulane University Health Sciences Center
Los Angeles, California, 70112
UMPC Department of Dermatology
Pittsburgh, Pennsylvania, 15213
North Shore University Health System
Skokie, Illinois, 60077
Germany3 sites
MENSINGDERMA Research GmbH
Hamburg, 22391
Universitätsklinikum Schleswig-Holstein
Kiel, 24105
Universitäts Hautklinik
Tübingen, 72076
Poland1 site
Cityclinic Przychodnia Lekarsko-Psychologiczna Matusiak Spółka Partnerska
Wroclaw
About this study
Official title: A Randomized, Part A Partial Blinded and Part B Double Blinded, Placebo-controlled 24-week Clinical Study to Evaluate the Efficacy and Safety of Nomacopan Therapy in Adult Patients With Bullous Pemphigoid Receiving Adjunct Oral Corticosteroid Therapy (ARREST-BP)
A phase III two-part study of nomacopan, a bifunctional inhibitor of complement component C5 and leukotriene B4 (LTB4), for the treatment of moderate and severe bullous pemphigoid. There is evidence that both terminal complement activation (via C5) and the lipid mediator LTB4 may have a central role in driving the disease. In this study patients will be randomized to receive either nomacopan plus oral corticosteroids (OCS) or placebo plus OCS for a treatment period of 24 weeks. OCS will be tapered over the course of the treatment if the symptoms of disease improve.
- Conditions
- Bullous Pemphigoid
- Being tested
- nomacopan (rVA576), Placebo
- Keywords
- pemphigoid, blistering skin disease, nomacopan, complement, leukotriene
- Medical subject headings
- Pemphigoid, Bullous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
nomacopan (rVA576)Drug
Nomacopan an inhibitor of complement C5 and LTB4
PlaceboOther
Placebo
| Study group | Role | What they receive |
|---|---|---|
| nomacopan (rVA576) | Active comparator | PART A:
High dose nomacopan (standard complement ablating doses on Day 1 followed by 45 mg qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day OCS qd
or
Low dose nomacopan (standard complement ablating doses on Day 1 followed by 15 mg qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day OCS
PART B:
Nomacopan (standard complement ablating doses on Day 1 followed by to be confirmed mg qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day OCS qd Receives: Drug: nomacopan (rVA576) |
| Placebo | Placebo comparator | PART A:
Placebo (matching standard complement ablating doses on Day 1 and then matching injection volume of 45mg dose qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day oral corticosteroid (OCS) qd
or
Placebo (matching standard complement ablating doses on Day 1 and then matching injection volume of 15mg dose qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day oral corticosteroid (OCS) qd
PART B:
Placebo (matching standard complement ablating doses on Day 1 and then matching injection volume of active dose qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day oral corticosteroid (OCS) qd Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Achievement of Complete Disease Remission
Proportion of patients in Complete Disease Remission
Measured: weeks 16 - 24
Secondary outcomes (10)
Cumulative oral corticosteroid, OCS, during treatment
Cumulative OCS used during treatment
Measured: Randomization to 24 weeks
Proportion of patients requiring rescue therapy
Proportion of patients requiring rescue therapy during the 24 weeks of treatment
Measured: Randomization to 24 weeks
Achievement Partial Disease Remission
Proportion of patients in Partial Disease Remission
Measured: weeks 16 - 24
Time to onset of Complete Disease Remission
Time (weeks) to onset of Complete Disease Remission
Measured: week 6 to 24
Duration of Complete and Partial Disease Remission
Duration (weeks) of Complete Disease Remission and Partial Disease Remission
Measured: week 6 to 24
Investigator Global Assessment (IGA) score
Proportion of patients with Investigator Global Assessment (IGA) score of 0 or 1
Measured: weeks 6 - 24
Adverse Events
Frequency, type and relationship of AEs to treatment
Measured: Day 1 to Week 28
Steroid-related AEs
Incidence of steroid-related AEs
Measured: Day 1 to Week 28
Dermatology Life Quality Index (DLQI)
Change from baseline in Dermatology Life Quality Index (DLQI)
Measured: Randomisation to week 24
Incidence of treatment-emergent anti-drug antibody (ADA) responses and titre and neutralising potential assessed in vitro at baseline and every 4 weeks
Incidence of treatment-emergent anti-drug antibody (ADA) responses and titre and neutralising potential assessed in vitro at baseline and then every 4 weeks
Measured: Day 1 to Week 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2021
- Study start6 May 2022
- Primary completion1 Aug 2022
- Study completion1 Aug 2022
Why it stopped: Akari has decided to discontinue AK802 study due to strategic resource allocation decisions.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bullous PemphigoidStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- AKARI Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05061771
- Sponsor's protocol ID
- AK802
- Acronym
- ARREST-BP
- Status last verified
- Oct 2022
- First submitted
- 15 Sep 2021
- Last updated
- 9 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Part A Partial Blinded and Part B Double Blinded, Placebo-controlled 24-week Clinical Study to Evaluate the Efficacy and Safety of Nomacopan Therapy in Adult Patients With Bullous Pemphigoid Receiving Adjunct Oral Corticosteroid Therapy (ARREST-BP)
- ClinicalTrials.gov ID
- NCT05061771
- Acronym / sponsor ID
- ARREST-BP / AK802
- Conditions
- Bullous Pemphigoid
- Keywords
- pemphigoid, blistering skin disease, nomacopan, complement, leukotriene
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Akari has decided to discontinue AK802 study due to strategic resource allocation decisions.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- AKARI TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 6 May 2022 (actual)
- Primary completion
- 1 Aug 2022 (actual)
- Study completion
- 1 Aug 2022 (actual)
- First posted
- 30 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A phase III two-part study of nomacopan, a bifunctional inhibitor of complement component C5 and leukotriene B4 (LTB4), for the treatment of moderate and severe bullous pemphigoid. There is evidence that both terminal complement activation (via C5) and the lipid mediator LTB4 may have a central role in driving the disease. In this study patients will be randomized to receive either nomacopan plus oral corticosteroids (OCS) or placebo plus OCS for a treatment period of 24 weeks. OCS will be tapered over the course of the treatment if the symptoms of disease improve.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| nomacopan (rVA576) | Active comparator | Drug: nomacopan (rVA576)PART A: High dose nomacopan (standard complement ablating doses on Day 1 followed by 45 mg qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day OCS qd or Low dose nomacopan (standard complement ablating doses on Day 1 followed by 15 mg qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day OCS PART B: Nomacopan (standard complement ablating doses on Day 1 followed by to be confirmed mg qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day OCS qd |
| Placebo | Placebo comparator | Other: PlaceboPART A: Placebo (matching standard complement ablating doses on Day 1 and then matching injection volume of 45mg dose qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day oral corticosteroid (OCS) qd or Placebo (matching standard complement ablating doses on Day 1 and then matching injection volume of 15mg dose qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day oral corticosteroid (OCS) qd PART B: Placebo (matching standard complement ablating doses on Day 1 and then matching injection volume of active dose qd) administered by subcutaneous injection, plus a starting dose of 0.5 mg/kg/day oral corticosteroid (OCS) qd |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male or female between 18 and 89 years of age inclusive at the time of consent with Karnofsky score of 50% or more at screening
- Male or female ≥90 years of age at the time of consent with Karnofsky score of 70% or more at screening
- Diagnosis of Bullous Pemphigoid either newly diagnosed or relapsing
- Patients with confirmed atypical Bullous Pemphigoid
- Bullous Pemphigoid classified as either moderate or severe on the basis of the Investigator Global Assessment (IGA) at randomisation
Key exclusion criteria (17 listed)
- Patients with recalcitrant BP that have never achieved CDA or who have never been in complete disease remission despite long term treatment with super potent topical steroid or oral cotricosteroid
- Epidermolysis bullosa acquisita, mucous membrane pemphigoid, or anti p200 pemphigoid
- Mucosal lesions BPDAI score accounts for ≥30% of total BPDAI activity score at randomisation
- BP considered to be drug induced, in particular diagnosis of BP made within two months of starting a drug well known to induce BP
- Treatment with BP-directed biologics including: a) Any cell-depleting agents including, but not limited to, rituximab within 12 months prior to baseline, b) Other biologics within five half-lives (if known) or 16 weeks prior to the baseline, whichever is longer, or c) Intravenous immunoglobulin within 16 weeks prior to the baseline.
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 |
|---|---|---|
| nomacopan (rVA576)Nomacopan an inhibitor of complement C5 and LTB4 | Drug | nomacopan (rVA576) |
| PlaceboPlacebo | Other | Placebo |
Primary outcome
Achievement of Complete Disease Remission
Time frame: weeks 16 - 24
Proportion of patients in Complete Disease Remission
10 secondary outcomes
Cumulative oral corticosteroid, OCS, during treatment
Time frame: Randomization to 24 weeks
Proportion of patients requiring rescue therapy
Time frame: Randomization to 24 weeks
Achievement Partial Disease Remission
Time frame: weeks 16 - 24
Time to onset of Complete Disease Remission
Time frame: week 6 to 24
Duration of Complete and Partial Disease Remission
Time frame: week 6 to 24
Investigator Global Assessment (IGA) score
Time frame: weeks 6 - 24
Adverse Events
Time frame: Day 1 to Week 28
Steroid-related AEs
Time frame: Day 1 to Week 28
Dermatology Life Quality Index (DLQI)
Time frame: Randomisation to week 24
Incidence of treatment-emergent anti-drug antibody (ADA) responses and titre and neutralising potential assessed in vitro at baseline and every 4 weeks
Time frame: Day 1 to Week 28
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2025.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
12 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing nomacopan (rVA576) in people with Bullous Pemphigoid, run by AKARI Therapeutics at 12 sites in 4 countries.
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 7 inclusion and 17 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: withdrawn, as of the sponsor's last update on 9 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Germany, Netherlands, Poland. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Achievement of Complete Disease Remission (time frame: weeks 16 - 24). 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 9 Apr 2025.