Nomacopan Therapy in Adult Patients With Bullous Pemphigoid Receiving Adjunct Oral Corticosteroid Therapy (ARREST-BP)

NCT05061771 | Withdrawn Phase 3 FDA Drug

• 1 other identifier: AK802
• Study Type: interventional
• Target: N/A
• Locations: 4 countries
• Sites: 12

Brief 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. 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

Keywords

pemphigoid; blistering skin disease; nomacopan; complement; leukotriene

Outcome Measures

Primary Outcomes (1)

• Achievement of Complete Disease Remission

  Proportion of patients in Complete Disease Remission

  weeks 16 - 24

Secondary Outcomes (10)

• Cumulative oral corticosteroid, OCS, during treatment

  Randomization to 24 weeks

• Proportion of patients requiring rescue therapy

  Randomization to 24 weeks

• Achievement Partial Disease Remission

  weeks 16 - 24

• Time to onset of Complete Disease Remission

  week 6 to 24

• Duration of Complete and Partial Disease Remission

  week 6 to 24

Study Arms (2)

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

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

Other: Placebo

Interventions

nomacopan (rVA576) DRUG

Nomacopan an inhibitor of complement C5 and LTB4

nomacopan (rVA576)

Placebo OTHER

Placebo

Placebo

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• AKARI Therapeutics: lead

Study Sites (12)

Tulane University Health Sciences Center

Los Angeles, California, 70112, United States

Location

North Shore University Health System

Skokie, Illinois, 60077, United States

Location

Dawes Fretzin Clinical Research Group LLC

Indianapolis, Indiana, 46250, United States

Location

David Fivenson MD PLC

Ann Arbor, Michigan, 48103, United States

Location

Duke Dermatology

Durham, North Carolina, 27710, United States

Location

Wright State Physicians 725 University Blvd.

Fairborn, Ohio, 45324, United States

Location

UMPC Department of Dermatology

Pittsburgh, Pennsylvania, 15213, United States

Location

MENSINGDERMA Research GmbH

Hamburg, 22391, Germany

Location

Universitätsklinikum Schleswig-Holstein

Kiel, 24105, Germany

Location

Universitäts Hautklinik

Tübingen, 72076, Germany

Location

University Medical Center Groningen

Groningen, 9700RB, Netherlands

Location

Cityclinic Przychodnia Lekarsko-Psychologiczna Matusiak Spółka Partnerska

Wroclaw, Poland

Location

MeSH Terms

Conditions

Pemphigoid, Bullous

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 15, 2021
• First Posted: September 30, 2021
• Study Start: May 6, 2022
• Primary Completion: August 1, 2022
• Study Completion: August 1, 2022
• Last Updated: April 9, 2025

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (7); DE (3); PL (1); NL (1)