- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04035733
rVA576 in Adult Mild to Moderate Bullous Pemphigoid Subjects
June 22, 2021 updated by: AKARI Therapeutics
A Phase IIa Open-label Single Arm Study of Safety and Efficacy of rVA576 in Adult Mild to Moderate Bullous Pemphigoid Subjects
Bullous pemphigoid (BP) is the most common of the autoimmune blistering skin diseases in Western Europe.
[Joly 2012]The study will recruit the new onset or relapsing mild to moderate BP patients.
The study population will consist of patients above the age of 18 years with an active episode of BP, confirmed by inclusion and exclusion criteria and who, in the opinion of the Investigator, would benefit from treatment with rVA576.
Recombinant rVA576 is a small protein complement C5 and LTB4 inhibitor, which prevents the cleavage of C5 by C5 convertase and thereby inhibits generation of C5b-9 the membrane attack complex (MAC), as well as preventing the release of the anaphylatoxin C5a.
rVA576 is effective in inhibiting terminal complement activity irrespective of the activating pathway (classical, lectin or alternative).
This Phase IIa open-label single-arm study will evaluate the safety and efficacy of rVA576 in adult mild to moderate Bullous Pemphigoid patients
Study Overview
Study Type
Interventional
Enrollment (Actual)
9
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Lubeck, Germany
- University of Lübeck
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Hanzeplein 1
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Groningen, Hanzeplein 1, Netherlands, 9700 RB
- UMCG Groningen
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Adult male or female ≥18-year-old patients
- Subject with newly presenting mild to moderate cutaneous bullous pemphigoid (BP)
- BPDAI global score at the screening of 10-56 (≥ 10 but <56)
- Subjects with a relapse of mild to moderate bullous pemphigoid are eligible if their disease was quiescent for at least 2 months before the current relapse.
Cutaneous bullous pemphigoid (BP) per standard diagnostic criteria:
- Clinical presentation (cutaneous blistering and/or itchy dermatosis), AND
- Direct immunofluorescence (DIF) studies.
- Karnofsky performance status ≥ 60%
- Adequate cardiac, renal, hepatic, neurological and psychiatric function as determined by the Investigator and demonstrated by screening laboratory evaluations, vital sign measurement, ECG recording and physical examination results.
- Women of childbearing potential (WOCBP) must agree to use effective contraception consistently throughout the study and have a negative serum pregnancy test at screening and a negative urine pregnancy test per the schedule of visits.
- Males with a childbearing potential partner must agree to use effective contraception consistently OR have had a vasectomy
- Willing and able to adhere to the study visit schedule and other protocol requirements.
- Willing and able to provide voluntary written informed consent
- Willing to receive immunisation against Neisseria meningitidis and antibiotic prophylaxis in accordance with applicable guidelines and local standard of care of the PI at the trial site
Exclusion Criteria:
- Patients with severe BP. Severe disease to defined as global BPDAI ≥ 56.
- Patients with refractory BP.
- Suspected drug-induced BP
- Concomitant skin conditions preventing physical evaluation of BP.
- Participation in a clinical trial of an investigational product within 6 weeks of screening.
- Known hypersensitivity to tick or to rVA576 and any of its excipients.
- BP patients on systemic corticosteroid or systemic immunomodulator or other treatment for the current BP episode (including azathioprine, dapsone, doxycycline, etc) provided the treatment cannot be discontinued before Day 1.
- Treatment with biologics (e.g. etanercept, adalimumab, ustekinumab, infliximab, intravenous immunoglobulin (IVIG) and rituximab or other anti-CD20 therapies) within 5 half-lives of the drugs prior to screening.
- Known hypersensitivity to mometasone furoate or to other corticosteroids or to any excipients in mometasone furoate
- Received rVA576 for the treatment of the current episode of BP prior to study entry.
- Patients with severe medical or surgical conditions at screening or Day 1 including, but not limited to cardiac, respiratory, renal, hepatic, haematological, gastrointestinal, endocrine, pulmonary, cardiac, neurologic, cerebral, psychiatric, or any other severe acute or chronic medical condition that may increase the risk associated with study participation/treatment or may interfere with the interpretation of study results and, in the Investigator's opinion, would make the patient inappropriate for study entry.
- Presence of any malignancy that has been under active treatment or in previous 5 years except for patients with the removal of uncomplicated basal cell carcinoma or cutaneous squamous cell carcinoma, who may take part in the study.
- Congenital or acquired immunodeficiency (e.g. common variable immunodeficiency, organ transplantation).
- Clinically significant vital sign measurements or ECG findings as determined by the Investigator.
- Clinically significant abnormal laboratory test results.
- The active or recent history of clinically significant infection within 1 month of Screening.
- Pregnant or breast-feeding, or planning to become pregnant during the study.
- Evidence of an active disease of hepatitis B (HBsAg positive or HBcAg positive) or hepatitis C (HCV ab positive), CMV (IgM positive) or human immunodeficiency virus (HIV) infection (HIV1/2 Ab positive)
- Active abuse of alcohol or drugs.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Open-label single arm study
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BP subjects will be treated with 30 mg once daily rVA576 regime for 6 weeks.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Safety parameter
Time Frame: 42 days
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Proportion of participants reporting grade 3, 4 and 5 adverse events, which are related/possibly related to rVA576 during the treatment period. Treatment emergent adverse events (TEAEs) consist of:
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42 days
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Efficacy Parameter
Time Frame: 42 days
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Change in BPDAI between baseline (Day 1) and Day 42.
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42 days
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Quality of life questionnaire
Time Frame: 42 days
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Change in quality of life questionnaire between baseline (Day 1) and Day 42
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42 days
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
September 25, 2018
Primary Completion (Actual)
March 12, 2020
Study Completion (Actual)
April 29, 2020
Study Registration Dates
First Submitted
April 12, 2019
First Submitted That Met QC Criteria
July 25, 2019
First Posted (Actual)
July 29, 2019
Study Record Updates
Last Update Posted (Actual)
June 28, 2021
Last Update Submitted That Met QC Criteria
June 22, 2021
Last Verified
June 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- AK 801
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Undecided
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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