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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open-label single arm study | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rVA576 | Drug | BP subjects will be treated with 30 mg once daily rVA576 regime for 6 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety parameter | 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:
| 42 days |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy Parameter | Change in BPDAI between baseline (Day 1) and Day 42. | 42 days |
| Quality of life questionnaire | Change in quality of life questionnaire between baseline (Day 1) and Day 42 |
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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:
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:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Lubeck | Lübeck | Germany | ||||
| UMCG Groningen |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35507334 | Derived | Sadik CD, Rashid H, Hammers CM, Diercks GFH, Weidinger A, Beissert S, Schauer F, Fettiplace J, Thaci D, Ngai Y, Nunn MA, Zillikens D, Horvath B. Evaluation of Nomacopan for Treatment of Bullous Pemphigoid: A Phase 2a Nonrandomized Controlled Trial. JAMA Dermatol. 2022 Jun 1;158(6):641-649. doi: 10.1001/jamadermatol.2022.1156. |
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| ID | Term |
|---|---|
| D010391 | Pemphigoid, Bullous |
| ID | Term |
|---|---|
| D012872 | Skin Diseases, Vesiculobullous |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
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A Phase IIa open-label single arm study of safety and efficacy of rVA576 in adult mild to moderate Bullous Pemphigoid subjects
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| 42 days |
| Groningen |
| Hanzeplein 1 |
| 9700 RB |
| Netherlands |
| D007154 | Immune System Diseases |