rVA576 in Adult Mild to Moderate Bullous Pemphigoid Subjects

• Register: ClinicalTrials.gov
• Last refreshed on: 5 July 2021
• Main ID: NCT04035733
• Date of registration: 12/04/2019
• Prospective Registration: No
• Primary sponsor: AKARI Therapeutics
• Public title: rVA576 in Adult Mild to Moderate Bullous Pemphigoid Subjects
• Scientific title: A Phase IIa Open-label Single Arm Study of Safety and Efficacy of rVA576 in Adult Mild to Moderate Bullous Pemphigoid Subjects
• Date of first enrolment: September 25, 2018
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04035733
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Germany, Netherlands

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Adult male or female =18-year-old patients

2. Subject with newly presenting mild to moderate cutaneous bullous pemphigoid (BP)

3. BPDAI global score at the screening of 10-56 (= 10 but <56)

4. 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.

5. Cutaneous bullous pemphigoid (BP) per standard diagnostic criteria:

1. Clinical presentation (cutaneous blistering and/or itchy dermatosis), AND

2. Direct immunofluorescence (DIF) studies.

6. Karnofsky performance status = 60%

7. 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.

8. 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.

9. Males with a childbearing potential partner must agree to use effective contraception
consistently OR have had a vasectomy

10. Willing and able to adhere to the study visit schedule and other protocol
requirements.

11. Willing and able to provide voluntary written informed consent

12. 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:

1. Patients with severe BP. Severe disease to defined as global BPDAI = 56.

2. Patients with refractory BP.

3. Suspected drug-induced BP

4. Concomitant skin conditions preventing physical evaluation of BP.

5. Participation in a clinical trial of an investigational product within 6 weeks of
screening.

6. Known hypersensitivity to tick or to rVA576 and any of its excipients.

7. 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.

8. 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.

9. Known hypersensitivity to mometasone furoate or to other corticosteroids or to any
excipients in mometasone furoate

10. Received rVA576 for the treatment of the current episode of BP prior to study entry.

11. 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.

12. 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.

13. Congenital or acquired immunodeficiency (e.g. common variable immunodeficiency, organ
transplantation).

14. Clinically significant vital sign measurements or ECG findings as determined by the
Investigator.

15. Clinically significant abnormal laboratory test results.

16. The active or recent history of clinically significant infection within 1 month of
Screening.

17. Pregnant or breast-feeding, or planning to become pregnant during the study.

18. 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)

19. Active abuse of alcohol or drugs.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Bullous Pemphigoid (BP)

Intervention(s)

Drug: rVA576

Primary Outcome(s)

Safety parameter [Time Frame: 42 days]

Secondary Outcome(s)

Efficacy Parameter [Time Frame: 42 days]

Quality of life questionnaire [Time Frame: 42 days]

Secondary ID(s)

AK 801

Source(s) of Monetary Support

Please refer to primary and secondary sponsors