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rVA576 in Adult Mild to Moderate Bullous Pemphigoid Subjects

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ClinicalTrials.gov Identifier: NCT04035733
Recruitment Status : Recruiting
First Posted : July 29, 2019
Last Update Posted : January 13, 2020
Sponsor:
Information provided by (Responsible Party):
AKARI Therapeutics

Tracking Information
First Submitted Date  ICMJE April 12, 2019
First Posted Date  ICMJE July 29, 2019
Last Update Posted Date January 13, 2020
Actual Study Start Date  ICMJE September 25, 2018
Estimated Primary Completion Date March 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 25, 2019)
Safety parameter [ Time Frame: 42 days ]
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:
  • Change from baseline in physical examination
  • ECG
  • Clinical laboratory tests
  • Vital signs The Common Terminology Criteria for Adverse Events (CTCAE v4.03) will be used to grade adverse events.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 25, 2019)
  • Efficacy Parameter [ Time Frame: 42 days ]
    Change in BPDAI between baseline (Day 1) and Day 42.
  • Quality of life questionnaire [ Time Frame: 42 days ]
    Change in quality of life questionnaire between baseline (Day 1) and Day 42
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE rVA576 in Adult Mild to Moderate Bullous Pemphigoid Subjects
Official Title  ICMJE A Phase IIa Open-label Single Arm Study of Safety and Efficacy of rVA576 in Adult Mild to Moderate Bullous Pemphigoid Subjects
Brief Summary 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
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
A Phase IIa open-label single arm study of safety and efficacy of rVA576 in adult mild to moderate Bullous Pemphigoid subjects
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Bullous Pemphigoid (BP)
Intervention  ICMJE Biological: rVA576
BP subjects will be treated with 30 mg once daily rVA576 regime for 6 weeks.
Study Arms  ICMJE Experimental: Open-label single arm study
Intervention: Biological: rVA576
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 25, 2019)
9
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 30, 2020
Estimated Primary Completion Date March 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 including self-injection.
  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. 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.
  8. Known hypersensitivity to mometasone furoate or to other corticosteroids or to any excipients in mometasone furoate
  9. Received rVA576 for the treatment of the current episode of BP prior to study entry.
  10. 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.
  11. 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.
  12. Congenital or acquired immunodeficiency (e.g. common variable immunodeficiency, organ transplantation).
  13. Clinically significant vital sign measurements or ECG findings as determined by the Investigator.
  14. Clinically significant abnormal laboratory test results.
  15. The active or recent history of clinically significant infection within 1 month of Screening.
  16. Pregnant or breast-feeding, or planning to become pregnant during the study.
  17. 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)
  18. Active abuse of alcohol or drugs.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Miles Nunn, MD 02080040267 Miles.Nunn@akaritx.com
Contact: Wynne Weston 02080040267 wynne.weston-davies@akaritx.com
Listed Location Countries  ICMJE Germany,   Netherlands
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04035733
Other Study ID Numbers  ICMJE AK 801
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party AKARI Therapeutics
Study Sponsor  ICMJE AKARI Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account AKARI Therapeutics
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP