Clinical Trial: Evaluation of Safety, Efficacy and Pharmacodynamic Effect of Bertilimumab in Patients With Bullous Pemphigoid

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: An Open-Label, Proof of Concept Study Designed to Evaluate the Safety, Efficacy and Pharmacodynamic Effect of Bertilimumab in Patients With Newly Diagnosed, Moderate to Extensive Bullous This is an open-label, proof-of-concept, single group study in adult patients with newly diagnosed, moderate to extensive BP.

The study will consist of three periods: a screening period of up to 2 weeks, an open-label treatment period lasting 4 weeks consisting of IV infusion of bertilimumab on Days 0 and 14 and 28, and a safety and efficacy follow-up period of approximately 13 weeks.

Patients will receive concomitant oral steroids during the treatment and follow-up period.

Detailed Summary:

This is an open-label, proof-of-concept, single group study in adult patients with newly diagnosed, moderate to extensive BP.

The study will consist of three periods: a screening period of up to 2 weeks, an open-label treatment period lasting 4 weeks consisting of IV infusion of bertilimumab on Days 0 and 14 and Day 28, and a safety and efficacy follow-up period of approximately 13 weeks.

Patients will receive concomitant oral steroids during the treatment and follow-up period. They will start on 30 mg prednisone daily (or equivalent). The initial dose will be maintained for at least 1 week, commencing on Day 0, until blister formation has ceased, crusts and erosions have disappeared and reepithelialization of lesions has started. The corticosteroid dose will then be reduced to 20 mg daily for 5 to 14 days. According to clinical response, this will be followed by corticosteroid dose reduction in 5 mg steps every 5 to 14 days until a dose of 10 mg daily is reached and then corticosteroid dose reduction in 2.5 mg steps every 5 to 14 days until the end of the study.

Sponsor: Immune Pharmaceuticals

Current Primary Outcome: Safety Endpoints [ Time Frame: participants will be followed for the duration of the study , an expected average of 118 days ]

Original Primary Outcome:

• Safety Measurements [ Time Frame: participants will be followed for the duration of the study , an expected average of 60 days ]

  Safety outcome would be assessed as the number of the following events which the participants experienced throughout the study:

  • Number if Adverse events (AE)
  • Injection site reactions after infusions
  • Abnormal Physical findings during the examination
  • Abnormal Vital signs (blood pressure, heart rate, temperature)
  • Abnormal ECG
  • Number of Concomitant medications taken
  • Abnormal Laboratory values
• Efficacy [ Time Frame: participants will be followed for the duration of the study , an expected average of 60 day ]
  Number of patients which achieved a reduction in BP Disease Area Index (BPDAI questionnaire) score, of at least 50%, 75% and 90%.
• Efficacy [ Time Frame: participants will be followed for the duration of the study , an expected average of 60 day ]
  Number of patients which decrease to prednisone dose of ≤ 10mg/day

Current Secondary Outcome:

• Efficacy Endpoints: : Proportion of patients who achieve a reduction in Bullous Pemphigoid Disease Area Index score of at least 50%, 75% and 90% at visit 6, 7, 8 or 9 compared to baseline [ Time Frame: participants will be followed for the duration of the study , an expected average of 84 days ]
  • BP Disease Area Index Proportion of patients who achieve a reduction in BPDAI score of at least 50%, 75% and 90% at visit 6, 7, 8 or 9 compared to baseline.
  • Proportion of patients who have tapered to prednisone dose of ≤ 10 mg/day at visit 9.

  • Proportion of patients who achieve control of disease activity at visit 6,7, 8 or 9

    o Control of disease activity is defined as the time when at least two of the following occur: new lesions cease to form, established lesions begin to heal and pruritic symptoms start to abate.

  • Mean time from baseline to control of disease activity
  • Change in BPDAI score at each scheduled measurement timepoint compared to baseline
  • Change in pruritus visual analogue score at each scheduled measurement timepoint compared to baseline
  • Change in QOL score at each scheduled measurement timepoint compared to baseline
• Efficacy Endpoint: Proportion of patients who have tapered to prednisone dose of ≤ 10 mg/day at visit 9 [ Time Frame: participants will be followed for the duration of the study , an expected average of 84 days ]

Original Secondary Outcome:

• Efficacy Measurements [ Time Frame: participants will be followed for the duration of the study , an expected average of 60 day ]
  • Number of patients who achieve control of disease activity untill visit 5 (Defined as a state in which new lesions cease to form and established lesions begin to heal and pruritic symptoms start to abate).
  • Mean time to control of disease activity (Defined as the time from baseline at which new lesions cease to form and established lesions begin to heal and pruritic symptoms start to abate).
  • Changes in BP Disease Area Index questionnaire score
  • Changes in pruritus visual analogue questionnaire score
  • Changes in Quality of life questionnaire score at each scheduled
• PHARMACODYNAMIC Measurements [ Time Frame: participants will be followed for the duration of the study , an expected average of 60 day ]

  PD measurements will be assessed through:

  • Changes in the titers of BP180 and BP230 autoantibody
  • Changes in the amount of eosinophils counted in the blood.

Information By: Immune Pharmaceuticals

Dates:
Date Received: July 27, 2014
Date Started: February 2016
Date Completion: April 2017
Last Updated: November 8, 2016
Last Verified: November 2016