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

NCT02226146 | Completed Phase 2 Results

• 1 other identifier: Immune/BRT/BP-01
• Study Type: interventional
• Target: 11
• Locations: 2 countries
• Sites: 8

Brief Summary

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

Conditions

Pemphigoid, Bullous

Keywords

Pemphigoid Bullous; Bullous; Blisters; SKIN DISEASES; BP

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Anti-Drug Antibodies

  Baseline up to 1 year

• Number of Participants With Treatment Emergent Adverse Events (TEAEs)

  An AE is any untoward medical occurrence in a participant, which does not necessarily have a causal relationship with the study treatment. TEAEs were defined as AEs that started on or after the first administration of study drug until the end of the follow up period. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.

  Baseline up to 1 year

Secondary Outcomes (4)

• Number of Participants Who Achieved at Least 50%, 70% and 90% Reduction From Baseline in Total Activity Score of the Bullous Pemphigoid Disease Area Index (BPDAI) Score

  Baseline, Day 84

• Number of Participants Who Had Tapered to Prednisone Dose of ≤ 10 mg/Day

  Baseline, Day 84

• Percentage of Reduction From Baseline in BPDAI Pruritis (Visual Analogue Scale [VAS]) Total Score

  Baseline, Day 84

• Percentage of Reduction From Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) Total Score

  Baseline, Day 84

Study Arms (1)

Bertilimumab

EXPERIMENTAL

Intravenous injection over 30 minutes of 10 mg/kg of Bertilimumab in physiological solution (PBS)

Biological: Bertilimumab

Interventions

Bertilimumab BIOLOGICAL

Bertilimumab

Eligibility Criteria

• Age: 60 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females, ≥ 60 years of age.
• Karnofsky performance status \> 60%
• Newly diagnosed, Bullous Pemphigoid per standard diagnostic criteria:
• Clinical presentation \[2\]
• Skin biopsy from a fresh blister showing subepidermal clefting and an inflammatory infiltrate consisting mainly of eosinophils
• Immunofluorescence (IF) studies performed on uninvolved skin collected approximately 1 cm away from a fresh blister showing linear deposition of IgG and/or C3 along the basement membrane zone.
• Moderate to extensive Bullous Pemphigoid defined by the mean number of new bullae and urticarial plaques that have appeared over the course of 3 days as determined by the investigator or referring physician (moderate disease defined by \> 1 and ≤ 10 new bullae daily and ≥ 5 urticarial plaques and extensive disease by \>10 new bullae daily) \[3\].
• Adequate cardiac, renal and hepatic function as determined by the Investigator and demonstrated by screening laboratory evaluations, vital sign measurement, ECG recording and physical examination results.
• Females of childbearing potential must agree to use effective contraception consistently throughout the study (such as hormonal contraception or two forms of barrier contraception) and have a negative serum pregnancy test at screening and a negative urine pregnancy test per the schedule of visits. Women are considered post-menopausal and not of childbearing potential if they have had 12 months of amenorrhea or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks previously.
• Males must have had a vasectomy or have expressed that they have no interest in fertility in the future.
• Fertile males must agree to use effective contraception consistently throughout the study and for a period of four months following the end of study drug administration.
• Willing and able to adhere to the study visit schedule and other protocol requirements.
• Willing and able to provide voluntary written informed consent or written informed consent from a legally authorized representative with assent from the patient.

You may not qualify if:

• Patients with severe medical or surgical conditions at screening or baseline including, but not limited to, severe dementia or mental impairment, severe stroke, severe cardiac insufficiency, severe arterial hypertension, severe or uncontrolled renal, hepatic, hematological, 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 (e.g., radiotherapy or chemotherapy) within the 2 years prior to baseline or is anticipated to require treatment during the study period (including follow up) with the exception of patients with 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, unless regarded by the Investigator as related to BP, including but not limited to:
• Hemoglobin level \<10.0 g/dL
• White blood cell count \< 3 x 103/μL
• Lymphocyte count \< 0.5 x 103/μL
• Platelet count \<100 x 103/μL or \>1200 x 103/μL
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3 the upper limit of normal (ULN)
• Alkaline phosphatase \>3 ULN
• Serum creatinine \>2 ULN
• Patients with mild, relapsed or refractory Bullous Pemphigoid. Mild disease defined by the mean number of new lesions that have appeared over the course of 3 days as determined by the investigator or referring physician, as follows: ≤ 1 bulla or \< 5 urticarial plaques.
• Concomitant skin conditions preventing physical evaluation of Bullous Pemphigoid.
• Active or recent history of clinically significant infection within 1 month of baseline.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• Immune Pharmaceuticals: collaborator

Study Sites (8)

Research Site

Iowa City, Iowa, United States

Location

Research Site

Buffalo, New York, United States

Location

Research Site

New York, New York, United States

Location

Research Site

Durham, North Carolina, United States

Location

Research Site

Cleveland, Ohio, 10900, United States

Location

Research Site

Salt Lake City, Utah, United States

Location

Research Site

Ramat Gan, 5262100, Israel

Location

Research Site

Tel Aviv, 64239, Israel

Location

MeSH Terms

Conditions

Pemphigoid, Bullous; Blister; Skin Diseases

Condition Hierarchy (Ancestors)

Skin Diseases, Vesiculobullous; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

Study conducted by Immune Pharmaceuticals Inc. and was acquired by Alexion Pharmaceuticals, Inc after study completion, database lock, and report generation.

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

855-752-2356

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 27, 2014
• First Posted: August 27, 2014
• Study Start: February 29, 2016
• Primary Completion: April 30, 2018
• Study Completion: April 30, 2018
• Last Updated: November 26, 2025
• Results First Posted: November 26, 2025

Record last verified: 2025-11

Locations

IL (2); US (6)