Pemphigoid, BullousPhase 2NCT02226146
Evaluation of Safety, Efficacy and Pharmacodynamic Effect of Bertilimumab in Patients With Bullous Pemphigoid
A Phase 2 study testing Bertilimumab in people with Pemphigoid, Bullous, run by Alexion Pharmaceuticals, Inc. at 8 sites in 2 countries.
Trial summary
This is an open-label, proof-of-concept, single group study in adult participants with newly diagnosed, moderate to extensive BP. Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Pemphigoid, Bullous
- What is being tested?
- Bertilimumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 8 sitesin 2 countries
- Study dates
- 29 Feb 2016to 30 Apr 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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
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- 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.
- Pregnant or breast-feeding, or planning to become pregnant during the study.
- Participation in a clinical trial of an investigational (unapproved) product within 4 weeks of baseline.
- Known hypersensitivity to bertilimumab or any of the drug excipients.
- Use of prednisone or other systemic steroids (excluding inhaled or ocular use of steroids) within 4 weeks prior to baseline. (Concomitant oral corticosteroids administered as part of the study protocol, from Day 0 onward, are allowed). Use of class 1 and 2 topical steroids (such as clobetasol propionate cream, reference Appendix D for further guidance) within 4 weeks prior to baseline. (Use of other topical steroids is allowed throughout the study at the discretion of the investigator).
- Treatment with immunosuppressants (e.g., azathioprine, methotrexate) within 4 weeks prior to baseline.
- Treatment with biologics (e.g., etanercept, adalimumab, ustekinumab, infliximab, intravenous Ig) within 4 months of baseline. Patients who have received rituximab within 1 year of baseline will be excluded from study participation.
- Treatment with macrolides or tetracyclines within 4 weeks prior to baseline.
- Received a vaccine or other immunostimulator within 4 weeks prior to baseline. Subject has current clinical, radiographic or laboratory evidence of active mycobacterium tuberculosis (TB) infection or prior evidence of active TB that, in the opinion of the investigator, has not been adequately treated or controlled and that represents a reactivation risk. If in the investigator's opinion the patient is at risk for latent TB, the patient should be evaluated for active/latent TB as applicable (e.g. PPD, QFT, and/or chest x-ray).
- 18. Evidence of an active disease of hepatitis B (HBsAg positive or HBcAb 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. 20. Any other condition, which in the opinion of the Investigator would place the patient at an unacceptable risk if participating in the study protocol.
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
United States6 sites
Research Site
Buffalo, New York
Research Site
Cleveland, Ohio, 10900
Research Site
Durham, North Carolina
Research Site
Iowa City, Iowa
Research Site
New York, New York
Research Site
Salt Lake City, Utah
About this study
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 Pemphigoid
This is an open-label, proof-of-concept, single group study in adult participants with newly diagnosed, moderate to extensive BP.
- Conditions
- Pemphigoid, Bullous
- Being tested
- Bertilimumab
- Keywords
- Pemphigoid Bullous, Bullous, Blisters, SKIN DISEASES, BP
- Medical subject headings
- Pemphigoid, Bullous, Blister, Skin Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BertilimumabBiological
| Study group | Role | What they receive |
|---|---|---|
| Bertilimumab | Experimental | Intravenous injection over 30 minutes of 10 mg/kg of Bertilimumab in physiological solution (PBS) Receives: Biological: Bertilimumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Anti-Drug Antibodies
Measured: 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'.
Measured: 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
BPDAI is a clinician completed tool that is used for independent disease severity assessment to measure disease extent in bullous pemphigoid. The total BPDAI activity score was calculated as the arithmetic sum of the 3 subcomponents - cutaneous blisters/erosions, cutaneous urticaria/erythema, and mucosal blisters/erosions. The BPDAI total activity gives an indication of disease activity, with score range from 0 (no disease activity) to 360 (severe disease activity). Higher scores indicated greater disease activity. Baseline was defined as the last measurement obtained before the treatment initiation.
Measured: Baseline, Day 84
Number of Participants Who Had Tapered to Prednisone Dose of ≤ 10 mg/Day
Measured: Baseline, Day 84
Percentage of Reduction From Baseline in BPDAI Pruritis (Visual Analogue Scale [VAS]) Total Score
The VAS sub-scores were defined as: 1. VAS Sub-Score I: How severe has your itching been over the last 24 hours. 2. VAS Sub-Score II: How severe has your itching been over the past week. 3. VAS Sub-Score III: How severe has your itching been over the past month. The Total Pruritus VAS score was defined as the sum over the three sub-scores. If at least one of the sub-scores is missing, the total is not defined. If value for the pruritus total score could not be determined than the value was left as blank for the calculation of the mean. Scores for the BPDAI-VAS ranged from 0 to 30, with higher scores indicating a worse condition.
Measured: Baseline, Day 84
Percentage of Reduction From Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) Total Score
ABQoL questionnaire assessed the impact of autoimmune bullous disease and their therapies on the daily life of participants. Scores range from 0 to 51 with a higher score representing a worse quality of life. If value for the ABQOL total score could not be determined than the value was left as blank for the calculation of the mean.
Measured: Baseline, Day 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Aug 2014 (estimated)
- Study start29 Feb 2016
- Primary completion30 Apr 2018
- Study completion30 Apr 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pemphigoid, BullousStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Immune Pharmaceuticals
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02226146
- Sponsor's protocol ID
- Immune/BRT/BP-01
- Status last verified
- Nov 2025
- First submitted
- 27 Jul 2014
- Last updated
- 26 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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 Pemphigoid
- ClinicalTrials.gov ID
- NCT02226146
- Acronym / sponsor ID
- Immune/BRT/BP-01
- Conditions
- Pemphigoid, Bullous
- Keywords
- Pemphigoid Bullous, Bullous, Blisters, SKIN DISEASES, BP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Immune Pharmaceuticals
- Study start
- 29 Feb 2016 (actual)
- Primary completion
- 30 Apr 2018 (actual)
- Study completion
- 30 Apr 2018 (actual)
- First posted
- 27 Aug 2014 (estimated)
- Results
- Posted 26 Nov 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label, proof-of-concept, single group study in adult participants with newly diagnosed, moderate to extensive BP.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bertilimumab | Experimental | Biological: BertilimumabIntravenous injection over 30 minutes of 10 mg/kg of Bertilimumab in physiological solution (PBS) |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Males or females, ≥ 60 years of age.
- Karnofsky performance status > 60%
- Newly diagnosed, Bullous Pemphigoid per standard diagnostic criteria:
- 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.
Key exclusion criteria (24 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bertilimumab | Biological | Bertilimumab |
Primary outcome
Number of Participants With Anti-Drug Antibodies
Time frame: Baseline up to 1 year
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline up to 1 year
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'.
4 secondary outcomes
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
Time frame: Baseline, Day 84
Number of Participants Who Had Tapered to Prednisone Dose of ≤ 10 mg/Day
Time frame: Baseline, Day 84
Percentage of Reduction From Baseline in BPDAI Pruritis (Visual Analogue Scale [VAS]) Total Score
Time frame: Baseline, Day 84
Percentage of Reduction From Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) Total Score
Time frame: Baseline, Day 84
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bertilimumab in people with Pemphigoid, Bullous, run by Alexion Pharmaceuticals, Inc. at 8 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Nov 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Anti-Drug Antibodies (time frame: Baseline up to 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Nov 2025.