Trial Outcomes & Findings for Evaluation of Safety, Efficacy and Pharmacodynamic Effect of Bertilimumab in Patients With Bullous Pemphigoid (NCT NCT02226146)

Results Overview

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'.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

11 participants

Primary outcome timeframe

Baseline up to 1 year

Results posted on

2025-11-26

Participant Flow

Participant milestones

Participant milestones
Measure	Bertilimumab + Prednisone Participants with moderate to extensive bullous pemphigoid (BP) received bertilimumab 10 milligrams/kilogram (mg/kg) intravenous (IV) infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Overall Study STARTED	11
Overall Study Received at Least 1 Dose of Study Drug	9
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Bertilimumab + Prednisone Participants with moderate to extensive bullous pemphigoid (BP) received bertilimumab 10 milligrams/kilogram (mg/kg) intravenous (IV) infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Overall Study Received rescue therapy	1
Overall Study Withdrawal by Subject	2

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bertilimumab + Prednisone n=9 Participants Participants with moderate to extensive BP received bertilimumab 10 mg/kg IV infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Age, Categorical >=65 years	8 Participants n=492 Participants
Sex: Female, Male Female	4 Participants n=492 Participants
Sex: Female, Male Male	5 Participants n=492 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=492 Participants
Race (NIH/OMB) Asian	0 Participants n=492 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=492 Participants
Race (NIH/OMB) Black or African American	1 Participants n=492 Participants
Race (NIH/OMB) White	8 Participants n=492 Participants
Race (NIH/OMB) More than one race	0 Participants n=492 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=492 Participants
Age, Categorical <=18 years	0 Participants n=492 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=492 Participants

PRIMARY outcome

Timeframe: Baseline up to 1 year

Population: The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.

Outcome measures

Outcome measures
Measure	Bertilimumab + Prednisone n=9 Participants Participants with moderate to extensive BP received bertilimumab 10 mg/kg IV infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Number of Participants With Anti-Drug Antibodies	0 Participants

PRIMARY outcome

Timeframe: Baseline up to 1 year

Population: The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.

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'.

Outcome measures

Outcome measures
Measure	Bertilimumab + Prednisone n=9 Participants Participants with moderate to extensive BP received bertilimumab 10 mg/kg IV infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	6 Participants

SECONDARY outcome

Timeframe: Baseline, Day 84

Population: The Efficacy population included all participants in the safety population who had the visit score on the BPDAI at baseline and at least one post baseline BPDAI score. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure.

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.

Outcome measures

Outcome measures
Measure	Bertilimumab + Prednisone n=7 Participants Participants with moderate to extensive BP received bertilimumab 10 mg/kg IV infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
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 50% Reduction	6 Participants
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 70% Reduction	5 Participants
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 90% Reduction	4 Participants

SECONDARY outcome

Timeframe: Baseline, Day 84

Population: The Efficacy population included all participants in the safety population who had the visit score on the BPDAI at baseline and at least one post baseline BPDAI score.

Outcome measures

Outcome measures
Measure	Bertilimumab + Prednisone n=9 Participants Participants with moderate to extensive BP received bertilimumab 10 mg/kg IV infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Number of Participants Who Had Tapered to Prednisone Dose of ≤ 10 mg/Day	5 Participants

SECONDARY outcome

Timeframe: Baseline, Day 84

Population: The Efficacy population included all participants in the safety population who had the visit score on the BPDAI at baseline and at least one post baseline BPDAI 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.

Outcome measures

Outcome measures
Measure	Bertilimumab + Prednisone n=9 Participants Participants with moderate to extensive BP received bertilimumab 10 mg/kg IV infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Percentage of Reduction From Baseline in BPDAI Pruritis (Visual Analogue Scale [VAS]) Total Score	8.6 percentage of reduction in BPDAI VAS Interval 3.0 to 19.0

SECONDARY outcome

Timeframe: Baseline, Day 84

Population: The Efficacy population included all participants in the safety population who had the visit score on the BPDAI at baseline and at least one post baseline BPDAI 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.

Outcome measures

Outcome measures
Measure	Bertilimumab + Prednisone n=9 Participants Participants with moderate to extensive BP received bertilimumab 10 mg/kg IV infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Percentage of Reduction From Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) Total Score	11 percentage of reduction in ABQOL score Interval 1.0 to 39.0

Adverse Events

Bertilimumab + Prednisone

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Bertilimumab + Prednisone n=9 participants at risk Participants with moderate to extensive BP received bertilimumab 10 mg/kg IV infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Vascular disorders Peripheral vascular disorder	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.

Other adverse events

Other adverse events
Measure	Bertilimumab + Prednisone n=9 participants at risk Participants with moderate to extensive BP received bertilimumab 10 mg/kg IV infusion biweekly and prednisone at a maximum dose of 30 mg orally per the investigator's discretion.
Infections and infestations Upper respiratory tract infection	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.
Reproductive system and breast disorders Night sweating	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.
General disorders Edema (bilateral)	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.
Musculoskeletal and connective tissue disorders Muscle cramps	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.
Injury, poisoning and procedural complications Fall	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.
Hepatobiliary disorders Gamma-Glutamyl Transferase increased	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Cyst at base of tongue	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.
Eye disorders Blurred vision	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.
Injury, poisoning and procedural complications Traumatic laceration right big toe	11.1% 1/9 • Baseline up to 1 year The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.

Additional Information

Alexion Pharmaceuticals, Inc.

Phone: 855-752-2356

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place