A Phase 2 interventional study of Bertilimumab in Pemphigoid, Bullous, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 8 sites in 2 countries. Open to participants aged 60 Years and older. Per ClinicalTrials.gov, last updated 2025-11-26.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 2, Interventional, and Treatment
This is an open-label, proof-of-concept, single group study in adult participants with newly diagnosed, moderate to extensive BP.
64 studies on the registry are indexed under Pemphigoid, Bullous; 8 are open to participants now.
This study's enrollment of 11 is below the median of 30 across 39 interventional studies indexed under Pemphigoid, Bullous.
Browse Pemphigoid, Bullous studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
First submitted before Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Newly diagnosed, Bullous Pemphigoid per standard diagnostic criteria:
Exclusion criteria:
Clinically significant abnormal laboratory test results, unless regarded by the Investigator as related to BP, including but not limited to:
18. Evidence of an active disease of (HBsAg positive or HBcAb positive) or (HCV ab positive), CMV (IgM positive) or (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.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Intravenous injection over 30 minutes of 10 mg/kg of Bertilimumab in physiological solution (PBS)
Biological: Bertilimumab
Number of Participants With Anti-Drug Antibodies
Time frame: 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'.
Time frame: Baseline up to 1 year
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.
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
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.
Time frame: 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.
Time frame: Baseline, Day 84
| Milestone | Bertilimumab + Prednisone |
|---|---|
| Started | 11 |
| Received at least 1 dose of study drug | 9 |
| Completed | 8 |
| Not completed | 3 |
| Withdrew: Received rescue therapy | 1 |
| Withdrew: Withdrawal by subject | 2 |
| Participants | Bertilimumab + Prednisone |
|---|---|
| Number of Participants With Anti-Drug Antibodies | 0 |
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'.
| Participants | Bertilimumab + Prednisone |
|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 6 |
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.
| Participants | Bertilimumab + Prednisone |
|---|---|
| 50% Reduction | 6 |
| 70% Reduction | 5 |
| 90% Reduction | 4 |
| Participants | Bertilimumab + Prednisone |
|---|---|
| Number of Participants Who Had Tapered to Prednisone Dose of ≤ 10 mg/Day | 5 |
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.
| percentage of reduction in BPDAI VAS | Bertilimumab + Prednisone |
|---|---|
| Percentage of Reduction From Baseline in BPDAI Pruritis (Visual Analogue Scale [VAS]) Total Score | 8.6 (3 to 19) |
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.
| percentage of reduction in ABQOL score | Bertilimumab + Prednisone |
|---|---|
| Percentage of Reduction From Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) Total Score | 11 (1 to 39) |
Collected over Baseline up to 1 year. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Bertilimumab + Prednisone | 0/9 (0%) | 1/9 (11.1%) | 6/9 (66.7%) |
| Event | Bertilimumab + Prednisone |
|---|---|
| Peripheral vascular disorderVascular disorders | 1/9 |
| Event | Bertilimumab + Prednisone |
|---|---|
| Blurred visionEye disorders | 1/9 |
| Traumatic laceration right big toeInjury, poisoning and procedural complications | 1/9 |
| Upper respiratory tract infectionInfections and infestations | 1/9 |
| Night sweatingReproductive system and breast disorders | 1/9 |
| Edema (bilateral)General disorders | 1/9 |
| Muscle crampsMusculoskeletal and connective tissue disorders | 1/9 |
| FallInjury, poisoning and procedural complications | 1/9 |
| Gamma-Glutamyl Transferase increasedHepatobiliary disorders | 1/9 |
| Cyst at base of tongueNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/9 |
The Safety analysis population consisted of all participants enrolled in the trial who received at least one infusion of bertilimumab 10 mg/kg.
| Age, Categorical(Participants) | Bertilimumab + Prednisone |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 1 |
| >=65 years | 8 |
| Sex: Female, Male(Participants) | Bertilimumab + Prednisone |
|---|---|
| Female | 4 |
| Male | 5 |
| Race (NIH/OMB)(Participants) | Bertilimumab + Prednisone |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 8 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is completed, as verified in Nov 2025. You cannot join it, but the record below documents what was studied.
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Alexion Pharmaceuticals, Inc. →