Bullous PemphigoidPhase 4NCT00472030

Efficacy and Safety of Omalizumab in Bullous Pemphigoid

A Phase 4 study testing Omalizumab and prednisone in people with Bullous Pemphigoid, run by University of Iowa at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

The primary objective is to test the safety and efficacy of Xolair in the treatment of the autoimmune blistering disease, bullous pemphigoid (BP). This is a pilot, open label case-control study. Patients treated with Xolair will be compared to patients receiving standard treatment with prednisone. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Bullous Pemphigoid
What is being tested?: Omalizumab and prednisone
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 2 participants
Where: 1 siteUnited States
Study dates: Aug 2007to Dec 2010
Phase: Phase 4
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 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients must have the clinical and histological findings consistent with bullous pemphigoid. Clinically this is defined as urticarial plaques and/or vesicles and bullae. Histologically patients must show characteristic eosinophilic spongiosis and/or subepidermal separation of the skin consistent with BP.
Patients must have either a positive direct (IgG and/or C3 at the BMZ) or indirect (IgG on the roof of salt-split skin) immunofluorescence microscopy features of pemphigoid.
Patients of both sexes, all races and ethnic backgrounds that are 18 years of age or older will be eligible to participate in this study.
Patients much have more than 5% total body surface involved, since patients with less extensive disease are often treated with topical measures only.

Who cannot join

Women of childbearing potential not using the contraception method(s) specified during this study. Women of childbearing potential must use proven birth control methods (such as - abstinence, birth control pills, intrauterine device, barrier method combined with gel or foam with spermicide, tubal ligation, or a partner who has had a vasectomy).
Women who are pregnant or breastfeeding.
Patients under the age of 18.
Patients unable to give informed consent.
Known sensitivity to study drug(s) or class of study drug(s).
Patients with severe medical condition(s) that in the view of the investigator prohibits participation in the study.

Show all 11Show fewer

Any cancer other than non-melanoma skin cancer in the past 5 years.
All non-melanoma skin cancers must have been adequately treated at entrance to the study.
Use of any other investigational agent in the last 30 days.
Treatment with prednisone in the past 2 weeks.
Weight or serum IgE levels that place the patient outside standard dosing guidelines for Xolair.

Trial locations and contacts

1 site in 1 country.

United States1 site

University of Iowa, Department of Dermatology
Iowa City, Iowa, 52242

Other trials in United States

About this study

Official title: An Open Label Case Series on the Effects of Xolair (Omalizumab) in Bullous Pemphigoid

The primary objective is to test the safety and efficacy of Xolair in the treatment of the autoimmune blistering disease, bullous pemphigoid (BP).

This is a pilot, open label case-control study. Patients treated with Xolair will be compared to patients receiving standard treatment with prednisone.

The enrollment period for the study is 24 weeks: 16 weeks active treatment and 8 additional weeks of observation.

Read the detailed description

Objectives: The primary objective is to test the safety and efficacy of Omalizumab (Xolair) in the treatment of the autoimmune blistering disease, bullous pemphigoid (BP).

Study Rationale: The current treatment for bullous pemphigoid is non-specific immunosuppression, causing great morbidity in these patients. Recently, pathogenic Immunoglogulin Class E autoantibodies have been identified in these patients. Development of a more targeted approach to treatment may reduce morbidity.

Methodology: This is a pilot, open-label case-control study. Patients treated with Omalizumab (Xolair) will be compared to patients receiving standard treatment with prednisone.

Number of centers and patients: This is a single center study that will enroll 12 patients.

Population: Bullous pemphigoid patients, meeting clinical, histological and immunologic criteria for the disease will be enrolled. Pregnant women, children less than 18 years of age, and patients unable to give consent will be excluded from this preliminary study.

Investigational drug: Xolair® (Omalizumab)

Study duration: 24 weeks: 16 weeks of active treatment, 8 additional weeks of observation

Evaluation criteria: Primary: 1. Time to cessation of new blister formation. 2. Percent body surface area of skin involved before and after treatment 3. Total and average daily dose of prednisone required in 30, 60 and 180 days after starting Xolair. Secondary: 1. Number of circulating eosinophils 2. Measurement of circulating anti-BMZ (basement membrane zone) autoantibodies 3. Histamine release assay.

Conditions: Bullous Pemphigoid
Being tested: Omalizumab, prednisone
Keywords: bullous pemphigoid, omalizumab, xolair, IgE, autoimmunity, skin disease
Medical subject headings: Pemphigoid, Bullous, Autoimmune Diseases, Skin Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

OmalizumabDrug
Also called Xolair
Patients will be treated with 150-375 milligrams of Omalizumab (Xolair), based on their baseline weight and serum Immunoglobulin E levels. Omalizumab will be administered subcutaneously on Day 1, and on Week 2, 4, 6, 8, 10, 12 and 14 treatment.
prednisoneDrug
Prednisone, to a maximum dose of 0.5 mg/kg/day.

Study groups in NCT00472030
Study group	Role	What they receive
Omalizumab	Experimental	Patients will be treated with 150-375 milligrams of Omalizumab (Xolair), based on their baseline weight and serum Immunoglobulin E levels. Omalizumab will be administered subcutaneously on Day 1, and on Week 2, 4, 6, 8, 10, 12 and 14 treatment. Receives: Drug: Omalizumab
Prednisone	Active comparator	The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day. Receives: Drug: prednisone

What is being measured

The primary outcome is the main question the study is designed to answer.

Median Time From First Dose of Omalizumab Treatment to Cessation of New Blisters.
The study subject underwent physical examination and was assessed for cessation of new blister formation via physical examination and photography.
Measured: Up to 24 weeks
Percent Decrease in the Total Body Surface Area Affected By Active Bullous Pemphigoid Skin Disease From Day 0 to Week 24.
Measurement of total body surface area affected by bullous pemphigoid active skin disease(active erosions, blisters, and/or lesions) was measured at Day 0 (prior to treatment with Omalizumab) and at 24 weeks (24 weeks is end of study).
Measured: Up to 24 weeks
Median Increase in Prednisone Dosage Measured at Week 4, 8 and 24 in Patients Treated With Omalizumab and in Patients Receiving Standard Therapy.
The total dose of prednisone required to control the bullous pemphigoid at week 4, 8 and 24 weeks was to be calculated in both arms of this study.
Measured: Week 4, Week 8 and Week 24

Secondary outcomes (4)

Decrease in Anti-BP180 IgG (Immunoglobulin G Anti-Bullous Pemphigoid 180 Antibody) Following Treatment With Omalizumab.
Anti-BP180 IgG levels were completed using an Elisa assay. Anti-BP180 IgG levels were obtained prior to baseline and at week 16
Measured: Up to 24 weeks
Decrease in Eosinophil Levels Following Treatment With Omalizumab.
The subject's eosinophil count measured at baseline was compared to the eosinophil count at week 8. A normal eosinophil count at the University of Iowa Hospital lab is 0-0.4 cells per microliter
Measured: Baseline, 24 weeks.
Decrease in Anti-BP230 Antibody IgG (Anti-bullous Pemphigoid 230 Antibody Immunoglobulin G) At Baseline and Week 16
Measured: Up to 24 weeks
Change in Histamine Release Assay Following Treatment With Omalizumab.
The histamine release assay measures the release of histamine which occurs upon stimulation of basophilic granulocytes depending upon their sensitivity to an allergen.
Measured: Up to 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Aug 2007Dec 2010

First posted10 May 2007 (estimated)
Study startAug 2007
Primary completionDec 2010
Study completionDec 2010

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.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Bullous PemphigoidStudies of the same condition, open and under way.
More trials run by University of IowaThe 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 4
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 2 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00472030
Sponsor's protocol ID: 100569
Status last verified: Sep 2012
First submitted: 8 May 2007
Last updated: 16 Oct 2012
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Dimson OG, Giudice GJ, Fu CL, Van den Bergh F, Warren SJ, Janson MM, Fairley JA. Identification of a potential effector function for IgE autoantibodies in the organ-specific autoimmune disease bullous pemphigoid. J Invest Dermatol. 2003 May;120(5):784-8. doi: 10.1046/j.1523-1747.2003.12146.x.
PubMed 12713582
Fairley JA, Fu CL, Giudice GJ. Mapping the binding sites of anti-BP180 immunoglobulin E autoantibodies in bullous pemphigoid. J Invest Dermatol. 2005 Sep;125(3):467-72. doi: 10.1111/j.0022-202X.2005.23853.x.
PubMed 16117787
Holgate ST, Djukanovic R, Casale T, Bousquet J. Anti-immunoglobulin E treatment with omalizumab in allergic diseases: an update on anti-inflammatory activity and clinical efficacy. Clin Exp Allergy. 2005 Apr;35(4):408-16. doi: 10.1111/j.1365-2222.2005.02191.x.
PubMed 15836747

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruitingPhase 1
Subconjunctival Humira for Boston Keratoprosthesis
Penetrating Keratoplasty, Multiple Graft Failure
RecruitingPhase 1
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP)
Enrolling by invitationPhase 1
Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP)
Active, not recruitingObservational study
Association Between Disease Severity and Clinical, Histopathological, and Eosinophilic Markers in Patients With Bullous Pemphigoid (BP-EPIC)
Bullous Pemphigoid (BP)
Active, not recruitingPhase 3
Randomized Clinical Trial Comparing the Safety and Efficacy of Rituximab Versus Oral Cyclophosphamide in Severe Forms of Mucous Membrane Pemphigoid
Severe Forms of Mucous Membrane Pemphigoid

All Bullous Pemphigoid trials

Study at a glance

From the registry record, last updated by the sponsor on 16 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open Label Case Series on the Effects of Xolair (Omalizumab) in Bullous Pemphigoid
ClinicalTrials.gov ID: NCT00472030
Acronym / sponsor ID: 100569
Conditions: Bullous Pemphigoid
Keywords: bullous pemphigoid, omalizumab, xolair, IgE, autoimmunity, skin disease
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 2 participants (actual)
Lead sponsor: University of IowaUniversity, hospital or other
Collaborators: Genentech, Inc.
Study start: Aug 2007
Primary completion: Dec 2010 (actual)
Study completion: Dec 2010 (actual)
First posted: 10 May 2007 (estimated)
Results: Posted 16 Oct 2012

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The primary objective is to test the safety and efficacy of Xolair in the treatment of the autoimmune blistering disease, bullous pemphigoid (BP).

This is a pilot, open label case-control study. Patients treated with Xolair will be compared to patients receiving standard treatment with prednisone.

The enrollment period for the study is 24 weeks: 16 weeks active treatment and 8 additional weeks of observation.

Detailed description

Objectives: The primary objective is to test the safety and efficacy of Omalizumab (Xolair) in the treatment of the autoimmune blistering disease, bullous pemphigoid (BP).

Methodology: This is a pilot, open-label case-control study. Patients treated with Omalizumab (Xolair) will be compared to patients receiving standard treatment with prednisone.

Number of centers and patients: This is a single center study that will enroll 12 patients.

Investigational drug: Xolair® (Omalizumab)

Study duration: 24 weeks: 16 weeks of active treatment, 8 additional weeks of observation

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Omalizumab	Experimental	Drug: OmalizumabPatients will be treated with 150-375 milligrams of Omalizumab (Xolair), based on their baseline weight and serum Immunoglobulin E levels. Omalizumab will be administered subcutaneously on Day 1, and on Week 2, 4, 6, 8, 10, 12 and 14 treatment.
Prednisone	Active comparator	Drug: prednisoneThe control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Patients must have the clinical and histological findings consistent with bullous pemphigoid. Clinically this is defined as urticarial plaques and/or vesicles and bullae. Histologically patients must show characteristic eosinophilic spongiosis and/or subepidermal separation of the skin consistent with BP.
Patients must have either a positive direct (IgG and/or C3 at the BMZ) or indirect (IgG on the roof of salt-split skin) immunofluorescence microscopy features of pemphigoid.
Patients of both sexes, all races and ethnic backgrounds that are 18 years of age or older will be eligible to participate in this study.
Patients much have more than 5% total body surface involved, since patients with less extensive disease are often treated with topical measures only.

Key exclusion criteria (11 listed)

Women of childbearing potential not using the contraception method(s) specified during this study. Women of childbearing potential must use proven birth control methods (such as - abstinence, birth control pills, intrauterine device, barrier method combined with gel or foam with spermicide, tubal ligation, or a partner who has had a vasectomy).
Women who are pregnant or breastfeeding.
Patients under the age of 18.
Patients unable to give informed consent.
Known sensitivity to study drug(s) or class of study drug(s).

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
OmalizumabAlso: XolairPatients will be treated with 150-375 milligrams of Omalizumab (Xolair), based on their baseline weight and serum Immunoglobulin E levels. Omalizumab will be administered subcutaneously on Day 1, and on Week 2, 4, 6, 8, 10, 12 and 14 treatment.	Drug	Omalizumab
prednisonePrednisone, to a maximum dose of 0.5 mg/kg/day.	Drug	Prednisone

Primary outcome

Median Time From First Dose of Omalizumab Treatment to Cessation of New Blisters.
Time frame: Up to 24 weeks
The study subject underwent physical examination and was assessed for cessation of new blister formation via physical examination and photography.
Percent Decrease in the Total Body Surface Area Affected By Active Bullous Pemphigoid Skin Disease From Day 0 to Week 24.
Time frame: Up to 24 weeks
Measurement of total body surface area affected by bullous pemphigoid active skin disease(active erosions, blisters, and/or lesions) was measured at Day 0 (prior to treatment with Omalizumab) and at 24 weeks (24 weeks is end of study).
Median Increase in Prednisone Dosage Measured at Week 4, 8 and 24 in Patients Treated With Omalizumab and in Patients Receiving Standard Therapy.
Time frame: Week 4, Week 8 and Week 24
The total dose of prednisone required to control the bullous pemphigoid at week 4, 8 and 24 weeks was to be calculated in both arms of this study.

4 secondary outcomes

Decrease in Anti-BP180 IgG (Immunoglobulin G Anti-Bullous Pemphigoid 180 Antibody) Following Treatment With Omalizumab.
Time frame: Up to 24 weeks
Decrease in Eosinophil Levels Following Treatment With Omalizumab.
Time frame: Baseline, 24 weeks.
Decrease in Anti-BP230 Antibody IgG (Anti-bullous Pemphigoid 230 Antibody Immunoglobulin G) At Baseline and Week 16
Time frame: Up to 24 weeks
Change in Histamine Release Assay Following Treatment With Omalizumab.
Time frame: Up to 24 weeks

Results and safety

Results posted to the registry on 16 Oct 2012, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Omalizumab Treatment Arm	2	1
Prednisone Standard Therapy Treatment Arm	0	0

Primary outcome: Median Time From First Dose of Omalizumab Treatment to Cessation of New Blisters.

Time frame: Up to 24 weeks. Number. Unit: weeks

Measure	Omalizumab Treatment Arm n = 1
Value	6

Population analysed: The population analyzed included one subject who received Omalizumab treatment on Day 1, Week 2,4,6,8,10,12 and 14 and completed assessments through week 24. A second subject enrolled in the study and received one treatment with Omalizumab and was terminated from the study per investigator at Week 4.

Primary outcome: Percent Decrease in the Total Body Surface Area Affected By Active Bullous Pemphigoid Skin Disease From Day 0 to Week 24.

Time frame: Up to 24 weeks. Number. Unit: percentage of active skin disease

Measure	Omalizumab Treatment Arm n = 1
Value	22.5

Primary outcome: Median Increase in Prednisone Dosage Measured at Week 4, 8 and 24 in Patients Treated With Omalizumab and in Patients Receiving Standard Therapy.

Time frame: Week 4, Week 8 and Week 24

Population analysed: Neither subject required treatment with Prednisone. Since we did not enroll any subject in the Prednisone Standard Therapy Treatment Arm we do not have any measurements for this outcome

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse event data were collected over a 24 week time period.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Omalizumab Treatment Arm	1 of 2 (50%)	1 of 2 (50%)	Not reported
Prednisone Standard Therapy Treatment Arm	0 of 0	0 of 0	Not reported

Limitations and caveats (from the sponsor): Limitations of this trial include low enrollment of research subjects and the need to withdraw a subject from the trial.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2012.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 12713582Dimson OG, Giudice GJ, Fu CL, Van den Bergh F, Warren SJ, Janson MM, Fairley JA. Identification of a potential effector function for IgE autoantibodies in the organ-specific autoimmune disease bullous pemphigoid. J Invest Dermatol. 2003 May;120(5):784-8. doi: 10.1046/j.1523-1747.2003.12146.x.
Background: PubMed 16117787Fairley JA, Fu CL, Giudice GJ. Mapping the binding sites of anti-BP180 immunoglobulin E autoantibodies in bullous pemphigoid. J Invest Dermatol. 2005 Sep;125(3):467-72. doi: 10.1111/j.0022-202X.2005.23853.x.
Background: PubMed 15836747Holgate ST, Djukanovic R, Casale T, Bousquet J. Anti-immunoglobulin E treatment with omalizumab in allergic diseases: an update on anti-inflammatory activity and clinical efficacy. Clin Exp Allergy. 2005 Apr;35(4):408-16. doi: 10.1111/j.1365-2222.2005.02191.x.

Related trials for Bullous Pemphigoid

Not yet recruitingPhase 1
Subconjunctival Humira for Boston Keratoprosthesis
Penetrating Keratoplasty, Multiple Graft Failure
RecruitingPhase 1
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP)
Enrolling by invitationPhase 1
Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP)
Active, not recruitingObservational study
Association Between Disease Severity and Clinical, Histopathological, and Eosinophilic Markers in Patients With Bullous Pemphigoid (BP-EPIC)
Bullous Pemphigoid (BP)
Active, not recruitingPhase 3
Randomized Clinical Trial Comparing the Safety and Efficacy of Rituximab Versus Oral Cyclophosphamide in Severe Forms of Mucous Membrane Pemphigoid
Severe Forms of Mucous Membrane Pemphigoid

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.

Janet A Fairley, MD principal investigator

University of Iowa

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Omalizumab and prednisone in people with Bullous Pemphigoid, run by University of Iowa at 1 site in United States.

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 4 inclusion and 11 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 16 Oct 2012. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

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 16 Oct 2012). 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?

Median Time From First Dose of Omalizumab Treatment to Cessation of New Blisters. (time frame: Up to 24 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00472030, which also lists 3 linked publications.

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 16 Oct 2012.