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Trial record 2 of 2 for:    "Bullous Pemphigoid" | "Antineoplastic Agents, Hormonal"

Efficacy and Safety of Omalizumab in Bullous Pemphigoid

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ClinicalTrials.gov Identifier: NCT00472030
Recruitment Status : Completed
First Posted : May 10, 2007
Results First Posted : October 16, 2012
Last Update Posted : October 16, 2012
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Janet Fairley, University of Iowa

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Bullous Pemphigoid
Interventions Drug: Omalizumab
Drug: prednisone
Enrollment 2
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Hide Arm/Group Description Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 14 weeks. The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 milligrams per kilogram per day (mg/kg/day).
Period Title: Overall Study
Started 2 0
Completed 1 0
Not Completed 1 0
Reason Not Completed
Physician Decision             1             0
Arm/Group Title Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm Total
Hide Arm/Group Description Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 16 weeks. The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day. Total of all reporting groups
Overall Number of Baseline Participants 2 0 2
Hide Baseline Analysis Population Description
[Not Specified]
Age Categorical  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 2 participants 0 participants 2 participants
<=18 years 0 0
Between 18 and 65 years 0 0
>=65 years 2 2
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 2 participants 0 participants 2 participants
78  (0) 78  (0)
Gender  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 2 participants 0 participants 2 participants
Female 2 2
Male 0 0
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 2 participants 0 participants 2 participants
2 2
1.Primary Outcome
Title Median Time From First Dose of Omalizumab Treatment to Cessation of New Blisters.
Hide Description The study subject underwent physical examination and was assessed for cessation of new blister formation via physical examination and photography.
Time Frame Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Omalizumab Treatment Arm
Hide Arm/Group Description:
Omalizumab 150-375 milligrams (mg) will be administered subcutaneously at Day 1, Week 2,4,6,8,10,12, and 14.
Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: weeks
6
2.Primary Outcome
Title Percent Decrease in the Total Body Surface Area Affected By Active Bullous Pemphigoid Skin Disease From Day 0 to Week 24.
Hide Description 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).
Time Frame Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Omalizumab Treatment Arm
Hide Arm/Group Description:
Research subjects enrolled to the Omalizumab treatment arm will be treated with Omalizumab on Day 1 and at Week 2,4,6,8,10,12,& 14.
Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: percentage of active skin disease
22.5
3.Primary Outcome
Title Median Increase in Prednisone Dosage Measured at Week 4, 8 and 24 in Patients Treated With Omalizumab and in Patients Receiving Standard Therapy.
Hide Description 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.
Time Frame Week 4, Week 8 and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
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
Arm/Group Title Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Hide Arm/Group Description:
Omalizumab 150-375 milligrams (mg) will be administered subcutaneously at Day 1, Week 2,4,6,8,10,12, and 14.
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Decrease in Anti-BP180 IgG (Immunoglobulin G Anti-Bullous Pemphigoid 180 Antibody) Following Treatment With Omalizumab.
Hide Description Anti-BP180 IgG levels were completed using an Elisa assay. Anti-BP180 IgG levels were obtained prior to baseline and at week 16
Time Frame Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Hide Arm/Group Description:
Omalizumab 150-375 milligrams (mg) will be administered subcutaneously at Day 1, Week 2,4,6,8,10,12, and 14.
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
Overall Number of Participants Analyzed 1 0
Measure Type: Number
Unit of Measure: units per milliliter
92.06
5.Secondary Outcome
Title Decrease in Eosinophil Levels Following Treatment With Omalizumab.
Hide Description 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
Time Frame Baseline, 24 weeks.
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Hide Arm/Group Description:
Patients with bullous pemphigoid will be treated with Omalizumab on Day 1, Week 2,4,6,8,10,12 and 14.
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 milligrams per kilogram per day (mg/kg/day).
Overall Number of Participants Analyzed 1 0
Measure Type: Number
Unit of Measure: cells/microliter
2.16
6.Secondary Outcome
Title Decrease in Anti-BP230 Antibody IgG (Anti-bullous Pemphigoid 230 Antibody Immunoglobulin G) At Baseline and Week 16
Hide Description [Not Specified]
Time Frame Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Hide Arm/Group Description:
Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 14 weeks.
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 milligrams per kilogram per day (mg/kg/day).
Overall Number of Participants Analyzed 1 0
Measure Type: Number
Unit of Measure: units per milliliter
62.6
7.Secondary Outcome
Title Change in Histamine Release Assay Following Treatment With Omalizumab.
Hide Description The histamine release assay measures the release of histamine which occurs upon stimulation of basophilic granulocytes depending upon their sensitivity to an allergen.
Time Frame Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
We were unable to complete this assay in our research subjects due to technical difficulties.
Arm/Group Title Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Hide Arm/Group Description:
Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 14 weeks.
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 milligrams per kilogram per day (mg/kg/day).
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Adverse event data were collected over a 24 week time period.
Adverse Event Reporting Description Subjects were underwent physical examination and/or had labwork taken at Baseline/Screening, Week 1,2,4,8,12,16, 20 & 24. Subjects were observed for signs of allergic reaction and vital signs were monitored for two hours after each injection of Omalizumab. Subjects were screened for adverse events throughout the 24 week period.
 
Arm/Group Title Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Hide Arm/Group Description Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 16 weeks. The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
All-Cause Mortality
Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/2 (50.00%)      0/0    
Cardiac disorders     
Hospitalization for Congestive Heart Failure Exacerbation  [1]  1/2 (50.00%)  1 0/0  0
Skin and subcutaneous tissue disorders     
Cellulitis  [2]  1/2 (50.00%)  1 0/0  0
Indicates events were collected by systematic assessment
[1]
Subject with known history/treatment of Congestive Heart Failure and chronic obstructive pulmonary disease. ON 8/25/2008, subject experienced shortness of breath with hospitalization, deemed not related to study drug.
[2]
Subject hospitalized for cellulitis at an outside hospital. Subject prescribed antibiotics for skin infection but did not take. Subsequent followup visit revealed Pemphigoid in the site Serious adverse event determined not to be related to study drug
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Omalizumab Treatment Arm Prednisone Standard Therapy Treatment Arm
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/2 (50.00%)      0/0    
General disorders     
Nasal Stuffiness  [1]  1/1 (100.00%)  1 0/0  0
Cough associated Nasal Drainage  [2]  1/1 (100.00%)  1 0/0  0
Skin and subcutaneous tissue disorders     
Ankle Edema  [3]  1/1 (100.00%)  1 0/0  0
Pruritis  [4]  1/1 (100.00%)  1 0/0  0
Staph Aureus Infection  [5]  1/1 (100.00%)  1 0/0  0
Staphylococcus infection  [6]  1/1 (100.00%)  1 0/0  0
Indicates events were collected by systematic assessment
[1]
Subject was diagnosed with nasal stuffiness which was rated as mild in severity, was rated unlikely related to the study drug, was not treated and resolved without sequelae.
[2]
Subject experienced a cough associated with nasal drainage which was rated as mild in severity, not treated, resolved without sequelae and rated as unlikely to be related to the study drug use.
[3]
Subject presented with ankle edema at baseline. This was rated as mild, not related to study drug, no action was taken or required and this resolved without sequelae.
[4]
Subject experienced pruritis at baseline. This was rated as moderately severe, unrelated to the study drug, was treated with concomitant medications and was continuing at the end of followup.
[5]
Subject was diagnosed with staph aureus infection which was cultured from a superficial skin erosion on the back. This was mild, unrelated to study drug, resolved without sequelae and was treated with concomitant medication.
[6]
Subject experienced a staphylococcus infection on the skin of the right toe. This was rated as mild in severity, resolved without sequelae, was rated unlikely to be related to study drug and required the use of concomitant medication.
Limitations of this trial include low enrollment of research subjects and the need to withdraw a subject from the trial.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Janet Fairley, M.D.
Organization: University of Iowa
Phone: 319-384-8560
EMail: janet-fairley@uiowa.edu
Layout table for additonal information
Responsible Party: Janet Fairley, University of Iowa
ClinicalTrials.gov Identifier: NCT00472030     History of Changes
Other Study ID Numbers: 100569
First Submitted: May 8, 2007
First Posted: May 10, 2007
Results First Submitted: June 26, 2012
Results First Posted: October 16, 2012
Last Update Posted: October 16, 2012