Trial Outcomes & Findings for Efficacy and Safety of Omalizumab in Bullous Pemphigoid (NCT NCT00472030)
NCT ID: NCT00472030
Last Updated: 2012-10-16
Results Overview
The study subject underwent physical examination and was assessed for cessation of new blister formation via physical examination and photography.
COMPLETED
PHASE4
2 participants
Up to 24 weeks
2012-10-16
Participant Flow
Participant milestones
| Measure |
Omalizumab Treatment Arm
Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 14 weeks.
|
Prednisone Standard Therapy Treatment Arm
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 Study
STARTED
|
2
|
0
|
|
Overall Study
COMPLETED
|
1
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Omalizumab Treatment Arm
Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 14 weeks.
|
Prednisone Standard Therapy Treatment Arm
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 Study
Physician Decision
|
1
|
0
|
Baseline Characteristics
Efficacy and Safety of Omalizumab in Bullous Pemphigoid
Baseline characteristics by cohort
| Measure |
Omalizumab Treatment Arm
n=2 Participants
Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 16 weeks.
|
Prednisone Standard Therapy Treatment Arm
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
|
Total
n=2 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Categorical
<=18 years
|
0 participants
n=5 Participants
|
—
|
0 participants
n=5 Participants
|
|
Age Categorical
Between 18 and 65 years
|
0 participants
n=5 Participants
|
—
|
0 participants
n=5 Participants
|
|
Age Categorical
>=65 years
|
2 participants
n=5 Participants
|
—
|
2 participants
n=5 Participants
|
|
Age Continuous
|
78 years
STANDARD_DEVIATION 0 • n=5 Participants
|
—
|
78 years
STANDARD_DEVIATION 0 • n=5 Participants
|
|
Gender
Female
|
2 participants
n=5 Participants
|
—
|
2 participants
n=5 Participants
|
|
Gender
Male
|
0 participants
n=5 Participants
|
—
|
0 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=5 Participants
|
—
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 24 weeksPopulation: 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.
The study subject underwent physical examination and was assessed for cessation of new blister formation via physical examination and photography.
Outcome measures
| Measure |
Omalizumab Treatment Arm
n=1 Participants
Omalizumab 150-375 milligrams (mg) will be administered subcutaneously at Day 1, Week 2,4,6,8,10,12, and 14.
|
Prednisone Standard Therapy Treatment Arm
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
|
|---|---|---|
|
Median Time From First Dose of Omalizumab Treatment to Cessation of New Blisters.
|
6 weeks
|
—
|
PRIMARY outcome
Timeframe: Up to 24 weeksPopulation: 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.
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).
Outcome measures
| Measure |
Omalizumab Treatment Arm
n=1 Participants
Omalizumab 150-375 milligrams (mg) will be administered subcutaneously at Day 1, Week 2,4,6,8,10,12, and 14.
|
Prednisone Standard Therapy Treatment Arm
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
|
|---|---|---|
|
Percent Decrease in the Total Body Surface Area Affected By Active Bullous Pemphigoid Skin Disease From Day 0 to Week 24.
|
22.5 percentage of active skin disease
|
—
|
PRIMARY outcome
Timeframe: Week 4, Week 8 and Week 24Population: 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
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 24 weeksPopulation: 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.
Anti-BP180 IgG levels were completed using an Elisa assay. Anti-BP180 IgG levels were obtained prior to baseline and at week 16
Outcome measures
| Measure |
Omalizumab Treatment Arm
n=1 Participants
Omalizumab 150-375 milligrams (mg) will be administered subcutaneously at Day 1, Week 2,4,6,8,10,12, and 14.
|
Prednisone Standard Therapy Treatment Arm
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
|
|---|---|---|
|
Decrease in Anti-BP180 IgG (Immunoglobulin G Anti-Bullous Pemphigoid 180 Antibody) Following Treatment With Omalizumab.
|
92.06 units per milliliter
|
—
|
SECONDARY outcome
Timeframe: Baseline, 24 weeks.Population: 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.
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
Outcome measures
| Measure |
Omalizumab Treatment Arm
n=1 Participants
Omalizumab 150-375 milligrams (mg) will be administered subcutaneously at Day 1, Week 2,4,6,8,10,12, and 14.
|
Prednisone Standard Therapy Treatment Arm
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
|
|---|---|---|
|
Decrease in Eosinophil Levels Following Treatment With Omalizumab.
|
2.16 cells/microliter
|
—
|
SECONDARY outcome
Timeframe: Up to 24 weeksPopulation: 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.
Outcome measures
| Measure |
Omalizumab Treatment Arm
n=1 Participants
Omalizumab 150-375 milligrams (mg) will be administered subcutaneously at Day 1, Week 2,4,6,8,10,12, and 14.
|
Prednisone Standard Therapy Treatment Arm
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
|
|---|---|---|
|
Decrease in Anti-BP230 Antibody IgG (Anti-bullous Pemphigoid 230 Antibody Immunoglobulin G) At Baseline and Week 16
|
62.6 units per milliliter
|
—
|
SECONDARY outcome
Timeframe: Up to 24 weeksPopulation: We were unable to complete this assay in our research subjects due to technical difficulties.
The histamine release assay measures the release of histamine which occurs upon stimulation of basophilic granulocytes depending upon their sensitivity to an allergen.
Outcome measures
Outcome data not reported
Adverse Events
Omalizumab Treatment Arm
Prednisone Standard Therapy Treatment Arm
Serious adverse events
| Measure |
Omalizumab Treatment Arm
n=2 participants at risk
Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 16 weeks.
|
Prednisone Standard Therapy Treatment Arm
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
|
|---|---|---|
|
Cardiac disorders
Hospitalization for Congestive Heart Failure Exacerbation
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected over a 24 week time period.
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.
|
—
0/0 • Adverse event data were collected over a 24 week time period.
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.
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
50.0%
1/2 • Number of events 1 • Adverse event data were collected over a 24 week time period.
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.
|
—
0/0 • Adverse event data were collected over a 24 week time period.
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.
|
Other adverse events
| Measure |
Omalizumab Treatment Arm
n=2 participants at risk
Patients with bullous pemphigoid will be treated with omalizumab for a total treatment period of 16 weeks.
|
Prednisone Standard Therapy Treatment Arm
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Ankle Edema
|
100.0%
1/1 • Number of events 1 • Adverse event data were collected over a 24 week time period.
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.
|
—
0/0 • Adverse event data were collected over a 24 week time period.
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.
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
100.0%
1/1 • Number of events 1 • Adverse event data were collected over a 24 week time period.
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.
|
—
0/0 • Adverse event data were collected over a 24 week time period.
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.
|
|
Skin and subcutaneous tissue disorders
Staph Aureus Infection
|
100.0%
1/1 • Number of events 1 • Adverse event data were collected over a 24 week time period.
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.
|
—
0/0 • Adverse event data were collected over a 24 week time period.
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.
|
|
General disorders
Nasal Stuffiness
|
100.0%
1/1 • Number of events 1 • Adverse event data were collected over a 24 week time period.
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.
|
—
0/0 • Adverse event data were collected over a 24 week time period.
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.
|
|
General disorders
Cough associated Nasal Drainage
|
100.0%
1/1 • Number of events 1 • Adverse event data were collected over a 24 week time period.
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.
|
—
0/0 • Adverse event data were collected over a 24 week time period.
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.
|
|
Skin and subcutaneous tissue disorders
Staphylococcus infection
|
100.0%
1/1 • Number of events 1 • Adverse event data were collected over a 24 week time period.
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.
|
—
0/0 • Adverse event data were collected over a 24 week time period.
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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60