Trial Outcomes & Findings for Ixekizumab in the Treatment of Bullous Pemphigoid (NCT NCT03099538)

NCT ID: NCT03099538

Last Updated: 2020-05-21

Results Overview

Median time to cessation of blister formation during the 12 weeks of therapy.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

Up to 12 weeks

Results posted on

2020-05-21

Participant Flow

Participant milestones

Participant milestones
Measure
Ixekizumab
Ixekizumab subcutaneous 160mg week 0, 80mg weeks 2, 4, 6, 8, 10, 12. Ixekizumab: Subcutaneous injection
Overall Study
STARTED
4
Overall Study
COMPLETED
1
Overall Study
NOT COMPLETED
3

Reasons for withdrawal

Reasons for withdrawal
Measure
Ixekizumab
Ixekizumab subcutaneous 160mg week 0, 80mg weeks 2, 4, 6, 8, 10, 12. Ixekizumab: Subcutaneous injection
Overall Study
Lack of Efficacy
3

Baseline Characteristics

Ixekizumab in the Treatment of Bullous Pemphigoid

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Ixekizumab
n=4 Participants
Ixekizumab subcutaneous 160mg week 0, 80mg weeks 2, 4, 6, 8, 10, 12. Ixekizumab: Subcutaneous injection
Age, Continuous
70.8 years
STANDARD_DEVIATION 6.45 • n=5 Participants
Sex: Female, Male
Female
4 Participants
n=5 Participants
Sex: Female, Male
Male
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
3 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
4 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
United States
4 participants
n=5 Participants

PRIMARY outcome

Timeframe: Up to 12 weeks

Population: Endpoint was not achieved as there was no cession of blisters.

Median time to cessation of blister formation during the 12 weeks of therapy.

Outcome measures

Outcome measures
Measure
Ixekizumab
n=4 Participants
Ixekizumab subcutaneous 160mg week 0, 80mg weeks 2, 4, 6, 8, 10, 12. Ixekizumab: Subcutaneous injection
Cessation of Blister Formation
NA days
Standard Deviation NA
Insufficient number of participants with events.

SECONDARY outcome

Timeframe: Week 0 and week 12

The change in Bullous Pemphigoid Disease Activity Index (BPDAI) from week 0 to 12 of treatment will be measured.The BPDAI is a standard scoring system for cutaneous disease activity and pruritus in BP. BPDAI is predictive of the likelihood of a subsequent flare. The BPDAI consists of scoring various types of lesions and creates a score ranging from 0 to 120. A score of greater than 56 is considered severe disease.

Outcome measures

Outcome measures
Measure
Ixekizumab
n=4 Participants
Ixekizumab subcutaneous 160mg week 0, 80mg weeks 2, 4, 6, 8, 10, 12. Ixekizumab: Subcutaneous injection
Change in Bullous Pemphigoid Disease Activity Index (BPDAI)
Week 0
24.8 units on a scale
Standard Deviation 17.71
Change in Bullous Pemphigoid Disease Activity Index (BPDAI)
Week 12
39 units on a scale
Standard Deviation 24.48

SECONDARY outcome

Timeframe: Epoch 1 (washout- up to 4 weeks) Epoch 2 (week 0 to week 12) Epoch 3 (week 12 to week 16)

Population: Only one subject remained in the study by Epoch 3 timepoint (week 12 to 16)

Average daily prednisone dose (mg) will be calculated for each Epoch.

Outcome measures

Outcome measures
Measure
Ixekizumab
n=4 Participants
Ixekizumab subcutaneous 160mg week 0, 80mg weeks 2, 4, 6, 8, 10, 12. Ixekizumab: Subcutaneous injection
Prednisone Dose (mg)
Epoch 1
0 mg/d
Standard Deviation 0
Prednisone Dose (mg)
Epoch 2
15.42 mg/d
Standard Deviation 9.17
Prednisone Dose (mg)
Epoch 3
0 mg/d
Standard Deviation 0

SECONDARY outcome

Timeframe: weeks 0 to 18

The clinical safety of Ixekizumab will be monitored with collection of vital signs, clinical examination, and clinical laboratory studies. Adverse events (AE) will be reported per the Common Terminology Criteria for Adverse Events (CTCAEv4.0), a descriptive terminology which can be utilized for AE reporting. A grading (severity) scale is provided for each AE Grade refers to the severity of the AE. The CTCAE displays Grades 1-5 with unique clinical descriptions of severity for each AE based on this general guideline: Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; Grade 4 Life-threatening consequences; urgent intervention indicated. Grade 5 Death related to AE.

Outcome measures

Outcome measures
Measure
Ixekizumab
n=4 Participants
Ixekizumab subcutaneous 160mg week 0, 80mg weeks 2, 4, 6, 8, 10, 12. Ixekizumab: Subcutaneous injection
Number of Participants With Treatment-related Adverse Events as Assessed by the Common Terminology Criteria for Adverse Events Version 4.0 (CTCAE v4.0).
Grade 1
1 Participants
Number of Participants With Treatment-related Adverse Events as Assessed by the Common Terminology Criteria for Adverse Events Version 4.0 (CTCAE v4.0).
Grade 2
0 Participants
Number of Participants With Treatment-related Adverse Events as Assessed by the Common Terminology Criteria for Adverse Events Version 4.0 (CTCAE v4.0).
Grade 3
0 Participants
Number of Participants With Treatment-related Adverse Events as Assessed by the Common Terminology Criteria for Adverse Events Version 4.0 (CTCAE v4.0).
Grade 4
0 Participants
Number of Participants With Treatment-related Adverse Events as Assessed by the Common Terminology Criteria for Adverse Events Version 4.0 (CTCAE v4.0).
Grade 5
0 Participants

SECONDARY outcome

Timeframe: week 0, 4, 8, 12

Population: This outcome measure was not achieved due to lack to sample to run assays.

We will measure the anti-BP180\&230 antibodies through out treatment. These assays will be completed using and ELISA assay. Mayo medical labs references for anti-BP180\&230 (\<9.0 Units negative, \> or = 9.0 Units positive)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: week 0, 4, 8, 12

Population: This outcome measure was not achieved due to lack of data.

Neutrophil and eosinophil counts will be monitored throughout therapy. Mayo medical labs reports normal neutrophil levels (1.70-7.00X10(9)/L) and normal eosinophil levels (0.05-0.5X10(9)/L)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: week 0, 4, 8, 12

Population: This outcome measure was not achieved due to lack of sample to run assays.

Multiplex cytokine analysis will be performed throughout therapy on Interleukin (IL)- 6, 17, 22, 23, Transforming growth factor beta (TGFb), and matrix-metalloprotease-9 (MMP9).

Outcome measures

Outcome data not reported

Adverse Events

Ixekizumab

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Ixekizumab
n=4 participants at risk
Ixekizumab subcutaneous 160mg week 0, 80mg weeks 2, 4, 6, 8, 10, 12. Ixekizumab: Subcutaneous injection
General disorders
Rib pain
25.0%
1/4 • Number of events 1 • Adverse events were collected for each subject from baseline until 30 days post-study participation, for a total of approximately four to five months.
General disorders
Skin Pain
25.0%
1/4 • Number of events 1 • Adverse events were collected for each subject from baseline until 30 days post-study participation, for a total of approximately four to five months.
Skin and subcutaneous tissue disorders
Pruritus
50.0%
2/4 • Number of events 2 • Adverse events were collected for each subject from baseline until 30 days post-study participation, for a total of approximately four to five months.
Eye disorders
Conjunctivitis
25.0%
1/4 • Number of events 1 • Adverse events were collected for each subject from baseline until 30 days post-study participation, for a total of approximately four to five months.
Eye disorders
Dry eyes
25.0%
1/4 • Number of events 1 • Adverse events were collected for each subject from baseline until 30 days post-study participation, for a total of approximately four to five months.
General disorders
Headache
25.0%
1/4 • Number of events 1 • Adverse events were collected for each subject from baseline until 30 days post-study participation, for a total of approximately four to five months.
Respiratory, thoracic and mediastinal disorders
Shortness of breath
25.0%
1/4 • Number of events 1 • Adverse events were collected for each subject from baseline until 30 days post-study participation, for a total of approximately four to five months.
Infections and infestations
Staphylococcal overgrowth
25.0%
1/4 • Number of events 1 • Adverse events were collected for each subject from baseline until 30 days post-study participation, for a total of approximately four to five months.
General disorders
Facial swelling
25.0%
1/4 • Number of events 1 • Adverse events were collected for each subject from baseline until 30 days post-study participation, for a total of approximately four to five months.

Additional Information

Aaron R. Mangold, MD

Mayo Clinic

Phone: 480-301-4256

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place