Trial Outcomes & Findings for Mepolizumab in Episodic Angioedema With Eosinophilia (NCT NCT04128371)

Last Updated: 2024-04-16

Results Overview

We measure the number and severity of clinical symptoms associated with episodic angioedema with eosinophilia (EAE) using the maximum of the patient reported daily angioedema activity score, a validated patient reported outcome measure. The daily score was used to measure changes in swelling within a cycle. Participants answered 5 questions each day that were scored from 0-3 for each item. The daily score consisted of a minimum score of 0 and a maximum score of 15, the sum of the 5 question answers. Higher scores indicate a worse outcome. The change of the daily angioedema activity score is measured as the average percent reduction in the maximum score over the 3 cycles (about 3 months) after treatment compared to the cycle (about 1 month) pre-treatment. The estimated percent change and its confidence interval was calculated from a quasi-Poisson model. The model estimates a multiplicative treatment effect on the baseline AAS, and that effect is translated into a percent change.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

1 month prior to treatment and 3 months after treatment

Results posted on

2024-04-16

Participant Flow

Participant milestones

Participant milestones
Measure	Active Treatment Participants with episodic angioedema with eosinophilia (EAE) received mepolizumab 700 mg intravenously monthly for three doses.
Overall Study STARTED	5
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Active Treatment Participants with episodic angioedema with eosinophilia (EAE) received mepolizumab 700 mg intravenously monthly for three doses.
Overall Study Lack of Efficacy	1

Baseline Characteristics

Mepolizumab in Episodic Angioedema With Eosinophilia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Active Treatment n=5 Participants Participants with episodic angioedema with eosinophilia (EAE) received mepolizumab 700 mg intravenously monthly for three doses.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 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	5 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

PRIMARY outcome

Timeframe: 1 month prior to treatment and 3 months after treatment

Population: The analyses included all participants during the cycle (about the 1 month) prior to treatment with up to 3 cycles (about 3 months) after treatment began. One patient withdrew after only 2 cycles post treatment and we used their values up until time of withdrawal.

Outcome measures

Outcome measures
Measure	Active Treatment n=5 Participants Participants with episodic angioedema with eosinophilia (EAE) received mepolizumab 700 mg intravenously monthly for three doses.
Percent Change in Maximum Daily Angioedema Activity Score (AAS)	16.5 Percent change Interval -64.6 to 57.7

SECONDARY outcome

Timeframe: 1 month prior to treatment and 1 month after treatment

Population: Analysis included all participants

Percent change in peak AEC is measured on each individual as the percentage change in peak AEC in the cycle following the first mepolizumab infusion compared to the peak AEC in the cycle prior to mepolizumab treatment. Those values are summarized by taking the mean of a transformation of each percent change in peak AEC, and back-transforming the results. Specifically, if x is the percent change in peak AEC, then the transformation is log(1-x/100). The estimated percent change and its confidence interval was calculated using the mean of the transformed values and using the associated one-sample t-test confidence interval on that mean, then translating those estimates and confidence intervals back to the percent change scale.

Outcome measures

Outcome measures
Measure	Active Treatment n=5 Participants Participants with episodic angioedema with eosinophilia (EAE) received mepolizumab 700 mg intravenously monthly for three doses.
Percent Change in Peak Absolute Eosinophil Count (AEC)	82.2 Percent change Interval -108.5 to 98.5

SECONDARY outcome

Timeframe: 1 month prior to treatment and 3 months after treatment

Population: 4 of the 5 participants were assessed at visit 8. One participant withdrew prior to visit 8, and we used their values at visit 7.

Percent change in peak AEC is measured on each individual as the percentage change in peak AEC by visit 8 (3 months) compared to the peak AEC in the cycle prior to mepolizumab treatment. Those values are summarized by taking the mean of a transformation of each percent change in peak AEC, and back-transforming the results. Specifically, if x is the percent change in peak AEC, then the transformation is log(1-x/100). The estimated percent change and its confidence interval was calculated using the mean of the transformed values and using the associated one-sample t-test confidence interval on that mean, then translating those estimates and confidence intervals back to the percent change scale.

Outcome measures

Outcome measures
Measure	Active Treatment n=5 Participants Participants with episodic angioedema with eosinophilia (EAE) received mepolizumab 700 mg intravenously monthly for three doses.
Percent Change in Peak Absolute Eosinophil Count (AEC)	96.2 Percent change Interval 71.4 to 99.5

Adverse Events

Active Treatment

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	Active Treatment n=5 participants at risk Participants with episodic angioedema with eosinophilia (EAE) received mepolizumab 700 mg intravenously monthly for three doses.
Gastrointestinal disorders Nausea	20.0% 1/5 • Up to 12 months
Infections and infestations Upper respiratory tract infection	20.0% 1/5 • Up to 12 months
Investigations Coronavirus test positive	40.0% 2/5 • Up to 12 months
Investigations Haemoglobin decreased	20.0% 1/5 • Up to 12 months
Nervous system disorders Paraesthesia	20.0% 1/5 • Up to 12 months
Respiratory, thoracic and mediastinal disorders Cough	20.0% 1/5 • Up to 12 months
Respiratory, thoracic and mediastinal disorders Pharyngitis	20.0% 1/5 • Up to 12 months

Additional Information

Dr. Paneez Khoury

National Institute of Allergy and Infectious Diseases

Phone: +1 301 402 3673

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place