Trial Outcomes & Findings for Mepolizumab As a Steroid-sparing Treatment Option in the Churg Strauss Syndrome (NCT NCT00527566)

NCT ID: NCT00527566

Last Updated: 2017-03-22

Results Overview

Side effects experienced by participants 1 to 2 days after Mepolizumab infusion.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: Participants were followed for the duration of the study, approximately 44 weeks
• Results posted on: 2017-03-22

Participant Flow

Participant milestones

Measure	Mepolizumab Intravenously infused 750 mg of mepolizumab 1x every 4 weeks for 16 weeks.
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Mepolizumab As a Steroid-sparing Treatment Option in the Churg Strauss Syndrome

Baseline characteristics by cohort

Measure	Mepolizumab n=10 Participants Intravenously infused 750 mg of mepolizumab 1x every 4 weeks for 16 weeks.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	48 years STANDARD_DEVIATION 5 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Race/Ethnicity, Customized Participants · # White Participants	7 Participants n=5 Participants
Race/Ethnicity, Customized Participants · Unknown	3 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: Participants were followed for the duration of the study, approximately 44 weeks

Side effects experienced by participants 1 to 2 days after Mepolizumab infusion.

Outcome measures

Measure	Mepolizumab n=10 Participants Intravenously infused 750 mg of mepolizumab 1x every 4 weeks for 16 weeks.	Non-treatment Phase The non-treatment phase consists of the wash-out phase and the safety monitoring phase.
Number of Participants With Indicated Side Effects Mild transient headaches	3 Participants	—
Number of Participants With Indicated Side Effects Mild pruritus	1 Participants	—
Number of Participants With Indicated Side Effects Urticaria, allergic reaction, worsening symptoms	0 Participants	—
Number of Participants With Indicated Side Effects Loose stool	1 Participants	—

PRIMARY outcome

Timeframe: 44 weeks

Number of participants who experienced specific symptoms during the trial.

Outcome measures

Measure	Mepolizumab n=10 Participants Intravenously infused 750 mg of mepolizumab 1x every 4 weeks for 16 weeks.	Non-treatment Phase The non-treatment phase consists of the wash-out phase and the safety monitoring phase.
Number of Participants Who Experienced Specific Symptoms Wheeze or cough	4 Participants	—
Number of Participants Who Experienced Specific Symptoms Sore throat	4 Participants	—
Number of Participants Who Experienced Specific Symptoms Nausea or abdominal discomfort	3 Participants	—
Number of Participants Who Experienced Specific Symptoms Sinusitis	6 Participants	—
Number of Participants Who Experienced Specific Symptoms Arthritis	1 Participants	—

SECONDARY outcome

Timeframe: 20 weeks

Outcome measures

Measure	Mepolizumab n=10 Participants Intravenously infused 750 mg of mepolizumab 1x every 4 weeks for 16 weeks.	Non-treatment Phase The non-treatment phase consists of the wash-out phase and the safety monitoring phase.
Steroid Dosing During Trial Baseline (Week 0)	14.3 Prednisone mg/day Interval 10.0 to 20.0	—
Steroid Dosing During Trial Week 12	5.6 Prednisone mg/day Interval 2.0 to 12.5	—
Steroid Dosing During Trial Week 20	4.9 Prednisone mg/day Interval 0.0 to 20.0	—

SECONDARY outcome

Timeframe: 20 weeks

The Asthma Control Questionnaire (ACQ) was one measure used to assess the prevalence of asthma symptoms the participant was having during the study. It is a series of 7 questions assessing how often, over the past two weeks, the participant wakes up from their asthma, how bad their symptoms were, etc. The greater the prevalence of symptoms, the higher the score. Each of the 7 questions is scored 0-6. The total score is calculated by adding the individual question scores and dividing the sum by 7.

Outcome measures

Measure	Mepolizumab n=10 Participants Intravenously infused 750 mg of mepolizumab 1x every 4 weeks for 16 weeks.	Non-treatment Phase The non-treatment phase consists of the wash-out phase and the safety monitoring phase.
Evaluate Overall Positive Change in Churg-Strauss Syndrome Via the Measures Outlined in Study Aims Week 0, baseline	1.40 units on a scale Interval 0.0 to 2.86	—
Evaluate Overall Positive Change in Churg-Strauss Syndrome Via the Measures Outlined in Study Aims Week 12, Treatment phase	1.76 units on a scale Interval 0.29 to 7.0	—
Evaluate Overall Positive Change in Churg-Strauss Syndrome Via the Measures Outlined in Study Aims Week 20, end of washout phase	1.30 units on a scale Interval 0.0 to 4.1	—

SECONDARY outcome

Timeframe: Treatment period (12 weeks)

Quantified the exacerbation rate (total number of exacerbations per day) of the participants during treatment with mepolizumab and without treatment. Exacerbations were characterized as any worsening of clinical disease requiring an increase of systemic corticosteroid therapy (e.g. prednisone) for asthma, respiratory symptoms, or underlying vasculitis.

Outcome measures

Measure	Mepolizumab n=10 Participants Intravenously infused 750 mg of mepolizumab 1x every 4 weeks for 16 weeks.	Non-treatment Phase n=10 Participants The non-treatment phase consists of the wash-out phase and the safety monitoring phase.
Efficacy- Exacerbation Rate	0.14 Exacerbation rate (Exacerbations/day)	0.69 Exacerbation rate (Exacerbations/day)

Adverse Events

Mepolizumab

Serious events: 1 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Measure	Mepolizumab n=10 participants at risk Intravenously infused 750 mg of mepolizumab 1x every 4 weeks for 16 weeks.
Musculoskeletal and connective tissue disorders Fractured Hip	10.0% 1/10 • Number of events 1

Other adverse events

Measure	Mepolizumab n=10 participants at risk Intravenously infused 750 mg of mepolizumab 1x every 4 weeks for 16 weeks.
Nervous system disorders Headache	40.0% 4/10 • Number of events 5
Gastrointestinal disorders Digestive System Issues	30.0% 3/10 • Number of events 3
Endocrine disorders Churg-Strauss Syndrome Flair	30.0% 3/10 • Number of events 5
Respiratory, thoracic and mediastinal disorders Sinusitis	30.0% 3/10 • Number of events 4
Respiratory, thoracic and mediastinal disorders Upper Respiratory Infection	70.0% 7/10 • Number of events 8
Respiratory, thoracic and mediastinal disorders Wheezing and Shortness of Breath	70.0% 7/10 • Number of events 8

Additional Information

Michael Wechsler

National Jewish Health

Phone: 617-285-4987

Email: wechslerm@NJHealth.org

Results disclosure agreements

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