Trial Outcomes & Findings for Efficacy and Safety of Miltefosine in Antihistamine Resistant Chronic Urticaria (NCT NCT01170949)

NCT ID: NCT01170949

Last Updated: 2016-12-26

Results Overview

The weekly UAS was calculated by adding the daily scores over one week. During the whole course of the study patients recorded the amount of wheals and the intensity of itching as well as the occurrence of swelling in ranges between 0 and 3. These daily scores were used to calculate urticaria activity scores (UAS) as follows: Daily UAS are calculated by adding the score points obtained for the symptom categories "number of wheals" and "intensity of pruritus". "Number of wheals" is scored as 0 = no wheals, 1 = some wheals (\<20), 2 = moderate number of wheals (20-50), 3 = more than 50 wheals. "Intensity of pruritus" is scored as 0 = no itching; 1 = mild itching, not irritating; 2 = moderate itching, normal daily activity and sleep is possible; and 3 = severe itching, normal daily activity and sleep is impaired. The maximum score is 42.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 76 participants
• Primary outcome timeframe: Day 28
• Results posted on: 2016-12-26

Participant Flow

Participant milestones

Measure	Miltefosine Week 1 - 50 mg miltefosine or placebo Week 2 - 100 mg miltefosine or placebo (1 capsule in the morning and one in the evening),if evidence of intolerability dose had to be reduced to 50 mg, those patients received 50 mg until the end of the treatment period Week 3 - 150 mg miltefosine or placebo (3 capsules, one in the morning, one at lunch and one in the evening) if evidence of intolerability dose had to be reduced to 100 mg, those patients received 100 mg until the end of the treatment period	Placebo Week 1 - 50 mg miltefosine or placebo Week 2 - 100 mg miltefosine or placebo (1 capsule in the morning and one in the evening),if evidence of intolerability dose had to be reduced to 50 mg, those patients received 50 mg until the end of the treatment period Week 3 - 150 mg miltefosine or placebo (3 capsules, one in the morning, one at lunch and one in the evening) if evidence of intolerability dose had to be reduced to 100 mg, those patients received 100 mg until the end of the treatment period
Overall Study STARTED	50	26
Overall Study COMPLETED	43	22
Overall Study NOT COMPLETED	7	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Safety of Miltefosine in Antihistamine Resistant Chronic Urticaria

Baseline characteristics by cohort

Measure	Miltefosine n=50 Participants	Placebo n=26 Participants	Total n=76 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	44 Participants n=5 Participants	22 Participants n=7 Participants	66 Participants n=5 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants	4 Participants n=7 Participants	10 Participants n=5 Participants
Age, Continuous	45.0 years STANDARD_DEVIATION 12.4 • n=5 Participants	42.6 years STANDARD_DEVIATION 12.5 • n=7 Participants	44 years STANDARD_DEVIATION 24.9 • n=5 Participants
Gender Female	28 Participants n=5 Participants	14 Participants n=7 Participants	42 Participants n=5 Participants
Gender Male	22 Participants n=5 Participants	12 Participants n=7 Participants	34 Participants n=5 Participants
Region of Enrollment Germany	50 participants n=5 Participants	26 participants n=7 Participants	76 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 28

Population: ITT = 73

The weekly UAS was calculated by adding the daily scores over one week. During the whole course of the study patients recorded the amount of wheals and the intensity of itching as well as the occurrence of swelling in ranges between 0 and 3. These daily scores were used to calculate urticaria activity scores (UAS) as follows: Daily UAS are calculated by adding the score points obtained for the symptom categories "number of wheals" and "intensity of pruritus". "Number of wheals" is scored as 0 = no wheals, 1 = some wheals (<20), 2 = moderate number of wheals (20-50), 3 = more than 50 wheals. "Intensity of pruritus" is scored as 0 = no itching; 1 = mild itching, not irritating; 2 = moderate itching, normal daily activity and sleep is possible; and 3 = severe itching, normal daily activity and sleep is impaired. The maximum score is 42.

Outcome measures

Measure	Miltefosine n=47 Participants Miltefosine: 50 or 100 or 150mg per day	Placebo n=26 Participants Placebo: Placebo
Urticaria Activity Score (% Change From Baseline)	-25.5 percentage of UAS baseline Standard Deviation 38.2	-10.1 percentage of UAS baseline Standard Deviation 33.4

Adverse Events

Miltefosine

Serious events: 0 serious events

Other events: 44 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 17 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Miltefosine n=50 participants at risk	Placebo n=26 participants at risk
Gastrointestinal disorders Abdominal discomfort	70.0% 35/50 • Adverse event data were collected at each Visit.	50.0% 13/26 • Adverse event data were collected at each Visit.
Gastrointestinal disorders Nausea	28.0% 14/50 • Number of events 28 • Adverse event data were collected at each Visit.	15.4% 4/26 • Adverse event data were collected at each Visit.
Gastrointestinal disorders Vomiting	20.0% 10/50 • Adverse event data were collected at each Visit.	3.8% 1/26 • Adverse event data were collected at each Visit.

Additional Information

Markus Magerl

University Charité Berlin

Phone: +4930450618304

Email: markus.magerl@charite.de

Results disclosure agreements

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