Trial Outcomes & Findings for Neuropeptide Treatment for Hot Flushes During the Menopause (NCT NCT02668185)

NCT ID: NCT02668185

Last Updated: 2021-05-03

Results Overview

To ensure accurate records, participants recorded their flushes in real time using either a tally chart on a piece of paper (n=34) or an application on their smartphone such as Tally Counter (Pixel Research Labs, Minneapolis-Saint Paul, MN, USA; n=3), and then collated their total number of flushes twice daily on waking to record previous overnight symptoms and before bed to record daytime symptoms.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

37 participants

Primary outcome timeframe

Final week of each 4 week treatment period

Results posted on

2021-05-03

Participant Flow

Participant milestones

Participant milestones
Measure
Active Drug First Then Placebo
2 week baseline period First NK3R antagonist - AZD4901 - 40mg orally bd - for 4 weeks intervention then 2 weeks washout period then second intervention with Matched placebo orally bd for 4 weeks with 2 weeks monitoring period
Placebo First Then Active Drug
2 week baseline period First intervention is Placebo - 40mg bd - for 4 weeks, then 2 weeks washout period, then second intervention with NK3R antagonist - AZD4901 - 40mg orally bd - for 4 weeks with 2 weeks monitoring period
Baseline Period (2 Weeks)
STARTED
20
17
Baseline Period (2 Weeks)
COMPLETED
20
17
Baseline Period (2 Weeks)
NOT COMPLETED
0
0
First Intervention (4 Weeks)
STARTED
20
17
First Intervention (4 Weeks)
COMPLETED
18
17
First Intervention (4 Weeks)
NOT COMPLETED
2
0
Washout Period (2 Weeks)
STARTED
18
17
Washout Period (2 Weeks)
COMPLETED
17
15
Washout Period (2 Weeks)
NOT COMPLETED
1
2
Second Intervention (4 Weeks)
STARTED
17
15
Second Intervention (4 Weeks)
COMPLETED
16
12
Second Intervention (4 Weeks)
NOT COMPLETED
1
3
Moitoring Period (2 Weeks)
STARTED
16
12
Moitoring Period (2 Weeks)
COMPLETED
16
12
Moitoring Period (2 Weeks)
NOT COMPLETED
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Active Drug First Then Placebo
2 week baseline period First NK3R antagonist - AZD4901 - 40mg orally bd - for 4 weeks intervention then 2 weeks washout period then second intervention with Matched placebo orally bd for 4 weeks with 2 weeks monitoring period
Placebo First Then Active Drug
2 week baseline period First intervention is Placebo - 40mg bd - for 4 weeks, then 2 weeks washout period, then second intervention with NK3R antagonist - AZD4901 - 40mg orally bd - for 4 weeks with 2 weeks monitoring period
First Intervention (4 Weeks)
Physician Decision
2
0
Washout Period (2 Weeks)
Adverse Event
1
0
Washout Period (2 Weeks)
Physician Decision
0
2
Second Intervention (4 Weeks)
Physician Decision
1
3

Baseline Characteristics

Neuropeptide Treatment for Hot Flushes During the Menopause

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Overall Participants
n=37 Participants
Crossover study - all participants
Age, Categorical
<=18 years
0 Participants
n=93 Participants
Age, Categorical
Between 18 and 65 years
37 Participants
n=93 Participants
Age, Categorical
>=65 years
0 Participants
n=93 Participants
Sex: Female, Male
Female
37 Participants
n=93 Participants
Sex: Female, Male
Male
0 Participants
n=93 Participants
Race/Ethnicity, Customized
White British
17 Participants
n=93 Participants
Race/Ethnicity, Customized
White European
2 Participants
n=93 Participants
Race/Ethnicity, Customized
White Other
1 Participants
n=93 Participants
Race/Ethnicity, Customized
White Unspecified
1 Participants
n=93 Participants
Race/Ethnicity, Customized
Asian, Pakistani
1 Participants
n=93 Participants
Race/Ethnicity, Customized
Black Carribean
5 Participants
n=93 Participants
Race/Ethnicity, Customized
Mixed white and black carribean
1 Participants
n=93 Participants
Region of Enrollment
United Kingdom
37 participants
n=93 Participants

PRIMARY outcome

Timeframe: Final week of each 4 week treatment period

Population: Whole group intention to treat analysis irrespective of treatment assignment order, adjusted means from crossover analysis with 95% CIs: percentage change in total no. hot flush frequency during final week of 4 week treatment period with MLE4901 and placebo vs with total no. hot flush frequency during the final week of the 2 week baseline period.

To ensure accurate records, participants recorded their flushes in real time using either a tally chart on a piece of paper (n=34) or an application on their smartphone such as Tally Counter (Pixel Research Labs, Minneapolis-Saint Paul, MN, USA; n=3), and then collated their total number of flushes twice daily on waking to record previous overnight symptoms and before bed to record daytime symptoms.

Outcome measures

Outcome measures
Measure
Active Drug
n=28 Participants
NK3R antagonist - AZD4901 - 40mg bd - for 4 weeks NK3R antagonist - AZD4901: Neurokinin 3 receptor antagonist
Placebo
n=28 Participants
Placebo - 40mg bd - for 4 weeks Placebo: Placebo
Total Number of Hot Flushes During the Final Week of Each 4 Weeks Treatment Period
19.35 hot flushes
Interval 15.99 to 23.42
49.01 hot flushes
Interval 40.81 to 58.56

SECONDARY outcome

Timeframe: Twice daily, morning and night for 14 weeks

Score on a scale - HF severity (rated as 1-nil, 2-mild, 3-moderate, 4-severe, as per Joffe 2014) recorded twice daily (day/night as described above for HF frequency). Scores are summed.

Outcome measures

Outcome measures
Measure
Active Drug
n=28 Participants
NK3R antagonist - AZD4901 - 40mg bd - for 4 weeks NK3R antagonist - AZD4901: Neurokinin 3 receptor antagonist
Placebo
n=28 Participants
Placebo - 40mg bd - for 4 weeks Placebo: Placebo
Hot Flush Severity
3.27 numerical rating of severity by particip
Interval 2.92 to 3.66
5.70 numerical rating of severity by particip
Interval 5.09 to 6.38

SECONDARY outcome

Timeframe: twice daily (day/night) for 14 weeks

Score on a scale - HF bother (rated as 1-none, 2-a little, 3-moderate, 4-a lot, as per Joffe 2014) will be recorded twice daily (day/night as described above for HF frequency). The data will be analysed as detailed above for the HF frequency. Scores are summed.

Outcome measures

Outcome measures
Measure
Active Drug
n=28 Participants
NK3R antagonist - AZD4901 - 40mg bd - for 4 weeks NK3R antagonist - AZD4901: Neurokinin 3 receptor antagonist
Placebo
n=28 Participants
Placebo - 40mg bd - for 4 weeks Placebo: Placebo
Hot Flush Bother
2.92 score on a scale
Interval 2.61 to 3.27
5.56 score on a scale
Interval 4.96 to 6.27

SECONDARY outcome

Timeframe: Daily at bedtime for 14 weeks

Score on a scale Menopause Specific Quality of Life (MENQOL) questionnaire 0=not bothered at all - 6=extremely bothered. 4 domains, mean calculated for set of questions in each domain. Overall score is a mean of the means. Highr scor = higher interference.

Outcome measures

Outcome measures
Measure
Active Drug
n=28 Participants
NK3R antagonist - AZD4901 - 40mg bd - for 4 weeks NK3R antagonist - AZD4901: Neurokinin 3 receptor antagonist
Placebo
n=28 Participants
Placebo - 40mg bd - for 4 weeks Placebo: Placebo
Hot Flush Interference
7.94 Score by participant on 'interference sc
Interval 5.76 to 10.95
26.48 Score by participant on 'interference sc
Interval 20.02 to 35.03

SECONDARY outcome

Timeframe: Once per week for 48 hours over 14 weeks

Mean number of flushes detected during the 48 hours by the skin conductance monitoring will be compared each week when the patients receive AZD4901 versus placebo

Outcome measures

Outcome measures
Measure
Active Drug
n=28 Participants
NK3R antagonist - AZD4901 - 40mg bd - for 4 weeks NK3R antagonist - AZD4901: Neurokinin 3 receptor antagonist
Placebo
n=28 Participants
Placebo - 40mg bd - for 4 weeks Placebo: Placebo
Skin Conductance Monitor Data.
16.22 Number of hot flushes detected by monito
Interval 13.99 to 18.8
26.91 Number of hot flushes detected by monito
Interval 23.16 to 31.27

Adverse Events

Active Drug First Then Placebo

Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths

Placebo First Then Active Drug

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

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Active Drug First Then Placebo
n=28 participants at risk
2 week baseline period First NK3R antagonist - AZD4901 - 40mg orally bd - for 4 weeks intervention then 2 weeks washout period then second intervention with Matched placebo orally bd for 4 weeks with 2 weeks monitoring period
Placebo First Then Active Drug
n=28 participants at risk
2 week baseline period First intervention is Placebo - 40mg bd - for 4 weeks, then 2 weeks washout period, then second intervention with NK3R antagonist - AZD4901 - 40mg orally bd - for 4 weeks with 2 weeks monitoring period
Hepatobiliary disorders
transaminase elevations by treatment and CTCAE grade
10.7%
3/28 • Number of events 3 • 10 weeks
0.00%
0/28 • 10 weeks

Additional Information

Professor Wajit Dhillo

Imperial College London

Phone: +44 (0)20 7594 2489

Results disclosure agreements

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