Trial Outcomes & Findings for A Study of the Efficacy and Safety of a 3-month Treatment Course of Ulipristal Acetate for the Treatment of Abnormal Uterine Bleeding Associated With Leiomyomas (NCT NCT02147197)
NCT ID: NCT02147197
Last Updated: 2019-04-30
Results Overview
Participants recorded bleeding in a daily diary. Absence of bleeding was defined as no bleeding days (i.e. no entries for bleeding or heavy bleeding; however, spotting was allowed), during the last 35 consecutive days on treatment in the 12-week Treatment Period.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
157 participants
Primary outcome timeframe
Last 35 consecutive days on treatment in the 12-week Treatment Period
Results posted on
2019-04-30
Participant Flow
Participant milestones
| Measure |
Placebo
Matching placebo tablet (5 mg and 10 mg), orally, once daily for 12 weeks.
|
UPA 5 mg
Ulipristal acetate (UPA) 5 mg tablet plus matching placebo 10 mg tablet, orally, once daily for 12 weeks.
|
UPA 10 mg
UPA 10 mg tablet plus matching placebo 5 mg tablet, orally, once daily for 12 weeks.
|
|---|---|---|---|
|
Overall Study
STARTED
|
56
|
53
|
48
|
|
Overall Study
COMPLETED
|
47
|
47
|
39
|
|
Overall Study
NOT COMPLETED
|
9
|
6
|
9
|
Reasons for withdrawal
| Measure |
Placebo
Matching placebo tablet (5 mg and 10 mg), orally, once daily for 12 weeks.
|
UPA 5 mg
Ulipristal acetate (UPA) 5 mg tablet plus matching placebo 10 mg tablet, orally, once daily for 12 weeks.
|
UPA 10 mg
UPA 10 mg tablet plus matching placebo 5 mg tablet, orally, once daily for 12 weeks.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
0
|
|
Overall Study
Withdrawal of Consent
|
4
|
3
|
2
|
|
Overall Study
Lost to Follow-up
|
2
|
3
|
1
|
|
Overall Study
Protocol Violation
|
1
|
0
|
3
|
|
Overall Study
Other Miscellaneous Reasons
|
1
|
0
|
3
|
Baseline Characteristics
A Study of the Efficacy and Safety of a 3-month Treatment Course of Ulipristal Acetate for the Treatment of Abnormal Uterine Bleeding Associated With Leiomyomas
Baseline characteristics by cohort
| Measure |
Placebo
n=56 Participants
Matching placebo tablet (5 mg and 10 mg), orally, once daily for 12 weeks.
|
UPA 5 mg
n=53 Participants
UPA 5 mg tablet plus matching placebo 10 mg tablet, orally, once daily for 12 weeks.
|
UPA 10 mg
n=48 Participants
UPA 10 mg tablet plus matching placebo 5 mg tablet, orally, once daily for 12 weeks.
|
Total
n=157 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
41.6 years
STANDARD_DEVIATION 5.1 • n=5 Participants
|
40.5 years
STANDARD_DEVIATION 5.9 • n=7 Participants
|
41.3 years
STANDARD_DEVIATION 5.1 • n=5 Participants
|
41.1 years
STANDARD_DEVIATION 5.4 • n=4 Participants
|
|
Sex: Female, Male
Female
|
56 Participants
n=5 Participants
|
53 Participants
n=7 Participants
|
48 Participants
n=5 Participants
|
157 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Last 35 consecutive days on treatment in the 12-week Treatment PeriodPopulation: Intent-to-Treat (ITT) Population included all randomized participants. Data was summarized as per the treatment assigned.
Participants recorded bleeding in a daily diary. Absence of bleeding was defined as no bleeding days (i.e. no entries for bleeding or heavy bleeding; however, spotting was allowed), during the last 35 consecutive days on treatment in the 12-week Treatment Period.
Outcome measures
| Measure |
Placebo
n=56 Participants
Matching placebo tablet (5 mg and 10 mg), orally, once daily for 12 weeks.
|
UPA 5 mg
n=53 Participants
UPA 5 mg tablet plus matching placebo 10 mg tablet, orally, once daily for 12 weeks.
|
UPA 10 mg
n=48 Participants
UPA 10 mg tablet plus matching placebo 5 mg tablet, orally, once daily for 12 weeks.
|
|---|---|---|---|
|
Percentage of Participants With Absence of Bleeding During the Last 35 Consecutive Days on Treatment
|
1.8 percentage of participants
Interval 0.0 to 10.9
|
47.2 percentage of participants
Interval 31.6 to 63.2
|
58.3 percentage of participants
Interval 41.2 to 74.1
|
PRIMARY outcome
Timeframe: From first dose up to the end of the 12-week Treatment PeriodPopulation: ITT Population included all randomized participants. Data was summarized as per the treatment assigned.
Time to absence of bleeding was defined as the duration in days from first dose to the first day in the time interval in which absence of bleeding occurs and persists through the last dose on treatment. The persistence of absence of bleeding occurred for a minimum of 35 consecutive days counting backward from the last dose on treatment in the 12-week Treatment Period.
Outcome measures
| Measure |
Placebo
n=56 Participants
Matching placebo tablet (5 mg and 10 mg), orally, once daily for 12 weeks.
|
UPA 5 mg
n=53 Participants
UPA 5 mg tablet plus matching placebo 10 mg tablet, orally, once daily for 12 weeks.
|
UPA 10 mg
n=48 Participants
UPA 10 mg tablet plus matching placebo 5 mg tablet, orally, once daily for 12 weeks.
|
|---|---|---|---|
|
Time to Absence of Bleeding on Treatment
|
NA days
The Median, upper and lower limit of 97.5% Confidence Interval were not estimated due to the low number of participants with events.
|
NA days
Interval 6.0 to
The Median and upper limit of 97.5% Confidence Interval were not estimated due to the low number of participants with events.
|
9.5 days
Interval 5.0 to
The upper limit of 97.5% Confidence Interval were not estimated due to the low number of participants with events.
|
SECONDARY outcome
Timeframe: Day 11 through the end of the 12-week Treatment PeriodPopulation: ITT Population included all randomized participants. Data was summarized as per the treatment assigned.
Participants recorded bleeding in a daily diary. Absence of bleeding was defined as no bleeding days (i.e. no entries for bleeding or heavy bleeding; however, spotting was allowed), starting from Day 11 through the end of the 12-week Treatment Period.
Outcome measures
| Measure |
Placebo
n=56 Participants
Matching placebo tablet (5 mg and 10 mg), orally, once daily for 12 weeks.
|
UPA 5 mg
n=53 Participants
UPA 5 mg tablet plus matching placebo 10 mg tablet, orally, once daily for 12 weeks.
|
UPA 10 mg
n=48 Participants
UPA 10 mg tablet plus matching placebo 5 mg tablet, orally, once daily for 12 weeks.
|
|---|---|---|---|
|
Percentage of Participants With Absence of Bleeding From Day 11 Through the End of Treatment
|
0.0 percentage of participants
Interval 0.0 to 7.5
|
43.4 percentage of participants
Interval 28.2 to 59.6
|
58.3 percentage of participants
Interval 41.2 to 74.1
|
SECONDARY outcome
Timeframe: Baseline (Day 1-4) to the end of 12-week Treatment PeriodPopulation: Data was summarized as per treatment assigned and included all participants with data at both Baseline and Week 12. The Baseline row shows the actual scores. The results at Week 12 are the difference in scores from Baseline to Week 12.
The UFS-QOL is a uterine fibroid-specific questionnaire consisting of 37 questions developed to evaluate symptoms of uterine fibroids and their impact on health-related quality of life in women with leiomyomas. The first 8 questions comprise the Symptom Severity subscale to assess symptoms experienced by women with uterine leiomyomas, the remaining 29 questions comprise 6 subscales (Concern, Activities, Energy/mood, Control, Self-consciousness, Sexual Function) which overall deal with women's feelings and experiences regarding impact of uterine leiomyoma symptoms on her life. Each item is scored between 1 and 5, where 1=none of the time or not at all and 5=all of the time or a very great deal. A Revised Activities subscale was created to include the most relevant items pertaining to physical and social activities with a total possible score of 0 to 100. Higher Revised Activities subscale scores indicate less impact on activities. A positive change from Baseline indicates improvement.
Outcome measures
| Measure |
Placebo
n=51 Participants
Matching placebo tablet (5 mg and 10 mg), orally, once daily for 12 weeks.
|
UPA 5 mg
n=50 Participants
UPA 5 mg tablet plus matching placebo 10 mg tablet, orally, once daily for 12 weeks.
|
UPA 10 mg
n=43 Participants
UPA 10 mg tablet plus matching placebo 5 mg tablet, orally, once daily for 12 weeks.
|
|---|---|---|---|
|
Change From Baseline in Uterine Fibroid Symptom and Health-Related Quality of Life Questionnaire (UFS-QOL) Revised Activities Subscale Score at the End of Treatment Period
Baseline
|
34.51 score on a scale
Standard Deviation 26.41
|
29.60 score on a scale
Standard Deviation 23.34
|
28.15 score on a scale
Standard Deviation 23.39
|
|
Change From Baseline in Uterine Fibroid Symptom and Health-Related Quality of Life Questionnaire (UFS-QOL) Revised Activities Subscale Score at the End of Treatment Period
Change from Baseline at Week 12
|
18.26 score on a scale
Standard Deviation 27.52
|
53.00 score on a scale
Standard Deviation 34.36
|
61.04 score on a scale
Standard Deviation 31.99
|
Adverse Events
Placebo
Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths
UPA 5 mg
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
UPA 10 mg
Serious events: 2 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=56 participants at risk
Matching placebo tablet (5 mg and 10 mg), orally, once daily for 12 weeks.
|
UPA 5 mg
n=53 participants at risk
UPA 5 mg tablet plus matching placebo 10 mg tablet, orally, once daily for 12 weeks.
|
UPA 10 mg
n=48 participants at risk
UPA 10 mg tablet plus matching placebo 5 mg tablet, orally, once daily for 12 weeks.
|
|---|---|---|---|
|
Gastrointestinal disorders
Haemorrhoids
|
0.00%
0/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
0.00%
0/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
2.1%
1/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
|
Vascular disorders
Hypertension
|
0.00%
0/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
0.00%
0/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
2.1%
1/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
|
Infections and infestations
Influenza
|
0.00%
0/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
0.00%
0/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
2.1%
1/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
|
Cardiac disorders
Angina pectoris
|
1.8%
1/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
0.00%
0/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
0.00%
0/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
|
Psychiatric disorders
Anxiety
|
1.8%
1/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
0.00%
0/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
0.00%
0/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
|
Infections and infestations
Cellulitis
|
0.00%
0/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
1.9%
1/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
0.00%
0/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
Other adverse events
| Measure |
Placebo
n=56 participants at risk
Matching placebo tablet (5 mg and 10 mg), orally, once daily for 12 weeks.
|
UPA 5 mg
n=53 participants at risk
UPA 5 mg tablet plus matching placebo 10 mg tablet, orally, once daily for 12 weeks.
|
UPA 10 mg
n=48 participants at risk
UPA 10 mg tablet plus matching placebo 5 mg tablet, orally, once daily for 12 weeks.
|
|---|---|---|---|
|
Vascular disorders
Hypertension
|
0.00%
0/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
3.8%
2/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
6.2%
3/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
3.8%
2/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
6.2%
3/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
|
Vascular disorders
Hot flush
|
0.00%
0/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
5.7%
3/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
4.2%
2/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Acne
|
1.8%
1/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
0.00%
0/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
6.2%
3/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Nausea
|
5.4%
3/56 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
1.9%
1/53 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
2.1%
1/48 • From study drug administration until the end of the 12-week off-treatment follow-up period (Up to 24 weeks)
Safety Population consisted of all randomized participants who received at least one dose of study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee A disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER