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Treatment of Uterine Fibroids With the Selective Progesterone Receptor Modulator CDB-2914

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ClinicalTrials.gov Identifier: NCT00290251
Recruitment Status : Completed
First Posted : February 10, 2006
Results First Posted : December 18, 2012
Last Update Posted : December 18, 2012
Sponsor:
Collaborator:
HRA Pharma
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Leiomyoma
Interventions Drug: ulipristal acetate
Drug: placebo
Enrollment 72
Recruitment Details  
Pre-assignment Details Seventy-two women were screened for eligibility before being randomized to treatment 1. Of these only 42 were eligible to continue in the study.
Arm/Group Title Ulipristal Acetate - 20 mg Ulipristal Acetate- 10 mg Placebo (PLC)
Hide Arm/Group Description Women received ulipristal acetate at a daily dose of 20 mg for 90 - 102 days or three menstrual cycles. Women received ulipristal acetate at a daily dose of 10 mg for 90 - 102 days or three menstrual cycles. Women received placebo capsules for 90 - 102 days or three menstrual cycles.
Period Title: Treatment 1 (Months 1 - 3)
Started 14 14 14
Completed 13 13 12
Not Completed 1 1 2
Reason Not Completed
Withdrawal by Subject             0             0             2
headache             0             1             0
Out of body experience             1             0             0
Period Title: Treatment 2 (Months 4 - 6)
Started 8 [1] 5 [2] 0 [3]
Completed 6 5 0
Not Completed 2 0 0
Reason Not Completed
Lost to Follow-up             2             0             0
[1]
2 crossed over from placebo; others in treatment 1 PLC or ulipristal acetate 20 did not continue
[2]
2 crossed over from placebo; others in treatment 1 PLC or ulipristal acetate 10 grp did not continue
[3]
Women were randomized from PLC to ulipristal acetate or continued same dose ulipristal acetate
Period Title: Elective Extension (to 12 Months no Rx)
Started 5 [1] 3 [1] 5 [1]
Completed 5 3 5
Not Completed 0 0 0
[1]
Extension study was elective and observational only; treatment arm given as last study drug group
Arm/Group Title Eligible Women
Hide Arm/Group Description All women who consented and were eligible
Overall Number of Baseline Participants 42
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 42 participants
<=18 years
0
   0.0%
Between 18 and 65 years
42
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 42 participants
Female
42
 100.0%
Male
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 42 participants
42
1.Primary Outcome
Title Shrinkage of Fibroids - Size of Fibroids
Hide Description The primary outcome, fibroid volume, was calculated by an ellipsoid formula (π/6xd1xd2xd3) using orthogonal three-dimensional measurements taken from pelvic MRI scan. Individual volumes were summed to assess total fibroid volume for each woman, which were log-transformed before analysis. Women with paired MRI results were included in this intent to treat analysis, even if they did not take all study medication. Fibroids were included if they were seen on both studies.The absolute change in cm3 between baseline and end of treatment was calculated and its log was used for statistics and reporting the results in the data table below.
Time Frame 3 months (baseline to end of treatment)
Hide Outcome Measure Data
Hide Analysis Population Description
Per protocol, including women with two MRIs regardless of whether they took all study medication
Arm/Group Title Ulipristal Acetate -20mg Ulipristal Acetate - 10 mg Placebo
Hide Arm/Group Description:
Women received ulipristal acetate at 20 mg/day for 90 - 102 days or three menstrual cycles.
Women received ulipristal acetate at 10 mg/day for 90 - 102 days or three menstrual cycles.
Women received placebo capsules for 90 - 102 days or three menstrual cycles.
Overall Number of Participants Analyzed 13 13 12
Mean (Standard Error)
Unit of Measure: logcm3
-0.27  (0.07) -0.18  (0.08) 0.07  (0.78)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ulipristal Acetate -20mg, Ulipristal Acetate - 10 mg, Placebo
Comments Ulipristal acetate groups one and two were first compared and found to be similar, so they were combined into a single treatment group for comparison to placebo group
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.01
Comments [Not Specified]
Method t-test, 2 sided
Comments Performed on log-transformed delta change
2.Secondary Outcome
Title Quality of Life
Hide Description The short form-36 (SF-36)and Uterine Fibroid Symptom Quality of Life (UFS-QOL) questionnaires were given before and at treatment end with scales of 0 - 100. SF-36 scales = mental and physical well-being. The UFS subscales are symptom severity, concern, activities, energy and mood, control, self-consciousness, sexual functioning compiled into an overall QOL score. Higher results indicate better QOL on all but symptom severity (higher = worse). The change in scores from baseline to end of treatment was calculated.
Time Frame 3 months (Baseline to end of treatment 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All completers received a questionnaire at the end of the three-month study. One woman in the placebo group did not complete the questionnaire.
Arm/Group Title Ulipristal Acetate -10 and 20mg Placebo
Hide Arm/Group Description:
Women received ulipristal acetate at a daily dose of 10 or 20 mg for 90 - 102 days or three menstrual cycles.
Women received placebo capsules for 90 - 102 days or three menstrual cycles.
Overall Number of Participants Analyzed 26 11
Mean (Standard Error)
Unit of Measure: units on a scale
SF-36 Physical Component 4.2  (1.2) -1.5  (2.0)
SF-36 Mental Component 4.1  (1.5) -2.2  (2.4)
UFS Symptom Severity Score -28.3  (4.2) -4.2  (6.5)
UFS Overall health related quality of life 27.8  (3.6) 8.6  (5.6)
UFS Concern subscore 46.1  (4.5) 12.1  (6.9)
UFS Energy/mood subscore 19.2  (3.7) 3.7  (5.8)
UFS Control subscore 20.3  (4.3) 9.1  (6.8)
UFS self-conscious subscore 19.0  (4.7) 15.8  (7.5)
UFS sexual function subscore 25.7  (5.5) 18.7  (8.5)
UFS activities subscore 83.9  (4.4) 56.1  (7.0)
UFS composite bleeding subscore 2.1  (0.2) 0.4  (0.3)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ulipristal Acetate -10 and 20mg, Placebo
Comments No sample size calculation was made.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.05
Comments The Delta of scores from treatment end to baseline was used to compare by ANOVA.
Method ANOVA
Comments Adjustment for age
Time Frame During treatment periods including cross-over treatments with active compound. The number at risk is larger than the starting number in the treatment groups because of crossover additions to the treatment groups
Adverse Event Reporting Description Women recorded specific adverse events on a calendar; there was also a write-in field. The percentage of days in which any event was experienced was calculated for baseline and treatment. The difference between the two was considered the "true" incidence of events.
 
Arm/Group Title CDB-2914 -20mg CDB-2914 - 10 mg Placebo
Hide Arm/Group Description Women received CDB-2914 at a daily dose of 20 mg for 90 - 102 days or three menstrual cycles. Women received CDB-2914 at a daily dose of 10 mg for 90 - 102 days or three menstrual cycles. Women received placebo capsules for 90 - 102 days or three menstrual cycles.
All-Cause Mortality
CDB-2914 -20mg CDB-2914 - 10 mg Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Hide Serious Adverse Events
CDB-2914 -20mg CDB-2914 - 10 mg Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/16 (0.00%)      0/16 (0.00%)      0/14 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
CDB-2914 -20mg CDB-2914 - 10 mg Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/16 (43.75%)      13/16 (81.25%)      7/14 (50.00%)    
Gastrointestinal disorders       
Nausea/vomiting  [1]  0/16 (0.00%)  0 1/16 (6.25%)  61 0/14 (0.00%)  0
Decreased appetitie  [2]  0/16 (0.00%)  0 0/16 (0.00%)  0 1/14 (7.14%)  8
Diarrhea  [2]  0/16 (0.00%)  0 1/16 (6.25%)  12 0/14 (0.00%)  0
General disorders       
Abdominal pain  [2]  2/16 (12.50%)  120 1/16 (6.25%)  34 1/14 (7.14%)  8
Pelvic pain  [2]  1/16 (6.25%)  5 0/16 (0.00%)  0 1/14 (7.14%)  8
Fatigue  [2]  0/16 (0.00%)  0 2/16 (12.50%)  74 2/14 (14.29%)  28
Headache  [2]  2/16 (12.50%)  19 2/16 (12.50%)  23 2/14 (14.29%)  21
Abdominal cramps  [2]  0/16 (0.00%)  0 1/16 (6.25%)  18 0/14 (0.00%)  0
Bloating  [2]  0/16 (0.00%)  0 0/16 (0.00%)  0 1/14 (7.14%)  8
Leg swelling * [3]  0/16 (0.00%)  0 1/16 (6.25%)  47 0/14 (0.00%)  0
Increased appetite * [4]  0/16 (0.00%)  0 1/16 (6.25%)  62 0/14 (0.00%)  0
Depersonalization * [5]  1/16 (6.25%)  1 0/16 (0.00%)  0 0/14 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Joint Pain  [2]  3/16 (18.75%)  76 1/16 (6.25%)  19 1/14 (7.14%)  13
Calf or Thigh pain  [2]  2/16 (12.50%)  28 1/16 (6.25%)  6 0/14 (0.00%)  0
Back pain  [6]  0/16 (0.00%)  0 1/16 (6.25%)  21 0/14 (0.00%)  0
Psychiatric disorders       
Mood changes  [2]  0/16 (0.00%)  0 1/16 (6.25%)  15 1/14 (7.14%)  6
Reproductive system and breast disorders       
Vaginal discharge   2/16 (12.50%)  95 2/16 (12.50%)  37 1/14 (7.14%)  42
Non-menses Vaginal Bleeding  [2]  1/16 (6.25%)  21 4/16 (25.00%)  129 0/14 (0.00%)  0
Breast pain  [2]  1/16 (6.25%)  8 3/16 (18.75%)  91 3/14 (21.43%)  19
Skin and subcutaneous tissue disorders       
Skin Rash  [1]  1/16 (6.25%)  8 4/16 (25.00%)  81 1/14 (7.14%)  8
Hot flashes  [1]  2/16 (12.50%)  105 2/16 (12.50%)  92 1/14 (7.14%)  11
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
[1]
Self-reported via calendar
[2]
self-assessed via calendar
[3]
self-assessed via calendar and write-in option
[4]
self-assessed via calendar using write-in option
[5]
Out of body experience
[6]
self-assessed by calendar
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Lynnette Nieman MD
Organization: NICHD, NIH
Phone: 301-496-8935
EMail: NiemanL@nih.gov
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) )
ClinicalTrials.gov Identifier: NCT00290251    
Other Study ID Numbers: 060090
1ZIAHD000637-17 ( U.S. NIH Grant/Contract )
06-CH-0090
First Submitted: February 10, 2006
First Posted: February 10, 2006
Results First Submitted: October 28, 2011
Results First Posted: December 18, 2012
Last Update Posted: December 18, 2012