Pelvic Pain Associated With Refractory Endometriosis
Conditions
Keywords
Menstrual pain, Refractory endometriosis, Pelvic pain
Brief summary
The purpose of this study is to determine efficacy, safety, tolerability and pharmacokinetics of multiple doses of BGS649 with concurrent daily administration of combined oral contraception in patients with refractory endometriosis
Interventions
DRUGBGS649
0.1 mg capsules for oral administration. A total dose of 3 capsules of BGS649 0.1mg will be administered at randomization, week 4, and week 8 at the study site.
DRUGPlacebo
Placebo matching BGS649 will be provided by Novartis as capsules for oral administration. Three capsules of placebo will be administered at randomization, week 4, and week 8 at the study site.
Sponsors
Novartis
Mereo BioPharma
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
FEMALE
Age
18 Years to 49 Years
Healthy volunteers
No
Inclusion criteria
* Must have surgical diagnosis of endometriosis est. w/in 5 years before screening. Subjects must have self reported moderate to severe pelvic pain at screening to be confirmed during the run-in-phase * Subjects with moderate to severe pelvic pain who were refractory to surgery (persistent or recurrent pelvic pain within 24 months after any therapeutic surgery) and at least two hormonal therapies from two different classes, including one approved hormonal therapy
Exclusion criteria
* Subjects who have undergone hysterectomy or bilateral oophorectomy. * Surgical treatment of endometriosis within 3 months before screening. * Subjects who are pregnant or who were pregnant within 3 months of visit one. * Subjects who are nursing or lactating * Subjects who are tobacco smokers. Other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Change in Baseline to Week 12 in Numeric Rating Scale Pelvic Pain Score | 12 weeks | Mean change from baseline to Month 3 (Week 12) in Number Rating Scale pelvic pain score. The Numeric Rating Scale is a scale between 0 and 10 where 0 is no pain and 10 is the worse pain you can imagine. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean Change From Baseline to 4 Weeks in Numeric Rating Scale Pelvic Pain Scores | 4 weeks | Mean change from baseline in Numeric Rating Scale pelvic pain scores to 4 weeks. The Numeric Rating Scale is a scale between 0 and 10 where 0 is no pain and 10 is the worse pain you can imagine. |
| Percentage of Patients Achieving a Response in Numeric Rating Scale Pelvic Pain Score | 12 weeks | The proportion (expressed as a percentage) of patients achieving a response in the Numeric Rating Scale pelvic pain score where a response is defined as greater than or equal to 2-point improvement from baseline or a greater than or equal to 30% improvement in the score. The Numeric Rating Scale is a scale between 0 and 10 where 0 is no pain and 10 is the worse pain you can imagine. |
| Mean Change From Baseline in Numeric Rating Scale Pelvic Pain Scores | 8 weeks | Mean change from baseline in Numeric Rating Scale pelvic pain scores to 8 weeks. Where the Numeric Rate Scale Pelvic Pain Score is a scale between 0 and 10, where 0 is no pain and is the worse pain you can imagine. |
| Percentage of Patients Achieving a Response in Their Pelvic Pain Score Measured on the Modified Biberoglu & Behrman Scale | 12 weeks | The proportion of patients (expressed as a percentage) achieving a response to their pelvic pain score measure on the Biberoglu and Behrman pelvic pain score where a response is defined as a greater than or equal to 1-point improvement from baseline. B & B scales allow for grading pain with a score between 0 and 4 with higher number being more severe pain. |
Countries
Puerto Rico, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| BGS649 Co-administered With Levora 28™ BGS649: 0.1 mg capsules for oral administration. A total dose of 3 capsules of BGS649 0.1mg administered at randomization, week 4, and week 8 at the study site. | 11 |
| Placebo Co-administered With Levora 28™ Placebo: Placebo matching BGS649 provided by Novartis as capsules for oral administration. Three capsules of placebo administered at randomization, week 4, and week 8 at the study site. | 16 |
| Total | 27 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Follow-up | Lost to Follow-up | 0 | 1 |
| Follow-up | Withdrawal by Subject | 0 | 1 |
| Treatment | Adverse Event | 0 | 1 |
| Treatment | Lack of Efficacy | 0 | 1 |
| Treatment | Lost to Follow-up | 1 | 1 |
| Treatment | Protocol Violation | 0 | 2 |
| Treatment | Withdrawal by Subject | 2 | 2 |
| Withdrawal | Lost to Follow-up | 0 | 1 |
| Withdrawal | Withdrawal by Subject | 0 | 1 |
Baseline characteristics
| Characteristic | BGS649 Co-administered With Levora 28™ | Placebo Co-administered With Levora 28™ | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 11 Participants | 16 Participants | 27 Participants |
| Age, Continuous | 29.4 years STANDARD_DEVIATION 5.75 | 29.4 years STANDARD_DEVIATION 5.67 | 29.4 years STANDARD_DEVIATION 5.59 |
| Body Mass Index | 27.9 kg/m^2 STANDARD_DEVIATION 4.98 | 25.6 kg/m^2 STANDARD_DEVIATION 4.11 | 26.6 kg/m^2 STANDARD_DEVIATION 4.54 |
| Race/Ethnicity, Customized Black | 1 Participants | 3 Participants | 4 Participants |
| Race/Ethnicity, Customized Caucasian | 5 Participants | 12 Participants | 17 Participants |
| Race/Ethnicity, Customized Other | 5 Participants | 1 Participants | 6 Participants |
| Region of Enrollment United States | 11 Participants | 16 Participants | 27 Participants |
| Sex: Female, Male Female | 11 Participants | 16 Participants | 27 Participants |
| Sex: Female, Male Male | 0 Participants | 0 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 10 / 12 | 10 / 15 |
| serious Total, serious adverse events | 1 / 12 | 2 / 15 |
Outcome results
Primary
Mean Change in Baseline to Week 12 in Numeric Rating Scale Pelvic Pain Score
Mean change from baseline to Month 3 (Week 12) in Number Rating Scale pelvic pain score. The Numeric Rating Scale is a scale between 0 and 10 where 0 is no pain and 10 is the worse pain you can imagine.
Time frame: 12 weeks
Population: Full analysis set. All patients randomised excluding those who were randomised inadvertently and did not receive study drug
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| BGS649 Co-administered With Levora 28™ | Mean Change in Baseline to Week 12 in Numeric Rating Scale Pelvic Pain Score | -0.7637 units on a scale | Standard Deviation 2.12386 |
| Placebo Co-administered With Levora 28™ | Mean Change in Baseline to Week 12 in Numeric Rating Scale Pelvic Pain Score | -2.1706 units on a scale | Standard Deviation 2.27246 |
Secondary
Mean Change From Baseline in Numeric Rating Scale Pelvic Pain Scores
Mean change from baseline in Numeric Rating Scale pelvic pain scores to 8 weeks. Where the Numeric Rate Scale Pelvic Pain Score is a scale between 0 and 10, where 0 is no pain and is the worse pain you can imagine.
Time frame: 8 weeks
Population: Full Analysis Set. All randomized patients excluding patients randomized inadvertently who did not receive drug (ITT)
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| BGS649 Co-administered With Levora 28™ | Mean Change From Baseline in Numeric Rating Scale Pelvic Pain Scores | -1.2840 score on a scale | Standard Deviation 2.18569 |
| Placebo Co-administered With Levora 28™ | Mean Change From Baseline in Numeric Rating Scale Pelvic Pain Scores | -2.0601 score on a scale | Standard Deviation 2.1181 |
Secondary
Mean Change From Baseline to 4 Weeks in Numeric Rating Scale Pelvic Pain Scores
Mean change from baseline in Numeric Rating Scale pelvic pain scores to 4 weeks. The Numeric Rating Scale is a scale between 0 and 10 where 0 is no pain and 10 is the worse pain you can imagine.
Time frame: 4 weeks
Population: Full Analysis Set. All randomized patients excluding patients randomized inadvertently who did not receive drug (ITT)
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| BGS649 Co-administered With Levora 28™ | Mean Change From Baseline to 4 Weeks in Numeric Rating Scale Pelvic Pain Scores | -0.6596 score on a scale | Standard Deviation 1.34546 |
| Placebo Co-administered With Levora 28™ | Mean Change From Baseline to 4 Weeks in Numeric Rating Scale Pelvic Pain Scores | -0.9282 score on a scale | Standard Deviation 1.05794 |
Secondary
Percentage of Patients Achieving a Response in Numeric Rating Scale Pelvic Pain Score
The proportion (expressed as a percentage) of patients achieving a response in the Numeric Rating Scale pelvic pain score where a response is defined as greater than or equal to 2-point improvement from baseline or a greater than or equal to 30% improvement in the score. The Numeric Rating Scale is a scale between 0 and 10 where 0 is no pain and 10 is the worse pain you can imagine.
Time frame: 12 weeks
Population: Full Analysis Set. All participants randomized except patients inadvertently randomized who didn't receive study drug (ITT principles)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| BGS649 Co-administered With Levora 28™ | Percentage of Patients Achieving a Response in Numeric Rating Scale Pelvic Pain Score | 9.1 percentage of participants |
| Placebo Co-administered With Levora 28™ | Percentage of Patients Achieving a Response in Numeric Rating Scale Pelvic Pain Score | 37.5 percentage of participants |
Secondary
Percentage of Patients Achieving a Response in Their Pelvic Pain Score Measured on the Modified Biberoglu & Behrman Scale
The proportion of patients (expressed as a percentage) achieving a response to their pelvic pain score measure on the Biberoglu and Behrman pelvic pain score where a response is defined as a greater than or equal to 1-point improvement from baseline. B & B scales allow for grading pain with a score between 0 and 4 with higher number being more severe pain.
Time frame: 12 weeks
Population: Full Analysis Set. All participants randomized except patients inadvertently randomized who didn't receive study drug (ITT principles)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| BGS649 Co-administered With Levora 28™ | Percentage of Patients Achieving a Response in Their Pelvic Pain Score Measured on the Modified Biberoglu & Behrman Scale | 0 percentage of participants |
| Placebo Co-administered With Levora 28™ | Percentage of Patients Achieving a Response in Their Pelvic Pain Score Measured on the Modified Biberoglu & Behrman Scale | 31.3 percentage of participants |