Endometriosis
Conditions
Brief summary
The proposed pilot study will estimate the efficacy and safety of a novel therapeutic agent, cabergoline, with a clinical standard therapy, norethindrone acetate, for the treatment of endometriosis-associated pain in young women with endometriosis.
Interventions
DRUGCabergoline
Sponsors
Boston Children's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
FEMALE
Age
15 Years to 40 Years
Healthy volunteers
No
Inclusion criteria
* Surgically confirmed endometriosis * Age between 15-40 y, and premenopausal * Pelvic pain score ≥3 on a Visual Analog Scaleover the last month
Exclusion criteria
* Use of other concurrent hormone medications (such as birth control pills) * Impaired liver function (ALT \> 2x normal) or liver disease (cirrhosis, hepatitis) * Pregnancy * Breast cancer * Active thromboembolic disease * Uncontrolled hypertension, history of cardiac valve disorder, history of fibrotic disorders
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Score in Worst Pain Over the Last Month | Baseline, 6 months | visual analog scale, minimum=0 and maximum=10 Higher numbers are a worse outcome Outcome measures is calculated as the value at 6 months minus value at baseline. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in Pain Interference Scores | Baseline, 6 months | Brief Pain Inventory Interference subscale is a 7-item self-report measure, designed to assess the extent to which pain interferes with various components of functioning, including physical and emotional functioning and sleep.The items in this scale can be grouped into those that assess physical functioning (general activity; walking ability; normal work, including both work outside the home and housework), those that assess emotional functioning (mood; relations with people; enjoyment of life), and a single item that assess the extent to which pain interferes with sleep. The arithmetic mean of the seven interference items is used as a measure of pain interference (i.e., how much a participant's pain interferes with her ability to complete activities of daily living and functioning). The score on the pain interference subscale ranges from 0-70. Higher scores are worse outcomes. Outcome measure calculated as the value at 6 months minus the value at baseline |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Control Norethindrone acetate 5 mg po daily x 6 months
Norethindrone acetate | 4 |
| Experimental cabergoline 0.5 mg PO twice weekly x 6 months
Cabergoline | 5 |
| Total | 9 |
Baseline characteristics
| Characteristic | Control | Total | Experimental |
|---|---|---|---|
| Age, Continuous | 25.2 years STANDARD_DEVIATION 8.7 | 24.8 years STANDARD_DEVIATION 5.8 | 24.8 years STANDARD_DEVIATION 2.8 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 2 Participants | 3 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 2 Participants | 5 Participants | 3 Participants |
| Region of Enrollment United States | 4 Participants | 9 Participants | 5 Participants |
| Sex: Female, Male Female | 4 Participants | 9 Participants | 5 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 | 0 / 4 | 0 / 5 |
| other Total, other adverse events | 4 / 4 | 5 / 5 |
| serious Total, serious adverse events | 0 / 4 | 0 / 5 |
Outcome results
Primary
Change in Score in Worst Pain Over the Last Month
visual analog scale, minimum=0 and maximum=10 Higher numbers are a worse outcome Outcome measures is calculated as the value at 6 months minus value at baseline.
Time frame: Baseline, 6 months
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| Norethindrone Acetate 5 mg | Change in Score in Worst Pain Over the Last Month | -0.5 score on a scale | Standard Deviation 1.5 |
| Cabergoline 0.5 mg | Change in Score in Worst Pain Over the Last Month | -5 score on a scale | Standard Deviation 2.5 |
Secondary
Changes in Pain Interference Scores
Brief Pain Inventory Interference subscale is a 7-item self-report measure, designed to assess the extent to which pain interferes with various components of functioning, including physical and emotional functioning and sleep.The items in this scale can be grouped into those that assess physical functioning (general activity; walking ability; normal work, including both work outside the home and housework), those that assess emotional functioning (mood; relations with people; enjoyment of life), and a single item that assess the extent to which pain interferes with sleep. The arithmetic mean of the seven interference items is used as a measure of pain interference (i.e., how much a participant's pain interferes with her ability to complete activities of daily living and functioning). The score on the pain interference subscale ranges from 0-70. Higher scores are worse outcomes. Outcome measure calculated as the value at 6 months minus the value at baseline
Time frame: Baseline, 6 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norethindrone Acetate 5 mg | Changes in Pain Interference Scores | 0.85 units on a scale | Standard Error 1.1 |
| Cabergoline 0.5 mg | Changes in Pain Interference Scores | 0 units on a scale | Standard Error 1.1 |