Trial Outcomes & Findings for Low-Dose Naltrexone in Combination With Standard Treatment in Women With Endometriosis (NCT NCT03970330)
NCT ID: NCT03970330
Last Updated: 2022-11-03
Results Overview
Pain is reported daily using the Visual Analog Scale, a 100mm horizontal line on which the patient's pain intensity is represented by a point between the extremities of 0 (no pain) and 100 (worst pain). The final outcome measure will be calculated as area under the curve from randomization through 12-weeks of intervention. AUC was calculated using the trapezoid rule. Calculated AUC per subject across this 12-week period can range from 0 - 8300.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
9 participants
Primary outcome timeframe
12 weeks
Results posted on
2022-11-03
Participant Flow
Participant milestones
| Measure |
Low-Dose Naltrexone
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
|---|---|---|
|
Overall Study
STARTED
|
5
|
4
|
|
Overall Study
COMPLETED
|
5
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Low-Dose Naltrexone in Combination With Standard Treatment in Women With Endometriosis
Baseline characteristics by cohort
| Measure |
Low-Dose Naltrexone
n=5 Participants
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
n=4 Participants
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
30 years
STANDARD_DEVIATION 9 • n=5 Participants
|
27.5 years
STANDARD_DEVIATION 3.4 • n=7 Participants
|
28.9 years
STANDARD_DEVIATION 6.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 weeksPain is reported daily using the Visual Analog Scale, a 100mm horizontal line on which the patient's pain intensity is represented by a point between the extremities of 0 (no pain) and 100 (worst pain). The final outcome measure will be calculated as area under the curve from randomization through 12-weeks of intervention. AUC was calculated using the trapezoid rule. Calculated AUC per subject across this 12-week period can range from 0 - 8300.
Outcome measures
| Measure |
Low-Dose Naltrexone
n=5 Participants
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
n=4 Participants
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
|---|---|---|
|
Pain Score Area Under the Curve (AUC)
|
2461 units on a scale*days
Standard Deviation 1445
|
1338 units on a scale*days
Standard Deviation 697
|
SECONDARY outcome
Timeframe: Baseline, 4, 8, 12, and 16 weeksQuality of life measured by the validated Endometriosis Health Profile (EHP-30) questionnaire. Scores range from 0 (best health status) to 100 (worst health status).
Outcome measures
| Measure |
Low-Dose Naltrexone
n=5 Participants
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
n=4 Participants
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
|---|---|---|
|
EHP-30 Score
Week 12
|
34.1 score on a scale
Standard Deviation 26.6
|
18.6 score on a scale
Standard Deviation 15.8
|
|
EHP-30 Score
Week 16
|
35.7 score on a scale
Standard Deviation 28.0
|
28.9 score on a scale
Standard Deviation 23.3
|
|
EHP-30 Score
Baseline
|
56.5 score on a scale
Standard Deviation 18.3
|
27.6 score on a scale
Standard Deviation 19.4
|
|
EHP-30 Score
Week 4
|
35.5 score on a scale
Standard Deviation 16.6
|
25.1 score on a scale
Standard Deviation 17.1
|
|
EHP-30 Score
Week 8
|
34.9 score on a scale
Standard Deviation 22.8
|
16.0 score on a scale
Standard Deviation 8.0
|
SECONDARY outcome
Timeframe: 4, 8, 12 and 16 weeksPatient's Global Impression of Change (PGIC) scale will be used to rate painful periods. This is a 7 point scale ranging from 1 (much worse) to 7 (much better).
Outcome measures
| Measure |
Low-Dose Naltrexone
n=5 Participants
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
n=4 Participants
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
|---|---|---|
|
PGIC Score (Painful Periods)
4 weeks
|
4.0 units on a scale
Interval 4.0 to 4.0
|
6.5 units on a scale
Interval 5.5 to 7.0
|
|
PGIC Score (Painful Periods)
8 weeks
|
4.0 units on a scale
Interval 4.0 to 4.0
|
6.5 units on a scale
Interval 5.5 to 7.0
|
|
PGIC Score (Painful Periods)
12 weeks
|
4.0 units on a scale
Interval 4.0 to 4.0
|
6.5 units on a scale
Interval 6.0 to 7.0
|
|
PGIC Score (Painful Periods)
16 weeks
|
4.0 units on a scale
Interval 4.0 to 5.0
|
4.5 units on a scale
Interval 4.0 to 5.5
|
SECONDARY outcome
Timeframe: 4, 8, 12 and 16 weeksPatient's Global Impression of Change (PGIC) scale will be used to rate nonmenstrual pelvic pain. This is a 7 point scale ranging from 1 (much worse) to 7 (much better).
Outcome measures
| Measure |
Low-Dose Naltrexone
n=5 Participants
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
n=4 Participants
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
|---|---|---|
|
PGIC Score (Nonmenstrual Pelvic Pain)
Week 4
|
5.0 units on a scale
Interval 4.0 to 6.0
|
6.5 units on a scale
Interval 5.5 to 7.0
|
|
PGIC Score (Nonmenstrual Pelvic Pain)
Week 8
|
4.0 units on a scale
Interval 4.0 to 6.0
|
6.5 units on a scale
Interval 5.5 to 7.0
|
|
PGIC Score (Nonmenstrual Pelvic Pain)
Week 12
|
4.0 units on a scale
Interval 4.0 to 6.0
|
6.5 units on a scale
Interval 6.0 to 7.0
|
|
PGIC Score (Nonmenstrual Pelvic Pain)
Week 16
|
4.0 units on a scale
Interval 4.0 to 6.0
|
5.5 units on a scale
Interval 4.5 to 6.0
|
SECONDARY outcome
Timeframe: 4, 8, 12 and 16 weeksPopulation: This measure was only assessed in patients who had intercourse during study treatment.
Patient's Global Impression of Change (PGIC) scale will be used to rate pelvic pain during sex. This is a 7 point scale ranging from 1 (much worse) to 7 (much better).
Outcome measures
| Measure |
Low-Dose Naltrexone
n=2 Participants
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
n=3 Participants
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
|---|---|---|
|
PGIC Score (Dyspareunia)
Week 12
|
4.0 units on a scale
Interval 4.0 to 4.0
|
6.0 units on a scale
Interval 4.0 to 6.0
|
|
PGIC Score (Dyspareunia)
Week 16
|
4.0 units on a scale
Interval 4.0 to 4.0
|
5.0 units on a scale
Interval 4.0 to 7.0
|
|
PGIC Score (Dyspareunia)
Week 4
|
4.0 units on a scale
Interval 4.0 to 4.0
|
6.0 units on a scale
Interval 4.0 to 6.0
|
|
PGIC Score (Dyspareunia)
Week 8
|
4.0 units on a scale
Interval 4.0 to 4.0
|
6.0 units on a scale
Interval 4.0 to 7.0
|
SECONDARY outcome
Timeframe: 12 weeksAverage # of ibuprofen 200 mg pills per week during the study treatment period
Outcome measures
| Measure |
Low-Dose Naltrexone
n=5 Participants
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
n=4 Participants
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
|---|---|---|
|
Ibuprofen Use
|
5.5 pills/week
Standard Deviation 3.9
|
1.2 pills/week
Standard Deviation 1.3
|
SECONDARY outcome
Timeframe: 12 weeksNumber of subjects who used oxycodone at any time during the study
Outcome measures
| Measure |
Low-Dose Naltrexone
n=5 Participants
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
n=4 Participants
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
|---|---|---|
|
Oxycodone Use
|
0 Participants
|
0 Participants
|
Adverse Events
Low-Dose Naltrexone
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Low-Dose Naltrexone
n=5 participants at risk
12-week intervention period of 4.5 mg daily naltrexone in combination with standard treatment of 5-15 mg daily norethindrone acetate
Naltrexone: 4.5mg daily dose, taken orally
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
|
Placebo
n=4 participants at risk
12-week intervention period of daily placebo in combination with standard treatment of 5-15 mg daily norethindrone acetate
Norethindrone Acetate: 5 - 15mg daily dose, taken orally
Placebo: daily placebo pill, taken orally
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Skin sensitivity
|
0.00%
0/5 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
25.0%
1/4 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
Gastrointestinal disorders
Gastritis
|
20.0%
1/5 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
0.00%
0/4 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/5 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
25.0%
1/4 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/5 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
25.0%
1/4 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
Skin and subcutaneous tissue disorders
Acne
|
0.00%
0/5 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
25.0%
1/4 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
Skin and subcutaneous tissue disorders
Poison Ivy
|
0.00%
0/5 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
25.0%
1/4 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
Infections and infestations
Sinus infection
|
0.00%
0/5 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
25.0%
1/4 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
Infections and infestations
Strep throat
|
0.00%
0/5 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
25.0%
1/4 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
General disorders
Insomnia
|
0.00%
0/5 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
25.0%
1/4 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
General disorders
Night sweats
|
40.0%
2/5 • Number of events 2 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
25.0%
1/4 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
General disorders
Vivid dreams
|
20.0%
1/5 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
0.00%
0/4 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
General disorders
Headaches
|
20.0%
1/5 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
0.00%
0/4 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
|
Injury, poisoning and procedural complications
Leg contusion
|
20.0%
1/5 • Number of events 1 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
0.00%
0/4 • 16 weeks
Adverse event collection occurred every 4 weeks (during study visits).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place