Trial Outcomes & Findings for IC PaIN Trial: Interstitial Cystitis Pain Improvement With Naltrexone (NCT NCT04313972)

NCT ID: NCT04313972

Last Updated: 2025-03-07

Results Overview

The effect of LDN (low-dose naltrexone) in decreasing symptoms associated with IC/PBS (interstitial cystitis/painful bladder syndrome) when treating with low-dose naltrexone as scored by the Interstitial Cystitis Symptom Index as compared to placebo. Scores are from 0 to 20, with lower scores indicating less symptoms associated with IC/PBS.

• Recruitment status: TERMINATED
• Study phase: PHASE4
• Target enrollment: 3 participants
• Primary outcome timeframe: six weeks
• Results posted on: 2025-03-07

Participant Flow

Participant milestones

Measure	Low-dose Naltrexone 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Overall Study STARTED	1	2
Overall Study COMPLETED	1	1
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Measure	Low-dose Naltrexone 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Overall Study Withdrawal by Subject	0	1

Baseline Characteristics

IC PaIN Trial: Interstitial Cystitis Pain Improvement With Naltrexone

Baseline characteristics by cohort

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=2 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks	Total n=3 Participants Total of all reporting groups
Age, Continuous	52 years n=5 Participants	52 years STANDARD_DEVIATION 8.5 • n=7 Participants	52 years STANDARD_DEVIATION 6 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	2 Participants n=7 Participants	3 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	1 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	2 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	1 Participants n=7 Participants	1 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	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 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	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: six weeks

Population: Excluding one participant who withdrew from the study.

The effect of LDN (low-dose naltrexone) in decreasing symptoms associated with IC/PBS (interstitial cystitis/painful bladder syndrome) when treating with low-dose naltrexone as scored by the Interstitial Cystitis Symptom Index as compared to placebo. Scores are from 0 to 20, with lower scores indicating less symptoms associated with IC/PBS.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Interstitial Cystitis Symptom Index	6 score on a scale	5 score on a scale

PRIMARY outcome

Timeframe: six weeks

Population: Excluding one participant who withdrew from the study.

The effect of LDN in decreasing pain associated with IC/PBS when treating with low-dose naltrexone as scored by visual analog scale as compared with placebo. Scores are from 0 to 10, with lower scores indicating less pain.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Visual Analog Scale	0 score on a scale	0 score on a scale

SECONDARY outcome

Timeframe: prior to initiating treatment and at the conclusion of 6 weeks of treatment

Population: Excluding one participant who withdrew from the study.

The changes in IC/PBS associated problems when treating interstitial cystitis/painful bladder syndrome with low-dose naltrexone as scored by the Interstitial Cystitis Problem Index, prior to initiating treatment and at the conclusion of 6 weeks of treatment. Scores are from 0-16, with lower scores indicating a better score, with fewer problems associated with IC/PBS. The change was calculated from two time points as the value prior to initiating treatment minus the value at the conclusion of 6 weeks of treatment.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Change in Interstitial Cystitis Problem Index	4 score on a scale	11 score on a scale

SECONDARY outcome

Timeframe: prior to initiating treatment and at the conclusion of 6 weeks of treatment

Population: Excluding one participant who withdrew from the study.

The change in number of patient voids during the day when comparing LDN against placebo as determined from 24 hour bladder diary performed by patient prior to initiating treatment and at the conclusion of 6 weeks of treatment. The change was calculated from two time points as the value prior to initiating treatment minus the value at the conclusion of 6 weeks of treatment.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Change in Urinary Frequency	8 voids per day	11 voids per day

SECONDARY outcome

Timeframe: prior to initiating treatment and at the conclusion of 6 weeks of treatment

Population: Excluding one participant who withdrew from the study.

The change in number of patient voids during at night when comparing LDN against placebo as determined from 24 hour bladder diary performed by patient prior to initiating treatment and at the conclusion of 6 weeks of treatment. The change was calculated from two time points as the value prior to initiating treatment minus the value at the conclusion of 6 weeks of treatment.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Change in Number of Nocturia	3 voids per night	2 voids per night

SECONDARY outcome

Timeframe: prior to initiating treatment and at the conclusion of 6 weeks of treatment

Population: Excluding one participant who withdrew from the study.

The change in pelvic pain and urgency/frequency symptoms as measured on the Pelvic Pain and Urgency/Frequency Patient Symptom Scale, prior to initiating treatment and at the conclusion of 6 weeks of treatment. Scores can range from 0-23, with higher scores indicating more severe symptoms associated with IC/PBS. The change was calculated from two time points as the value prior to initiating treatment minus the value at the conclusion of 6 weeks of treatment.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Change in Pelvic Pain and Urgency/Frequency Symptoms	3 score on a scale	7 score on a scale

SECONDARY outcome

Timeframe: prior to initiating treatment and at the conclusion of 6 weeks of treatment

Population: Excluding one participant who withdrew from the study.

The change in pelvic pain and urgency/frequency bother as measured on the Pelvic Pain and Urgency/Frequency Patient Symptom Scale, prior to initiating treatment and at the conclusion of 6 weeks of treatment. Scores can range from 0-12, with higher values corresponding to more bother from IC/PBS symptoms. The change was calculated from two time points as the value prior to initiating treatment minus the value at the conclusion of 6 weeks of treatment.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Change in Pelvic Pain and Urgency/Frequency Bother	1 score on a scale	7 score on a scale

SECONDARY outcome

Timeframe: six weeks

Population: Excluding one participant who withdrew from the study.

Patient perceived quality of life as measured by the medical outcomes study short form 36 (SF-36). Higher scores indicate a more favorable health status. There are 8 subscales, which when averaged, include a score from 0-100.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Patient Perceived Quality of Life: SF-36	86 score on a scale	84 score on a scale

SECONDARY outcome

Timeframe: six weeks

Population: Excluding one participant who withdrew from the study.

The percentage of patients complaining of adverse effects from LDN including vivid dreams, nightmares, insomnia, GI disturbances such as stomach cramps or diarrhea, agitation, anxiety, flu-like symptoms and headaches.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Adverse Effects	0 Participants	0 Participants

SECONDARY outcome

Timeframe: six weeks

Population: Excluding one participant who withdrew from the study.

The percentage of patients who discontinue the study medication measured by the tolerability survey.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Medication Tolerability	0 Participants	0 Participants

SECONDARY outcome

Timeframe: six weeks

Population: Excluding one participant who withdrew from the study.

The number of days with pain medication use while using LDN, as determined by a pain medication diary.

Outcome measures

Measure	Low-dose Naltrexone n=1 Participants 2mg low-dose naltrexone capsules low-dose naltrexone: 2mg of low-dose naltrexone nightly for 2 weeks, then 4mg of low-dose naltrexone nightly for 4 weeks	Placebo n=1 Participants Placebo capsules Placebo oral tablet: 1 capsule nighty for 2 weeks, then 2 capsules nightly for 4 weeks
Number of Days With Pain Medications	13 days	42 days

Adverse Events

Low-dose Naltrexone

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Urogynecology Research Coordinator

Endeavor Health

Phone: 847-570-4729

Email: JLee5@northshore.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place