Dopamine Receptor Agonist Therapy for Pain Relief in Women Suffering From Endometriosis: A Pilot Study

• Sponsor: Boston Children's Hospital (Other)
• Overall Status: Completed
• CT.gov ID: NCT02542410
• Collaborator: (none)
• Enrollment: 10
• Location: 1
• Arms: 2
• Duration (Months): 28
• Patients Per Site Per Month: 0.4

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Endometriosis	Drug: Cabergoline; Drug: Norethindrone acetate	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 10 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Dopamine Receptor Agonist Therapy for Pain Relief in Women Suffering From Endometriosis: A Pilot Study
• Study Start Date: May 1, 2016
• Actual Primary Completion Date: Sep 1, 2018
• Actual Study Completion Date: Sep 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Control Norethindrone acetate 5 mg po daily x 6 months	Drug: Norethindrone acetate; Other Names: Aygestin
Experimental: Experimental cabergoline 0.5 mg PO twice weekly x 6 months	Drug: Cabergoline; Other Names: Dostinex

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. 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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 15 Years to 40 Years
• Sexes Eligible for Study: Female
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital Boston	Boston	Massachusetts	United States	02115

Sponsors and Collaborators

• Boston Children's Hospital

Investigators

None specified.

Study Documents (Full-Text)

• Study Protocol - Jun 11, 2019
• Statistical Analysis Plan - Jun 11, 2019

More Information

Publications

None provided.

• Responsible Party: Amy DiVasta, MD, Principal Investigator, Boston Children's Hospital
• ClinicalTrials.gov Identifier: NCT02542410
• Other Study ID Numbers: P00018628
• First Posted: Sep 7, 2015
• Last Update Posted: Feb 5, 2020
• Last Verified: Jan 1, 2020

• Additional relevant MeSH terms: Endometriosis; Cabergoline; Norethindrone; Norethindrone Acetate

Study Results

Participant Flow

Arm/Group Title	Norethindrone Acetate 5 mg Daily	Cabergoline 0.5 mg Twice Weekly
Arm/Group Description	Norethindrone acetate 5 mg po daily x 6 months Norethindrone acetate	cabergoline 0.5 mg PO twice weekly x 6 months Cabergoline
Period Title: Overall Study
STARTED	5	5
COMPLETED	3	4
NOT COMPLETED	2	1

Baseline Characteristics

Arm/Group Title	Control	Experimental	Total
Arm/Group Description	Norethindrone acetate 5 mg po daily x 6 months Norethindrone acetate	cabergoline 0.5 mg PO twice weekly x 6 months Cabergoline	Total of all reporting groups
Overall Participants	4	5	9
Age (years) [Median (Standard Deviation) ]
Median (Standard Deviation) [years]	25.2 (8.7)	24.8 (2.8)	24.8 (5.8)
Sex: Female, Male (Count of Participants)
Female	4 100%	5 100%	9 100%
Male	0 0%	0 0%	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	1 20%	1 11.1%
Asian	0 0%	0 0%	0 0%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%
Black or African American	2 50%	1 20%	3 33.3%
White	2 50%	3 60%	5 55.6%
More than one race	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%
Region of Enrollment (Count of Participants)
United States	4 100%	5 100%	9 100%

Outcome Measures

1. Primary Outcome

Title	Change in Score in Worst Pain Over the Last Month
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Norethindrone Acetate 5 mg	Cabergoline 0.5 mg
Arm/Group Description	Norethindrone acetate 5 mg po daily x 6 months Norethindrone acetate	cabergoline 0.5 mg PO twice weekly x 6 months Cabergoline
Measure Participants	4	5
Median (Standard Deviation) [score on a scale]	-0.5 (1.5)	-5 (2.5)

2. Secondary Outcome

Title	Changes in Pain Interference Scores
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Norethindrone Acetate 5 mg	Cabergoline 0.5 mg
Arm/Group Description	Norethindrone acetate 5 mg po daily x 6 months Norethindrone acetate	cabergoline 0.5 mg PO twice weekly x 6 months Cabergoline
Measure Participants	4	5
Mean (Standard Error) [units on a scale]	0.85 (1.1)	0 (1.1)

Adverse Events

Time Frame	6 MONTHS
Arm/Group Title	Control		Experimental
Arm/Group Description	Norethindrone acetate 5 mg po daily x 6 months Norethindrone acetate		cabergoline 0.5 mg PO twice weekly x 6 months Cabergoline
All Cause Mortality
	Control		Experimental
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/4 (0%)		0/5 (0%)
Serious Adverse Events
	Control		Experimental
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/4 (0%)		0/5 (0%)
Other (Not Including Serious) Adverse Events
	Control		Experimental
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	4/4 (100%)		5/5 (100%)
Gastrointestinal disorders
NAUSEA	1/4 (25%)	1	5/5 (100%)	5
General disorders
FATIGUE	1/4 (25%)	1	2/5 (40%)	5
Reproductive system and breast disorders
IRREGULAR VAGINAL BLEEDING	2/4 (50%)	2	2/5 (40%)	3

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT 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.