Trial Outcomes & Findings for Effects of Riluzole on CNS Glutamate and Fatigue in Breast Cancer Survivors With High Inflammation (NCT NCT02796755)

NCT ID: NCT02796755

Last Updated: 2021-04-14

Results Overview

Magnetic resonance spectroscopy (MRS) is a specialized technique associated with magnetic resonance imaging (MRI). MRS is a non-invasive way to obtain biochemical information about the tissues of the human body. Participants underwent single voxel (3-dimensional volume X pixel) MRS to measure CNS glutamate before and after 1 and 8 weeks of riluzole or placebo treatment. Voxels were placed in the right and left basal ganglia and the dorsal anterior cingulate cortex (dACC), well known targets of inflammatory cytokines on the brain, and cytokine effects on these brain regions have been associated with symptoms of fatigue and cognitive dysfunction as well as reduced motivation. MRS has shown that chronic exposure to the inflammatory cytokine interferon (IFN)-alpha leads to increased CNS glutamate (as reflected by the glutamate/creatine (Glu/Cr) ratio) which correlated with symptoms of fatigue and cognitive dysfunction.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

30 participants

Primary outcome timeframe

Baseline, Week 1, Week 8

Results posted on

2021-04-14

Participant Flow

Participants were enrolled beginning April 2016 and all follow-up visits were complete by October 24, 2019. Participants were enrolled from the patient population at the Emory University Winship Cancer Institute in Atlanta, Georgia.

Participant milestones

Participant milestones
Measure
Riluzole Arm
Participants took a daily oral dose of 100 mg of riluzole (50 mg two times per day) for 8 weeks. Participants were instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Placebo Arm
Participants took a daily oral dose of 100 mg of a placebo that appears identical to riluzole (50 mg two times per day), for 8 weeks. Participants will be instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Overall Study
STARTED
15
15
Overall Study
COMPLETED
13
15
Overall Study
NOT COMPLETED
2
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Riluzole Arm
Participants took a daily oral dose of 100 mg of riluzole (50 mg two times per day) for 8 weeks. Participants were instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Placebo Arm
Participants took a daily oral dose of 100 mg of a placebo that appears identical to riluzole (50 mg two times per day), for 8 weeks. Participants will be instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Overall Study
Adverse Event
2
0

Baseline Characteristics

Effects of Riluzole on CNS Glutamate and Fatigue in Breast Cancer Survivors With High Inflammation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Riluzole Arm
n=15 Participants
Participants took a daily oral dose of 100 mg of riluzole (50 mg two times per day) for 8 weeks. Participants were instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Placebo Arm
n=15 Participants
Participants took a daily oral dose of 100 mg of a placebo that appears identical to riluzole (50 mg two times per day), for 8 weeks. Participants will be instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Total
n=30 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Age, Categorical
Between 18 and 65 years
15 Participants
n=99 Participants
15 Participants
n=107 Participants
30 Participants
n=206 Participants
Age, Categorical
>=65 years
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Age, Continuous
54.2 years
STANDARD_DEVIATION 6.7 • n=99 Participants
53.7 years
STANDARD_DEVIATION 5.3 • n=107 Participants
53.9 years
STANDARD_DEVIATION 5.9 • n=206 Participants
Sex: Female, Male
Female
15 Participants
n=99 Participants
15 Participants
n=107 Participants
30 Participants
n=206 Participants
Sex: Female, Male
Male
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
15 Participants
n=99 Participants
15 Participants
n=107 Participants
30 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Black or African American
11 Participants
n=99 Participants
7 Participants
n=107 Participants
18 Participants
n=206 Participants
Race (NIH/OMB)
White
4 Participants
n=99 Participants
8 Participants
n=107 Participants
12 Participants
n=206 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Region of Enrollment
United States
15 Participants
n=99 Participants
15 Participants
n=107 Participants
30 Participants
n=206 Participants

PRIMARY outcome

Timeframe: Baseline, Week 1, Week 8

Magnetic resonance spectroscopy (MRS) is a specialized technique associated with magnetic resonance imaging (MRI). MRS is a non-invasive way to obtain biochemical information about the tissues of the human body. Participants underwent single voxel (3-dimensional volume X pixel) MRS to measure CNS glutamate before and after 1 and 8 weeks of riluzole or placebo treatment. Voxels were placed in the right and left basal ganglia and the dorsal anterior cingulate cortex (dACC), well known targets of inflammatory cytokines on the brain, and cytokine effects on these brain regions have been associated with symptoms of fatigue and cognitive dysfunction as well as reduced motivation. MRS has shown that chronic exposure to the inflammatory cytokine interferon (IFN)-alpha leads to increased CNS glutamate (as reflected by the glutamate/creatine (Glu/Cr) ratio) which correlated with symptoms of fatigue and cognitive dysfunction.

Outcome measures

Outcome measures
Measure
Riluzole Arm
n=15 Participants
Participants took a daily oral dose of 100 mg of riluzole (50 mg two times per day) for 8 weeks. Participants were instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Placebo Arm
n=15 Participants
Participants took a daily oral dose of 100 mg of a placebo that appears identical to riluzole (50 mg two times per day), for 8 weeks. Participants will be instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Central Nervous System (CNS) Glutamate Measured by Magnetic Resonance Spectroscopy (MRS)
Baseline dorsal anterior cingulate cortex (dACC)
1.26 Glu/Cr
Standard Deviation 0.09
1.2 Glu/Cr
Standard Deviation 0.15
Central Nervous System (CNS) Glutamate Measured by Magnetic Resonance Spectroscopy (MRS)
Week 1 dACC
1.26 Glu/Cr
Standard Deviation 1.26
1.27 Glu/Cr
Standard Deviation 0.12
Central Nervous System (CNS) Glutamate Measured by Magnetic Resonance Spectroscopy (MRS)
Week 8 dACC
1.27 Glu/Cr
Standard Deviation 0.12
1.27 Glu/Cr
Standard Deviation 0.07
Central Nervous System (CNS) Glutamate Measured by Magnetic Resonance Spectroscopy (MRS)
Baseline left basal ganglia
0.85 Glu/Cr
Standard Deviation 0.09
0.91 Glu/Cr
Standard Deviation 0.12
Central Nervous System (CNS) Glutamate Measured by Magnetic Resonance Spectroscopy (MRS)
Week 1 left basal ganglia
0.93 Glu/Cr
Standard Deviation 0.16
0.96 Glu/Cr
Standard Deviation 0.13
Central Nervous System (CNS) Glutamate Measured by Magnetic Resonance Spectroscopy (MRS)
Week 8 left basal ganglia
0.96 Glu/Cr
Standard Deviation 0.2
0.98 Glu/Cr
Standard Deviation 0.11
Central Nervous System (CNS) Glutamate Measured by Magnetic Resonance Spectroscopy (MRS)
Baseline right basal ganglia
0.9 Glu/Cr
Standard Deviation 0.09
0.92 Glu/Cr
Standard Deviation 0.14
Central Nervous System (CNS) Glutamate Measured by Magnetic Resonance Spectroscopy (MRS)
Week 1 right basal ganglia
0.9 Glu/Cr
Standard Deviation 0.08
0.89 Glu/Cr
Standard Deviation 0.11
Central Nervous System (CNS) Glutamate Measured by Magnetic Resonance Spectroscopy (MRS)
Week 8 right basal ganglia
0.93 Glu/Cr
Standard Deviation 0.13
0.93 Glu/Cr
Standard Deviation 0.1

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 2, 4, 8

The Multidimensional Fatigue Inventory (MFI) is a 20-item scale used to evaluate the presence and severity of fatigue among subjects by self-reports. The MFI assesses 5 dimensions of fatigue, including general fatigue, physical fatigue, mental fatigue, reduced activity, and reduced motivation. Participants respond to fatigue related statements using a 5 point scale where 1 = "yes, that is true" and 5 = "no, that is not true". Total scores range from 20 to 100 and higher scores indicate greater fatigue.

Outcome measures

Outcome measures
Measure
Riluzole Arm
n=15 Participants
Participants took a daily oral dose of 100 mg of riluzole (50 mg two times per day) for 8 weeks. Participants were instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Placebo Arm
n=15 Participants
Participants took a daily oral dose of 100 mg of a placebo that appears identical to riluzole (50 mg two times per day), for 8 weeks. Participants will be instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Multidimensional Fatigue Inventory (MFI) Score
Week 8
58.92 score on a scale
Standard Deviation 16.47
46.50 score on a scale
Standard Deviation 13.85
Multidimensional Fatigue Inventory (MFI) Score
Baseline
66.90 score on a scale
Standard Deviation 10.99
58.90 score on a scale
Standard Deviation 13.86
Multidimensional Fatigue Inventory (MFI) Score
Week 1
63.80 score on a scale
Standard Deviation 12.10
53.50 score on a scale
Standard Deviation 15.80
Multidimensional Fatigue Inventory (MFI) Score
Week 2
62.00 score on a scale
Standard Deviation 14.60
53.21 score on a scale
Standard Deviation 16.85
Multidimensional Fatigue Inventory (MFI) Score
Week 4
61.07 score on a scale
Standard Deviation 12.40
51.14 score on a scale
Standard Deviation 15.64

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 2, 4, 8

PROMIS-Fatigue Short Form is a 7-item scale developed by the Patient-Reported Outcome Measurement Information System (PROMIS), a part of the NIH Roadmap Initiative which is focused on developing a publicly available resource of standardized, accurate, and efficient outcome measures of symptoms, distress, and functioning. The criterion for a minimally clinically important difference in patients with advanced-stage cancer is a 3 to 5 point difference in raw score. Recommendations for high priority research on cancer-related fatigue recommend use of the PROMIS fatigue scale to allow comparison of results across studies. Respondents indicate how much they agree with the item statements on a scale from 1 (not at all) to 5 (very much). Total scores range from 7 to 35 with higher scores indicating greater fatigue.

Outcome measures

Outcome measures
Measure
Riluzole Arm
n=15 Participants
Participants took a daily oral dose of 100 mg of riluzole (50 mg two times per day) for 8 weeks. Participants were instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Placebo Arm
n=15 Participants
Participants took a daily oral dose of 100 mg of a placebo that appears identical to riluzole (50 mg two times per day), for 8 weeks. Participants will be instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Patient-Reported Outcomes Measurement Information System (PROMIS) - Fatigue Short Form Score
Baseline
25.1 score on a scale
Standard Deviation 3.8
21.8 score on a scale
Standard Deviation 5.0
Patient-Reported Outcomes Measurement Information System (PROMIS) - Fatigue Short Form Score
Week 1
22.5 score on a scale
Standard Deviation 4.3
18.71 score on a scale
Standard Deviation 5.06
Patient-Reported Outcomes Measurement Information System (PROMIS) - Fatigue Short Form Score
Week 2
23.7 score on a scale
Standard Deviation 5.5
17.07 score on a scale
Standard Deviation 6.18
Patient-Reported Outcomes Measurement Information System (PROMIS) - Fatigue Short Form Score
Week 4
21.6 score on a scale
Standard Deviation 5.8
17.36 score on a scale
Standard Deviation 6.07
Patient-Reported Outcomes Measurement Information System (PROMIS) - Fatigue Short Form Score
Week 8
18.8 score on a scale
Standard Deviation 6.1
15.07 score on a scale
Standard Deviation 6.11

Adverse Events

Riluzole Arm

Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths

Placebo Arm

Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Riluzole Arm
n=15 participants at risk
Participants took a daily oral dose of 100 mg of riluzole (50 mg two times per day) for 8 weeks. Participants were instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
Placebo Arm
n=15 participants at risk
Participants took a daily oral dose of 100 mg of a placebo that appears identical to riluzole (50 mg two times per day), for 8 weeks. Participants will be instructed to take the study medication on an empty stomach (1 hour before or 2 hours after meals).
General disorders
Headache
26.7%
4/15 • Number of events 4 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
13.3%
2/15 • Number of events 2 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
Gastrointestinal disorders
Gastrointestinal distress
40.0%
6/15 • Number of events 6 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
20.0%
3/15 • Number of events 3 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
Skin and subcutaneous tissue disorders
Itch/Rash
13.3%
2/15 • Number of events 2 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
0.00%
0/15 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
General disorders
Fatigue
6.7%
1/15 • Number of events 1 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
0.00%
0/15 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
General disorders
Irritability
6.7%
1/15 • Number of events 1 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
6.7%
1/15 • Number of events 1 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
General disorders
Paresthesia
0.00%
0/15 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
6.7%
1/15 • Number of events 1 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
General disorders
Increased white blood cell count
0.00%
0/15 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
6.7%
1/15 • Number of events 1 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
Surgical and medical procedures
Venipuncture complications
13.3%
2/15 • Number of events 2 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.
0.00%
0/15 • Information on adverse events was collected from the time consent to participate in the study was given through the Week 8 study visit.

Additional Information

Andrew H Miller, MD

Emory University

Phone: 404-727-8260

Results disclosure agreements

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