Trial Outcomes & Findings for Fenofibrate Treatment in SCI (NCT NCT02455336)

Last Updated: 2019-06-26

Results Overview

To determine the efficacy of fenofibrate monotherapy after 2 months of treatment to improve the lipoprotein profile; a successful response will be defined as a 25% reduction in the serum TG concentration at 2 months.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

23 participants

Primary outcome timeframe

two months from initiating drug treatment

Results posted on

2019-06-26

Participant Flow

Participant milestones

Participant milestones
Measure	Fenofibrate Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive once daily fenofibrate therapy (i.e., 145 mg) for 4 months Fenofibrate: Fenofibrate is a peroxisome proliferator-activated receptor alpha agonist that is demonstrated to reduce triglyceride concentrations in the blood.	No Intervention Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive no therapy for 4 months No intervention: A cohort of participants will be randomized to receive no study drug, but will engage in study encounters.
Overall Study STARTED	15	8
Overall Study COMPLETED	10	6
Overall Study NOT COMPLETED	5	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Fenofibrate Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive once daily fenofibrate therapy (i.e., 145 mg) for 4 months Fenofibrate: Fenofibrate is a peroxisome proliferator-activated receptor alpha agonist that is demonstrated to reduce triglyceride concentrations in the blood.	No Intervention Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive no therapy for 4 months No intervention: A cohort of participants will be randomized to receive no study drug, but will engage in study encounters.
Overall Study Withdrawal by Subject	0	2
Overall Study Adverse Event	3	0
Overall Study Non-responder to treatment	2	0

Baseline Characteristics

Fenofibrate Treatment in SCI

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fenofibrate n=10 Participants Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive once daily fenofibrate therapy (i.e., 145 mg) for 4 months Fenofibrate: Fenofibrate is a peroxisome proliferator-activated receptor alpha agonist that is demonstrated to reduce triglyceride concentrations in the blood.	No Intervention n=8 Participants Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive no therapy for 4 months No intervention: A cohort of participants will be randomized to receive no study drug, but will engage in study encounters.	Total n=18 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants	8 Participants n=7 Participants	18 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	49 years STANDARD_DEVIATION 14 • n=5 Participants	44 years STANDARD_DEVIATION 13 • n=7 Participants	46 years STANDARD_DEVIATION 14 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants	8 Participants n=7 Participants	17 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	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants	8 Participants n=7 Participants	16 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: two months from initiating drug treatment

Outcome measures

Outcome measures
Measure	Fenofibrate n=10 Participants Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive once daily fenofibrate therapy (i.e., 145 mg) for 4 months Fenofibrate: Fenofibrate is a peroxisome proliferator-activated receptor alpha agonist that is demonstrated to reduce triglyceride concentrations in the blood.	No Intervention n=8 Participants Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive no therapy for 4 months No intervention: A cohort of participants will be randomized to receive no study drug, but will engage in study encounters.
Triglyceride Concentration (Percent Change From Baseline)	-40 percent change from baseline Standard Deviation 12	-2 percent change from baseline Standard Deviation 16

SECONDARY outcome

Timeframe: four months from initiating drug treatment

To determine the efficacy of fenofibrate monotherapy to lower TG concentration at 4 months of treatment, when the peak therapeutic efficacy to drug treatment has been reported to occur.

Outcome measures

Outcome measures
Measure	Fenofibrate n=10 Participants Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive once daily fenofibrate therapy (i.e., 145 mg) for 4 months Fenofibrate: Fenofibrate is a peroxisome proliferator-activated receptor alpha agonist that is demonstrated to reduce triglyceride concentrations in the blood.	No Intervention n=8 Participants Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive no therapy for 4 months No intervention: A cohort of participants will be randomized to receive no study drug, but will engage in study encounters.
Triglyceride Concentration (Percent Change From Baseline)	-40 percent change from baseline Standard Deviation 20	7 percent change from baseline Standard Deviation 14

OTHER_PRE_SPECIFIED outcome

Timeframe: 4 months

Documentation and description of adverse events will be obtained in subjects who have received drug treatment compared to events occurring in the control group.

Outcome measures

Outcome measures
Measure	Fenofibrate n=15 Participants Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive once daily fenofibrate therapy (i.e., 145 mg) for 4 months Fenofibrate: Fenofibrate is a peroxisome proliferator-activated receptor alpha agonist that is demonstrated to reduce triglyceride concentrations in the blood.	No Intervention n=8 Participants Subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive no therapy for 4 months No intervention: A cohort of participants will be randomized to receive no study drug, but will engage in study encounters.
Adverse Event Profile Elevated liver enzymes	2 Participants	0 Participants
Adverse Event Profile Gastrointestinal discomfort	2 Participants	0 Participants

Adverse Events

Fenofibrate

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

No Intervention

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Fenofibrate n=15 participants at risk Twenty subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive once daily fenofibrate therapy (i.e., 145 mg) for 4 months Fenofibrate: Fenofibrate is a peroxisome proliferator-activated receptor alpha agonist that is demonstrated to reduce triglyceride concentrations in the blood.	No Intervention n=8 participants at risk Ten subjects with adverse TG concentrations (i.e., paraplegia: \>/=135 mg/dl; tetraplegia \>/=115 mg/dl) will be randomized to receive no therapy for 4 months No intervention: A cohort of participants will be randomized to receive no study drug, but will engage in study encounters.
Hepatobiliary disorders Elevated liver enzymes	13.3% 2/15 • Number of events 2 • All blood sample results were reviewed by the study physician for the presence of adverse findings at each monthly visit during the 4 months of the treatment trial. The presence of an adverse finding in liver function tests, kidney function, CBC w/diff, or a change in the patient self-report health status were submitted to the Institutional Review Board (IRB) as an adverse event; the nature/severity of the event may have resulted in discontinuation of the subject from the study drug and continued participation in the trial, which was contingent upon the judgement of the study physician and the advice of the IRB.	0.00% 0/8 • All blood sample results were reviewed by the study physician for the presence of adverse findings at each monthly visit during the 4 months of the treatment trial. The presence of an adverse finding in liver function tests, kidney function, CBC w/diff, or a change in the patient self-report health status were submitted to the Institutional Review Board (IRB) as an adverse event; the nature/severity of the event may have resulted in discontinuation of the subject from the study drug and continued participation in the trial, which was contingent upon the judgement of the study physician and the advice of the IRB.
Gastrointestinal disorders Gastrointestinal discomfort	13.3% 2/15 • Number of events 2 • All blood sample results were reviewed by the study physician for the presence of adverse findings at each monthly visit during the 4 months of the treatment trial. The presence of an adverse finding in liver function tests, kidney function, CBC w/diff, or a change in the patient self-report health status were submitted to the Institutional Review Board (IRB) as an adverse event; the nature/severity of the event may have resulted in discontinuation of the subject from the study drug and continued participation in the trial, which was contingent upon the judgement of the study physician and the advice of the IRB.	0.00% 0/8 • All blood sample results were reviewed by the study physician for the presence of adverse findings at each monthly visit during the 4 months of the treatment trial. The presence of an adverse finding in liver function tests, kidney function, CBC w/diff, or a change in the patient self-report health status were submitted to the Institutional Review Board (IRB) as an adverse event; the nature/severity of the event may have resulted in discontinuation of the subject from the study drug and continued participation in the trial, which was contingent upon the judgement of the study physician and the advice of the IRB.

Additional Information

Michael F. La Fountaine, EdD, ATC, FACSM

James J. Peters VA Medical Center

Phone: 718-584-9000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place