Trial Outcomes & Findings for Safety and Efficacy of Baclofen for Treatment of Muscle Spasms in Patients With Cirrhosis: A Pilot Study (NCT NCT02298868)

NCT ID: NCT02298868

Last Updated: 2015-02-05

Results Overview

Proportion of patients with headache at any time during the 4 weeks of therapy

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

4 weeks of active therapy

Results posted on

2015-02-05

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Overall Study STARTED	10
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Overall Study Adverse Event	1
Overall Study Death	1
Overall Study Patient underwent transplant	1

Baseline Characteristics

Safety and Efficacy of Baclofen for Treatment of Muscle Spasms in Patients With Cirrhosis: A Pilot Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Sex: Female, Male Female	5 Participants n=99 Participants
Sex: Female, Male Male	5 Participants n=99 Participants
Region of Enrollment United States	10 participants n=99 Participants

PRIMARY outcome

Timeframe: 4 weeks of active therapy

Proportion of patients with headache at any time during the 4 weeks of therapy

Outcome measures

Outcome measures
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability (Headache)	3 participants

PRIMARY outcome

Timeframe: 4 weeks of active therapy

Proportion of patients with nausea at any time during the 4 weeks of therapy

Outcome measures

Outcome measures
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability (Nausea)	4 participants

PRIMARY outcome

Timeframe: 4 weeks of active therapy

Proportion of patients with dizziness at any time during the 4 weeks of therapy

Outcome measures

Outcome measures
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability (Dizziness)	2 participants

PRIMARY outcome

Timeframe: 4 weeks of active therapy

Proportion of patients with endephalopathy at any time during the 4 weeks of therapy

Outcome measures

Outcome measures
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability (Encephalopathy)	0 participants

PRIMARY outcome

Timeframe: 4 weeks of active therapy

Proportion of patients with somnolence at any time during the 4 weeks of therapy

Outcome measures

Outcome measures
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability (Somnolence)	1 participants

SECONDARY outcome

Timeframe: Baseline to 4 weeks of therapy

Patients undertook a muscle cramp questionnaire prior to treatment that measured frequency in the number of days in a week that a subject experienced muscle cramps. This measures was repeated after 4 weeks of therapy and reported as the mean decrease in frequency of muscle cramps (4 weeks of therapy-Baseline).

Outcome measures

Outcome measures
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Efficacy of Baclofen to Change Frequency of Muscle Cramps in Patients With Cirrhosis at the End of 4 Weeks of Therapy	-4.4 days/week Standard Deviation 3.3

SECONDARY outcome

Timeframe: Baseline to end of 4 weeks of therapy

Patients undertook a muscle cramp questionnaire prior to treatment that measured severity in a 0-10 analog scale (0 is no pain and 10 is most severe pain). These measures were repeated after 4 weeks of therapy and the difference was assessed (4 weeks of therapy-Baseline).

Outcome measures

Outcome measures
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Efficacy of Baclofen to Change Severity of Muscle Cramps in Patients With Cirrhosis After 4 Weeks of Therapy	-6 units on a scale Standard Deviation 2.9

SECONDARY outcome

Timeframe: End of treatment (week 4) to end of washout (week 7)

Subjects undertook a muscle cramp questionnaire prior to treatment that measured frequency in the number of days in a week that a subject experienced muscle cramps and repeated this measure at the end of baclofen therapy (end of week 4). Subjects then underwent a 1 week taper of baclofen and a subsequent two week washout period. At the end of the washout period (end of week 7) the subjects underwent a final muscle cramp questionnaire to reassess frequency of muscle cramps. This was then compared to the end of therapy frequency to document the increase in muscle cramps after stopping baclofen. (week 7 result - week 4 result)

Outcome measures

Outcome measures
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Change in Frequency of Muscle Cramps After Washout Period	3.0 days/week Standard Deviation 3.9

SECONDARY outcome

Timeframe: End of treatment (week 4) to end of washout (week 7)

Subjects undertook a muscle cramp questionnaire prior to treatment that measured severity on a 0-10 analog scale (0 is no pain, 10 is most severe pain) and repeated this measure at the end of baclofen therapy (end of week 4). Subjects then underwent a 1 week taper of baclofen and a subsequent two week washout period. At the end of the washout period (end of week 7) the subjects underwent a final muscle cramp questionnaire to reassess severity of muscle cramps. This was then compared to the end of therapy severity to document the increase in muscle cramps after stopping baclofen. (week 7 result - week 4 result)

Outcome measures

Outcome measures
Measure	Treatment n=10 Participants All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Change in Severity of Muscle Cramps After Washout Period	4.0 units on a scale Standard Deviation 4.0

Adverse Events

Treatment

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Treatment n=10 participants at risk All patients will be administered a standing dose of Baclofen initially at 5 mg three times a day for the first week and then increased to 10 mg three times a day for the next 3 weeks with a tapering dose the final week, a total of 5 weeks of therapy.
Nervous system disorders Headache	30.0% 3/10
Gastrointestinal disorders Nausea	40.0% 4/10
Nervous system disorders Dizziness	20.0% 2/10
Nervous system disorders Somnolence	10.0% 1/10

Additional Information

Dr. Zachary Henry

University of Virginia

Phone: 434-243-2718

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place