Trial Outcomes & Findings for Pilot Study to Assess Dimethyl Fumarate Related GI Symptom Mitigation (NCT NCT02217982)

NCT ID: NCT02217982

Last Updated: 2017-03-23

Results Overview

The primary endpoint will be severity of GI events as measured by the MAGIS scale for subjects in the treatment arm compared to the standard therapy arm.

• Recruitment status: TERMINATED
• Study phase: PHASE4
• Target enrollment: 5 participants
• Primary outcome timeframe: 7 Weeks
• Results posted on: 2017-03-23

Participant Flow

Participant milestones

Measure	Control Group Patients randomized to the standard therapy arm will be instructed to follow the normal dosing regimen for DMF with a food bolus of their choice prior to dosing. If severe symptoms (MAGIS \>6.5) are noted at any time post randomization in any MAGIS category, crossover to the treatment arm will be allowed. Both groups will be asked to rate their GI symptoms over the past 24 hours using the MAGIS scale once daily.	Treatment Arm Patients who are randomized to the treatment arm will be instructed to take 125 mg simethicone and one tablespoon of a high fat food (peanut butter)10 minutes prior to each DMF dose. If the average MAGIS score is greater than 3.5 in the diarrhea category they will also be instructed to take 2 mg loperamide three times daily. Simethicone Loperamide Peanut Butter
Overall Study STARTED	1	4
Overall Study COMPLETED	0	3
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Measure	Control Group Patients randomized to the standard therapy arm will be instructed to follow the normal dosing regimen for DMF with a food bolus of their choice prior to dosing. If severe symptoms (MAGIS \>6.5) are noted at any time post randomization in any MAGIS category, crossover to the treatment arm will be allowed. Both groups will be asked to rate their GI symptoms over the past 24 hours using the MAGIS scale once daily.	Treatment Arm Patients who are randomized to the treatment arm will be instructed to take 125 mg simethicone and one tablespoon of a high fat food (peanut butter)10 minutes prior to each DMF dose. If the average MAGIS score is greater than 3.5 in the diarrhea category they will also be instructed to take 2 mg loperamide three times daily. Simethicone Loperamide Peanut Butter
Overall Study Trial discontinued	1	0
Overall Study Adverse Event	0	1

Baseline Characteristics

Pilot Study to Assess Dimethyl Fumarate Related GI Symptom Mitigation

Baseline characteristics by cohort

Measure	Control Group n=1 Participants Patients randomized to the standard therapy arm will be instructed to follow the normal dosing regimen for DMF with a food bolus of their choice prior to dosing. If severe symptoms (MAGIS \>6.5) are noted at any time post randomization in any MAGIS category, crossover to the treatment arm will be allowed. Both groups will be asked to rate their GI symptoms over the past 24 hours using the MAGIS scale once daily.	Treatment Arm n=4 Participants Patients who are randomized to the treatment arm will be instructed to take 125 mg simethicone and one tablespoon of a high fat food (peanut butter)10 minutes prior to each DMF dose. If the average MAGIS score is greater than 3.5 in the diarrhea category they will also be instructed to take 2 mg loperamide three times daily. Simethicone Loperamide Peanut Butter	Total n=5 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	1 Participants n=5 Participants	4 Participants n=7 Participants	5 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	4 Participants n=7 Participants	5 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	1 Participants n=5 Participants	4 Participants n=7 Participants	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: 7 Weeks

Population: Trial was shut down early as not enough patients qualified with GI symptoms their first 2 weeks after initiating DMF therapy

The primary endpoint will be severity of GI events as measured by the MAGIS scale for subjects in the treatment arm compared to the standard therapy arm.

Outcome measures

Measure	Control Group Patients randomized to the standard therapy arm will be instructed to follow the normal dosing regimen for DMF with a food bolus of their choice prior to dosing. If severe symptoms (MAGIS \>6.5) are noted at any time post randomization in any MAGIS category, crossover to the treatment arm will be allowed. Both groups will be asked to rate their GI symptoms over the past 24 hours using the MAGIS scale once daily.	Treatment Arm Patients who are randomized to the treatment arm will be instructed to take 125 mg simethicone and one tablespoon of a high fat food (peanut butter)10 minutes prior to each DMF dose. If the average MAGIS score is greater than 3.5 in the diarrhea category they will also be instructed to take 2 mg loperamide three times daily. Simethicone Loperamide Peanut Butter
Reported GI Symptoms	0	0

SECONDARY outcome

Timeframe: 7 Weeks

Population: Not enough patients qualified and the study was terminated early.

The secondary objective is to assess the reduction in diarrhea in the treatment group compared to the control.

Outcome measures

Measure	Control Group Patients randomized to the standard therapy arm will be instructed to follow the normal dosing regimen for DMF with a food bolus of their choice prior to dosing. If severe symptoms (MAGIS \>6.5) are noted at any time post randomization in any MAGIS category, crossover to the treatment arm will be allowed. Both groups will be asked to rate their GI symptoms over the past 24 hours using the MAGIS scale once daily.	Treatment Arm Patients who are randomized to the treatment arm will be instructed to take 125 mg simethicone and one tablespoon of a high fat food (peanut butter)10 minutes prior to each DMF dose. If the average MAGIS score is greater than 3.5 in the diarrhea category they will also be instructed to take 2 mg loperamide three times daily. Simethicone Loperamide Peanut Butter
Diarrhea Reduction	0	0

OTHER_PRE_SPECIFIED outcome

Timeframe: 7 Weeks

The tertiary objective is to gather further data regarding pre-existing conditions (GE reflex, gastric bypass, stomach ulcer, etc) and their possible relationship to GI symptoms when initiating DMF.

Outcome measures

Outcome data not reported

Adverse Events

Control Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Treatment Arm

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

Tammy Hoyt

Rocky Mountain MS Research Group

Phone: 8014085711

Email: thoyt@rmmsc.com

Results disclosure agreements

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