Trial Outcomes & Findings for Efficacy of Open Label Placebo in Children With FGIDs (NCT NCT02389998)

Last Updated: 2021-07-23

Results Overview

Change in mean pain score comparing both treatment arms to the baseline using the Visual analogue scale. (Scale 0-100mm) The scale reflects severity of the pain going from no pain (0) to maximum pain (100mm). Therefore the higher the number the more severe the pain is

Recruitment status

COMPLETED

Study phase

Target enrollment

31 participants

Primary outcome timeframe

It will be assessed at the end of the 3-week and 6-week treatment periods (at the end of each treatment arm prior to crossover to the next arm of treatment)

Results posted on

2021-07-23

Participant Flow

Cross over study

Participant milestones

Participant milestones
Measure	Placebo First Crossover to no Treatment After 3 Weeks Arm 1: Subjects take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary for a period of three weeks. Subjects will also have access to hyoscyamine as a rescue medication. Placebo Suspension: The study is divided into three phases: 1 one-week baseline assessment followed by 2 three-week study phases (phase A and phase B). Phase A will require subjects to take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary. In phase B subjects will not take the placebo. After 3 weeks in initial phase (either Phase A or B), subjects will switch to the alternate phase and continue the study for another 3 weeks. Hyoscyamine is available as a rescue medication during Phase A and Phase B. Half of the subjects will be randomized to begin with Phase A and half will be randomized to begin with Phase B. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.	No Treatment First Crossover to Placebo After 3 Weeks Arm 2: Subjects receive no treatment but have access to hyoscyamine as a rescue medication. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.
Overall Study STARTED	16	15
Overall Study COMPLETED	15	15
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo First Crossover to no Treatment After 3 Weeks Arm 1: Subjects take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary for a period of three weeks. Subjects will also have access to hyoscyamine as a rescue medication. Placebo Suspension: The study is divided into three phases: 1 one-week baseline assessment followed by 2 three-week study phases (phase A and phase B). Phase A will require subjects to take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary. In phase B subjects will not take the placebo. After 3 weeks in initial phase (either Phase A or B), subjects will switch to the alternate phase and continue the study for another 3 weeks. Hyoscyamine is available as a rescue medication during Phase A and Phase B. Half of the subjects will be randomized to begin with Phase A and half will be randomized to begin with Phase B. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.	No Treatment First Crossover to Placebo After 3 Weeks Arm 2: Subjects receive no treatment but have access to hyoscyamine as a rescue medication. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.
Overall Study car accident	1	0

Baseline Characteristics

Efficacy of Open Label Placebo in Children With FGIDs

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo First Followed by no Treatment n=15 Participants Arm 1: Subjects take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary for a period of three weeks. Subjects will also have access to hyoscyamine as a rescue medication. Placebo Suspension: The study is divided into three phases: 1 one-week baseline assessment followed by 2 three-week study phases (phase A and phase B). Phase A will require subjects to take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary. In phase B subjects will not take the placebo. After 3 weeks in initial phase (either Phase A or B), subjects will switch to the alternate phase and continue the study for another 3 weeks. Hyoscyamine is available as a rescue medication during Phase A and Phase B. Half of the subjects will be randomized to begin with Phase A and half will be randomized to begin with Phase B. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.	No Treatment First Followed by Placebo n=15 Participants Arm 2: Subjects receive no treatment but have access to hyoscyamine as a rescue medication. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.	Total n=30 Participants Total of all reporting groups
Age, Categorical <=18 years	15 Participants n=5 Participants	15 Participants n=7 Participants	30 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 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	13 Participants n=5 Participants	12 Participants n=7 Participants	25 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	3 Participants n=7 Participants	5 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	1 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) White	13 Participants n=5 Participants	14 Participants n=7 Participants	27 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
Functional abdominal pain	15 Participants n=5 Participants	15 Participants n=7 Participants	30 Participants n=5 Participants

PRIMARY outcome

Timeframe: It will be assessed at the end of the 3-week and 6-week treatment periods (at the end of each treatment arm prior to crossover to the next arm of treatment)

Population: A total of 30 patients completed the study. All of them received placebo or no treatment in a randomized crossover study. All measurements during placebo are combined. All measurements during no treatment are combined. Statistcal models were adjusted for order of treatment

Outcome measures

Outcome measures
Measure	Placebo n=30 Participants Arm 1: Subjects take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary for a period of three weeks. Subjects will also have access to hyoscyamine as a rescue medication. Placebo Suspension: The study is divided into three phases: 1 one-week baseline assessment followed by 2 three-week study phases (phase A and phase B). Phase A will require subjects to take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary. In phase B subjects will not take the placebo. After 3 weeks in initial phase (either Phase A or B), subjects will switch to the alternate phase and continue the study for another 3 weeks. Hyoscyamine is available as a rescue medication during Phase A and Phase B. Half of the subjects will be randomized to begin with Phase A and half will be randomized to begin with Phase B. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.	No Treatment n=30 Participants Arm 2: Subjects receive no treatment but have access to hyoscyamine as a rescue medication. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.
The Primary Outcome Measure Was Mean Daily Pain	39.9 pain scale by VAS in mm Standard Deviation 18	45.14 pain scale by VAS in mm Standard Deviation 14.7

SECONDARY outcome

Timeframe: 3 weeks of placebo vs 3 weeks of no treatment

Population: All patients

The number of medications used as rescue during each one of the periods of the study were counted

Outcome measures

Outcome measures
Measure	Placebo n=30 Participants Arm 1: Subjects take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary for a period of three weeks. Subjects will also have access to hyoscyamine as a rescue medication. Placebo Suspension: The study is divided into three phases: 1 one-week baseline assessment followed by 2 three-week study phases (phase A and phase B). Phase A will require subjects to take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary. In phase B subjects will not take the placebo. After 3 weeks in initial phase (either Phase A or B), subjects will switch to the alternate phase and continue the study for another 3 weeks. Hyoscyamine is available as a rescue medication during Phase A and Phase B. Half of the subjects will be randomized to begin with Phase A and half will be randomized to begin with Phase B. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.	No Treatment n=30 Participants Arm 2: Subjects receive no treatment but have access to hyoscyamine as a rescue medication. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.
Use of Rescue Medications	2.0 pill count number Standard Error 2.95	3.8 pill count number Standard Error 5.05

SECONDARY outcome

Timeframe: Following 1-week baseline and 3-week and 6-week treatment periods

Compared clinical global improvement during each phase of the study The following question was used: Overall, how do you feel your problem is? (better, same, or worse)." Patients were then divided in 2 groups: improved (if they answered better) vs not improved (if they answered same/worse)

Outcome measures

Outcome measures
Measure	Placebo n=30 Participants Arm 1: Subjects take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary for a period of three weeks. Subjects will also have access to hyoscyamine as a rescue medication. Placebo Suspension: The study is divided into three phases: 1 one-week baseline assessment followed by 2 three-week study phases (phase A and phase B). Phase A will require subjects to take 1/4 teaspoon placebo suspension 2 times a day (morning and night), and a third dose if necessary. In phase B subjects will not take the placebo. After 3 weeks in initial phase (either Phase A or B), subjects will switch to the alternate phase and continue the study for another 3 weeks. Hyoscyamine is available as a rescue medication during Phase A and Phase B. Half of the subjects will be randomized to begin with Phase A and half will be randomized to begin with Phase B. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.	No Treatment n=30 Participants Arm 2: Subjects receive no treatment but have access to hyoscyamine as a rescue medication. Hyoscyamine: While not an intervention of interest to our study, patients will have hyoscyamine available as a rescue medication throughout the study. This can be taken on a PRN basis for breakthrough pain a maximum of 4x daily.
Clinical Global Improvement Improvement	14 Participants	9 Participants
Clinical Global Improvement No improvement	16 Participants	21 Participants

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

No Treatment

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

Dr Samuel Nurko

Boston Children's Hospital

Phone: 6173556055

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place