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Efficacy of Open Label Placebo in Children With FGIDs (Placebo)

Primary Purpose

Functional Abdominal Pain, Functional Dyspepsia, Irritable Bowel Syndrome

Status

Completed

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

Placebo Suspension

Hyoscyamine

About this trial

This is an interventional treatment trial for Functional Abdominal Pain focused on measuring Functional Pain, Abdominal Pain, Irritable Bowel Syndrome, IBS, Functional Dyspepsia, FGID, Functional Gastrointestinal Disorders

Eligibility Criteria

8 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Age 8 to 21 years.
Diagnosis of functional abdominal pain, irritable bowel syndrome or functional dyspepsia made by a pediatric gastroenterologist according to Rome III Criteria.
Mean daily intensity of pain of 25 mm in the week prior to the initiation of the study, based on the Visual Analogue Scale
Children will not be excluded if they are adhering to any specific diet. Children will be asked to report any specific established diet prior to the study or dietary modifications that could have been made during the course of the study.
Normal laboratory tests including complete blood count, erythrocyte sedimentation rate, albumin, serum amylase, lipase, liver enzymes, urine analysis, stool examination for occult blood and ova and parasites one month prior the initiation of the study. Urinary culture will be obtained if the symptoms or urinalysis suggest the possibility of a urinary infection.
Normal lactose breath test or history of lack of resolution of symptoms on a lactose-free diet (2 weeks).
Patients receiving psychological treatment, hypnosis, biofeedback or guided imagery will not be excluded of the study if those were started at least one month prior to the initiation of the study and are not planned to be discontinued during the length of the trial. Patients will need to be prescribed hyoscyamine (clinically indicated) to be considered for this study, as the placebo will be in addition to their prescribed medication.

Exclusion Criteria:

Inclusion criteria not met.
Evidence of organic gastrointestinal disease, hepatic disorders, urinary or cardiac disease.
Children below the 5th percentile for weight or height.
Hemoccult positive stools.
Patients with diagnosis of Inflammatory Bowel Disease, hyperthyroidism, CHF, cardiac arrhythmias, prostatic hypertrophy, autonomic neuropathy, biliary tract disease, children with spastic paralysis or chronic lung disease (we will consult a pulmonologist concerning the inclusion of children with chronic lung disease).
Patients who are taking any of the following drugs: AbobotulinumtoxinA, Acetylcholinesterase Inhibitors (Central), Cannabinoids, OnabotulinumtoxinA, Potassium Chloride, Pramlintide, RimabotulinumtoxinB, Secretin. Patients receiving antidepressant or anticholinergic drugs will be excluded from the study. PPIs will be allowed as long as the patient had been on a stable dose for at least 12 weeks.
Patients planning to change their diet during the time of the study will be excluded. Children will be asked to report any specific established diet prior to the study or dietary modifications that could have been made during the course of the study.
Patients planning to start psychological treatment, hypnosis, biofeedback, or guided imagery during the course of the study or have started any of these within the month prior to consent.
The participant is pregnant or is planning to become pregnant throughout the course of the research study

Sites / Locations

Boston Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Other

Arm Label

Placebo

No Treatment

Arm Description

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.

Arm 2: Subjects receive no treatment but have access to hyoscyamine as a rescue medication.

Outcomes

Primary Outcome Measures

The Primary Outcome Measure Was Mean Daily Pain

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

Secondary Outcome Measures

Use of Rescue Medications

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

Clinical Global Improvement

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)

Full Information

NCT ID

NCT02389998

First Posted

February 25, 2015

Last Updated

July 2, 2021

Sponsor

Boston Children's Hospital

Collaborators

Nationwide Children's Hospital

1. Study Identification

Unique Protocol Identification Number

NCT02389998

Brief Title

Efficacy of Open Label Placebo in Children With FGIDs

Acronym

Placebo

Official Title

Evaluation of the Efficacy of Open Label Placebo in Children With FGIDs (Functional Gastrointestinal Disorders)

Study Type

Interventional

2. Study Status

Record Verification Date

July 2021

Overall Recruitment Status

Completed

Study Start Date

July 2014 (Actual)

Primary Completion Date

July 2019 (Actual)

Study Completion Date

July 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Boston Children's Hospital

Collaborators

Nationwide Children's Hospital

4. Oversight

5. Study Description

Brief Summary

This study is aimed at investigating the efficacy of placebo for symptom relief in children with abdominal pain related functional gastrointestinal disorders.

Detailed Description

The purpose of this research study is to see if prescribing an open label placebo to children with functional gastrointestinal disorders will help improve symptoms and their overall quality of life. Open label means you/your child are aware you are taking liquid placebo drops and not an active medication. Symptoms associated with functional gastrointestinal disorders (FGIDs) of children and adolescents are commonly encountered symptoms in general pediatrics and pediatric gastroenterology. The FGIDs the investigators are studying include functional abdominal pain, irritable bowel syndrome, and functional dyspepsia. The liquid placebo drops contain no active medication. Recent research studies have shown improvement in gastrointestinal symptoms after taking liquid placebo drops in both children and adults with FGIDs. A randomized research study for a medication used to treat children with FGIDs showed a very significant placebo effect, meaning patients receiving placebo also experienced improvement in their symptoms. Randomized refers to the fact that subjects were randomly selected to receive either the study medication or placebo. A recent adult study gave adult patients a placebo and told them it was a placebo, and these adults also had significant symptom improvement. The goal of this study is to further explore using open label (or non-deceptive) placebo use to treat children with FGIDs.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Functional Abdominal Pain, Functional Dyspepsia, Irritable Bowel Syndrome, Functional Gastrointestinal Disorders

Keywords

Functional Pain, Abdominal Pain, Irritable Bowel Syndrome, IBS, Functional Dyspepsia, FGID, Functional Gastrointestinal Disorders

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Crossover Assignment

Masking

None (Open Label)

Masking Description

Open placebo. No masking

Allocation

Randomized

Enrollment

31 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Placebo

Arm Type

Experimental

Arm Description

Arm Title

No Treatment

Arm Type

Other

Arm Description

Arm 2: Subjects receive no treatment but have access to hyoscyamine as a rescue medication.

Intervention Type

Other

Intervention Name(s)

Placebo Suspension

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

Hyoscyamine

Intervention Description

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.

Primary Outcome Measure Information:

Title

The Primary Outcome Measure Was Mean Daily Pain

Description

Time Frame

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)

Secondary Outcome Measure Information:

Title

Use of Rescue Medications

Description

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

Time Frame

3 weeks of placebo vs 3 weeks of no treatment

Title

Clinical Global Improvement

Description

Time Frame

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

8 Years

Maximum Age & Unit of Time

21 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Age 8 to 21 years. Diagnosis of functional abdominal pain, irritable bowel syndrome or functional dyspepsia made by a pediatric gastroenterologist according to Rome III Criteria. Mean daily intensity of pain of 25 mm in the week prior to the initiation of the study, based on the Visual Analogue Scale Children will not be excluded if they are adhering to any specific diet. Children will be asked to report any specific established diet prior to the study or dietary modifications that could have been made during the course of the study. Normal laboratory tests including complete blood count, erythrocyte sedimentation rate, albumin, serum amylase, lipase, liver enzymes, urine analysis, stool examination for occult blood and ova and parasites one month prior the initiation of the study. Urinary culture will be obtained if the symptoms or urinalysis suggest the possibility of a urinary infection. Normal lactose breath test or history of lack of resolution of symptoms on a lactose-free diet (2 weeks). Patients receiving psychological treatment, hypnosis, biofeedback or guided imagery will not be excluded of the study if those were started at least one month prior to the initiation of the study and are not planned to be discontinued during the length of the trial. Patients will need to be prescribed hyoscyamine (clinically indicated) to be considered for this study, as the placebo will be in addition to their prescribed medication. Exclusion Criteria: Inclusion criteria not met. Evidence of organic gastrointestinal disease, hepatic disorders, urinary or cardiac disease. Children below the 5th percentile for weight or height. Hemoccult positive stools. Patients with diagnosis of Inflammatory Bowel Disease, hyperthyroidism, CHF, cardiac arrhythmias, prostatic hypertrophy, autonomic neuropathy, biliary tract disease, children with spastic paralysis or chronic lung disease (we will consult a pulmonologist concerning the inclusion of children with chronic lung disease). Patients who are taking any of the following drugs: AbobotulinumtoxinA, Acetylcholinesterase Inhibitors (Central), Cannabinoids, OnabotulinumtoxinA, Potassium Chloride, Pramlintide, RimabotulinumtoxinB, Secretin. Patients receiving antidepressant or anticholinergic drugs will be excluded from the study. PPIs will be allowed as long as the patient had been on a stable dose for at least 12 weeks. Patients planning to change their diet during the time of the study will be excluded. Children will be asked to report any specific established diet prior to the study or dietary modifications that could have been made during the course of the study. Patients planning to start psychological treatment, hypnosis, biofeedback, or guided imagery during the course of the study or have started any of these within the month prior to consent. The participant is pregnant or is planning to become pregnant throughout the course of the research study

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Samuel Nurko, MD, MPH

Organizational Affiliation

Physician, Boston Children's Hospital

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Miguel Saps, MD

Organizational Affiliation

Nationwide Children's Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

Boston Children's Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

35099543

Citation

Nurko S, Saps M, Kossowsky J, Zion SR, Di Lorenzo C, Vaz K, Hawthorne K, Wu R, Ciciora S, Rosen JM, Kaptchuk TJ, Kelley JM. Effect of Open-label Placebo on Children and Adolescents With Functional Abdominal Pain or Irritable Bowel Syndrome: A Randomized Clinical Trial. JAMA Pediatr. 2022 Apr 1;176(4):349-356. doi: 10.1001/jamapediatrics.2021.5750. Erratum In: JAMA Pediatr. 2022 May 1;176(5):528.

Results Reference

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Efficacy of Open Label Placebo in Children With FGIDs

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