Efficacy of Open Label Placebo in Children With FGIDs

NCT02389998 | Completed Results

• 1 other identifier: P00003158
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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

  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 Outcomes (2)

• Use of Rescue Medications

  3 weeks of placebo vs 3 weeks of no treatment

• Clinical Global Improvement

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

Study Arms (2)

Placebo

EXPERIMENTAL

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.

Other: Placebo Suspension; Drug: Hyoscyamine

No Treatment

OTHER

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

Drug: Hyoscyamine

Interventions

Placebo Suspension OTHER

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.

Placebo

Hyoscyamine DRUG

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; Placebo

Eligibility Criteria

• Age: 8 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• Evidence of organic gastrointestinal disease, hepatic disorders, urinary or cardiac disease.
• Children below the 5th percentile for weight or height.
• Hemoccult positive stools.
• 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

Sponsors & Collaborators

• Boston Children's Hospital: lead
• Nationwide Children's Hospital: collaborator

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Related Publications (1)

• 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.

  PMID: 35099543 DERIVED

MeSH Terms

Conditions

Irritable Bowel Syndrome; Gastrointestinal Diseases; Abdominal Pain

Interventions

Hyoscyamine

Condition Hierarchy (Ancestors)

Colonic Diseases, Functional; Colonic Diseases; Intestinal Diseases; Digestive System Diseases; Pain; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Digestive

Intervention Hierarchy (Ancestors)

Atropine; Atropine Derivatives; Tropanes; Azabicyclo Compounds; Aza Compounds; Organic Chemicals; Belladonna Alkaloids; Solanaceous Alkaloids; Alkaloids; Heterocyclic Compounds; Bridged Bicyclo Compounds, Heterocyclic; Heterocyclic Compounds, Bridged-Ring

Results Point of Contact

• Title: Dr Samuel Nurko
• Organization: Boston Children's Hospital

samuel.nurko@childrens.harvard.edu

6173556055

Study Officials

• Samuel Nurko, MD, MPH

  Physician, Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

• Miguel Saps, MD

  Nationwide Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: Open placebo. No masking
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Attending Physician; Director, Center for Motility and Functional Gastrointestinal Disorders

Study Record Dates

• First Submitted: February 25, 2015
• First Posted: March 17, 2015
• Study Start: July 1, 2014
• Primary Completion: July 1, 2019
• Study Completion: July 1, 2019
• Last Updated: July 23, 2021
• Results First Posted: July 23, 2021

Record last verified: 2021-07

Locations

US (1)