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

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ClinicalTrials.gov Identifier: NCT02389998
Recruitment Status : Recruiting
First Posted : March 17, 2015
Last Update Posted : October 24, 2018
Sponsor:
Collaborator:
Nationwide Children's Hospital
Information provided by (Responsible Party):
Samuel Nurko, Boston Children’s Hospital

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

Condition or disease Intervention/treatment Phase
Functional Abdominal Pain Functional Dyspepsia Irritable Bowel Syndrome Functional Gastrointestinal Disorders Other: Placebo Suspension Drug: Hyoscyamine Not Applicable

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.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Masking Description: Open placebo. No masking
Primary Purpose: Treatment
Official Title: Evaluation of the Efficacy of Open Label Placebo in Children With FGIDs (Functional Gastrointestinal Disorders)
Actual Study Start Date : July 2014
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Placebo
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
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.

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




Primary Outcome Measures :
  1. Satisfactory Relief of Symptoms and Overall Improvement (Patient Reported Outcome Measures) [ Time Frame: Week 7 ]
    Satisfactory relief of symptoms and overall improvement in pain based on patient overall assessment and satisfaction with treatment will be assessed following the two treatment phases.


Secondary Outcome Measures :
  1. Catechol-O-methyl transferase (COMT) measurement [ Time Frame: Visit 2 (week 2) ]
    We will measure COMT levels via a saliva sample taken at visit 2 (week 2).

  2. Change in Results of Functional Disability Inventory (FDI) [ Time Frame: Following 1-week baseline and 3-week and 6-week treatment periods ]
    We will compare the results from the FDI after 1, 3, and 6 weeks in the study

  3. Change in Results of the Pediatric Quality of Life Questionnaire (PedsQoL) [ Time Frame: Following 1-week baseline and 3-week and 6-week treatment periods ]
    We will compare the results from the PedsQoL after 1, 3, and 6 weeks in the study

  4. Change in Results of the Gastrointestinal Pediatric Quality of Life Questionnaire (GI PedsQoL) [ Time Frame: Following 1-week baseline and 3-week and 6-week treatment periods ]
    We will compare the results from the Peds GI QoL after 1, 3, and 6 weeks in the study

  5. Change in Results of the Children's Somatization Inventory (CSI) [ Time Frame: Following 1-week baseline and 3-week and 6-week treatment periods ]
    We will compare the results from the CSI after 1, 3, and 6 weeks in the study



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Ages Eligible for Study:   8 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age 8 to 21 years.
  2. Diagnosis of functional abdominal pain, irritable bowel syndrome or functional dyspepsia made by a pediatric gastroenterologist according to Rome III Criteria.
  3. Mean daily intensity of pain of 25 mm in the week prior to the initiation of the study, based on the Word-Graphic Rating Scale score.
  4. 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.
  5. 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.
  6. Normal lactose breath test or history of lack of resolution of symptoms on a lactose-free diet (2 weeks).
  7. 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:

  1. Inclusion criteria not met.
  2. Evidence of organic gastrointestinal disease, hepatic disorders, urinary or cardiac disease.
  3. Children below the 5th percentile for weight or height.
  4. Hemoccult positive stools.
  5. 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).
  6. 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.
  7. 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.
  8. 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.
  9. The participant is pregnant or is planning to become pregnant throughout the course of the research study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02389998


Contacts
Contact: Samuel Nurko, MD 617-3556055 samuel.nurko@childrens.harvard.edu
Contact: Kelsey Hawthorne, BS 617-355-5998 kelsey.hawthorne@childrens.harvard.edu

Locations
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Boston Children’s Hospital
Nationwide Children's Hospital
Investigators
Principal Investigator: Samuel Nurko, MD, MPH Physician, Boston Children's Hospital
Principal Investigator: Miguel Saps, MD Nationwide Children's Hospital

Responsible Party: Samuel Nurko, Attending Physician; Director, Center for Motility and Functional Gastrointestinal Disorders, Boston Children’s Hospital
ClinicalTrials.gov Identifier: NCT02389998     History of Changes
Other Study ID Numbers: P00003158
First Posted: March 17, 2015    Key Record Dates
Last Update Posted: October 24, 2018
Last Verified: October 2018

Keywords provided by Samuel Nurko, Boston Children’s Hospital:
Functional Pain
Abdominal Pain
Irritable Bowel Syndrome
IBS
Functional Dyspepsia
FGID
Functional Gastrointestinal Disorders

Additional relevant MeSH terms:
Dyspepsia
Irritable Bowel Syndrome
Abdominal Pain
Digestive System Diseases
Gastrointestinal Diseases
Colonic Diseases, Functional
Colonic Diseases
Intestinal Diseases
Signs and Symptoms, Digestive
Signs and Symptoms
Pain
Neurologic Manifestations
Nervous System Diseases
Hyoscyamine
Adjuvants, Anesthesia
Anti-Arrhythmia Agents
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Mydriatics
Parasympatholytics
Muscarinic Antagonists
Cholinergic Antagonists
Cholinergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action