Clinical trial NCT01902368: Celiac Disease Screening
Official title: A Prospective Trial of Celiac Disease Screening
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Celiac Disease
Keywords: Gluten free diet
Lead sponsor: Beth Israel Deaconess Medical Center
Sponsor class: OTHER
First posted: 2013-07-18
Enrollment: 14
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SCREENING

Brief summary: The purpose of this study is to see if it makes sense to test people for celiac disease who have a first or second degree relative (parent, sibling, child, grandparent, aunt or uncle) with celiac disease. The investigators will check to see what differences there are in the health and quality of life between those who know they have celiac disease and start the gluten free diet and those who do not.

Detailed description: Our overall hypothesis is that first and second degree relatives of individuals with celiac disease benefit from screening and diagnosis of celiac disease. Secondary hypotheses are:

1. The number needed to test and cost to benefit one person though celiac disease screening are within acceptable ranges of 33 tested and \< $10,000 spent.
2. Treatment does not lead to adverse metabolic changes.
3. Intestinal biopsy is unnecessary for accurate diagnosis in a substantial subset of adults.

Aim 1. Determine the effect of screen detected celiac disease on health related quality of life.

* Evaluate change in health related quality of life at one year in participants randomized to the screen detected, early diagnosis cohort compared to the screen detected, delayed diagnosis, clinically detected and non-celiac control cohorts.
* Evaluate change in symptoms, psychological well-being and burden of treatment at one year in the screen detected, early diagnosis cohort compared to the screen detected, delayed diagnosis, clinically detected and non-celiac control cohorts.
* Evaluate the cost per diagnosis and the number needed to test for the diagnosis of individuals who will have a clinically meaningful improvement in health related quality of life attributable to treatment of celiac disease.

Aim 2: Assess the effect of screen detected celiac disease on nutritional and metabolic indices.

* Compare changes bone density, body mass index, Reynolds Cardiovascular Risk Score, and nutritional indices at one year in the screen detected, early diagnosis cohort compared to the screen detected, delayed diagnosis, clinically detected and non-celiac control cohorts.

Aim 3: Evaluate the reliability of using serologic tests in combination with intestinal fatty acid binding protein vs. intestinal biopsy to confirm celiac disease diagnosis in adults.

* Prospectively assess the sensitivity and specificity of a novel non-invasive celiac diagnostic algorithm in comparison to the current gold standard of small intestinal biopsy histology.
* Model the cost of modified, non-invasive celiac testing vs. classical testing with endoscopic biopsy in both screen-detected and clinically identified celiac disease.

Eligibility criteria: Inclusion Criteria:

* Be ambulatory, community dwelling, 18 to 80 years, inclusive
* For the screening cohort:

  * Have a first or second degree family member with known biopsy-proven celiac disease.
  * Have not been on a gluten-free diet in the past 6 months
  * Have not received a prior diagnosis of celiac disease at any time
* For the clinically detected cohort

  * Have biopsy proven celiac disease detected based on clinical symptoms and on a gluten free diet for less than 1 month.

Exclusion Criteria:

* For the screen detected cohort, have significantly severe symptoms (as judged by the investigator) at screening which preclude randomization;
* Be on a gluten-free diet or have been on a gluten-free diet within the past 6 months.
* Be pregnant or planning pregnancy in the study time period
* Be taking corticosteroids or immunomodulators
* Have a history of significant concomitant gastrointestinal disease or other comorbidity judged by the investigator to potentially interfere with study outcomes
* Be unable or unwilling to cooperate with the study protocol
* Have insufficient knowledge of English to complete study surveys

Primary outcomes:
measure: change in health related quality of life
timeFrame: Baseline, 3, 6, 9 and 12 months
Secondary outcomes:
measure: change in celiac symptoms
timeFrame: Baseline, 3, 6, 9 and 12 months
measure: change in bone density
timeFrame: Baseline and 12 months
measure: change in psychological well-being
timeFrame: Baseline, 3, 6, 9, and 12 months
measure: change in burden of treatment
timeFrame: baseline, 3, 6, 9, and 12 months
Other outcomes:
Locations:
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
Study officials:
affiliation: Beth Israel Deaconess Medical Center
name: Daniel Leffler, MD, MS
role: PRINCIPAL_INVESTIGATOR
References: