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Budesonide in Patients With Immune Mediated Enteropathies

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ClinicalTrials.gov Identifier: NCT03866538
Recruitment Status : Recruiting
First Posted : March 7, 2019
Last Update Posted : October 19, 2020
Sponsor:
Information provided by (Responsible Party):
Joseph A. Murray, M.D., Mayo Clinic

Brief Summary:
Researchers are trying to determine if withdrawal of budesonide therapy in patients with immune-mediated enteropathies doing well on therapy will result in worsening symptoms, histology, quality of life, and micronutrient/nutritional status when compared to continued therapy.

Condition or disease Intervention/treatment Phase
Enteropathy Celiac Disease CVID Enteropathy Collagenous Sprue Autoimmune Enteropathy Drug: Withdrawal of Oral Budesonide Phase 4

Detailed Description:

This study aims to evaluate patients with small intestinal diseases caused by the immune system (refractory celiac disease type 1, CVID enteropathy, autoimmune enteropathy, and collagenous enteropathy) who have had improvement in symptoms and small intestine healing with oral budesonide.

Patients who meet inclusion criteria and agree to enter the study will be included for 12 weeks or until they choose to discontinue or there are concerns for safety.

At the beginning of the study, patients will undergo questionnaires, blood draw, urine and stool collections, physical exam, and an upper scope (esophagogastroduodenoscopy) with biopsies from the small intestine. They will then be randomized to either continued therapy on their current dose and formulation of budesonide (medication provided by the study) or to withdrawal of the medication over 2 weeks and then no medication during the study. Daily questionnaire on symptoms will be recorded.

Patients will be assessed in the clinical research unit monthly during the study with a questionnaire, physical exam, blood draw, and urine/stool collections.

Finally, at the end of the trial (12 weeks) or at the time of withdrawal from the trial, the patients will complete questionnaires, physical exam, blood collection, urine/stool collection, and upper scope (esophagogastroduodenoscopy) with small bowel biopsies.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Randomized, open-label withdrawal of budesonide versus continued budesonide therapy in patients with immune-mediated enteropathies that have had symptomatic and histologic improvement
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label Withdrawal Trial of Budesonide in Patients With Immune Mediated Enteropathies
Actual Study Start Date : September 10, 2019
Estimated Primary Completion Date : September 1, 2021
Estimated Study Completion Date : December 31, 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Budesonide

Arm Intervention/treatment
No Intervention: Continued Budesonide
Patients in this arm continue budesonide at the dose they were taking at the time of enrollment in the trial
Experimental: Withdrawal of Budesonide
Patients are weaned off of budesonide over 2 weeks and continued off of the medication for the duration of the trial
Drug: Withdrawal of Oral Budesonide
The intervention arm will taper and stop budesonide therapy (withdrawal).




Primary Outcome Measures :
  1. Change in Histology [ Time Frame: 12 weeks ]
    Investigators will determine change in small bowel villi after withdrawal of budesonide compared to continued therapy to assess small intestinal injury. This will be accomplished by esophagogastroduodenoscopy (upper scope) with biopsies from the small intestine.


Secondary Outcome Measures :
  1. Change in Symptoms [ Time Frame: 12 weeks ]
    Symptoms (monitored daily) will be compared between patients that withdraw from budesonide compared to those that continue budesonide therapy. Symptoms will be measured on the Celiac Disease Symptom Diary (CDSD) questionnaire. First question asks if subject has diarrhea and how many times. Second question asks if subject has spontaneous bowel movement and how many. Third question asks if subject had abdominal pain and to rate on a scale from 0 to 10 with higher numbers being worse pain. Fourth question asks if experiencing bloating and if it is very mild, mild, moderate, severe, or very severe. Fifth question asks if subject has nausea and if it is ranked very mild, mild, moderate, severe, or very severe. The sixth question asks if the subject has experienced tiredness and to rank very mild, mild, moderate, severe or very severe. Each question is evaluated for changes or trends over time, and a composite score including multiple questions can be calculated and compared.

  2. Change in Quality of Life: Celiac Disease Quality of Life Questionnaire (CD-QOL) [ Time Frame: 12 weeks ]
    Quality of life will be assessed monthly comparing those that withdraw from budesonide compared to those that continue therapy. This will be assessed by the Celiac Disease Quality of Life Questionnaire (CD-QOL). This survey includes 20 questions with scores for each ranging from 1 to 5, with higher numbers indicating improved quality of life. Total scores will range from 20 to 100 with higher numbers indicating better quality of life.

  3. Change in Weight [ Time Frame: 12 weeks ]
    Weight in kilograms will be monitored throughout the study. Changes will be compare between those withdrawing from budesonide compared to those continuing therapy.

  4. Change in Iron [ Time Frame: 12 weeks ]
    Iron in the blood will be compared to those that withdraw from budesonide compared to those that continue therapy. This will be assessed at the initiation and completion of the trial.

  5. Change in Vitamin D [ Time Frame: 12 weeks ]
    25-hydroxy vitamin D in the blood will be compared to those that withdraw from budesonide compared to those that continue therapy. This will be assessed at the initiation and completion of the trial.

  6. Change in Vitamin B12 [ Time Frame: 12 weeks ]
    Vitamin B12 in the blood will be compared to those that withdraw from budesonide compared to those that continue therapy. This will be assessed at the initiation and completion of the trial.

  7. Change in Folate [ Time Frame: 12 weeks ]
    Folate in the blood will be compared to those that withdraw from budesonide compared to those that continue therapy. This will be assessed at the initiation and completion of the trial.

  8. Change in Zinc [ Time Frame: 12 weeks ]
    Zinc in the blood will be compared to those that withdraw from budesonide compared to those that continue therapy. This will be assessed at the initiation and completion of the trial.

  9. Change in Copper [ Time Frame: 12 weeks ]
    Copper in the blood will be compared to those that withdraw from budesonide compared to those that continue therapy. This will be assessed at the initiation and completion of the trial.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  • Patients will be recruited from the Division of Gastroenterology and Hepatology and the Mayo Clinic in Rochester, MN.
  • Adult patients with immune mediated enteropathies who have had improvement in symptoms and histology on oral budesonide therapy.

Exclusion Criteria

  • Age <18 years
  • Positive stool gluten testing in patients with refractory celiac disease
  • Small bowel malignancy or history of small bowel malignancy
  • Refractory celiac disease type 2
  • Post-transplant lymphoproliferative disorder associated enteropathy
  • No prior improvement in symptoms and histology with budesonide therapy
  • Discontinuation of budesonide therapy prior to the trial
  • Other concurrent systemic corticosteroids
  • Other immune mediating medications, for example but not limited to azathioprine, 6-mercaptopurine, cyclosporine, methotrexate, anti-TNF monoclonal antibodies, alpha-4 beta-7 integrin inhibiting monoclonal antibody, interleukin 12/23 inhibiting monoclonal antibody, JAK inhibitors.

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): NCT03866538


Contacts
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Contact: Carol T Van Dyke, CCRP 507-266-7842 VanDyke.Carol@mayo.edu

Locations
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United States, Minnesota
Mayo Clinic in Rochester Recruiting
Rochester, Minnesota, United States, 55905
Contact: Carol T Van Dyke, CCRP    507-266-7842    VanDyke.Carol@mayo.edu   
Principal Investigator: Joseph A Murray, MD         
Sponsors and Collaborators
Mayo Clinic
Investigators
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Principal Investigator: Joseph A Murray, MD Mayo Clinic
Additional Information:
Publications:

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Responsible Party: Joseph A. Murray, M.D., Principal Investigator, Mayo Clinic
ClinicalTrials.gov Identifier: NCT03866538    
Other Study ID Numbers: 18-008639
First Posted: March 7, 2019    Key Record Dates
Last Update Posted: October 19, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Celiac Disease
Intestinal Diseases
Collagenous Sprue
Malabsorption Syndromes
Gastrointestinal Diseases
Digestive System Diseases
Metabolic Diseases
Budesonide
Anti-Inflammatory Agents
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists