Study of orBec® as Monotherapy in the Treatment of Patients With Upper GI Symptoms Caused by Chronic Graft Versus Host Disease (GVHD)

Study Overview
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Brief Summary
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Use of an oral topically-active glucocorticoid with limited side effects may control the gastrointestinal inflammatory process of GVHD and minimize glucocorticoid exposure.

Official Title
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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of the Safety and Efficacy of orBec® (Oral Beclomethasone 17,21-Dipropionate) Administered as Monotherapy in the Treatment of Upper Gastrointestinal (GI) Symptoms Caused by GVHD in Patients With Chronic GI GVHD

Conditions
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Chronic Gastrointestinal Graft vs Host Disease

Intervention / Treatment
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* Drug: orBec
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Receipt of allogeneic hematopoietic cell transplant >100 days prior to consent Documented cGVHD as defined by the NIH consensus criteria in at least one organ system other than the GI tract (for example, cGVHD of the oral cavity would qualify as a criterion) Endoscopic findings consistent with GI GVHD Must be able to swallow tablets Must be able to read and understand informed consent Adequate birth control methods for the duration of the study Exclusion Criteria: >500 mL/day of diarrhea on any 1 day within 3 days prior to the first dose of study drug GI infection Multi-organ failure or other condition that, in the opinion of the investigator, would compromise the patient's ability to complete the study. HIV seropositivity Pregnant or nursing female Use of any investigational drug to treat chronic GVHD within 28 days of the first dose of study drug Evidence of recurrent or progressing malignant disorder that was the indication for HCT

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>Control | Drug: Placebo<br> <br> |
| Experimental: orBec<br>Investigational drug | Drug: orBec<br> <br> * Other names: oral beclomethasone 17,21-dipropionate;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Gastrointestinal (GI) Graft-vs-Host Disease (GVHD) Symptoms | Patients who achieved a Complete Response (CR) of their GI GVHD Symptoms during the Part 1, 16 week treatment period and who remained a CR at the end of the 16 week treatment period were to be eligible to continue into Part 2 of the study. All others were to discontinue. GI GVHD was assessed using a composite score based on the symptoms of satiety, nausea/vomiting, and anorexia. Each symptom was scored on a scale of 0-3, such that the minimum score = 0 and the maximum score = 9. At entry, all subjects must have a score of ≥ 3. A CR will be defined as a composite score of 0. | 16 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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BDP, orBec, GVHD, beclomethasone dipropionate, bone marrow transplant, hematopoietic cell transplant, HCT, stem cell transplant, marrow transplant, SCT, beclomethasone 17,21-dipropionate