Clinical trial NCT00887263: Efficacy and Safety Study of Budesonide to Treat Oral Chronic Graft vs Host Disease (cGvHD)
Official title: Double-blind, Randomised, Placebo-controlled Multicentre Phase III Clinical Study Followed by Open-label Phase on the Efficacy and Tolerability of Budesonide 3 mg Effervescent Tablet in Patients With Resistant Oral Chronic GvHD
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Oral Chronic Graft vs Host Disease
Lead sponsor: Dr. Falk Pharma GmbH
Sponsor class: INDUSTRY
First posted: 2009-04-23
Enrollment: 186
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to compare the efficacy and tolerability of budesonide 3 mg effervescent tablet (9 mg/day) compared to placebo for the treatment of patients with resistant oral cGvHD.


Eligibility criteria: Inclusion Criteria:

* Karnofsky \>= 70
* Oral chronic GvHD after allogeneic haematopoietic stem cell transplantation
* Oral cGvHD of erosive and/or ulcerative type
* NIH scale \>= 3
* Resistant oral cGvHD with no oral response to conventional primary treatment

Exclusion Criteria:

* Uncertain diagnosis of resistant oral cGvHD
* Symptomatic oral cGvHD of hyperkeratotic type solely
* Current active oral bacterial, viral, or fungal infection
* Unwilling to forego concurrent treatment for mucosal lesions and/or related oral pain
* Requiring addition of new systemic therapy including steroids, or radiation therapy
* Local intestinal infection
* Abnormal hepatic function or liver cirrhosis
* If careful medical monitoring is not ensured: tuberculosis, cardiovascular disease, diabetes mellitus, osteoporosis, active peptic ulcer disease, glaucoma, cataract, infection
* Second line treatment of oral cGvHD with topical steroids

Primary outcomes:
measure: Rate of patients with objective response
timeFrame: 12 weeks
Secondary outcomes:
measure: Rate of complete/partial response, stable disease, progressive disease
timeFrame: 12 weeks
measure: Time to initial objective response
timeFrame: x weeks
measure: Rate of subjective improvement
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Regensburg
country: Germany
facility: University of Regensburg
city: Jerusalem
country: Israel
facility: The Hebrew University-Hadassah School of Dental Medicine, Department of Oral Medicine
Study officials:
affiliation: University of Rochester Medical Center, Division of Oral Medicine, Eastman Institute for Oral Health
name: Sharon Elad, DMD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Elad S, Or R, Garfunkel AA, Shapira MY. Budesonide: a novel treatment for oral chronic graft versus host disease. Oral Surg Oral Med Oral Pathol Oral Radiol Endod. 2003 Mar;95(3):308-11. doi: 10.1067/moe.2003.23.
citation: Elad S, Zeevi I, Finke J, Koldehoff M, Schwerdtfeger R, Wolff D, Mohrbacher R, Levitt M, Greinwald R, Shapira MY. Improvement in oral chronic graft-versus-host disease with the administration of effervescent tablets of topical budesonide-an open, randomized, multicenter study. Biol Blood Marrow Transplant. 2012 Jan;18(1):134-40. doi: 10.1016/j.bbmt.2011.06.001. Epub 2011 Jun 12.