oral chronic graft versus host disease
Conditions
Interventions
Product Name: Budesonide effervescent tablet
Pharmaceutical Form: Effervescent tablet
INN or Proposed INN: budesonide
CAS Number: 51333-22-3
Concentration unit: mg milligram(s)
Concentration type: equ
Sponsors
Dr. Falk Pharma GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Signed informed consent 2. Man or woman between 18 and 75 years of age 3. Karnofsky >= 70 4. Modified OMRS = 20 5. Oral chronic GvHD after allogeneic haematopoietic stem cell transplantation (cGvHD defined as GvHD present beyond day 100 post-transplantation) 6. Oral cGvHD confirmed by surgical biopsy when necessary (diagnosis of oral cGvHD is based on clinical presentation, evidence of systemic GvHD, and when necessary, positive oral mucosal biopsy) 7. Symptomatic oral cGvHD of erosive and ulcerative type 8. Resistant oral cGvHD with no oral response defined as the lack of partial response to conventional primary treatment (i.e. systemic prednisone and/or cyclosporine) after 4 weeks or no need for systemic immunosuppression due to oral cGvHD only, and with oral cGvHD as the only symptom of cGvHD for at least 4 weeks 9. In case of conventional primary treatment (i.e. systemic prednisone and/or cyclosporine) dosage unchanged within the last 4 weeks before inclusion (dose reduction possible) 10. In case of treatment with tacrolimus (FK506), sirolimus (rapamycin), or mycophenolate mofetil (MMF) dosage unchanged within the last 4 weeks before inclusion (dose reduction/dose adjustment to plasma concentration possible) 11. Negative pregnancy test at baseline visit week 0 in females of childbearing potential 12. Male or female patients with reproductive potential must use an approved contraceptive method during study treatment evaluation Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Uncertain diagnosis of resistant oral cGvHD 2. Symptomatic oral cGvHD of hyperkeratotic type solely 3. Current active oral bacterial, viral, or fungal infection 4. Unwilling to forego concurrent treatment for mucosal lesions and/or related oral pain (including viscous lidocaine, topical antifungals, or mouthwashes) 5. Requiring addition of new systemic therapy including steroids, or radiation therapy 6. Local intestinal infection 7. Abnormal hepatic function or liver cirrhosis (ALT or AST > 3 x ULN) 8. If careful medical monitoring is not ensured: tuberculosis, cardiovascular disease, diabetes mellitus, osteoporosis, active peptic ulcer disease, glaucoma, cataract, infection 9. Second line treatment of oral cGvHD with topical steroids (e.g. dexamethasone, beclomethasone) within the last 12 weeks 10. Use of inhaled steroids for the treatment of e.g. COPD or bronchiolitis 11. Treatment with low dose total lymphoid irradiation, intraoral PUVA, extracorporeal photochemotherapy, thalidomide, pentostatin within the last 4 weeks 12. Treatment with ketoconazole, itraconazole or other CYP3A inhibitors except fluconazole, voriconazole, cyclosporine, tacrolimus/FK506 (dose changes are not permitted during study) 13. Unable to demonstrate appropriate use of study medication 14. Existing or intended pregnancy or lactation 15. Known intolerance/hypersensitivity to study drugs or drugs of similar chemical structure or pharmacological profile 16. Participation in another clinical trial for the treatment of cGvHD within the last 30 days or simultaneous, or previous participation in this trial 17. Well-founded doubt about the patient’s cooperation, e.g. because of addiction to alcohol or drugs
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the rate of objective response for various dosing-protocols at the final/withdrawal visit;Secondary Objective: To assess efficacy using several scores To assess subjective improvement To assess safety (e.g., adverse events, lab parameters) To assess patients’ acceptance of the study drug ;Primary end point(s): Rate of objective response at the final/withdrawal visit using modified OMRS (oral mucosa rating scale) | — |
Countries
Germany
Outcome results
None listed