Recipients refractory or resistant chronic GVHD to therapy with corticosteroid
Conditions
Interventions
Patients were orally administered Tamibarotene (AM80G) (4 mg/day) daily for 24 weeks.
Sponsors
Okayama University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients diagnosed as chronic GVHD according to diagnostic criteria proposed by National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic GVHD. 1. Aged 18 to 84 years at consent 2. Patients with Karnofsky Performance Scale of 60 or more 3. Patient or guardian willing and able to provide informed consent 4. Steroid-refractory or steroid-resistant chronic GVHD defined as: Inability to begin prednisone therapy at a dose of greater than 0.5 mg/kg/day or equivalent dose if on another steroid. (1) Progression of chronic GVHD while being treated with prednisone therapy at a dose of greater than 1 mg/kg/day for 2 weeks. (2) Failure to improve after 4 weeks while being treated with prednisone therapy at a dose of greater than 0.5 mg/kg/day for at least 2 weeks. (3) Flare of chronic GVHD while tapering of steroids. (4) steroid-dependent chronic GVHD received prednisone therapy at a dose of 10 mg/day or equivalent dose if on another steroid. 5. Stated willingness to use contraception during the study and through 2 years for wowen and 6 months for men after the final dose of study treatment.
Exclusion criteria
Exclusion criteria: 1. Mental illness which needs psychiatric medication or are judged by physician as being unsuitable 2. Active, uncontrolled infection (not responding to antibiotics, antifungal or antiviral therapies) 3. Patients with severe liver dysfunction (CTCAE ver.4.0 Grade 3 or more, excepting liver GVHD) 4. Patients with previous experience with Am80 or other retinoid in the past 12 weeks 5. Patients who are allergic or hypersensitive to Am80 or other retinoid. 6. Patients who are pregnant, patients who have possibility of being pregnant, or patients who are breastfeeding. 7. Pulmonary: DLCO <40% or FEV1% <40% 8. Patients with life expectancy of less than 6 months. 9. Patients who are enrolled in other clinical studies in the past 3 months. 10. Other patients who are judged by physician as being unsuitable
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Probability of failure-free survival (FFS) and complete/partial response at 24 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Probability of FFS and complete/partial response at 12 weeks 2.Probability of FFS and overall survival rate at 1 year 3.Proportion of patients who are FFS and have at least 50% reduction in baseline steroid dose at 24 weeks | — |
Countries
Japan
Contacts
Public ContactHisakazu Nishimori
Okayama University Hospital Department of Hematology and Oncology
Outcome results
None listed