This trial is active, not recruiting.

Condition graft vs host disease
Treatment imatinib
Phase phase 1
Sponsor Stanford University
Collaborator Novartis
Start date September 2008
End date August 2009
Trial size 15 participants
Trial identifier NCT00760981, BMT195, SU-07112008-1254


To determine if subjects with steroid refractory cGVHD can tolerate imatinib mesylate and whether their cGVHD responds to imatinib mesylate.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment

Primary Outcomes

The frequency of adverse events graded according to the CTCAE will be the primary endpoint
time frame: Subjects will be monitored at 1, 4, 8, 16, and 24 weeks.

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria:A. Subject has cGVHD requiring systemic therapy occurring >100 days after hematopoietic cell transplant diagnosed with at least one diagnostic feature from Appendix A. B. Subject has active cGVHD with either: 1. Persistent steroid dependence defined as the inability to taper steroid treatment to less than 0.25 mg/kg/d prednisone or its equivalent for at least 3 months 2. Progression of cGVHD signs and symptoms on steroid therapy equivalent to prednisone 0.5 mg/kg/d for at least 1 month. C. Subject has at least one of the following manifestations with which to follow progression of disease or response to imatinib: 1. Skin changes (rash, sclerosis, fasciitis, or ulceration) 2. Abnormal eye wetness <= 5 mm as measured by Schirmer's test 3. Oral mucosal changes (erythema, lichenoid changes, ulcers, or mucoceles) 4. Thrombocytopenia (platelets <150,000/uL). 5. Abnormal liver function testing (alkaline phosphatase, AST, ALT, or total bilirubin > ULN). 6. Bronchiolitis obliterans (diagnosed by a > 5% annual decline in FEV1 with the lowest post-transplant FEV1/FVC < 0.8 and an appropriate CT scan or lung biopsy, see Appendix A for details) D. Subject may have previously any received immunosuppressive therapies for cGVHD. Continuing treatment with steroids and any one or none of the following is allowed: cyclosporine, tacrolimus, sirolimus, mycophenolate mofetil, or extracorporeal photopheresis. E. Subject has been on a fixed dose of steroids or a fixed dose of steroids and one other immunosuppressant (cyclosporine, tacrolimus, sirolimus, mycophenolate mofetil, or extracorporeal photopheresis) for >= 30 days before starting imatinib. F. Subject has a life expectancy >= 6 months. G. Subject has the ability to understand and willingness to sign a written informed consent document. H. Subject has a Karnofsky performance status^3 50% (Appendix B). I. Subject is ³ 18 years of age. J. If a female with reproductive potential (defined as having at least 1 menstrual period in the past 12 months), the subject must have a negative pregnancy test performed <= 7 days before starting study drug. K. If a female with reproductive potential, the subject agrees to use contraception for the duration of the trial. L. Subject has a total bilirubin < 1.5X ULN. M. Subject has an aspartate transaminase (AST), alanine aminotransferase (ALT), and alkaline phosphatase < 2.5X ULN. N. Subject has an absolute neutrophil count > 500/uL (growth factor supplementation is allowed). O. Subject has a hematocrit > 26% (transfusion support is allowed). P. Subject has a platelet count > 20,000/uL. Exclusion Criteria:A. Subject has received another investigational agent <= 30 days before starting the study drug. B. Subject has an on-going, intercurrent illness such as an infection not responsive to antibiotics, antiviral medicines, or antifungal medicines. C. Subject has progressive malignant disease. D. Subject has a secondary malignancy that has not been effectively treated within the past 5 years (except localized basal cell or squamous cell carcinoma). E. Subject has imatinib intolerance or allergy. F. Subject is breast-feeding. G. Subject is not willing to comply with treatment or response evaluation. H. Subject has received an allogeneic cell product (including DLI or hematopoietic cell boost) <= 100 days before starting study drug. I. The subject's steroid and/or immunosuppressant dose has changed <= 30 days before starting study drug.

Additional Information

Official title A Pilot Study of Imatinib Mesylate in Steroid Refractory Chronic Graft Versus Host Disease
Principal investigator David Miklos
Trial information was received from ClinicalTrials.gov and was last updated in February 2010.
Information provided to ClinicalTrials.gov by Stanford University.