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A Pilot Study of Imatinib Mesylate in Steroid Refractory Chronic Graft Versus Host Disease

Phase 1
18 Years
Open (Enrolling)
Graft vs Host Disease

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Trial Information

A Pilot Study of Imatinib Mesylate in Steroid Refractory Chronic Graft Versus Host Disease

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 >

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

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

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.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The frequency of adverse events graded according to the CTCAE will be the primary endpoint

Outcome Time Frame:

Subjects will be monitored at 1, 4, 8, 16, and 24 weeks.

Safety Issue:


Principal Investigator

David Miklos

Investigator Role:

Principal Investigator

Investigator Affiliation:

Stanford University


United States: Institutional Review Board

Study ID:




Start Date:

September 2008

Completion Date:

December 2009

Related Keywords:

  • Graft Vs Host Disease
  • Graft vs Host Disease



Stanford University School of MedicineStanford, California  94305-5317
Fred Hutchinson Cancer Research Center (FHCRC)Seattle, Washington  98109