Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder

Vanderbilt University Medical Center

Status and phase: Completed; Phase 3

Conditions: Graft Versus Host Disease; Multiple Myeloma and Plasma Cell Neoplasm; Chronic Myeloproliferative Disorders; Lymphoma; Leukemia; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms

Treatments: Drug: cyclosporine ophthalmic emulsion; Other: placebo

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT00755040

VICC BMT 0766

VU-VICC-BMT-0766

P30CA068485 (U.S. NIH Grant/Contract)

VU-080786

Details and patient eligibility

About

RATIONALE: Cyclosporine eye drops may prevent graft-versus-host disease of the eye in patients who have undergone donor stem cell transplant for hematologic cancer or bone marrow failure disorder.

PURPOSE: This randomized phase I trial is studying how well cyclosporine eye drops work in preventing graft-versus-host disease of the eye in patients who have undergone donor stem cell transplant for hematologic cancer or bone marrow failure disorder.

Full description

OBJECTIVES:

Primary

• To assess the efficacy of cyclosporine ophthalmic emulsion (Restasis®) in the prevention of ocular graft-versus-host disease in patients who have undergone allogeneic stem cell transplantation for hematologic malignancies or bone marrow failure disorders.

Secondary

• To correlate the Ocular Surface Disease Index with clinical ophthalmologic examination.

OUTLINE: This is a multicenter study. Patients are stratified according to age, type of transplant (related vs unrelated), and intensity of transplant (ablative vs other). Patients are randomized to 1 of 2 treatment arms.

• Arm I: Patients receive cyclosporine ophthalmic emulsion (Restasis®) drops in each eye twice daily for up to 1 year after transplant.
• Arm II: Patients receive placebo ophthalmic drops in each eye twice daily for up to 1 year after transplant.

Patients in both arms may also receive artificial tear drops at least twice daily as clinically necessary.

Enrollment: 164 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age greater than or equal to 18 years at time of enrollment
• Day 80-120 after first allogeneic stem cell transplant for hematologic malignancies or -marrow failure disorder at time of study enrollment
• Signed informed consent
• Willing to adhere to protocol requirements

Exclusion criteria

• history of non-compliance
• diagnosis of ocular GVHD at time of study enrollment
• documented dry eye prior to onset of stem cell transplant
• significant non- GVHD ocular problems that precludes participation in study
• life expectancy of lesser than 6 months at time of enrollment (viz. grade 4 acute GVHD, florid progression or relapse of underlying disease)
• history of documented ocular infections prior to stem cell transplant or during transplant (i.e. history of herpetic keratitis)
• females who are pregnant or breastfeeding

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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164 participants in 2 patient groups, including a placebo group

Ocular Cyclosporine (Restasis)

Experimental group

Description:

Patients receive cyclosporine ophthalmic emulsion (Restasis®) drops in each eye twice daily for up to 1 year after transplant.

Treatment:

Drug: cyclosporine ophthalmic emulsion

Placebo

Placebo Comparator group

Description:

Patients receive placebo ophthalmic drops in each eye twice daily for up to 1 year after transplant.

Treatment:

Other: placebo