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
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ClinicalTrials.gov Identifier: NCT00755040 |
Recruitment Status
:
Completed
First Posted
: September 18, 2008
Results First Posted
: December 8, 2016
Last Update Posted
: March 7, 2017
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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.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Chronic Myeloproliferative Disorders Graft Versus Host Disease Leukemia Lymphoma Multiple Myeloma and Plasma Cell Neoplasm Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms | Drug: cyclosporine ophthalmic emulsion Other: placebo | Phase 3 |
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.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 164 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Prevention |
Official Title: | Randomized Placebo Controlled Double Blind Study of Restasis Versus Placebo in Primary Prevention of Ocular GVHD After Allogeneic Stem Cell Transplantation |
Study Start Date : | October 2008 |
Actual Primary Completion Date : | December 2014 |
Actual Study Completion Date : | May 2016 |

Arm | Intervention/treatment |
---|---|
Experimental: Ocular Cyclosporine (Restasis)
Patients receive cyclosporine ophthalmic emulsion (Restasis®) drops in each eye twice daily for up to 1 year after transplant.
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Drug: cyclosporine ophthalmic emulsion
Given as eye drops
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Placebo Comparator: Placebo
Patients receive placebo ophthalmic drops in each eye twice daily for up to 1 year after transplant.
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Other: placebo
Given as eye drops
|
- Number of Patients That Develop Ocular GVHD While on Study in the Two Arms (Ocular Cyclosporine (Restasis) vs. Placebo) [ Time Frame: Up to 2 years after transplantation ]Data analysis will be performed on an intention-to-treat basis. Logistic regression will be used to estimate the odds ratio and 95% confidence interval of the two treatment groups with the adjustment of baseline covariates. Outcome comparisons for categorical/dichotomous variables will be assessed by 2 test or Fisher's exact test where expected cell frequencies were <5; continuous variables will be compared by X/2 test or by Mann-Whitney U test where the data are strongly skewed.
- Numerical Score of Ocular Surface Disease Index (OSDI) and Development or Lack of Ocular GVHD (by Ophthalmologic Examination) as Measured by Odds Ratio in a Linear Regression Model. [ Time Frame: 1 year after transplant ]OSDI is a patient reported questionnaire consisting of 12 questions which are scored from 0 to 4. The total scored is computed and depending on the number of questions answered scores are computed which are then categorized into mild, moderate or severe dry eye symptoms. This tool should adequately reflect the morbidity of the dry eye symptoms.

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Ages Eligible for Study: | 18 Years and older (Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00755040
United States, California | |
Stanford University | |
Palo Alto, California, United States, 94305 | |
United States, Illinois | |
Norwestern Memorial Hospital | |
Chicago, Illinois, United States, 60611 | |
United States, Tennessee | |
Vanderbilt-Ingram Cancer Center | |
Nashville, Tennessee, United States, 37232-6838 | |
United States, Texas | |
M. D. Anderson Cancer Center | |
Houston, Texas, United States, 77030 | |
United States, Washington | |
Fred Hutchinson Cancer Research Center | |
Seattle, Washington, United States, 989109 |
Principal Investigator: | Madan Jagasia, MD | Vanderbilt-Ingram Cancer Center |
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Madan Jagasia, MD, Associate Professor of Medicine; Director, Outpatient Transplant Program; Section Chief, Hematology and Stem Cell Transplant; Hematologist/Oncologist, Vanderbilt-Ingram Cancer Center |
ClinicalTrials.gov Identifier: | NCT00755040 History of Changes |
Other Study ID Numbers: |
VICC BMT 0766 P30CA068485 ( U.S. NIH Grant/Contract ) VU-VICC-BMT-0766 VU-080786 |
First Posted: | September 18, 2008 Key Record Dates |
Results First Posted: | December 8, 2016 |
Last Update Posted: | March 7, 2017 |
Last Verified: | January 2017 |
Keywords provided by Madan Jagasia, MD, Vanderbilt-Ingram Cancer Center:
graft versus host disease stage III adult Burkitt lymphoma stage III adult diffuse large cell lymphoma stage III adult diffuse mixed cell lymphoma stage III adult diffuse small cleaved cell lymphoma stage III adult Hodgkin lymphoma stage III adult immunoblastic large cell lymphoma stage III adult lymphoblastic lymphoma stage III grade 1 follicular lymphoma stage III grade 2 follicular lymphoma stage III grade 3 follicular lymphoma stage III mantle cell lymphoma stage III marginal zone lymphoma stage III small lymphocytic lymphoma stage IV adult Burkitt lymphoma |
stage IV adult diffuse large cell lymphoma stage IV adult diffuse mixed cell lymphoma stage IV adult diffuse small cleaved cell lymphoma stage IV adult Hodgkin lymphoma stage IV adult immunoblastic large cell lymphoma stage IV adult lymphoblastic lymphoma stage IV grade 1 follicular lymphoma stage IV grade 2 follicular lymphoma stage IV grade 3 follicular lymphoma stage IV mantle cell lymphoma stage IV marginal zone lymphoma stage IV small lymphocytic lymphoma extranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue nodal marginal zone B-cell lymphoma splenic marginal zone lymphoma |
Additional relevant MeSH terms:
Lymphoma Syndrome Leukemia Neoplasms Multiple Myeloma Neoplasms, Plasma Cell Myelodysplastic Syndromes Preleukemia Graft vs Host Disease Myeloproliferative Disorders Plasmacytoma Myelodysplastic-Myeloproliferative Diseases Neoplasms by Histologic Type Lymphoproliferative Disorders Lymphatic Diseases |
Immunoproliferative Disorders Immune System Diseases Disease Pathologic Processes Hemostatic Disorders Vascular Diseases Cardiovascular Diseases Paraproteinemias Blood Protein Disorders Hematologic Diseases Hemorrhagic Disorders Bone Marrow Diseases Precancerous Conditions Cyclosporins Cyclosporine |