A Phase I Clinical Trial to Study the Safety of a Sustained-Release Subconjunctival Cyclosporine Implant for Ocular Graft-vs-Host Disease (GVHD1)
The objective of this randomized pilot study is to investigate the safety and potential
efficacy of a CsA implant in participants with lacrimal gland involvement and aqueous tear
deficiency related to GVHD. Safety will be evaluated in terms of adverse events related to
the implant. Efficacy will be evaluated by changes in Schirmer tear test (with anesthesia).
Secondary efficacy evaluation will include changes in corneal and conjunctival staining
grades, best-corrected visual acuity (BCVA), the Ocular Surface Diseases Index (OSDI),
changes in conjunctival GVHD grades, tear break-up time and meibomian gland dysfunction.
Participants with active systemic GVHD with aqueous tear deficiency associated with lacrimal
gland dysfunction following allogeneic hematopoietic SCT who are nine years of age or older
are eligible for inclusion in this pilot study. The study will involve surgical placement
of the CsA implant into the subconjunctival space adjacent to the lacrimal gland of one eye
in each participant. Participants older than 12 years of age will be randomized to receive
one of two implant release rates. However, all participants under age 12 will not be
randomized and will only be eligible to receive the smaller, lower dose implant. On each
anniversary study visit a participant will be given the opportunity to retain the implant on
an annual basis. This will be based upon the determination of clinical success (defined in
the Study Design section below) combined with a participant's report of symptom improvement.
The Implant will be removed if the Investigator believes the implant is harmful or if the
participant requests the implant to be removed.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
United States: Federal Government
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Bethesda, Maryland 20892|