A Randomized, Controlled, Double-Masked, Clinical Trial of Autologous Serum Eye Drops for Severe Ocular Chronic Graft-versus-Host Disease (GVHD) in Hematopoietic Stem Cell Transplant (HSCT) Patients

• Status: Terminated
• Trial end date: 2016-01-01
• Target enrollment: 0
• Participant gender: All

Summary

Some eye diseases can be helped by eye drops made from a person's own blood. These eye drops are called autologous serum eye drops, or ASEDs. ASEDs have been studied in only a few people with graft vs. host disease (GVHD) affecting the eye and were found to be helpful with few side effects.

The purpose of this study was to determine whether ASEDs are safe and more effective than control (normal saline) and can help with eye symptoms in people with severe chronic eye GVHD.

Each participant in this study was to have blood drawn to prepare ASEDs specifically for the participant. Each participant was scheduled to receive ASEDs for 3 months and placebo eye drops (salt water) for 3 months. Participants did not know when they were receiving the ASEDs and when they were receiving placebo eye drops.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Eye Institute (NEI)
• Collaborators: The Emmes Company, LLC; The EMMES Corporation
• Treatments: Ophthalmic Solutions; Tetrahydrozoline

Criteria

Inclusion Criteria

1. Participant must be 18 years of age or older.

2. Participant must understand and sign the protocol's informed consent document.

3. Participant must have severe ocular Graft-versus-host Disease (GVHD) in one (unilateral) or both (bilateral) eyes with the following characteristics in the study eye:

1. Combined score of modified Oxford punctate keratopathy grading and National Institutes of Health (NIH)/National Eye Institute (NEI) visual analogue scale of ≥ 4, and

2. Composite assessment scale (CAS) score of ≥ 3, and

3. Schirmer's tear test without anesthesia of ≤ 5 mm, and

4. Not responsive to standard medical treatment for at least three months prior to randomization. Standard medical treatment includes cyclosporine (Restasis®) ophthalmic emulsion (if tolerated), steroid drops (unless contraindicated), lubricating drops and ointments.

4. Participant is enrolled in an NIH study at the National Cancer Institute (NCI) or National Heart, Lung and Blood Institute (NHLBI).

5. Participant is willing and able to supply an adequate amount of blood to create the autologous serum eye drops (ASEDs).

Exclusion Criteria

1. Participant is unable to comply with study procedures or follow-up visits.

2. Participant is seropositive with positive nucleic acid confirmatory tests for human immunodeficiency virus-1/2 (HIV-1/2), human T lymphotropic virus-I/II (HTLV-I/II), hepatitis C virus (HCV), and/or hepatitis B virus (HBV) without confirmed history of vaccination.

3. Participant has GVHD proliferative keratopathy, uveitis or GVHD retinopathy in either eye.

4. Participant has an active ocular infection in either eye.

5. Participant has an allergy to dilating or anesthetic eye drops.

6. Participant has used Boston Scleral Lens (or similar lenses) in either eye or has used ASEDs in either eye within the past two months. Participants who have used the Boston Scleral Lens (or similar lenses) or ASEDs in either eye who did not respond to treatment and have stopped using them for at least two months are eligible.