Study for the Treatment of Ocular Chronic Graft-Versus-Host Disease (GVHD) With Amniotic Fluid Eye Drops (AFED)

Phase 1

Completed

• Conditions: Ocular Graft Versus Host Disease
• Interventions: Other: Saline Solution; Biological: Amniotic Fluid Eye Drops (AFED)

• Registration Number: NCT03298815
• Lead Sponsor: University of Utah

Brief Summary

A Randomized, Double-blinded, Placebo-Controlled Study for the Treatment of Ocular Chronic Graft Verses Host Disease with Processed Amniotic Fluid (pAF) Drops.

Detailed Description

This is a randomized (to each eye, within patient), double-blinded, placebo-controlled study of the efficacy of Processed Amniotic Fluid (pAF) in patients with hematologic malignancies who have undergone Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) and are diagnosed with Chronic Graft Verses Host Disease of the eye.

Study Timeline

• Study First Submitted: 2017-09-13
• Study First Submitted QC: 2017-09-26
• Study First Posted: 2017-10-02
• Study Start: 2019-12-20
• Primary Completion: 2022-12-16
• Study Completion: 2023-02-24
• Results First Submitted: 2025-03-11
• Status Verified: 2025-04
• Results First Submitted QC: 2025-04-07
• Last Update Submitted: 2025-04-07
• Results First Posted: 2025-04-09
• Last Update Posted: 2025-04-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

• Patients diagnosed within 5 years after hematopoietic stem cell transplant for any disease, with any graft and any conditioning regimen with at least one of the following symptoms:

  1. Dry eye symptoms partially affecting (requiring lubricant drops > 3 x per day or punctal plugs, or thermally cauterized puncta) or significantly affecting (special eyeware to relieve pain) activities of daily living (ADL)
  2. Unable to work because of ocular symptoms
  3. Loss of vision due to keratoconjunctivitis sicca (KCS)

• Patients may be using bilateral scleral lenses and/or bilateral punctal plugs at the time of accrual.

• Patients who are 18 years of age or older.

• Willing and able to provide informed consent.

Exclusion Criteria

• Patients who have any other reversible cause for dry eye at the time of accrual.
• More than 3 lines of therapy beyond corticosteroids with or without calcineurin inhibitors or sirolimus
• Relapsed malignancy at time of accrual after the most recent transplantation
• A difference in dryness between both eyes of more than 2 points of the grading provided by the International Dry Eye Workshop (DEWS) 2007 report
• Patients who are pregnant or plan to become pregnant while participating in the study.
• Patients who are not willing to discontinue the use of any eye drops, with the exception of non-medicated lubricant eye drops (artificial tears). All eye drops (excluding non-medicated lubricant eye drops) must be stopped at least seven days before treatment with pAF.
• Inability to comply with the investigational plan and visit schedule for any reason, in the judgement of the investigator.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Saline Solution - All participants, One eye	Saline Solution	-
Amniotic Fluid Eye Drops (AFED) - All participants, One eye	Amniotic Fluid Eye Drops (AFED)	-

Primary Outcome Measures

Name	Time	Method
Overall Response Rate	30 days	Response is a composite of the NIH Consensus Conference (CC) for assessment of response in chronic GVHD (eye score) and the International Dry Eye Workshop (DEWS) grading scale. Responders would be defined as a one point improvement in the dry eye grading scale (DEWS), without worsening in the eye score.

Secondary Outcome Measures

Name	Time	Method
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 100 Days	baseline and 100 days	Change in Quality of Life questionnaire from baseline to 100 days. FACT-G is a combination score of physical, social/family, emotional, and functional well-being. The score ranges from 0 to 108. The higher the score, the better the quality of life.
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 30 Days	baseline and 30 days	Change in ocular score using a four-point scale from 0 (no dry eye symptoms) to 3 (severe dry eye symptoms) from baseline to 30 days.
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 60 Days	baseline and 60 days	Change in ocular score using a four-point scale from 0 (no dry eye symptoms) to 3 (severe dry eye symptoms).
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 100 Days	baseline and 100 days	Change in ocular score using a four-point scale from 0 (no dry eye symptoms) to 3 (severe dry eye symptoms).
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline to 30 Days	baseline and 30 days	Ophthalmologic assessment to determine changes in dry eye signs/symptoms each graded on a scale of 1 to 4 with 1 being non/mild to 4 being most severe for a total score.
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 60 Days	baseline and 60 days	Ophthalmologic assessment to determine changes in dry eye signs/symptoms each graded on a scale of 1 to 4 with 1 being non/mild to 4 being most severe for a total score.
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 100 Days	baseline and 100 days	Ophthalmologic assessment to determine changes in dry eye signs/symptoms each graded on a scale of 1 to 4 with 1 being non/mild to 4 being most severe for a total score.
Change in Pain Assessment From Baseline to 60 Days	baseline and 60 days	Change in patient reported pain level per treatment eye using 0-10 pain rating scale with zero being no pain to 10 being very severe pain. Each individual pain score was used to estimate a regression slope for each eye with pain as the outcome and day of pAF treatments as the explanatory variable.
Changes in Visual Acuity	baseline, 30 days, 60 days, and 100 days	Ophthalmologic assessment to determine changes in visual acuity related to the administration of pAF.
Effects on the Corneal Surface	Up to 100 days	Ophthalmologic assessment to determine the effects of treatment with pAF on the corneal surface of patients with ocular chronic GVHD.
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 30 Days	baseline and 30 days	Change in Quality of Life questionnaire from baseline to 30 days. FACT-G is a combination score of physical, social/family, emotional, and functional well-being. The score ranges from 0 to 108. The higher the score, the better the quality of life.
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 60 Days	baseline and 60 days	Change in Quality of Life questionnaire from baseline to 60 days. FACT-G is a combination score of physical, social/family, emotional, and functional well-being. The score ranges from 0 to 108. The higher the score, the better the quality of life.

Trial Locations

Locations (1)

University of Utah Huntsman Cancer Institute; 🇺🇸 Salt Lake City, Utah, United States