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

A Randomized, Double-Blinded, Placebo-Controlled Study for the Treatment of Ocular Chronic Graft-Versus-Host Disease (GVHD) With Amniotic Fluid Eye Drops (AFED)

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

Study Overview

• Status: Completed
• Conditions: Ocular Graft Versus Host Disease
• Intervention / Treatment: Biological: Amniotic Fluid Eye Drops (AFED); Other: Saline Solution

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 Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • University of Utah Huntsman Cancer Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 after 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Amniotic Fluid Eye Drops (AFED) - All participants, One eye	Biological: Amniotic Fluid Eye Drops (AFED); One drop (0.25 mL) in one eye twice daily for up to 3 months
Placebo Comparator: Saline Solution - All participants, One eye	Other: Saline Solution; One drop (0.25 mL) in the other eye twice daily for up to 3 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Response Rate	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.	100 days
Safety of pAF in patients with chronic ocular GVHD: CTCAE version 4	Serious adverse events and ocular adverse events using CTCAE version 4	100 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Functional Assessment of Cancer Therapy: General (FACT-G)	Change in Quality of Life questionnaire using a five-point scale from 0 (not at all) to 4 (very much).	30 days
Functional Assessment of Cancer Therapy: General (FACT-G)	Change in Quality of Life questionnaire using a five-point scale from 0 (not at all) to 4 (very much).	60 days
Functional Assessment of Cancer Therapy: General (FACT-G)	Change in Quality of Life questionnaire using a five-point scale from 0 (not at all) to 4 (very much).	100 days
National Institutes of Health (NIH) Consensus Criteria (CC) ocular score of chronic GVHD	Change in ocular score using a four-point scale from 0 (no dry eye symptoms) to 3 (severe dry eye symptoms).	30 days
National Institutes of Health (NIH) Consensus Criteria (CC) ocular score of chronic GVHD	Change in ocular score using a four-point scale from 0 (no dry eye symptoms) to 3 (severe dry eye symptoms).	60 days
National Institutes of Health (NIH) Consensus Criteria (CC) ocular score of chronic GVHD	Change in ocular score using a four-point scale from 0 (no dry eye symptoms) to 3 (severe dry eye symptoms).	100 days
Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading	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.	30 days
Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading	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.	60 days
Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading	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.	100 days
Pain Assessment	Change in patient reported pain level using 0-10 pain rating scale with zero being no pain to 10 being very severe pain.	60 days
Changes in Visual Acuity	Ophthalmologic assessment to determine changes in visual acuity related to the administration of pAF.	Up to 100 days
Effects on the Corneal Surface	Ophthalmologic assessment to determine the effects of treatment with pAF on the corneal surface of patients with ocular chronic GVHD.	Up to 100 days

Collaborators and Investigators

• Sponsor: University of Utah
• Investigators: Principal Investigator: Catherine J. Lee, MD, University of Utah

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2019
• Primary Completion (Actual): December 16, 2022
• Study Completion (Actual): February 24, 2023

Study Registration Dates

• First Submitted: September 13, 2017
• First Submitted That Met QC Criteria: September 26, 2017
• First Posted (Actual): October 2, 2017

Study Record Updates

• Last Update Posted (Actual): July 7, 2023
• Last Update Submitted That Met QC Criteria: July 5, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Immune System Diseases; Lung Diseases; Bronchial Diseases; Lung Diseases, Obstructive; Bronchitis; Bronchiolitis Obliterans; Bronchiolitis; Organizing Pneumonia; Graft vs Host Disease; Bronchiolitis Obliterans Syndrome; Pharmaceutical Solutions; Ophthalmic Solutions
• Other Study ID Numbers: 103515

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No