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

NCT03298815 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: 103515
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Ocular Graft Versus Host Disease

Outcome Measures

Primary Outcomes (1)

• 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.

  30 days

Secondary Outcomes (12)

• Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 30 Days

  baseline and 30 days

• Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 60 Days

  baseline and 60 days

• Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 100 Days

  baseline and 100 days

• 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 National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 60 Days

  baseline and 60 days

Study Arms (2)

Amniotic Fluid Eye Drops (AFED) - All participants, One eye

ACTIVE COMPARATOR

Biological: Amniotic Fluid Eye Drops (AFED)

Saline Solution - All participants, One eye

PLACEBO COMPARATOR

Other: Saline Solution

Interventions

Amniotic Fluid Eye Drops (AFED) BIOLOGICAL

One drop (0.25 mL) in one eye twice daily for up to 3 months

Amniotic Fluid Eye Drops (AFED) - All participants, One eye

Saline Solution OTHER

One drop (0.25 mL) in the other eye twice daily for up to 3 months

Saline Solution - All participants, One eye

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• 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)
• Unable to work because of ocular symptoms
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Utah: lead

Study Sites (1)

University of Utah Huntsman Cancer Institute

Salt Lake City, Utah, 84112, United States

Location

MeSH Terms

Interventions

Saline Solution

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Limitations and Caveats

Main limitation of this study was the randomization of eyes within subjects as some had mild asymmetric disease between eyes.

Results Point of Contact

• Title: Catherine Lee, MD
• Organization: University of Utah

Catherine.Lee@hci.utah.edu

206-667-4922

Study Officials

• Catherine J. Lee, MD

  University of Utah

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: September 13, 2017
• First Posted: October 2, 2017
• Study Start: December 20, 2019
• Primary Completion: December 16, 2022
• Study Completion: February 24, 2023
• Last Updated: April 9, 2025
• Results First Posted: April 9, 2025

Record last verified: 2025-04

Locations

US (1)