Trial Outcomes & Findings for Study for the Treatment of Ocular Chronic Graft-Versus-Host Disease (GVHD) With Amniotic Fluid Eye Drops (AFED) (NCT NCT03298815)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

15 participants

Primary outcome timeframe

30 days

Results posted on

2025-04-09

Participant Flow

Unit of analysis: eyes

Participant milestones

Participant milestones
Measure	Amniotic Fluid Eye Drops (AFED) Subjects received both Amniotic Fluid Eye Drops and Saline Solution. One eye was randomized to receive amniotic fluid drops and the other eye received saline solution drops. Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution Subjects received both Amniotic Fluid Eye Drops and Saline Solution. One eye was randomized to receive amniotic fluid drops and the other eye received saline solution drops. Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Overall Study STARTED	15 15	15 15
Overall Study COMPLETED	15 15	15 15
Overall Study NOT COMPLETED	0 0	0 0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Participants n=15 Participants Subjects received both Amniotic Fluid Eye Drops and Saline Solution. Each were randomized to one eye. Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Age, Continuous	61 years n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	14 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants
Baseline Functional Assessment of Cancer Therapy - General (FACT-G) Score	88 units on a scale n=5 Participants
Subject wears contacts (scleral lenses)	6 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=12 eyes Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye n=12 eyes Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Overall Response Rate	3 eyes	6 eyes

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=15 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 30 Days	-1 Change in score on a scale Interval -6.0 to 2.7	—

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=15 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 60 Days	2.8 score on a scale Interval -1.8 to 3.2	—

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=15 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 100 Days	3.0 score on a scale Interval -4.0 to 10.3	—

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=13 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 30 Days Better results for pAF eye vs Placebo eye	3 Participants	—
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 30 Days Better results for Placebo eye vs pAF eye	0 Participants	—
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 30 Days Same Directional Results for Both Eyes	10 Participants	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=15 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 60 Days Better results for pAF eye vs. Placebo eye	1 Participants	—
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 60 Days Better results for Placebo eye vs pAF eye	0 Participants	—
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 60 Days Same Directional Results for Both Eyes	14 Participants	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=15 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 100 Days Better results for pAF eye vs Placeob eye	2 Participants	—
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 100 Days Better results for Placebo eye vs pAF eye	0 Participants	—
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 100 Days Same Directional Results for Both Eyes	13 Participants	—

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=13 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline to 30 Days Better results for pAF eye vs Placebo eye	3 Participants	—
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline to 30 Days Better results for Placebo eye vs pAF eye	5 Participants	—
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline to 30 Days Same Directional Results for Both Eyes	5 Participants	—

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=14 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 60 Days Better results for pAF eye vs Placebo eye	0 Participants	—
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 60 Days Better results for Placebo eye vs pAF eye	8 Participants	—
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 60 Days Same Directional Results for Both eyes	6 Participants	—

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=13 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 100 Days Better results for pAF eye vs Placebo eye	1 Participants	—
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 100 Days Better results for Placebo eye vs pAF eye	5 Participants	—
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 100 Days Same Directional Results for Both Eyes	7 Participants	—

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=15 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye n=15 Participants Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Change in Pain Assessment From Baseline to 60 Days	-0.004 regression slope Interval -0.029 to 0.019	-0.010 regression slope Interval -0.021 to 0.009

SECONDARY outcome

Timeframe: baseline, 30 days, 60 days, and 100 days

Population: One participant at the 30 day and 100 day visit did not complete visual acuity assessments.

Ophthalmologic assessment to determine changes in visual acuity related to the administration of pAF.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=14 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Changes in Visual Acuity Change from baseline to 100 days · Better results for pAF eye vs Placebo eye	3 Participants	—
Changes in Visual Acuity Change from baseline to 30 days · Better results for pAF eye vs Placebo eye	5 Participants	—
Changes in Visual Acuity Change from baseline to 30 days · Better results for Placebo eye vs pAF eye	2 Participants	—
Changes in Visual Acuity Change from baseline to 30 days · Same directional results for both eyes	6 Participants	—
Changes in Visual Acuity Change from baseline to 60 days · Better results for pAF eye vs Placebo eye	2 Participants	—
Changes in Visual Acuity Change from baseline to 60 days · Better results for Placebo eye vs pAF eye	5 Participants	—
Changes in Visual Acuity Change from baseline to 60 days · Same directional results for both eyes	7 Participants	—
Changes in Visual Acuity Change from baseline to 100 days · Better results for Placebo eye vs pAF eye	2 Participants	—
Changes in Visual Acuity Change from baseline to 100 days · Same directional results for both eyes	8 Participants	—

SECONDARY outcome

Timeframe: Up to 100 days

Population: A participant missed the 30 day visit and another missed the 100 day visit.

Ophthalmologic assessment to determine the effects of treatment with pAF on the corneal surface of patients with ocular chronic GVHD.

Outcome measures

Outcome measures
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=14 Participants Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Effects on the Corneal Surface 60 day visit · Same directional results for both eyes	7 Participants	—
Effects on the Corneal Surface 30 day visit · Better results for pAF eye vs Placebo eye	1 Participants	—
Effects on the Corneal Surface 30 day visit · Better results for Placebo eye vs pAF eye	7 Participants	—
Effects on the Corneal Surface 30 day visit · Same directional results for both eyes	5 Participants	—
Effects on the Corneal Surface 60 day visit · Better results for pAF eye vs Placebo eye	3 Participants	—
Effects on the Corneal Surface 60 day visit · Better results for Placebo eye vs pAF eye	4 Participants	—
Effects on the Corneal Surface 100 day visit · Better results for pAF eye vs Placebo eye	0 Participants	—
Effects on the Corneal Surface 100 day visit · Better results for Placebo eye vs pAF eye	3 Participants	—
Effects on the Corneal Surface 100 day visit · Same directional results for both eyes	10 Participants	—

Adverse Events

Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Saline Solution - All Participants, One Eye

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye n=15 participants at risk Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months	Saline Solution - All Participants, One Eye n=15 participants at risk Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months
Eye disorders Eye Disorders	6.7% 1/15 • Number of events 1 • Adverse Events are collected through the whole duration of the trial through day 100.	6.7% 1/15 • Number of events 1 • Adverse Events are collected through the whole duration of the trial through day 100.
Eye disorders Eye disorders	6.7% 1/15 • Number of events 1 • Adverse Events are collected through the whole duration of the trial through day 100.	0.00% 0/15 • Adverse Events are collected through the whole duration of the trial through day 100.
General disorders General disorders and administration site conditions	6.7% 1/15 • Number of events 1 • Adverse Events are collected through the whole duration of the trial through day 100.	0.00% 0/15 • Adverse Events are collected through the whole duration of the trial through day 100.

Additional Information

Catherine Lee, MD

University of Utah

Phone: 206-667-4922

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place