Ocular Graft Versus Host DiseasePhase 1/2NCT03298815
Study for the Treatment of Ocular Chronic Graft-Versus-Host Disease (GVHD) With Amniotic Fluid Eye Drops (AFED)
A Phase 1/2 study testing Amniotic Fluid Eye Drops (AFED) and Saline Solution in people with Ocular Graft Versus Host Disease, run by University of Utah at 1 site in United States.
Trial summary
A Randomized, Double-blinded, Placebo-Controlled Study for the Treatment of Ocular Chronic Graft Verses Host Disease with Processed Amniotic Fluid (pAF) Drops. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Ocular Graft Versus Host Disease
- What is being tested?
- Amniotic Fluid Eye Drops (AFED) and Saline Solution
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Dec 2019to 24 Feb 2023
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Utah Huntsman Cancer Institute
Salt Lake City, Utah, 84112
About this study
Official title: 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.
Read the 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.
- Conditions
- Ocular Graft Versus Host Disease
- Being tested
- Amniotic Fluid Eye Drops (AFED), Saline Solution
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Amniotic Fluid Eye Drops (AFED)Biological
One drop (0.25 mL) in one eye twice daily for up to 3 months
Saline SolutionOther
One drop (0.25 mL) in the other eye twice daily for up to 3 months
| Study group | Role | What they receive |
|---|---|---|
| Amniotic Fluid Eye Drops (AFED) - All participants, One eye | Active comparator | Not described Receives: Biological: Amniotic Fluid Eye Drops (AFED) |
| Saline Solution - All participants, One eye | Placebo comparator | Not described Receives: Other: Saline Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 30 days
Secondary outcomes (12)
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 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.
Measured: baseline and 30 days
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 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.
Measured: baseline and 60 days
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 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.
Measured: baseline and 100 days
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 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.
Measured: baseline and 30 days
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 60 Days
Change in ocular score using a four-point scale from 0 (no dry eye symptoms) to 3 (severe dry eye symptoms).
Measured: baseline and 60 days
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 100 Days
Change in ocular score using a four-point scale from 0 (no dry eye symptoms) to 3 (severe dry eye symptoms).
Measured: baseline and 100 days
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline to 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.
Measured: baseline and 30 days
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From 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.
Measured: baseline and 60 days
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From 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.
Measured: baseline and 100 days
Change in Pain Assessment From Baseline to 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.
Measured: baseline and 60 days
Changes in Visual Acuity
Ophthalmologic assessment to determine changes in visual acuity related to the administration of pAF.
Measured: baseline, 30 days, 60 days, and 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.
Measured: Up to 100 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2017
- Study start20 Dec 2019
- Primary completion16 Dec 2022
- Study completion24 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Ocular Graft Versus Host DiseaseStudies of the same condition, open and under way.
- More trials run by University of UtahThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Utah
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Catherine Lee, University of Utah
- Investigators
- Catherine J. Lee, MD, Principal investigator, University of Utah
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03298815
- Sponsor's protocol ID
- 103515
- Status last verified
- Apr 2025
- First submitted
- 13 Sep 2017
- Last updated
- 9 Apr 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blinded, Placebo-Controlled Study for the Treatment of Ocular Chronic Graft-Versus-Host Disease (GVHD) With Amniotic Fluid Eye Drops (AFED)
- ClinicalTrials.gov ID
- NCT03298815
- Acronym / sponsor ID
- 103515
- Conditions
- Ocular Graft Versus Host Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- University of UtahUniversity, hospital or other
- Study start
- 20 Dec 2019 (actual)
- Primary completion
- 16 Dec 2022 (actual)
- Study completion
- 24 Feb 2023 (actual)
- First posted
- 2 Oct 2017 (actual)
- Results
- Posted 9 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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:
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Amniotic Fluid Eye Drops (AFED)One drop (0.25 mL) in one eye twice daily for up to 3 months | Biological | Amniotic Fluid Eye Drops (AFED) - All participants, One eye |
| Saline SolutionOne drop (0.25 mL) in the other eye twice daily for up to 3 months | Other | Saline Solution - All participants, One eye |
Primary outcome
Overall Response Rate
Time frame: 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.
12 secondary outcomes
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 30 Days
Time frame: baseline and 30 days
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 60 Days
Time frame: baseline and 60 days
Change in Functional Assessment of Cancer Therapy: General (FACT-G) From Baseline to 100 Days
Time frame: baseline and 100 days
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 30 Days
Time frame: baseline and 30 days
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 60 Days
Time frame: baseline and 60 days
Change in National Institutes of Health (NIH) Consensus Criteria (CC) Ocular Score of Chronic GVHD From Baseline to 100 Days
Time frame: baseline and 100 days
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline to 30 Days
Time frame: baseline and 30 days
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 60 Days
Time frame: baseline and 60 days
Change in Dry Eye Workshop (DEWS) 2007 Dry Eye Severity Grading From Baseline and 100 Days
Time frame: baseline and 100 days
Change in Pain Assessment From Baseline to 60 Days
Time frame: baseline and 60 days
Changes in Visual Acuity
Time frame: baseline, 30 days, 60 days, and 100 days
Effects on the Corneal Surface
Time frame: Up to 100 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Catherine J. Lee, MD principal investigator
University of Utah
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Amniotic Fluid Eye Drops (AFED) and Saline Solution in people with Ocular Graft Versus Host Disease, run by University of Utah at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (time frame: 30 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Apr 2025.