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A Randomized, Double-blinded, Placebo-Controlled Study for the Treatment of Ocular Chronic Graft Verses Host Disease with Processed Amniotic Fluid (pAF) Drops.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Amniotic Fluid Eye Drops (AFED) - All participants, One eye | Active Comparator |
| |
| Saline Solution - All participants, One eye | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Amniotic Fluid Eye Drops (AFED) | Biological | One drop (0.25 mL) in one eye twice daily for up to 3 months |
|
| 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. | 30 days |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | baseline and 30 days |
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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:
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:
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| Name | Affiliation | Role |
|---|---|---|
| Catherine J. Lee, MD | University of Utah | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Utah Huntsman Cancer Institute | Salt Lake City | Utah | 84112 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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 |
| FG001 | 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 |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | All 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 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Count of Units | eyes | 30 days | eyes | eyes |
|
Adverse Events are collected through the whole duration of the trial through day 100.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Amniotic Fluid Eye Drops (AFED) - All Participants, One Eye | Amniotic Fluid Eye Drops (AFED): One drop (0.25 mL) in one eye twice daily for up to 3 months |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eye Disorders | Eye disorders | MedDRA (26.1) | Systematic Assessment |
Main limitation of this study was the randomization of eyes within subjects as some had mild asymmetric disease between eyes.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Catherine Lee, MD | University of Utah | 206-667-4922 | Catherine.Lee@hci.utah.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 14, 2022 | Nov 28, 2023 | Prot_001.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 13, 2019 | Nov 28, 2023 | SAP_002.pdf |
| ICF | No | No | Yes | Informed Consent Form | Jun 11, 2021 | Nov 28, 2023 | ICF_003.pdf |
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| ID | Term |
|---|---|
| D000077330 | Saline Solution |
| ID | Term |
|---|---|
| D000077324 | Crystalloid Solutions |
| D007552 | Isotonic Solutions |
| D012996 | Solutions |
| D004364 | Pharmaceutical Preparations |
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| Saline Solution | Other | 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 | 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. | 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. | 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. | 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). | 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). | 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. | 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. | 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. | 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. | baseline and 60 days |
| Changes in Visual Acuity | Ophthalmologic assessment to determine changes in visual acuity related to the administration of pAF. | 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. | Up to 100 days |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Baseline Functional Assessment of Cancer Therapy - General (FACT-G) Score | The FACT-G is a 27-item patient-reported questionnaire used to assess the impact of cancer therapy on quality of life. The questionnaire has 5-point Likert scale ranging from 0 (not at all) to 4 (very much) for every question. Questions are added together for an overall score. Higher scores represent better quality of life (range from 0 to 108). | Median | Full Range | units on a scale |
|
| Subject wears contacts (scleral lenses) | Count of Participants | Participants |
|
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|
|
| Secondary | 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. | Posted | Median | Inter-Quartile Range | Change in score on a scale | baseline and 30 days |
|
|
|
|
| Secondary | 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. | Posted | Median | Inter-Quartile Range | score on a scale | baseline and 60 days |
|
|
|
|
| Secondary | 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. | Posted | Median | Inter-Quartile Range | score on a scale | baseline and 100 days |
|
|
|
|
| Secondary | 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. | Posted | Count of Participants | Participants | baseline and 30 days |
|
|
|
|
| Secondary | 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). | Posted | Count of Participants | Participants | baseline and 60 days |
|
|
|
|
| Secondary | 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). | Posted | Count of Participants | Participants | baseline and 100 days |
|
|
|
|
| Secondary | 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. | Posted | Count of Participants | Participants | baseline and 30 days |
|
|
|
|
| Secondary | 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. | Posted | Count of Participants | Participants | baseline and 60 days |
|
|
|
|
| Secondary | 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. | Posted | Count of Participants | Participants | baseline and 100 days |
|
|
|
|
| Secondary | 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. | Posted | Median | Inter-Quartile Range | regression slope | baseline and 60 days |
|
|
|
|
| Secondary | Changes in Visual Acuity | Ophthalmologic assessment to determine changes in visual acuity related to the administration of pAF. | One participant at the 30 day and 100 day visit did not complete visual acuity assessments. | Posted | Count of Participants | Participants | baseline, 30 days, 60 days, and 100 days |
|
|
|
|
| Secondary | 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. | A participant missed the 30 day visit and another missed the 100 day visit. | Posted | Count of Participants | Participants | Up to 100 days |
|
|
|
|
| 0 |
| 15 |
| 0 |
| 15 |
| 3 |
| 15 |
| EG001 | Saline Solution - All Participants, One Eye | Saline Solution: One drop (0.25 mL) in the other eye twice daily for up to 3 months | 0 | 15 | 0 | 15 | 1 | 15 |
| Eye disorders | Eye disorders | MedDRA (26.1) | Systematic Assessment | Eye inflammation |
|
| General disorders and administration site conditions | General disorders | MedDRA (26.1) | Systematic Assessment | Application site pain |
|
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| Same directional results for both eyes |
|
| Change from baseline to 60 days |
|
|
| Change from baseline to 100 days |
|
|
60 day visit
| Superiority |
| Wilcoxon (Mann-Whitney) | 0.750 | 100 day visit | Superiority |
| Same directional results for both eyes |
|
| 60 day visit |
|
|
| 100 day visit |
|
|
60 day visit
| Superiority |
| Wilcoxon (Mann-Whitney) | 0.250 | 100 day visit | Superiority |