Graft-versus-host DiseasePhase 1NCT02561520
Safety and Efficacy of Autologous PRP and PPP Eye Drops in the Treatment of Ocular GVHD
A Phase 1 study testing PRP eye drops and PPP eye drops in people with Graft-versus-host Disease, run by Ladan Espandar at 1 site in United States.
Trial summary
The purpose of this study is to evaluate the safety and efficacy of autologous Platelet Rich Plasma (PRP) and Platelet Poor Plasma (PPP) eye drops four times a day in the treatment of ocular graft versus host disease (O-GVHD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Graft-versus-host Disease
- What is being tested?
- PRP eye drops and PPP eye drops
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2016to Sep 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Age ≥18 years.
- Willing and able to provide written informed consent.
- Willing and able to comply with study assessments for the full duration of the study.
- Diagnosis of ocular GVHD.
- Minimum corneal fluorescein staining of 4 (NEI grading scheme, 0-15) in at least one eye.
- In good stable overall health.
Who cannot join
- Remission from primary cancer in more than 5 years.
- History of thrombocytopenia (platelet<50,000) in the last 2 weeks before study entry.
- Ocular or periocular malignancy.
- Significant change, as judged by the PI, in systemic immunosuppressive regimen before 2 weeks of study entry.
- Any change in dosage of tetracycline compounds (tetracycline, doxycycline, and minocycline) within the last month.
- Any change in frequency of preserved anti-glaucoma medications before 2 weeks of study entry.
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- Current use of topical steroids more than twice a day.
- Change in frequency of topical cyclosporine and/or topical kineret within the last month.
- Signs of current infection, including fever and current treatment with antibiotics.
- Intra-ocular surgery or ocular laser surgery within the last 3 months.
- Has worn contact lenses, except for bandage contact lens or rigid gas permeable lens or scleral contact lens, for the last 2 weeks prior to the study or would be unable to stay off contact lenses for the study duration.
- Any condition (including language barrier) that precludes patient's ability to comply with study requirements including completion of study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Safety and Efficacy of Autologous Platelet Rich Plasma and Platelet Poor Plasma Eye Drops in the Treatment of Ocular Graft-Versus-Host Disease
The purpose of this study is to evaluate the safety and efficacy of autologous Platelet Rich Plasma (PRP) and Platelet Poor Plasma (PPP) eye drops four times a day in the treatment of ocular graft versus host disease (O-GVHD). In addition to their current medication (except autologous serum drops), patients will receive PRP and PPP drops.
Read the detailed description
Ocular involvement can be quite symptomatic in patients with chronic graft-versus-host disease (GVHD). The impact of ocular GVHD on quality of life (QOL) in patients with chronic GVHD has been studied in a prospective, multicenter, longitudinal, observational study and showed that ocular GVHD affects 57% of patients within 2 years of chronic GVHD diagnosis. Strong evidence suggested that ocular GVHD is associated with worse overall health-related QOL. Significant worsening of vision-related QOL in ocular GVHD has been reported. Ocular GVHD is devastating and there is no effective treatment available so far. The importance of this study is that for the first time in the nation, our institute will evaluate the safety and efficacy of topical autologous blood product (PRP and PPP) to treat ocular surface disease associated with ocular GVHD.
- Conditions
- Graft-versus-host Disease
- Being tested
- PRP eye drops, PPP eye drops
- Keywords
- platelet rich plasma, platelet poor plasma, ocular GVHD
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PRP eye dropsBiological
Also called Platelet rich plasma
Eye drops 4x a day, patients will start this eye drop first.
PPP eye dropsBiological
Also called Platelet poor plasma
Eye drops 4x a day, patients will start this eye drops after PRP.
| Study group | Role | What they receive |
|---|---|---|
| PRP and PPP | Experimental | PRP eye drops and PPP eye drops will be prepared from patient's own blood by Magellan technology. Patients will receive eye drops in sterile amber glass droppers. Patients will be instructed to keep refrigerated each bottle after opening for 7 days and keep frozen the unopened bottles up to 30 days. Receives: Biological: PRP eye drops; Biological: PPP eye drops |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Related Adverse Events
Safety and tolerability of topical autologous PRP and PPP four times a day will be monitored by the occurrence of systemic and ocular adverse events in addition to symptoms directly related to the instillation or use of the autologous blood products. The severity of each adverse event and relation to the study medication will be graded possibly, probably, or definitely related. Tolerability measures will be graded from trace to severe using a direct query method at each visit.
Measured: 8 Weeks
Secondary outcomes (7)
Efficacy of topical autologous PRP and PPP as measured by the National Eye Institute (NEI) grading scale
To evaluate the efficacy of topical autologous PRP and PPP four times a day in reducing signs of dry eye in ocular GVHD, cornea fluorescein staining will be used using NEI grading system.
Measured: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by Tear Film Break Up Time (TBUT)
To evaluate the efficacy of topical autologous PRP and PPP four times a day in reducing signs of dry eye in ocular GVHD,Tear Film Break Up Time (TBUT) will be used.
Measured: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by Schirmer Test I
To evaluate the efficacy of topical autologous PRP and PPP four times a day in reducing signs of dry eye in ocular GVHD, Schirmer test without topical anesthetic will be used.
Measured: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by expression of cellular markers of inflammation using real-time polymerase chain reaction (RT-PCR)
To evaluate the efficacy of topical autologous PRP and PPP four times a day in reducing signs of dry eye in ocular GVHD, expression of cellular markers of inflammation such as (intercellular adhesion molecule-1 (ICAM-1), interleukin IL-1b, IL-2, IL-6, IL-8, IL-10, IL-17, IL-23, interferon IFN-g and tumor necrosis factor TNF-α) will be used using real-time polymerase chain reaction (RT-PCR) on schirmer filter papers.
Measured: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by expression of cellular markers of inflammation using flow cytometry (FC)
To evaluate the efficacy of topical autologous PRP and PPP four times a day in reducing signs of dry eye in ocular GVHD, expression of cellular markers of inflammation such as (intercellular adhesion molecule-1 (ICAM-1), interleukin IL-1b, IL-2, IL-6, IL-8, IL-10, IL-17, IL-23, interferon IFN-g and tumor necrosis factor TNF-α) will be used using flow cytometry (FC) on schirmer filter papers.
Measured: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by Ocular Surface Disease Index (OSDI) questionnaire
To evaluate the efficacy of topical autologous PRP and PPP four times a day in reducing symptoms of dry eye in ocular GVHD by Surface Disease Index (OSDI) questionnaire
Measured: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by National Eye Institute-Visual Function Questionnaire (NEI-VFQ-25)
To evaluate the efficacy of topical autologous PRP and PPP four times a day in reducing symptoms of dry eye in ocular GVHD by National Eye Institute-Visual Function Questionnaire (NEI-VFQ-25)
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2015 (estimated)
- Study startDec 2016
- Primary completionSep 2018 (estimated)
- Study completionSep 2018 (estimated)
Why it stopped: Study did not enroll participants is being withdrawn from the system.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft-versus-host DiseaseStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ladan Espandar
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ladan Espandar, University of Pittsburgh
- Investigators
- Ladan Espandar, MD, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02561520
- Sponsor's protocol ID
- PRO15070211
- Status last verified
- Aug 2017
- First submitted
- 17 Sep 2015
- Last updated
- 28 Aug 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Autologous Platelet Rich Plasma and Platelet Poor Plasma Eye Drops in the Treatment of Ocular Graft-Versus-Host Disease
- ClinicalTrials.gov ID
- NCT02561520
- Acronym / sponsor ID
- PRO15070211
- Conditions
- Graft-versus-host Disease
- Keywords
- platelet rich plasma, platelet poor plasma, ocular GVHD
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study did not enroll participants is being withdrawn from the system.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Ladan EspandarUniversity, hospital or other
- Study start
- Dec 2016
- Primary completion
- Sep 2018 (estimated)
- Study completion
- Sep 2018 (estimated)
- First posted
- 28 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety and efficacy of autologous Platelet Rich Plasma (PRP) and Platelet Poor Plasma (PPP) eye drops four times a day in the treatment of ocular graft versus host disease (O-GVHD). In addition to their current medication (except autologous serum drops), patients will receive PRP and PPP drops.
Detailed description
Ocular involvement can be quite symptomatic in patients with chronic graft-versus-host disease (GVHD). The impact of ocular GVHD on quality of life (QOL) in patients with chronic GVHD has been studied in a prospective, multicenter, longitudinal, observational study and showed that ocular GVHD affects 57% of patients within 2 years of chronic GVHD diagnosis. Strong evidence suggested that ocular GVHD is associated with worse overall health-related QOL. Significant worsening of vision-related QOL in ocular GVHD has been reported. Ocular GVHD is devastating and there is no effective treatment available so far. The importance of this study is that for the first time in the nation, our institute will evaluate the safety and efficacy of topical autologous blood product (PRP and PPP) to treat ocular surface disease associated with ocular GVHD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PRP and PPP | Experimental | Biological: PRP eye drops, Biological: PPP eye dropsPRP eye drops and PPP eye drops will be prepared from patient's own blood by Magellan technology. Patients will receive eye drops in sterile amber glass droppers. Patients will be instructed to keep refrigerated each bottle after opening for 7 days and keep frozen the unopened bottles up to 30 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age ≥18 years.
- Willing and able to provide written informed consent.
- Willing and able to comply with study assessments for the full duration of the study.
- Diagnosis of ocular GVHD.
- Minimum corneal fluorescein staining of 4 (NEI grading scheme, 0-15) in at least one eye.
Key exclusion criteria (12 listed)
- Remission from primary cancer in more than 5 years.
- History of thrombocytopenia (platelet<50,000) in the last 2 weeks before study entry.
- Ocular or periocular malignancy.
- Significant change, as judged by the PI, in systemic immunosuppressive regimen before 2 weeks of study entry.
- Any change in dosage of tetracycline compounds (tetracycline, doxycycline, and minocycline) within the last month.
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 |
|---|---|---|
| PRP eye dropsAlso: Platelet rich plasmaEye drops 4x a day, patients will start this eye drop first. | Biological | PRP and PPP |
| PPP eye dropsAlso: Platelet poor plasmaEye drops 4x a day, patients will start this eye drops after PRP. | Biological | PRP and PPP |
Primary outcome
Number of Participants With Treatment-Related Adverse Events
Time frame: 8 Weeks
Safety and tolerability of topical autologous PRP and PPP four times a day will be monitored by the occurrence of systemic and ocular adverse events in addition to symptoms directly related to the instillation or use of the autologous blood products. The severity of each adverse event and relation to the study medication will be graded possibly, probably, or definitely related. Tolerability measures will be graded from trace to severe using a direct query method at each visit.
7 secondary outcomes
Efficacy of topical autologous PRP and PPP as measured by the National Eye Institute (NEI) grading scale
Time frame: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by Tear Film Break Up Time (TBUT)
Time frame: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by Schirmer Test I
Time frame: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by expression of cellular markers of inflammation using real-time polymerase chain reaction (RT-PCR)
Time frame: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by expression of cellular markers of inflammation using flow cytometry (FC)
Time frame: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by Ocular Surface Disease Index (OSDI) questionnaire
Time frame: 8 weeks
Efficacy of topical autologous PRP and PPP as measured by National Eye Institute-Visual Function Questionnaire (NEI-VFQ-25)
Time frame: 8 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2017.
- Individual participant dataSharing planned: Undecided
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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.
Ladan Espandar, MD principal investigator
University of Pittsburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PRP eye drops and PPP eye drops in people with Graft-versus-host Disease, run by Ladan Espandar 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 6 inclusion and 12 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: withdrawn, as of the sponsor's last update on 28 Aug 2017. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Number of Participants With Treatment-Related Adverse Events (time frame: 8 Weeks). 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 28 Aug 2017.