Graft Versus Host DiseasePhase 3oGVHDNCT03591874
Study of Brimonidine Tartrate Nanoemulsion Eye Drops in Patients With Ocular Graft-vs-Host Disease
A Phase 3 study testing Brimonidine Tartrate in people with Graft Versus Host Disease and Ocular Surface Disease, run by Ocugen at 13 sites in United States.
Trial summary
This study evaluates the use of Brimonidine tartrate nanoemulsion eye drop solution in the treatment of ocular Graft Verses Host Disease (oGVHD). Two thirds of participants will receive Brimonidine and one third will receive ophthalmic buffered saline (placebo). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Graft Versus Host Disease, Ocular Surface Disease, oGVHD
- What is being tested?
- Brimonidine Tartrate
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 59 participants
- Where
- 13 sitesUnited States
- Study dates
- 23 Dec 2018to 30 Apr 2020
- Phase
- Phase 3
- 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
- Men or women ≥ 18 years of age
- Diagnosis of definite oGVHD using the International Chronic Ocular GVHD Consensus Group Revised Diagnostic criteria in at least one eye.
- Ocular Discomfort score ≥ 3
- Validated Bulbar Redness score ≥ 40 in both eyes
- Subjects who are capable and willing to provide informed consent and follow study instructions
- Intraocular pressure (IOP) ≥ 5 mmHg and ≤22 mmHg in each eye
- Women who are not pregnant or lactating or post-menopausal or have undergone a sterilization procedure
Who cannot join
- Allergic to brimonidine or any similar products, or excipients of brimonidine
- Currently receiving any brimonidine or other treatment for glaucoma
- Receiving or have received any investigational drug or device within 30 days of screening
- Current use of contact lenses 14 days prior to screening
- Active ocular infection or ocular allergies
- Any history of eyelid surgery or ocular surgery within the past 3 months
Show all 9Show fewer
- Corneal epithelial defect larger than 1 mm (squared) in either eye
- Received corticosteroid-containing eye drops within 14 days prior to screening or planned use during the study
- Any change in corticosteroid-containing eye drops, systemic corticosteroids/immunosuppressives, topical ocular antibiotics, cyclosporine ophthalmic emulsion 0.05% (Restasis®), cyclosporine ophthalmic solution 0.09% (Cequa®) or lifitegrast ophthalmic solution 5% (Xiidra®), or autologous serum tears within 30 days prior to screening or planned change during the study
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
University of Michigan, Kellogg Eye Center
Ann Arbor, Michigan, 48105
Emory Eye Center
Atlanta, Georgia, 30322
Ohio State University
Columbus, Ohio, 43210
Duke University Medical Center
Durham, North Carolina, 27708
University of Wisconsin Dept. of Ophthalmology and Visual Sciences
Madison, Wisconsin, 53705
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Byers Eye Institute at Stanford University
Palo Alto, California, 94303
University of Penn Scheie Eye Institute,
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Phoenix, Arizona, 85054
Univeristy of Pittsburgh Medical Center Eye Center
Pittsburgh, Pennsylvania, 15213
OHSU Casey Eye Institute | Cornea Division
Portland, Oregon, 97239
University of Kansas Medical Center
Prairie Village, Kansas, 66208
About this study
Official title: A Phase 3 Randomized, Placebo-Controlled, Double-Masked, Multicenter, Safety and Efficacy Study of Brimonidine Tartrate Nanoemulsion Eye Drops in Patients With Ocular Graft-vs-Host Disease
This study evaluates the use of Brimonidine tartrate nanoemulsion eye drop solution in the treatment of ocular Graft Verses Host Disease (oGVHD). Two thirds of participants will receive Brimonidine and one third will receive ophthalmic buffered saline (placebo).
Read the detailed description
Ocular GVHD (oGVHD) is a common complication that occurs in 40-60% of patients who have undergone allogeneic bone marrow transplants. Driven by inflammation, oGVHD can result in damage to the ocular surface and tear-producing glands, which over time significantly diminishes quality of life and restricts daily activities due to visual impairment.
Early studies in animals and humans indicate that Brimonidine nano-emulsion, also known as OCU300, may relieve the signs and symptoms of oGVHD. These symptoms include blurry vision, foreign body sensation, burning sensation, severe light sensitivity, chronic conjunctivitis (pink or red eye), dry eyes and eye pain.
This study will be a randomized, placebo-controlled, double-masked, multicenter phase 3 study in the United States conducted at approximately 15 centers. Upon meeting the eligibility criteria, enrolled subjects with a diagnosis of definite oGVHD will be randomly assigned in a 2:1 (test: control) fashion to receive either Brimonidine Nanoemulsion Eye Drops 0.18% investigational product (test) or ophthalmic buffered saline (placebo).
- Conditions
- Graft Versus Host Disease, Ocular Surface Disease, oGVHD, Ocular Graft vs Host Disease, Brimonidine Tartrate, Lubricant Eye Drops
- Being tested
- Brimonidine Tartrate, Placebos
- Keywords
- ogvhd, ocular graft vs host, Ocular Surface Disease, Ocular Graft vs Host Disease, Brimonidine Tartrate, Lubricant Eye Drops, Ophthalmic Solutions, Graft vs Host Disease
- 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.
Brimonidine TartrateDrug
Also called OCU-300
Brimonidine Tartrate Nanoemulsion Eye Drops given 2 times a day for 12 weeks.
PlacebosDrug
Also called Placebo (For Brimonidine Tartrate)
Opthalmic buffered saline solution Eye Drops given 2 times a day for 12 weeks.
| Study group | Role | What they receive |
|---|---|---|
| OCU-300 | Experimental | Brimonidine Tartrate Nanoemulsion Eye Drops 0.18% given 2 times a day for 12 weeks. Receives: Drug: Brimonidine Tartrate |
| Placebos | Placebo comparator | Placebo - Ophthalmic buffered saline Eye Drops given 2 times a day for 12 weeks. Receives: Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to Day 84 in Validated Bulbar Redness (VBR) Score
The VBR consists of a set of ten images illustrating different degrees of ocular redness, ranging from normal to severe, and each image is assigned a value from 10 (least redness) to 100 (most redness). The bulbar conjunctival injection of the participant's eye (nasal and temporal) was examined via slit-lamp examination and compared to the reference images in the VBR and graded accordingly.
Measured: Baseline, Day 84
Change From Baseline to Day 84 in Ocular Discomfort Visual Analog Scale (VAS) Score
Participants were asked to rate their worst ocular pain/discomfort in the preceding 24 hours using a 10-point scale ranging from "None" (score=0) to "Unbearable/Excruciating" (score=10).
Measured: Baseline, Day 84
Secondary outcomes (1)
Change From Baseline to Day 84 in Symptom Assessment iN Dry Eye (SANDE) Questionnaire Scores
The SANDE questionnaire is a short VAS assessment that quantifies both severity and frequency of current dry eye symptoms. The SANDE is comprised of two questions, and each question employs a 100-mm horizontal linear VAS. The measurement of symptom frequency ranges from "rarely" to "all of the time", and the symptom severity from "very mild" to "very severe". Data collected from the SANDE questionnaire was calculated by multiplying the frequency score by the severity score and obtaining the square root. The result is the Overall SANDE score which ranges from 0 to 100, with 100 being the maximal amount of dry eye symptoms and 0 being the minimal amount of dry eye symptoms.
Measured: Baseline, Day 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2018
- Study start23 Dec 2018
- Primary completion30 Apr 2020
- Study completion30 Apr 2020
Why it stopped: Primary endpoint was not met.
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 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This will be a randomized, placebo-controlled, double-masked, multicenter phase 3 study.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ocugen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03591874
- Sponsor's protocol ID
- OCU-300-301
- Acronym
- oGVHD
- Status last verified
- Jun 2022
- First submitted
- 9 Jul 2018
- Last updated
- 6 Jul 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: There is no current plan to share participant data with other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Placebo-Controlled, Double-Masked, Multicenter, Safety and Efficacy Study of Brimonidine Tartrate Nanoemulsion Eye Drops in Patients With Ocular Graft-vs-Host Disease
- ClinicalTrials.gov ID
- NCT03591874
- Acronym / sponsor ID
- oGVHD / OCU-300-301
- Conditions
- Graft Versus Host Disease, Ocular Surface Disease, oGVHD, Ocular Graft vs Host Disease, Brimonidine Tartrate, Lubricant Eye Drops
- Keywords
- ogvhd, ocular graft vs host, Ocular Surface Disease, Ocular Graft vs Host Disease, Brimonidine Tartrate, Lubricant Eye Drops, Ophthalmic Solutions, Graft vs Host Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Primary endpoint was not met.
- Enrolment
- 59 participants (actual)
- Lead sponsor
- OcugenIndustry (pharma, biotech, devices)
- Study start
- 23 Dec 2018 (actual)
- Primary completion
- 30 Apr 2020 (actual)
- Study completion
- 30 Apr 2020 (actual)
- First posted
- 19 Jul 2018 (actual)
- Results
- Posted 6 Jul 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study evaluates the use of Brimonidine tartrate nanoemulsion eye drop solution in the treatment of ocular Graft Verses Host Disease (oGVHD). Two thirds of participants will receive Brimonidine and one third will receive ophthalmic buffered saline (placebo).
Detailed description
Ocular GVHD (oGVHD) is a common complication that occurs in 40-60% of patients who have undergone allogeneic bone marrow transplants. Driven by inflammation, oGVHD can result in damage to the ocular surface and tear-producing glands, which over time significantly diminishes quality of life and restricts daily activities due to visual impairment.
Early studies in animals and humans indicate that Brimonidine nano-emulsion, also known as OCU300, may relieve the signs and symptoms of oGVHD. These symptoms include blurry vision, foreign body sensation, burning sensation, severe light sensitivity, chronic conjunctivitis (pink or red eye), dry eyes and eye pain.
This study will be a randomized, placebo-controlled, double-masked, multicenter phase 3 study in the United States conducted at approximately 15 centers. Upon meeting the eligibility criteria, enrolled subjects with a diagnosis of definite oGVHD will be randomly assigned in a 2:1 (test: control) fashion to receive either Brimonidine Nanoemulsion Eye Drops 0.18% investigational product (test) or ophthalmic buffered saline (placebo).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This will be a randomized, placebo-controlled, double-masked, multicenter phase 3 study.
| Arm | Type | Receives |
|---|---|---|
| OCU-300 | Experimental | Drug: Brimonidine TartrateBrimonidine Tartrate Nanoemulsion Eye Drops 0.18% given 2 times a day for 12 weeks. |
| Placebos | Placebo comparator | Drug: PlacebosPlacebo - Ophthalmic buffered saline Eye Drops given 2 times a day for 12 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Men or women ≥ 18 years of age
- Diagnosis of definite oGVHD using the International Chronic Ocular GVHD Consensus Group Revised Diagnostic criteria in at least one eye.
- Ocular Discomfort score ≥ 3
- Validated Bulbar Redness score ≥ 40 in both eyes
- Subjects who are capable and willing to provide informed consent and follow study instructions
Key exclusion criteria (9 listed)
- Allergic to brimonidine or any similar products, or excipients of brimonidine
- Currently receiving any brimonidine or other treatment for glaucoma
- Receiving or have received any investigational drug or device within 30 days of screening
- Current use of contact lenses 14 days prior to screening
- Active ocular infection or ocular allergies
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 |
|---|---|---|
| Brimonidine TartrateAlso: OCU-300Brimonidine Tartrate Nanoemulsion Eye Drops given 2 times a day for 12 weeks. | Drug | OCU-300 |
| PlacebosAlso: Placebo (For Brimonidine Tartrate)Opthalmic buffered saline solution Eye Drops given 2 times a day for 12 weeks. | Drug | Placebos |
Primary outcome
Change From Baseline to Day 84 in Validated Bulbar Redness (VBR) Score
Time frame: Baseline, Day 84
The VBR consists of a set of ten images illustrating different degrees of ocular redness, ranging from normal to severe, and each image is assigned a value from 10 (least redness) to 100 (most redness). The bulbar conjunctival injection of the participant's eye (nasal and temporal) was examined via slit-lamp examination and compared to the reference images in the VBR and graded accordingly.
Change From Baseline to Day 84 in Ocular Discomfort Visual Analog Scale (VAS) Score
Time frame: Baseline, Day 84
Participants were asked to rate their worst ocular pain/discomfort in the preceding 24 hours using a 10-point scale ranging from "None" (score=0) to "Unbearable/Excruciating" (score=10).
1 secondary outcome
Change From Baseline to Day 84 in Symptom Assessment iN Dry Eye (SANDE) Questionnaire Scores
Time frame: Baseline, Day 84
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. There is no current plan to share participant data with other researchers.
Related trials for Graft Versus Host Disease
Active, not recruitingPhase 2
De-escalated Cyclophosphamide (PTCy) and Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis
Graft-versus-host Disease
RecruitingEarly Phase 1
HMCT/CT2401: Abatacept GVHD Prophylaxis Following Omidubicel HCT
Hematologic Malignancy
RecruitingObservational study
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
Graft-Versus-Host Disease, Graft-versus-leukemia
RecruitingPhase 1/2
The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
Peripheral Blood Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation
RecruitingPhase 1/2
Phase I/II Study of Pacritinib, A JAK2/IRAK1/CSF1R Inhibitor, in Refractory Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Graft vs Host Disease
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.
The record lists no contact. Use the official record to reach the sponsor.
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Brimonidine Tartrate in people with Graft Versus Host Disease and Ocular Surface Disease, run by Ocugen at 13 sites 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 9 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: stopped early, as of the sponsor's last update on 6 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 6 Jul 2022). 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?
Change From Baseline to Day 84 in Validated Bulbar Redness (VBR) Score (time frame: Baseline, Day 84). The study lists 2 primary outcomes in all. 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 6 Jul 2022.