Neurotrophic KeratopathyPhase 3NCT02600429
Assessment of the Safety and Efficacy Study of RGN-259 Ophthalmic Solutions for Neurotrophic Keratopathy : SEER-1
A Phase 3 study testing RGN-259 in people with Neurotrophic Keratopathy, run by ReGenTree, LLC at 11 sites in United States.
Trial summary
The objective of this study is to assess the safety and efficacy of RGN-259 Ophthalmic Solution compared to placebo for the treatment of NK. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neurotrophic Keratopathy
- What is being tested?
- RGN-259
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 18 participants
- Where
- 11 sitesUnited States
- Study dates
- 17 Sep 2015to 9 Mar 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
- Be male or female of any race, at least 18 years of age
- Have provided verbal and written informed consent.
- Be able and willing to follow instructions, including participation in all study assessments and visits;
- Have stage 2 or 3 neurotrophic keratopathy in at least one eye If a female of childbearing potential, have a negative urine pregnancy test at Visit 1 and agree to use an adequate method of birth control throughout the study period.
Who cannot join
- Have any clinically significant slit lamp findings at Visit 1 that in the opinion of the investigator may interfere with the study parameters;
- Have significant blepharitis, meibomian gland dysfunction (MGD), lid margin inflammation or active ocular allergy that requires treatment
- Have a lid function abnormality (ex. Lagophthalmos) which, in the opinion of the investigator, is the primary cause of the persistent epithelial defect;
- Be diagnosed with ongoing ocular infection (bacterial, viral or fungal) or active inflammation (e.g. follicular conjunctivitis) not related to NK
- Anticipate the use of fluoroquinolone-containing antibiotic eye drops during the study;
- Have used contact lenses (excluding therapeutic contact lenses) within 14 days prior to Visit 1 or anticipates use of contact lenses during the study period;
- Have an uncontrolled systemic disease that in the opinion of the investigator may interfere with the study parameters;
- Anticipate a change in immunosuppressive therapy during the course of the study;
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Vision Institute
Colorado Springs, Colorado, 80907
Eye Center of Northern Colorado
Fort Collins, Colorado, 80525
Midwest Cornea Associates, LLC
Indianapolis, Indiana, 46290
Hull Eye Center
Lancaster, California, 93534
Central Maine Eye Care
Lewiston, Maine, 04240
Koffler Vision Group
Lexington, Kentucky, 40509
Richard Eiferman, MD, PSC
Louisville, Kentucky, 40205
The Eye Care Institute
Louisville, Kentucky, 40206
Insight Vision Group
Parker, Colorado, 80134
Black Hills Regional Eye Institute
Rapid City, South Dakota, 57701
Medical Faculty Associates, Inc.
Washington D.C., District of Columbia, 20037
About this study
Official title: Phase 3, Multi-Center, Randomized, Double Masked, Placebo Controlled Clinical Study to Assess the Safety and Efficacy of RGN-259 Ophthalmic Solution for the Treatment of Neurotrophic Keratopathy: SEER-1
The objective of this study is to assess the safety and efficacy of RGN-259 Ophthalmic Solution compared to placebo for the treatment of NK.
Read the detailed description
Neurotrophic keratopathy (NK) is a degenerative corneal disease that occurs as a result of partial or total impairment of trigeminal innervation. The resulting loss of corneal sensitivity (anesthesia) leads to a reduction in lacrimation and a decline in status, metabolism, and mitosis of corneal epithelial cells. Previous studies (physician-sponsored studies) used to treat to nine patients with NK, six of whom had discrete geographic, non-healing lesions, and three of whom had punctate lesions and the study result reported.
- Conditions
- Neurotrophic Keratopathy
- Being tested
- RGN-259, Placebo
- Keywords
- Neurotrophic Keratopathy, NK
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RGN-259Drug
Also called Tβ4
A preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into affected eye(s), five times a day for 4 weeks.
PlaceboDrug
Also called Vehicle Control
It is composed of the same excipients as RGN-259 but does not contain Tβ4
| Study group | Role | What they receive |
|---|---|---|
| RGN-259 | Experimental | It is a preservative-free, sterile eye drop solution containing Tβ4 Receives: Drug: RGN-259 |
| Placebo | Placebo comparator | It is composed of the same excipients as RGN-259 but does not contain Tβ4. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Subjects Achieving Complete Healing at Day 29.
Percentage of subjects achieving complete healing of the persistent epithelial defect as determined by corneal fluorescein staining at day 29 after first dosing.
Measured: 29 days after first dosing
Secondary outcomes (5)
Percentage of Subjects Achieving Complete Healing at 8, 15, 22, 36, 43 Days
Percentage of subjects achieving complete healing of the Persistent Epithelial Defect(PED) determined by corneal fluorescein staining at 8, 15, 22, 36, 43 days after first dosing.
Measured: 8, 15, 22, 36, 43 days after first dosing
Epithelial Defect Measurement and Classification as Stage 1, 2 or 3 Using Mackie Classification.
Epithelial Defect Measurement and Classification as stage 1, 2 or 3 using Mackie Classification at 8, 15, 22, 29, 36, 43 days after first dosing
Measured: 8, 15, 22, 29, 36, 43 days after first dosing
Tear Film Break-up Time at 29, 36, 43 Days
Tear Film Break-up Time at 29, 36, 43 days after first dosing
Measured: 29, 36, 43 days after first dosing
Ocular Discomfort by Questionnaire at 8, 15, 22, 29, 36, 43 Days After First Dosing
Ocular Discomfort by Questionnaire at 8, 15, 22, 29, 36, 43 Days After First Dosing at Visits 2, 3, 4, 5, 6, and 7 (The scale used to determine the difference in Ocular Discomfort by questionnaire on each visit is from 0(None) to 5(Most). This outcome was calculated from two time points as the value at the later time point minus the value at the first dosing points and the lower value are considered to be a better outcome. and the all relevant time points used in the calculation in the Time Frame was 8, 15, 22, 29, 36, 43 days.)
Measured: 8, 15, 22, 29, 36, 43 days after first dosing
Visual Acuity(logMAR) at 8, 15, 22, 29, 36, 43 Days
Visual acuity(logMAR) at 8, 15, 22, 29, 36, 43 days
(The Visual acuity was assessed by LogMAR calculation method. In the case of the LogMAR method, Each letter has a score value of 0.02 log units. Since there are 5 letters per line, the total score for a line on the LogMAR chart represents a change of 0.1 log units. and The lower value are considered to be a better outcome.)
The formula used in calculating the score is:
LogMAR VA = 0.1 + LogMAR value of the best line read - 0.02 X (number of optotypes read)
used to determine the difference in Ocular Discomfort by questionnaire on each visit is the ORA scale: 0 None to 5: Most
And as this outcome was calculated from two time points as the value at the later time point minus the value at the first dosing points, The lower value are considered to be a better outcome. and the all relevant time points used in the calculation in the Time Frame was 8, 15, 22, 29, 36, 43 days.)
Measured: 8, 15, 22, 29, 36, 43 days after first dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2015 (estimated)
- Study start17 Sep 2015
- Primary completion20 Nov 2019
- Study completion9 Mar 2020
Why it stopped: Business decision
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 Neurotrophic KeratopathyStudies 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
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- ReGenTree, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02600429
- Sponsor's protocol ID
- RGN-NK-301
- Status last verified
- Jul 2023
- First submitted
- 26 Oct 2015
- Last updated
- 30 Aug 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3, Multi-Center, Randomized, Double Masked, Placebo Controlled Clinical Study to Assess the Safety and Efficacy of RGN-259 Ophthalmic Solution for the Treatment of Neurotrophic Keratopathy: SEER-1
- ClinicalTrials.gov ID
- NCT02600429
- Acronym / sponsor ID
- RGN-NK-301
- Conditions
- Neurotrophic Keratopathy
- Keywords
- Neurotrophic Keratopathy, NK
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Business decision
- Enrolment
- 18 participants (actual)
- Lead sponsor
- ReGenTree, LLCIndustry (pharma, biotech, devices)
- Study start
- 17 Sep 2015 (actual)
- Primary completion
- 20 Nov 2019 (actual)
- Study completion
- 9 Mar 2020 (actual)
- First posted
- 9 Nov 2015 (estimated)
- Results
- Posted 14 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to assess the safety and efficacy of RGN-259 Ophthalmic Solution compared to placebo for the treatment of NK.
Detailed description
Neurotrophic keratopathy (NK) is a degenerative corneal disease that occurs as a result of partial or total impairment of trigeminal innervation. The resulting loss of corneal sensitivity (anesthesia) leads to a reduction in lacrimation and a decline in status, metabolism, and mitosis of corneal epithelial cells. Previous studies (physician-sponsored studies) used to treat to nine patients with NK, six of whom had discrete geographic, non-healing lesions, and three of whom had punctate lesions and the study result reported.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RGN-259 | Experimental | Drug: RGN-259It is a preservative-free, sterile eye drop solution containing Tβ4 |
| Placebo | Placebo comparator | Drug: PlaceboIt is composed of the same excipients as RGN-259 but does not contain Tβ4. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Be male or female of any race, at least 18 years of age
- Have provided verbal and written informed consent.
- Be able and willing to follow instructions, including participation in all study assessments and visits;
- Have stage 2 or 3 neurotrophic keratopathy in at least one eye If a female of childbearing potential, have a negative urine pregnancy test at Visit 1 and agree to use an adequate method of birth control throughout the study period.
Key exclusion criteria (8 listed)
- Have any clinically significant slit lamp findings at Visit 1 that in the opinion of the investigator may interfere with the study parameters;
- Have significant blepharitis, meibomian gland dysfunction (MGD), lid margin inflammation or active ocular allergy that requires treatment
- Have a lid function abnormality (ex. Lagophthalmos) which, in the opinion of the investigator, is the primary cause of the persistent epithelial defect;
- Be diagnosed with ongoing ocular infection (bacterial, viral or fungal) or active inflammation (e.g. follicular conjunctivitis) not related to NK
- Anticipate the use of fluoroquinolone-containing antibiotic eye drops during 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 |
|---|---|---|
| RGN-259Also: Tβ4A preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into affected eye(s), five times a day for 4 weeks. | Drug | RGN-259 |
| PlaceboAlso: Vehicle ControlIt is composed of the same excipients as RGN-259 but does not contain Tβ4 | Drug | Placebo |
Primary outcome
Percentage of Subjects Achieving Complete Healing at Day 29.
Time frame: 29 days after first dosing
Percentage of subjects achieving complete healing of the persistent epithelial defect as determined by corneal fluorescein staining at day 29 after first dosing.
5 secondary outcomes
Percentage of Subjects Achieving Complete Healing at 8, 15, 22, 36, 43 Days
Time frame: 8, 15, 22, 36, 43 days after first dosing
Epithelial Defect Measurement and Classification as Stage 1, 2 or 3 Using Mackie Classification.
Time frame: 8, 15, 22, 29, 36, 43 days after first dosing
Tear Film Break-up Time at 29, 36, 43 Days
Time frame: 29, 36, 43 days after first dosing
Ocular Discomfort by Questionnaire at 8, 15, 22, 29, 36, 43 Days After First Dosing
Time frame: 8, 15, 22, 29, 36, 43 days after first dosing
Visual Acuity(logMAR) at 8, 15, 22, 29, 36, 43 Days
Time frame: 8, 15, 22, 29, 36, 43 days after first dosing
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Aug 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
11 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 RGN-259 in people with Neurotrophic Keratopathy, run by ReGenTree, LLC at 11 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 4 inclusion and 8 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 30 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 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 14 Aug 2023). 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?
Percentage of Subjects Achieving Complete Healing at Day 29. (time frame: 29 days after first dosing). 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 30 Aug 2023.