Diabetic Macular EdemaPhase 1NCT02281292
A Study of the Safety and Tolerability of Intravitreal LKA651 in Patients With Diabetic Macular Edema
A Phase 1 study testing LKA651 ophthalmic solution and Ranibizumab ophthalmic solution in people with Diabetic Macular Edema, run by Alcon Research.
Trial summary
The purpose of this study is to evaluate the initial safety of intravitreal (IVT) LKA651 and potential for use alone or in combination with Ranibizumab ophthalmic solution (Lucentis®) for the treatment of Diabetic Macular Edema (DME) in patients with symptomatic disease. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Diabetic Macular Edema
- What is being tested?
- LKA651 ophthalmic solution and Ranibizumab ophthalmic solution
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Dec 2015to Jun 2017
- 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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent must be obtained.
- Type 1 or type 2 diabetes that is actively managed by a physician and hemoglobin A1C ≤ 12% at screening/eligibility.
- Negative pregnancy test results at screening/eligibility and pre-injection on treatment day.
- Diabetic macular edema (DME) with center involvement in at least one eye, including those with focal or diffuse DME.
- ETDRS letter score in the study eye of 55 letters or worse (approximate Snellen equivalent of 20/80). The non-study eye (fellow eye) should be ≥ 60 letters or better (approximate Snellen equivalent of 20/63) at Day 1 pre-dose.
- Safe to withhold treatment of the study eye with laser photocoagulation, intravitreal steroid injection, or intravitreal vascular endothelial growth factor (VEGF) inhibitor (Part 1) for the duration of the study.
- Other protocol-defined inclusion criteria may apply.
Who cannot join
- Proliferative diabetic retinopathy in the study eye, with the exception of tufts of neovascularization less than 1 disc area with no vitreous hemorrhage.
- Any progressive disease of the retina (e.g. uveitis, rod-cone dystrophy) or optic nerve (e.g. glaucoma) other than diabetic retinopathy.
- Any active infection involving ocular adnexa including infectious conjunctivitis, keratitis, scleritis, endophthalmitis, as well as idiopathic or autoimmune-associated uveitis in either eye.
- Ocular disorders in the study eye that may confound interpretation of study results, including retinal vascular occlusion, retinal detachment, macular hole, or choroidal neovascularization of any cause (e.g., AMD, ocular histoplasmosis, or pathologic myopia).
- Cataract surgery in the study eye, Yttrium-Aluminum-Garnet (YAG) laser capsulotomy or any intraocular surgery within the past 6 months preceding Day 1.
- Use of systemic anticoagulant therapy during the study, e.g., warfarin, heparin, etc. The use of aspirin is not an exclusion.
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- Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, whichever is longer; or longer if required by local regulations.
- Pregnant or nursing (lactating) women.
- Women of child-bearing potential, unless using highly effective methods of contraception during dosing of study treatment.
- Other protocol-defined exclusion criteria may apply.
About this study
Official title: A Randomized, Sham Controlled, Single Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Activity of Intravitreal LKA651 in Patients With Diabetic Macular Edema
The purpose of this study is to evaluate the initial safety of intravitreal (IVT) LKA651 and potential for use alone or in combination with Ranibizumab ophthalmic solution (Lucentis®) for the treatment of Diabetic Macular Edema (DME) in patients with symptomatic disease.
Read the detailed description
This first-in-human study will be conducted in 2 parts. Part 1 is an ascending dose design to assess the safety, tolerability, pharmacodynamics and pharmacokinetics of various single IVT doses of LKA651 in up to 48 subjects with diabetic macular edema. Subjects will be randomized to receive active or sham injections in a 3:1 ratio. A total of 6 cohorts (8 subjects per cohort) may be enrolled in Part 1. Each subject will participate in a screening/eligibility period (up to 60 days), a treatment period (single day), and an 84 day follow up period. A total of up to 11 visits will take place, all on an out-patient basis. An independent data monitoring committee (DMC) will be chartered to review cumulative safety data and approve each dose escalation and cohort progression in this first-in-human trial.
Part 2 is a double-masked design to assess the safety/tolerability, pharmacokinetics and pharmacodynamics of a single IVT dose of LKA651 when co-administered with Lucentis®. A total of up to 3 cohorts (8 subjects per cohort) may be enrolled in Part 2. Each subject will participate in a screening/eligibility period (up to 60 days), a treatment period (combination therapy, single day), and an 84 day follow up period. A total of up to 11 visits will take place, all on an out-patient basis. For the LKA651 vs sham injections, the unmasked ophthalmologist is not permitted to do any of the assessments except for the injection (and an inspection of the injection site immediately following). All other ocular assessments after randomization will be conducted by a second ophthalmologist masked to the type of injection (active or sham). The Lucentis injection (Part 2) is given open label to all patients following either the LKA651 or sham injection.
- Conditions
- Diabetic Macular Edema
- Being tested
- LKA651 ophthalmic solution, Ranibizumab ophthalmic solution, Sham injection
- Keywords
- Diabetic Macular Edema, Intravitreal, Retina, Ophthalmology, Lucentis, OCT
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LKA651 ophthalmic solutionBiological
Ranibizumab ophthalmic solutionBiological
Also called Lucentis®
Sham injectionBiological
Mock injection administered as an empty hub without needle
| Study group | Role | What they receive |
|---|---|---|
| LKA651 (Part 1) | Experimental | LKA651 ophthalmic solution in 1 of 5 concentrations, administered as a single IVT injection in the study eye Receives: Biological: LKA651 ophthalmic solution |
| Sham injection (Part 1) | Placebo comparator | Sham injection in the study eye Receives: Biological: Sham injection |
| LKA651 and Lucentis (Part 2) | Experimental | LKA651 ophthalmic solution in 1 of 3 concentrations, administered as a single IVT injection in the study eye, followed by ranibizumab ophthalmic solution, 0.5 mg injection in the same eye 30 minutes later Receives: Biological: LKA651 ophthalmic solution; Biological: Ranibizumab ophthalmic solution |
| Sham injection and Lucentis (Part 2) | Placebo comparator | Sham injection in the study eye, followed by ranibizumab ophthalmic solution, 0.5 mg injection in the same eye 30 minutes later Receives: Biological: Ranibizumab ophthalmic solution; Biological: Sham injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with a serious adverse event (SAE) that, in the opinion of the investigator, is related to the study drug
Measured: Up to Day 85
Number of subjects experiencing a non-serious adverse event
Measured: Up to Day 85
Secondary outcomes (7)
The area under the serum concentration-time curve from time zero to the time of the last quantifiable concentration [mass x time / volume] (AUC(0-tlast))
Contingent upon observed serum concentration levels
Measured: Up to Day 85
The area under the serum concentration-time curve from time zero to time 't' where t is a defined time point after administration [mass x time / volume] (AUC (0-t))
Contingent upon observed serum concentration levels
Measured: Up to Day 85
The observed maximum serum concentration following drug administration [mass / volume] (Cmax)
Contingent upon observed serum concentration levels
Measured: Up to Day 85
The time to reach the maximum serum concentration after drug administration [time] (Tmax)
Contingent upon observed serum concentration levels
Measured: Up to Day 85
The dose normalized observed maximum serum concentration following drug administration [mass*dose / volume] (Cmax/D)
Contingent upon observed serum concentration levels
Measured: Up to Day 85
The dose-normalized area under the serum concentration-time curve from time zero to the time of the last quantifiable concentration [mass*dose x time / volume] (AUC/D)
Measured: Up to Day 85
Central subfield thickness
Contingent upon observed serum concentration levels
Measured: Up to Day 85
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2014 (estimated)
- Study startDec 2015
- Primary completionJun 2017 (estimated)
- Study completionJun 2017 (estimated)
Why it stopped: Management Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetic Macular EdemaStudies of the same condition, open and under way.
- More trials run by Alcon ResearchThe 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alcon Research
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Novartis Institutes for BioMedical Research
- Responsible party
- Sponsor
- Investigators
- Sr. Clinical Scientist, CSI, ID/Ophtha, Study director, Alcon Research
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02281292
- Sponsor's protocol ID
- LKA651X-2101
- Status last verified
- Dec 2015
- First submitted
- 30 Oct 2014
- Last updated
- 11 Dec 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Sham Controlled, Single Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Activity of Intravitreal LKA651 in Patients With Diabetic Macular Edema
- ClinicalTrials.gov ID
- NCT02281292
- Acronym / sponsor ID
- LKA651X-2101
- Conditions
- Diabetic Macular Edema
- Keywords
- Diabetic Macular Edema, Intravitreal, Retina, Ophthalmology, Lucentis, OCT
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Management Decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Alcon ResearchIndustry (pharma, biotech, devices)
- Collaborators
- Novartis Institutes for BioMedical Research
- Study start
- Dec 2015
- Primary completion
- Jun 2017 (estimated)
- Study completion
- Jun 2017 (estimated)
- First posted
- 3 Nov 2014 (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 initial safety of intravitreal (IVT) LKA651 and potential for use alone or in combination with Ranibizumab ophthalmic solution (Lucentis®) for the treatment of Diabetic Macular Edema (DME) in patients with symptomatic disease.
Detailed description
This first-in-human study will be conducted in 2 parts. Part 1 is an ascending dose design to assess the safety, tolerability, pharmacodynamics and pharmacokinetics of various single IVT doses of LKA651 in up to 48 subjects with diabetic macular edema. Subjects will be randomized to receive active or sham injections in a 3:1 ratio. A total of 6 cohorts (8 subjects per cohort) may be enrolled in Part 1. Each subject will participate in a screening/eligibility period (up to 60 days), a treatment period (single day), and an 84 day follow up period. A total of up to 11 visits will take place, all on an out-patient basis. An independent data monitoring committee (DMC) will be chartered to review cumulative safety data and approve each dose escalation and cohort progression in this first-in-human trial.
Part 2 is a double-masked design to assess the safety/tolerability, pharmacokinetics and pharmacodynamics of a single IVT dose of LKA651 when co-administered with Lucentis®. A total of up to 3 cohorts (8 subjects per cohort) may be enrolled in Part 2. Each subject will participate in a screening/eligibility period (up to 60 days), a treatment period (combination therapy, single day), and an 84 day follow up period. A total of up to 11 visits will take place, all on an out-patient basis. For the LKA651 vs sham injections, the unmasked ophthalmologist is not permitted to do any of the assessments except for the injection (and an inspection of the injection site immediately following). All other ocular assessments after randomization will be conducted by a second ophthalmologist masked to the type of injection (active or sham). The Lucentis injection (Part 2) is given open label to all patients following either the LKA651 or sham injection.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| LKA651 (Part 1) | Experimental | Biological: LKA651 ophthalmic solutionLKA651 ophthalmic solution in 1 of 5 concentrations, administered as a single IVT injection in the study eye |
| Sham injection (Part 1) | Placebo comparator | Biological: Sham injectionSham injection in the study eye |
| LKA651 and Lucentis (Part 2) | Experimental | Biological: LKA651 ophthalmic solution, Biological: Ranibizumab ophthalmic solutionLKA651 ophthalmic solution in 1 of 3 concentrations, administered as a single IVT injection in the study eye, followed by ranibizumab ophthalmic solution, 0.5 mg injection in the same eye 30 minutes later |
| Sham injection and Lucentis (Part 2) | Placebo comparator | Biological: Ranibizumab ophthalmic solution, Biological: Sham injectionSham injection in the study eye, followed by ranibizumab ophthalmic solution, 0.5 mg injection in the same eye 30 minutes later |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Written informed consent must be obtained.
- Type 1 or type 2 diabetes that is actively managed by a physician and hemoglobin A1C ≤ 12% at screening/eligibility.
- Negative pregnancy test results at screening/eligibility and pre-injection on treatment day.
- Diabetic macular edema (DME) with center involvement in at least one eye, including those with focal or diffuse DME.
- ETDRS letter score in the study eye of 55 letters or worse (approximate Snellen equivalent of 20/80). The non-study eye (fellow eye) should be ≥ 60 letters or better (approximate Snellen equivalent of 20/63) at Day 1 pre-dose.
Key exclusion criteria (10 listed)
- Proliferative diabetic retinopathy in the study eye, with the exception of tufts of neovascularization less than 1 disc area with no vitreous hemorrhage.
- Any progressive disease of the retina (e.g. uveitis, rod-cone dystrophy) or optic nerve (e.g. glaucoma) other than diabetic retinopathy.
- Any active infection involving ocular adnexa including infectious conjunctivitis, keratitis, scleritis, endophthalmitis, as well as idiopathic or autoimmune-associated uveitis in either eye.
- Ocular disorders in the study eye that may confound interpretation of study results, including retinal vascular occlusion, retinal detachment, macular hole, or choroidal neovascularization of any cause (e.g., AMD, ocular histoplasmosis, or pathologic myopia).
- Cataract surgery in the study eye, Yttrium-Aluminum-Garnet (YAG) laser capsulotomy or any intraocular surgery within the past 6 months preceding Day 1.
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 |
|---|---|---|
| LKA651 ophthalmic solution | Biological | LKA651 (Part 1), LKA651 and Lucentis (Part 2) |
| Ranibizumab ophthalmic solutionAlso: Lucentis® | Biological | LKA651 and Lucentis (Part 2), Sham injection and Lucentis (Part 2) |
| Sham injectionMock injection administered as an empty hub without needle | Biological | Sham injection (Part 1), Sham injection and Lucentis (Part 2) |
Primary outcome
Number of subjects with a serious adverse event (SAE) that, in the opinion of the investigator, is related to the study drug
Time frame: Up to Day 85
Number of subjects experiencing a non-serious adverse event
Time frame: Up to Day 85
7 secondary outcomes
The area under the serum concentration-time curve from time zero to the time of the last quantifiable concentration [mass x time / volume] (AUC(0-tlast))
Time frame: Up to Day 85
The area under the serum concentration-time curve from time zero to time 't' where t is a defined time point after administration [mass x time / volume] (AUC (0-t))
Time frame: Up to Day 85
The observed maximum serum concentration following drug administration [mass / volume] (Cmax)
Time frame: Up to Day 85
The time to reach the maximum serum concentration after drug administration [time] (Tmax)
Time frame: Up to Day 85
The dose normalized observed maximum serum concentration following drug administration [mass*dose / volume] (Cmax/D)
Time frame: Up to Day 85
The dose-normalized area under the serum concentration-time curve from time zero to the time of the last quantifiable concentration [mass*dose x time / volume] (AUC/D)
Time frame: Up to Day 85
Central subfield thickness
Time frame: Up to Day 85
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 11 Dec 2015.
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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.
Sr. Clinical Scientist, CSI, ID/Ophtha study director
Alcon Research
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing LKA651 ophthalmic solution and Ranibizumab ophthalmic solution in people with Diabetic Macular Edema, run by Alcon Research.
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 10 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 11 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.
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 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 subjects with a serious adverse event (SAE) that, in the opinion of the investigator, is related to the study drug (time frame: Up to Day 85). 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 11 Dec 2015.