Diabetic Macular EdemaPhase 1/2NCT04565756

A Study to Evaluate the Safety and Tolerability of EXN407

A Phase 1/2 study testing EXN407 in people with Diabetic Macular Edema, run by Exonate Limited at 11 sites in Australia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Diabetic Macular Edema
What is being tested?: EXN407
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 48 participants
Where: 11 sitesAustralia
Study dates: 5 Nov 2020to 29 Nov 2022
Phase: Phase 1/2
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

Subject is at least 18 years of age inclusive, at the time of signing the informed consent.
BCVA better than or equal to 69 ETDRS score (approximate Snellen equivalent 20/40 or 6/12) in the study eye using the ETDRS visual acuity scale at Screening or BCVA less than 69 ETDRS score (approximate Snellen equivalent 20/50 or 6/15) but who, in the Investigator's opinion, is unsuitable for treatment with anti-VEGF by intravitreal injection or refuses it. Subjects should have no more than a 7-letter difference in BCVA at Screening and baseline visit.
Ocular media is consistent with SD-OCT imaging and cataracts are not expected in the subject for the duration of the study.
The subject has no other retinal disease.
Subject or the subject's partner successfully demonstrates their ability to self-administer/administer eye drops at Screening, with multiple attempts allowed at the discretion of the Investigator.

Who cannot join

Any other retinal disease in the study eye, other than centre involved DMO or diabetic retinopathy.
Poor vision (VA 6/60 or worse) in the contralateral eye.
Intraocular inflammation (including trace or greater) in the study eye. History of idiopathic or autoimmune uveitis in either eye.
Use of intravitreal anti-VEGF drugs including ranibizumab, bevacizumab, aflibercept in the study eye within 6 months of the Screening Visit, or in the fellow (non study) eye within 3 months of the Screening Visit. Use of topical corticosteroids or topical non-steroidal anti-inflammatory agents in the study eye within 28 days of the Screening Visit. Use of intravitreal corticosteroids in either eye or systemic steroids within 12 months of the Screening Visit. Prior use of Iluvien (without time limitation).
Within 180 days prior to the Screening visit, use of medications known to be toxic to the retina, lens or optic nerve (e.g. desferoximine, chloroquine/hydrochloroquine, chlorpromazine, phenothiazines, tamoxifen, and ethambutol).
History of (within 90 days of Screening date) cerebral vascular accident (stroke) or MI.

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Significant renal impairment including subjects on chronic renal dialysis and subjects with a history of nephrectomy or kidney transplant (regardless of renal function).
History of anaphylaxis, anaphylactoid (resembling anaphylaxis) reactions, or severe allergic responses.
Positive pregnancy test (all female subjects of childbearing potential must have a urine β-human chorionic gonadotropin [hCG] pregnancy test performed at Screening and within 7 days prior to randomisation) or is known to be pregnant or lactating.
Known to have, or history of a positive test result for, hepatitis B or C, HIV, syphilis, tuberculosis, or COVID-19.

Trial locations and contacts

11 sites in 1 country.

City, postcode or hospital

11 sites in 1 country

Australia11 sites

Adelaide Eye and Retina Centre
Adelaide, South Australia, 5000
Macquarie University
Macquarie, New South Wales, 2109
Centre for Eye Research Australia (CERA)
Melbourne, Victoria, 3002
Retinology Institute
Melbourne, Victoria, 3146
Lions Eye Institute
Nedlands, Western Australia, 6009
Marsden Eye Specialists
Parramatta, New South Wales, 2150
Sydney Eye Hospital/Save Sight Institution
Sydney, New South Wales, 2000
Strathfield Retina Clinic
Sydney, New South Wales, 2135
Sydney Retina Clinic & Day Surgery
Sydney
Newcastle Eye Hospital Foundation
Waratah, New South Wales, 2298
Princess Alexandra Hospital
Woolloongabba, Queensland, 4102

Other trials in Australia

About this study

Official title: A Randomised, Double-Masked Vehicle-Controlled, Multiple Dose, Dose Escalation Study To Evaluate The Safety and Tolerability of EXN407 in Subjects With Centre Involved Diabetic Macular Oedema Secondary to Diabetes Mellitus

This first in human (FIH), Phase Ib/II study of EXN407 is a randomised, double-masked, vehicle-controlled, multiple dose, dose-escalating study to evaluate the safety and tolerability of EXN407 in subjects with centre involved Diabetic Macular Oedema (DMO), with Centre-subfield macular thickness (CMT) between 280-420 µm and Best corrected visual acuity (BCVA) better than or equal to 69 ETDRS score (approximate Snellen equivalent 20/40 (6/12 letters) in the study eye, which is considered secondary to diabetes mellitus.

This study will provide a basis for further clinical development of EXN407 ophthalmic solution.

Read the detailed description

EXN407 or vehicle-control solution administered unilaterally to the study eye only. To select the study eye the Investigator examines the subjects and identifies which eye exhibits centre involved DMO with a CMT between 280-420 μm (as determined by SD-OCT). Further, all other inclusion/exclusion criteria required to be met.

Dose Escalation Cohorts The study assesses the safety and tolerability of EXN407 in eligible subjects with center involved Diabetic Macular Oedema (DMO) at up to 3 escalating dose (concentration) levels and placebo (vehicle) consisting of an excipient formulation adjusted for osmolality. EXN407 or vehicle-control administered as a single 30 μL drop by unilateral eye drop administration to the study eye only, and the drops dosed BID for 7 days. Subjects assessed throughout the treatment period for safety, tolerability, and efficacy at Follow-up visits. Each of the ascending dose subject cohorts consists of 4 subjects (3 subjects randomised to receive EXN407 and 1 subject randomised to receive placebo).

Dose Expansion Cohort The highest well tolerated dose of EXN407 (as recommended by the DEC) is evaluated in a subject expansion cohort in eligible subjects with center involved Diabetic Macular Oedema (DMO) which consists of up to a maximum of 40 subjects (randomised to receive EXN407 at the selected dose or vehicle at a 2:1 drug: placebo ratio). Each eligible subject in the expansion cohort to receive study drug for up to 84 days, resulting in a total of 168 doses (168 single drops of 30 μL volume) of EXN407 or vehicle.

Conditions: Diabetic Macular Edema
Being tested: EXN407

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

EXN407Drug
EXN407 or placebo will be administered as a single 30 microliters drop twice a day, by unilateral eye drop administration to the study eye only.

Study groups in NCT04565756
Study group	Role	What they receive
Dose Escalation Cohort 1	Experimental	Each subject will receive a low-dose 0.5 mg/mL (0.05%) of EXN407 or placebo twice a day in 14 doses over a 7 day period. Receives: Drug: EXN407
Dose Escalation Cohort 2	Experimental	Each subject will receive a mid-dose 1 mg/mL (0.1%) of EXN407 or placebo twice a day in 14 doses over a 7 day period. Receives: Drug: EXN407
Dose Escalation Cohort 3	Experimental	Each subject will receive a high-dose 1.5 mg/mL (0.15%) of EXN407 or placebo twice a day in 14 doses over a 7 day period. Receives: Drug: EXN407
Dose Expansion Cohort	Experimental	The highest well-tolerated dose of EXN407 will be evaluated where subjects will receive EXN407 at the selected dose or placebo twice a day for up to 84 days resulting in a total of 168 doses Receives: Drug: EXN407

What is being measured

The primary outcome is the main question the study is designed to answer.

The Primary Objective of the Study is to Evaluate the Ocular Safety and Tolerability (by Incidence of Ocular Adverse Events) of EXN407 Ophthalmic Solution in Dose Escalation Phase.
Number of Participants with Ocular Treatment Emergent Adverse Events (TEAEs)
Measured: Assessed starting from Day 1 of treatment to Day 36 in Dose Escalation.
The Primary Objective of the Study is to Evaluate the Ocular Safety and Tolerability (by Incidence of Ocular Adverse Events) of EXN407 Ophthalmic Solution in Dose Expansion Phase.
Number of Participants with Ocular Treatment Emergent Adverse Events (TEAEs)
Measured: Assessed starting from Day 1 of treatment to Day 113 in Dose Expansion phase.

Secondary outcomes (6)

To Evaluate the Systemic Pharmacokinetics of EXN407 Ophthalmic Solution in Subjects With DMO Secondary to Diabetes Mellitus by Tmax.
Measured by characterizing the PK profile by estimating the time of the maximum plasma drug concentration (Tmax) of EXN407 in plasma.
Measured: Blood draws for PK will be collected only on Day 3(Dose Escalation) and Day 8(Dose Expansion) at the following timepoints: pre-dose and 15 mins, 30 mins, 1, 2, 3,and 4 hours post-dose.
To Evaluate the Systemic Pharmacokinetics of EXN407 Ophthalmic Solution in Subjects With DMO Secondary to Diabetes Mellitus by Cmax
Measured by characterizing the PK profile by estimating the maximum observed drug plasma concentration (Cmax) of EXN407 in plasma.
Measured: Blood draws for PK will be collected only on Day 3(Dose Escalation) and Day 8(Dose Expansion) at the following timepoints: pre-dose and 15 mins, 30 mins, 1, 2, 3,and 4 hours post-dose.
To Evaluate the Systemic Pharmacokinetics of EXN407 Ophthalmic Solution in Subjects With DMO Secondary to Diabetes Mellitus by AUC.
Measured by characterizing the PK profile by estimating the area under the curve (AUC) of EXN407 in plasma.
Measured: Blood draws for PK will be collected only on Day 3(Dose Escalation) and Day 8(Dose Expansion) at the following timepoints: pre-dose and 15 mins, 30 mins, 1, 2, 3,and 4 hours post-dose.
To Evaluate the Systemic Pharmacokinetics of EXN407 Ophthalmic Solution in Subjects With DMO Secondary to Diabetes Mellitus by t½.
Measured by characterizing the PK profile by estimating the apparent elimination half-life (t½) of EXN407 in plasma.
Measured: Blood draws for PK will be collected only on Day 3(Dose Escalation) and Day 8(Dose Expansion) at the following timepoints: pre-dose and 15 mins, 30 mins, 1, 2, 3,and 4 hours post-dose.
To Evaluate Changes in Ocular Functional Measures as Assessed Using Ophthalmoscopy.
Measured by the changes from baseline in ophthalmic examination finding through ophthalmoscopy (BCVA (Letters))
Measured: From Day 1 of Treatment to End of Treatment i.e. assessed upto 36 Days/EOS in Dose Escalation and upto 4 months(113 days/EOS) in Dose Expansion.
To Evaluate Changes in Ocular Structural Measures as Assessed Using Ophthalmoscopy.
Measured by the changes from baseline in ophthalmic examination finding through ophthalmoscopy (corneal thickness).
Measured: From Day 1 of Treatment to End of Treatment i.e. assessed upto 36 Days/EOS in Dose Escalation and up to 4 months(113 days) in Dose Expansion.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

5 Nov 202029 Nov 2022

First posted25 Sep 2020
Study start5 Nov 2020
Primary completion25 Oct 2022
Study completion29 Nov 2022

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 Diabetic Macular EdemaStudies 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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 48 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04565756
Sponsor's protocol ID: PQ-110-001
Status last verified: Apr 2025
First submitted: 26 Aug 2020
Last updated: 9 May 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
Real World Evidence in China: Faricimab Use in Diabetic Macular Edema, Retinal Vein Occlusion, and Neovascular Age-Related Macular Degeneration (The Farseeing Study)
Diabetic Macular Edema, Neovascular Age-related Macular Degeneration
Active, not recruitingPhase 2
Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With and Without Diabetic Retinopathy
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RecruitingPhase 3
A Phase 3 Trial for Intravitreal Injection of 4D-150 in Adults With Diabetic Macular Edema (DME).
Diabetic Macular Edema (DME)
RecruitingPhase 2
Oral N-Acetylcysteine on Diabetic Macular Edema
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All Diabetic Macular Edema trials

Study at a glance

From the registry record, last updated by the sponsor on 9 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomised, Double-Masked Vehicle-Controlled, Multiple Dose, Dose Escalation Study To Evaluate The Safety and Tolerability of EXN407 in Subjects With Centre Involved Diabetic Macular Oedema Secondary to Diabetes Mellitus
ClinicalTrials.gov ID: NCT04565756
Acronym / sponsor ID: PQ-110-001
Conditions: Diabetic Macular Edema
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 48 participants (actual)
Lead sponsor: Exonate LimitedIndustry (pharma, biotech, devices)
Collaborators: Novotech (Australia) Pty Limited
Study start: 5 Nov 2020 (actual)
Primary completion: 25 Oct 2022 (actual)
Study completion: 29 Nov 2022 (actual)
First posted: 25 Sep 2020 (actual)
Results: Posted 9 May 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This study will provide a basis for further clinical development of EXN407 ophthalmic solution.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: Double blind (participant, investigator)
Number of arms: 4

Arm	Type	Receives
Dose Escalation Cohort 1	Experimental	Drug: EXN407Each subject will receive a low-dose 0.5 mg/mL (0.05%) of EXN407 or placebo twice a day in 14 doses over a 7 day period.
Dose Escalation Cohort 2	Experimental	Drug: EXN407Each subject will receive a mid-dose 1 mg/mL (0.1%) of EXN407 or placebo twice a day in 14 doses over a 7 day period.
Dose Escalation Cohort 3	Experimental	Drug: EXN407Each subject will receive a high-dose 1.5 mg/mL (0.15%) of EXN407 or placebo twice a day in 14 doses over a 7 day period.
Dose Expansion Cohort	Experimental	Drug: EXN407The highest well-tolerated dose of EXN407 will be evaluated where subjects will receive EXN407 at the selected dose or placebo twice a day for up to 84 days resulting in a total of 168 doses

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

Subject is at least 18 years of age inclusive, at the time of signing the informed consent.
BCVA better than or equal to 69 ETDRS score (approximate Snellen equivalent 20/40 or 6/12) in the study eye using the ETDRS visual acuity scale at Screening or BCVA less than 69 ETDRS score (approximate Snellen equivalent 20/50 or 6/15) but who, in the Investigator's opinion, is unsuitable for treatment with anti-VEGF by intravitreal injection or refuses it. Subjects should have no more than a 7-letter difference in BCVA at Screening and baseline visit.
Ocular media is consistent with SD-OCT imaging and cataracts are not expected in the subject for the duration of the study.
The subject has no other retinal disease.
Subject or the subject's partner successfully demonstrates their ability to self-administer/administer eye drops at Screening, with multiple attempts allowed at the discretion of the Investigator.

Key exclusion criteria (10 listed)

Any other retinal disease in the study eye, other than centre involved DMO or diabetic retinopathy.
Poor vision (VA 6/60 or worse) in the contralateral eye.
Intraocular inflammation (including trace or greater) in the study eye. History of idiopathic or autoimmune uveitis in either eye.
Use of intravitreal anti-VEGF drugs including ranibizumab, bevacizumab, aflibercept in the study eye within 6 months of the Screening Visit, or in the fellow (non study) eye within 3 months of the Screening Visit. Use of topical corticosteroids or topical non-steroidal anti-inflammatory agents in the study eye within 28 days of the Screening Visit. Use of intravitreal corticosteroids in either eye or systemic steroids within 12 months of the Screening Visit. Prior use of Iluvien (without time limitation).
Within 180 days prior to the Screening visit, use of medications known to be toxic to the retina, lens or optic nerve (e.g. desferoximine, chloroquine/hydrochloroquine, chlorpromazine, phenothiazines, tamoxifen, and ethambutol).

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
EXN407EXN407 or placebo will be administered as a single 30 microliters drop twice a day, by unilateral eye drop administration to the study eye only.	Drug	Dose Escalation Cohort 1, Dose Escalation Cohort 2, Dose Escalation Cohort 3, Dose Expansion Cohort

Primary outcome

The Primary Objective of the Study is to Evaluate the Ocular Safety and Tolerability (by Incidence of Ocular Adverse Events) of EXN407 Ophthalmic Solution in Dose Escalation Phase.
Time frame: Assessed starting from Day 1 of treatment to Day 36 in Dose Escalation.
Number of Participants with Ocular Treatment Emergent Adverse Events (TEAEs)
The Primary Objective of the Study is to Evaluate the Ocular Safety and Tolerability (by Incidence of Ocular Adverse Events) of EXN407 Ophthalmic Solution in Dose Expansion Phase.
Time frame: Assessed starting from Day 1 of treatment to Day 113 in Dose Expansion phase.
Number of Participants with Ocular Treatment Emergent Adverse Events (TEAEs)

6 secondary outcomes

To Evaluate the Systemic Pharmacokinetics of EXN407 Ophthalmic Solution in Subjects With DMO Secondary to Diabetes Mellitus by Tmax.
Time frame: Blood draws for PK will be collected only on Day 3(Dose Escalation) and Day 8(Dose Expansion) at the following timepoints: pre-dose and 15 mins, 30 mins, 1, 2, 3,and 4 hours post-dose.
To Evaluate the Systemic Pharmacokinetics of EXN407 Ophthalmic Solution in Subjects With DMO Secondary to Diabetes Mellitus by Cmax
Time frame: Blood draws for PK will be collected only on Day 3(Dose Escalation) and Day 8(Dose Expansion) at the following timepoints: pre-dose and 15 mins, 30 mins, 1, 2, 3,and 4 hours post-dose.
To Evaluate the Systemic Pharmacokinetics of EXN407 Ophthalmic Solution in Subjects With DMO Secondary to Diabetes Mellitus by AUC.
Time frame: Blood draws for PK will be collected only on Day 3(Dose Escalation) and Day 8(Dose Expansion) at the following timepoints: pre-dose and 15 mins, 30 mins, 1, 2, 3,and 4 hours post-dose.
To Evaluate the Systemic Pharmacokinetics of EXN407 Ophthalmic Solution in Subjects With DMO Secondary to Diabetes Mellitus by t½.
Time frame: Blood draws for PK will be collected only on Day 3(Dose Escalation) and Day 8(Dose Expansion) at the following timepoints: pre-dose and 15 mins, 30 mins, 1, 2, 3,and 4 hours post-dose.
To Evaluate Changes in Ocular Functional Measures as Assessed Using Ophthalmoscopy.
Time frame: From Day 1 of Treatment to End of Treatment i.e. assessed upto 36 Days/EOS in Dose Escalation and upto 4 months(113 days/EOS) in Dose Expansion.
To Evaluate Changes in Ocular Structural Measures as Assessed Using Ophthalmoscopy.
Time frame: From Day 1 of Treatment to End of Treatment i.e. assessed upto 36 Days/EOS in Dose Escalation and up to 4 months(113 days) in Dose Expansion.

Results and safety

Results posted to the registry on 9 May 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Dose Escalation EXN407 Cohort 1	3	3
Dose Escalation EXN407 Cohort 2	3	3
Dose Escalation EXN407 Cohort 3	3	3
Dose Escalation Pooled Placebo Cohort	4	4
Dose Expansion EXN407 Cohort	23	22
Dose Expansion Placebo Cohort	12	11

Primary outcome: The Primary Objective of the Study is to Evaluate the Ocular Safety and Tolerability (by Incidence of Ocular Adverse Events) of EXN407 Ophthalmic Solution in Dose Escalation Phase.

Time frame: Assessed starting from Day 1 of treatment to Day 36 in Dose Escalation.. Count of participants. Unit: Participants

Measure	Dose Escalation EXN407 Cohort 1 Study Eye n = 3	Dose Escalation EXN407 Cohort 1 Contralateral Eye n = 3	Dose Escalation EXN407 Cohort 1, Bilateral Eye n = 3	Dose Escalation EXN407 Cohort 2 Study Eye n = 3	Dose Escalation EXN407 Cohort 2 Contralateral Eye n = 3	Dose Escalation EXN407 Cohort 2 Bilateral Eye n = 3	Dose Escalation EXN407 Cohort 3 Study Eye n = 3	Dose Escalation EXN407 Cohort 3 Contralateral Eye n = 3	Dose Escalation EXN407 Cohort 3 Bilateral Eye n = 3	Pooled Placebo (Cohort 1, Cohort 2, Cohort 3) Study Eye n = 4	Pooled Placebo (Cohort 1, Cohort 2, Cohort 3) Contralateral Eye n = 4	Pooled Placebo (Cohort 1, Cohort 2, Cohort) Bilateral Eye n = 4
Value	2	2	2	0	1	0	0	0	0	0	0	0

Population analysed: The Safety population comprised all randomised subjects who received any amount of study drug. Summaries, listings, and analyses were based on the treatment actually received. Screen failures and randomised subjects who did not receive any medication were excluded from the safety analysis set. The Safety population was used for the summaries of all safety assessments.

Primary outcome: The Primary Objective of the Study is to Evaluate the Ocular Safety and Tolerability (by Incidence of Ocular Adverse Events) of EXN407 Ophthalmic Solution in Dose Expansion Phase.

Time frame: Assessed starting from Day 1 of treatment to Day 113 in Dose Expansion phase.. Count of participants. Unit: Participants

Measure	Dose Expansion EXN407 Cohort Study Eye n = 23	Dose Expansion EXN407 Cohort Contralateral Eye n = 23	Dose Expansion EXN407 Cohort Bilateral Eye n = 23	Dose Expansion Placebo Cohort Study Eye n = 12	Dose Expansion Placebo Cohort Contralateral Eye n = 12	Dose Expansion Placebo Cohort Bilateral Eye n = 12
Value	5	3	2	3	1	1

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: Each subject in the Dose Escalation cohorts (numbered Cohorts 1, 2, 3, etc.) received EXN407 or vehicle BID over a 7-day period Each subject in the Dose Expansion received EXN407 or vehicle BID over an 84-day period. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Dose Escalation EXN407 Cohort 1	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
Dose Escalation EXN407 Cohort 2	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
Dose Escalation EXN407 Cohort 3	0 of 3 (0%)	1 of 3 (33.3%)	0 of 3
Dose Escalation Pooled Placebo Cohort	0 of 4 (0%)	1 of 4 (25%)	0 of 4
Dose Expansion EXN407 Cohort	2 of 23 (8.7%)	11 of 23 (47.8%)	0 of 23
Dose Expansion Placebo Cohort	1 of 12 (8.3%)	9 of 12 (75%)	0 of 12

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

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Active, not recruitingObservational study
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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.

Mark Gillies, Prof principal investigator

Sydney Eye Hospital/Save Sight Institution

Andrew Chang, A/Prof principal investigator

Sydney Retina Clinic & Day Surgery

Sanjeewa Wickremasinghe,, Prof principal investigator

Centre for Eye Research Australia (CERA)

11 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing EXN407 in people with Diabetic Macular Edema, run by Exonate Limited at 11 sites in Australia.

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 5 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: finished, as of the sponsor's last update on 9 May 2025. 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 Australia. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 9 May 2025). 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?

The Primary Objective of the Study is to Evaluate the Ocular Safety and Tolerability (by Incidence of Ocular Adverse Events) of EXN407 Ophthalmic Solution in Dose Escalation Phase. (time frame: Assessed starting from Day 1 of treatment to Day 36 in Dose Escalation.). 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?

On the ClinicalTrials.gov record for NCT04565756.

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 9 May 2025.