The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03630315




Registration number
NCT03630315
Ethics application status
Date submitted
1/06/2018
Date registered
14/08/2018
Date last updated
15/08/2019

Titles & IDs
Public title
CLN-0046: Treatment of AMD Subjects With OTX-TKI
Scientific title
A Phase 1 Open-Label, Dose Escalation Study of OTX-TKI for Intravitreal Use in Subjects With Neovascular Age-Related Macular Degeneration (AMD)
Secondary ID [1] 0 0
CLN-0046
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neovascular Age-related Macular Degeneration 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - OTX-TKI

Experimental: Dose 1 (Low Dose) - Dose 1 will be a low dose of OTX-TKI.

Experimental: Cohort Expansion - This cohort will expand to include more subjects.

Experimental: Dose 2 (High Dose) - Dose 2 will be a higher dose of OTX-TKI.


Treatment: Drugs: OTX-TKI
OTX-TKI is a dried polyethylene glycol-based hydrogel fiber containing dispersed microcrystals of a small molecule tyrosine kinase inhibitor (TKI).

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment emergent adverse events for each subject - All adverse events from screening through end of study will be captured
Timepoint [1] 0 0
9 months
Secondary outcome [1] 0 0
Determine the Maximum Tolerated Dose of the OTX-TKI injection - A Central Reading Center will evaluate multiple imaging modalities to confirm evidence of biological activity for subjects that have been treated with the OTX-TKI injection in 3 cohorts. If all subjects tolerate the lower dosage in cohort 1, additional subjects will be treated at a higher dosage in cohort 2. All data will be evaluated by the DSMC who, in concert with the Medical Monitor, will determine the Maximally Tolerated Dose and treat an additional cohort of subjects in cohort 3 using the MTD OTX-TKI Injection.
Timepoint [1] 0 0
9 months

Eligibility
Key inclusion criteria
- Are at least 50 years of age

- Are eligible for standard therapy

- Have active primary CNVM secondary to AMD, either newly diagnosed or previously
treated with documented response to anti-VEGF therapy in the study eye [primary
subfoveal CNV secondary to AMD including juxtafoveal lesions that affect the fovea]
documented by FA and SD-OCT

- Are female who is postmenopausal for at least 12 months prior to screening or
surgically sterile; or male or female of childbearing potential willing to use two
forms of adequate contraception

- Are able and willing to comply with all study requirements and visits
Minimum age
50 Years
Maximum age
100 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Have previous laser photocoagulation to the center of the fovea in the study eye

- Have participated in any study involving an investigational drug either in the U.S. or
outside the U.S. within the past 30 days

- Are an employee of the site that is directly involved in the management,
administration, or support of the study, or be an immediate family member of the same

- Have a presence of a disease other than CNVM due to AMD in the study eye that could
affect vision or safety assessments

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Brisbane Site 1 - Brisbane
Recruitment hospital [2] 0 0
Melbourne Site 1 - Melbourne
Recruitment hospital [3] 0 0
Sydney Site 1 - Sydney
Recruitment hospital [4] 0 0
Sydney Site 2 - Sydney
Recruitment hospital [5] 0 0
Sydney Site 3 - Sydney
Recruitment postcode(s) [1] 0 0
- Brisbane
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- Sydney

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Ocular Therapeutix, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
To evaluate the safety, tolerability and efficacy of OTX-TKI for intravitreal use, in
subjects who have neovascular age-related macular degeneration (AMD).
Trial website
https://clinicaltrials.gov/show/NCT03630315
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Project Manager
Address 0 0
Country 0 0
Phone 0 0
1-781-357-4000
Fax 0 0
Email 0 0
clinicalaffairs@ocutx.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03630315