CLN-0046: Treatment of AMD Subjects With OTX-TKI

A Phase 1 Open-Label, Dose Escalation Study of OTX-TKI for Intravitreal Use in Subjects With Neovascular Age-Related Macular Degeneration (AMD)

To evaluate the safety, tolerability and efficacy of OTX-TKI for intravitreal use, in subjects who have neovascular age-related macular degeneration (AMD).

Study Overview

• Status: Completed
• Conditions: Neovascular Age-related Macular Degeneration
• Intervention / Treatment: Drug: OTX-TKI; Drug: Anti-VEGF

Detailed Description

A multi-center, dose escalation, Phase 1, safety, tolerability and efficacy study to evaluate three dose groups of the OTX-TKI implant to treat subjects with a diagnosis of primary subfoveal neovascularization secondary to AMD.

• Study Type: Interventional
• Enrollment (Actual): 29
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • Adelaide, Australia
    • Ocular Therapeutix, Inc.
  • Albury, Australia
    • Ocular Therapeutix, Inc.
  • Melbourne, Australia
    • Ocular Therapeutix, Inc.
  • Site 1
    • Sydney, Site 1, Australia
      • Ocular Therapeutix, Inc.
  • Site 2
    • Sydney, Site 2, Australia
      • Ocular Therapeutiux, Inc.
  • Site 3
    • Sydney, Site 3, Australia
      • Ocular Therapeutiux, Inc.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 50 years to 100 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1 (Low Dose); Subjects will receive a low dose of OTX-TKI	Drug: OTX-TKI; OTX-TKI is a dried polyethylene glycol-based hydrogel fiber containing dispersed microcrystals of a small molecule tyrosine kinase inhibitor (TKI).
Experimental: Cohort 2 (Middle Dose); Subjects will receive a middle dose of OTX-TKI.	Drug: OTX-TKI; OTX-TKI is a dried polyethylene glycol-based hydrogel fiber containing dispersed microcrystals of a small molecule tyrosine kinase inhibitor (TKI).
Experimental: Cohort 3 (High Dose); Subjects will receive a high dose of OTX-TKI.	Drug: OTX-TKI; OTX-TKI is a dried polyethylene glycol-based hydrogel fiber containing dispersed microcrystals of a small molecule tyrosine kinase inhibitor (TKI).
Experimental: Cohort 3 (Anti-VEGF); Subjects will receive OTX-TKI plus a single anti-VEGF injection	Drug: OTX-TKI; OTX-TKI is a dried polyethylene glycol-based hydrogel fiber containing dispersed microcrystals of a small molecule tyrosine kinase inhibitor (TKI).; Drug: Anti-VEGF; Standard of care therapy used to block vascular endothelial growth factor; Other Names: aflibercept; bevacizumab; ranibizumab
Experimental: Cohort 4 (High Dose); Subjects will receive a high dose of OTX-TKI.	Drug: OTX-TKI; OTX-TKI is a dried polyethylene glycol-based hydrogel fiber containing dispersed microcrystals of a small molecule tyrosine kinase inhibitor (TKI).
Experimental: Cohort 4 (Anti-VEGF); Subjects will receive OTX-TKI plus a single anti-VEGF injection	Drug: OTX-TKI; OTX-TKI is a dried polyethylene glycol-based hydrogel fiber containing dispersed microcrystals of a small molecule tyrosine kinase inhibitor (TKI).; Drug: Anti-VEGF; Standard of care therapy used to block vascular endothelial growth factor; Other Names: aflibercept; bevacizumab; ranibizumab

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment emergent adverse events for each subject	All adverse events from screening through end of study will be captured	9 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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. If all subjects tolerate lower dosages, additional subjects will be treated at higher dosages. All data will be evaluated by the DSMC who, in concert with the Medical Monitor, will determine the Maximally Tolerated Dose.	9 months

Collaborators and Investigators

• Sponsor: Ocular Therapeutix, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 18, 2019
• Primary Completion (Actual): January 10, 2024
• Study Completion (Actual): January 10, 2024

Study Registration Dates

• First Submitted: June 1, 2018
• First Submitted That Met QC Criteria: August 9, 2018
• First Posted (Actual): August 14, 2018

Study Record Updates

• Last Update Posted (Actual): July 10, 2024
• Last Update Submitted That Met QC Criteria: July 9, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration; Wet Macular Degeneration; Physiological Effects of Drugs; Antineoplastic Agents; Antineoplastic Agents, Immunological; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Ranibizumab; Bevacizumab; Aflibercept
• Other Study ID Numbers: CLN-0046

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No