Clinical trial NCT04537884: Safety and Tolerability Study of UBX1325 in Patients With Diabetic Macular Edema or Neovascular Age-Related Macular Degeneration
Official title: A Phase 1, Open-Label, Single Ascending Dose Study to Assess the Safety and Tolerability of a Single Intravitreal Injection of UBX1325 in Patients With Diabetic Macular Edema or Neovascular Age-Related Macular Degeneration
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Diabetic Macular Edema
Neovascular Age-related Macular Degeneration
Keywords: Retinal disease
Macular edema
Diabetes mellitus
Diabetic macular edema
Neovascular Age-Related Macular Degeneration
Lead sponsor: Unity Biotechnology, Inc.
Sponsor class: INDUSTRY
First posted: 2020-09-03
Enrollment: 19
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: A study to evaluate safety, tolerability, and pharmacokinetics of a single intravitreal injection of UBX1325 in patients diagnosed with diabetic macular edema (DME) or neovascular Age-Related Macular Degeneration.

Detailed description: This is a Phase 1, open-label, first-in-human (FIH), single-ascending dose (SAD) study consisting of approximately 4 cohorts. The total number of patients will be at least 3 per cohort plus 3 additional patients in the maximum tolerated dose (MTD) cohort in order to address the primary objective. Up to an additional 6 nAMD patients will be enrolled in the highest dose cohort. A single dose of UBX1325 will be administered intravitreally and all patients will be followed for approximately 6 months.

Eligibility criteria: Inclusion Criteria:

* nAMD patients who, in the opinion of the Investigator, are inadequately responding to or failed current treatment options.
* Active choroidal neovascularization (CNV) associated with age-related macular degeneration as evidenced on FA and SD-OCT at Day 1, including presence of intraretinal or subretinal fluid.
* BCVA in the study eye (most affected) of 70 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or worse at screening and on Day 1.
* Patients who have the capacity to give informed consent and who are willing and able to comply with all study-related procedures and assessments.

Exclusion Criteria:

* Any ocular/intraocular/periocular infection or inflammation in either eye in the past 4 weeks prior to screening.
* Subretinal hemorrhage with bleeding area ≥4 disc area in the study eye
* Concomitant therapy with anti-VEGF therapies (e.g., Avastin®, Lucentis®, or Eylea®) or previous use of these agents in the study eye within approximately 28 days of study enrollment
* Any retinovascular disease or retinal degeneration other than nAMD in the study eye.
* History of systemic and intraocular steroid use for 6 months prior to Day 1. The use of intravitreal nonbiodegradable steroid implants (ex. Iluvien®, Yutiq®, Retisert®) is prohibited.
* Significant media opacities, including cataract, which might interfere with VA, assessment of toxicity, or fundus imaging.
* Any uncontrolled medical condition that, in the opinion of the investigator, would preclude participation in this study.

Primary outcomes:
measure: Ocular and systemic safety and tolerability of a single intravitreal injection of UBX1325 evaluated by the incidence of dose limiting toxicities (DLTs) and treatment emergent adverse events (TEAEs)
timeFrame: 24 weeks
Secondary outcomes:
measure: Plasma concentration of UBX1325 following a single intravitreal injection
timeFrame: up to 24 hours post dose
Other outcomes:
Locations:
city: Beverly Hills
country: United States
facility: Retina-Vitreous Associates Medical Group
state: California
city: Huntington Beach
country: United States
facility: Salehi Retina Institute
state: California
city: Miami
country: United States
facility: MedEye Associates
state: Florida
city: Sarasota
country: United States
facility: Sarasota Retina Institute
state: Florida
city: Winter Haven
country: United States
facility: Center for Retina and Macular Disease
state: Florida
city: Indianapolis
country: United States
facility: MidWest Eye Institute
state: Indiana
city: Houston
country: United States
facility: Houston Eye Associates
state: Texas
city: San Antonio
country: United States
facility: Medical Center Ophthalmology Associates
state: Texas
Study officials:
affiliation: UNITY Biotechnology
name: Jamie Dananberg, MD
role: STUDY_CHAIR
References: