Clinical trial NCT06467344: Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
Official title: ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Stargardt Disease
Cone Rod Dystrophy
Juvenile Macular Degeneration
Stargardt Disease 1
Keywords: ABCA4
ABCA4-related retinopathy
Stargardt Disease
Stargardt macular dystrophy
Cone rod dystrophy
Gene editing
RNA
Gene Therapy
Exon editing
IRD
Inherited retinal disease
Inherited retinal dystrophy
Inherited retinal degeneration
Lead sponsor: Ascidian Therapeutics, Inc
Sponsor class: INDUSTRY
First posted: 2024-06-21
Enrollment: 13
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.

Detailed description: This is an open-label, single ascending dose study of ACDN-01 in adult participants with ABCA4-related retinopathy. The study is designed to evaluate the safety, tolerability, and evidence of biological effect of SAD levels (low, medium, and high) of ACDN-01 when delivered subretinally.

Participants will be followed on study for 2 years for the primary safety and preliminary efficacy endpoints, after which they will continue in the study in a 3-year long-term follow-up period, for a total study duration of 5 years.

Eligibility criteria: Key Inclusion Criteria:

* Presence of mutations in the ABCA4 gene
* ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
* Area of atrophy located in the macula of the study eye
* BCVA of 20/80 (0.6 logMAR) or worse

Key Exclusion Criteria:

* The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy
* Retinal disease other than ABCA4-related retinopathy
* Presence of a medical condition (systemic or ophthalmic), psychiatric condition, including substance abuse disorder, or physical examination or laboratory finding that may in the opinion of the principal investigator and sponsor preclude adherence to the scheduled study visits, safe participation in the study, or affect the results of the study.

Primary outcomes:
measure: Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
timeFrame: 12 months
Secondary outcomes:
measure: Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.
timeFrame: 12 months
measure: To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).
timeFrame: 24 months
measure: To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).
timeFrame: 24 months
Other outcomes:
measure: To assess for evidence of preliminary efficacy of ACDN-01 based on best corrected visual acuity.
timeFrame: 24 months
measure: To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures.
timeFrame: 24 months
measure: To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
timeFrame: 24 months
measure: To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
timeFrame: 24 months
measure: To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
timeFrame: 24 months
measure: To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
timeFrame: 24 months
measure: To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
timeFrame: 24 months
Locations:
city: San Francisco
country: United States
facility: University of San Francisco
state: California
city: Gainesville
country: United States
facility: Vitreo Retinal Associates
state: Florida
city: Miami
country: United States
facility: Bascom Palmer Eye Institute
state: Florida
city: Baltimore
country: United States
facility: Wilmer Eye Institute at John Hopkins
state: Maryland
city: Boston
country: United States
facility: Massachusetts Eye and Ear
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Kellogg Eye Center
state: Michigan
city: Cincinnati
country: United States
facility: Cincinnati Eye Institute
state: Ohio
city: Portland
country: United States
facility: Casey Eye Institute OHSU
state: Oregon
city: Dallas
country: United States
facility: Retina Foundation of the Southwest
state: Texas
city: Houston
country: United States
facility: Retina Consultants of Texas
state: Texas
Study officials:
affiliation: Ascidian Therapeutics
name: Alia Rashid
role: STUDY_DIRECTOR
References: