Study to Evaluate ACDN-01 in ABCA4-related Retinopathy (STELLAR)

Stargardt Disease Clinical Trial

— 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06467344
• Other study ID #: ACDN-01-001
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: June 29, 2024
• Est. completion date: December 1, 2030

Study information

• Verified date: June 2024
• Source: Ascidian Therapeutics, Inc
• Contact: Associate Director, Clinical Operations
• Phone: 207-573-0412
• Email: researchtrials[@]ascidian-tx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 13
• Est. completion date: December 1, 2030
• Est. primary completion date: August 1, 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cone Rod Dystrophy
• Juvenile Macular Degeneration
• Macular Degeneration
• Retinal Diseases
• Stargardt Disease
• Stargardt Disease 1

Intervention

Drug:
• ACDN-01
ACDN-01 is an AAV-based vector carrying a DNA construct encoding for an ABCA4 RNA exon editor. One time administration is via subretinal injection.

Locations

Country	Name	City	State
United States	Cincinnati Eye Institute	Cincinnati	Ohio
United States	Retina Foundation of the Southwest	Dallas	Texas
United States	Vitreo Retinal Associates	Gainesville	Florida
United States	Retina Consultants of Texas	Houston	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Ascidian Therapeutics, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	To assess for evidence of preliminary efficacy of ACDN-01 based on best corrected visual acuity.	Best Corrected Visual Acuity is measured by number of ETDRS letters read in each eye compared to baseline.	24 months
Other	To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures.	Patient-reported outcome measures will be measured by overall and subscale scores on the Michigan Retinal -Degeneration Questionnaire (MRDQ).	24 months
Other	To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.	Contrast sensitivity is measured using Pelli-Robson and qCSF tests and will measure number of letters read and change in AUC (area under curve).	24 months
Other	To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.	Retinal sensitivity will be measured by change in the intensity (dB), number and location of detectable points.	24 months
Primary	Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.	To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy.	12 months
Secondary	Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.	To determine maximum tolerated dose (MTD) for subsequent clinical evaluation.	12 months
Secondary	To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).	To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in the area of macular atrophy in mm2 as measured on FAF over 24 months.	24 months
Secondary	To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).	To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in change in thickness in retinal layers as measured on OCT, over 24 months.	24 months