Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

Phase 2

Completed

• Conditions: Stargardt's Macular Dystrophy
• Interventions: Drug: avacincaptad pegol; Drug: Sham

• Registration Number: NCT03364153
• Lead Sponsor: Astellas Pharma Global Development, Inc.

Brief Summary

The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-12-01
• Study First Submitted QC: 2017-12-01
• Study First Posted: 2017-12-06
• Study Start: 2018-01-12
• Primary Completion: 2025-03-31
• Study Completion: 2025-03-31
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-18
• Last Update Posted: 2025-05-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 121

Inclusion Criteria

• At least two pathogenic mutations of ATP-Binding Cassette (ABC)A4 gene confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory
• Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive

Exclusion Criteria

• Macular atrophy secondary to any condition other than STGD1 in either eye
• Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior intravitreal treatment for any indication in either eye
• Participation in an interventional study of a vitamin A derivative </= 3 months prior to screening
• Presence of intraocular inflammation, macular hole, pathologic myopia, epiretinal membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or aphakia
• Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region
• Diabetes mellitus
• Hemoglobin A1c (HbA1c) value of >/=6.5%
• Stroke within 12 months of trial entry
• Any major surgical procedure within one month of trial entry or anticipated during the trial
• Any treatment with an investigational agent in the past 60 days for any condition
• Women who are pregnant or nursing
• Known serious allergies to the fluorescein dye used in angiography, povidone iodine, or to the components of the avacincaptad pegol formulation

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
avacincaptad pegol	avacincaptad pegol	Participants will receive avacincaptad pegol monthly for up to 17 Months.
Sham	Sham	Participants will receive a matching sham monthly for up to 17 Months.

Primary Outcome Measures

Name	Time	Method
Rate of change in the area of ellipsoid zone defect	Baseline up to 18 Months	The area of ellipsoid zone defect will be measured by en face spectral domain-optical coherence tomography (SD-OCT).

Secondary Outcome Measures

Name	Time	Method
Time to persistent vision loss	Baseline up to 18 Months	Vision loss is defined as BCVA loss \>/= 10, 15 or 20 letters from Baseline at two or more consecutive visits through Month 18.
Emergence of at least one new atrophic lesion (DDAF)	Up to 18 Months	The DDAF will be measured by FAF.
Number of participants with Adverse Events (AEs)	Up to 18 Months	An AE is defined as any untoward medical occurrence in a participant including unfavorable and unintended signs, symptoms or disease temporally associated with the use of a medicinal product and which does not necessarily have to have a causal relationship to this treatment.; AEs include illnesses with onset during the trial, or exacerbations of pre-existing illnesses. Exacerbation of pre-existing illness is defined as a significant increase in the severity of the illness as compared to the start of the trial and should be considered when a patient requires new or additional treatment for that illness.
Number of participants with vital sign abnormalities and/or AEs	Up to 18 Months	Number of participants with potentially clinically significant vital sign values.
Number of participants with ophthalmic abnormalities and/or AEs	Up to 18 Months	Number of participants with potentially clinically significant ophthalmic variables.
Number of participants with 12-Lead electrocardiogram (ECG) abnormalities and/or AEs	Up to 18 Months	Number of participants with potentially clinically significant 12-Lead ECG values.
Number of participants with laboratory value abnormalities and/or AEs	Up to 18 Months	Number of participants with potentially clinically significant laboratory values.
Change in best corrected visual acuity	Baseline up to 18 Months	Best corrected visual acuity (BCVA) will be measured by Early Treatment Diabetic Retinopathy Study \[ETDRS\] letters chart.
Change in photopic and/or mesopic macular sensitivity	Baseline up to 18 Months	The photopic and/or mesopic macular sensitivity will be measured by microperimetry (optional assessment).
Rate of change in the area of atrophic lesion (definite decrease in autofluorescence)	Baseline up to 18 Months	The definite decrease in autofluorescence (DDAF) will be measured by fundus autofluorescence (FAF).
Rate of change in the thickness of the outer nuclear layer	Baseline up to 18 Months	The thickness of outer nuclear layer will be measured by a horizontal scan through the foveal center using SD-OCT.

Trial Locations

Locations (41)

Retinal Research Institute; 🇺🇸 Phoenix, Arizona, United States

Jules Stein Eye Institute/ David Geffen School of Medicine; 🇺🇸 Los Angeles, California, United States

VitreoRetinal Associates; 🇺🇸 Gainesville, Florida, United States

Retina Specialty Institute; 🇺🇸 Pensacola, Florida, United States

Wilmer Eye Institute, Johns Hopkins; 🇺🇸 Baltimore, Maryland, United States

Ophthalmic Consultants of Boston; 🇺🇸 Boston, Massachusetts, United States

University of Michigan/Kellogg Eye Center; 🇺🇸 Ann Arbor, Michigan, United States

The Retina Center; 🇺🇸 Minneapolis, Minnesota, United States

Retina Center of NJ, LLC.; 🇺🇸 Bloomfield, New Jersey, United States

Casey Eye Institute/Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

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