Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
- Conditions
- Stargardt's Macular Dystrophy
- Interventions
- Drug: avacincaptad pegolDrug: 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
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 121
- 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
- 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 Mean rate of change in the area of ellipsoid zone defect up to 18 Months The ellipsoid zone will be measured through the foveal center with an 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.
Mean 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).
Mean change in photopic sensitivity Baseline up to 18 Months Photopic sensitivity will be measured by microperimetry.
Mean 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.
Emergence of at least one new atrophic lesion (DDAF) Up to 18 Months The DDAF will be measured by FAF.
Mean rate of change in the thickness of the outer nuclear layer Baseline up to 18 Months The outer nuclear layer will be measured by a horizontal scan through the foveal center at the position of maximum width of ellipsoid zone loss measured by SD-OCT.
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.
Mean rate of change in the horizontal width of undetectable ellipsoid zone Baseline up to 18 Months The ellipsoid zone will be measured by a horizontal scan through the foveal center with SD-OCT.
Mean change in mesopic macular sensitivity Baseline up to 18 Months Mesopic macular sensitivity will be measured by microperimetry.
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 laboratory value abnormalities and/or AEs Up to 18 Months Number of participants with potentially clinically significant laboratory values.
Trial Locations
- Locations (41)
Retina Specialty Institute
🇺🇸Pensacola, Florida, United States
Wilmer Eye Institute, Johns Hopkins
🇺🇸Baltimore, Maryland, United States
Centre ophtalmologique des Quinzes Vingts
🇫🇷Paris, France
Rabin Medical Center, Beilinson campus
🇮🇱Petah tikva, Israel
Creteil University Eye Clinic University Paris EST
🇫🇷Créteil, France
Jules Stein Eye Institute/ David Geffen School of Medicine
🇺🇸Los Angeles, California, United States
Ospedale San Raffaele
🇮🇹Milano, Italy
UPMC Eye Center
🇺🇸Pittsburgh, Pennsylvania, United States
AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia,
🇮🇹Bologna, Italy
Retina Foundation of the Southwest
🇺🇸Dallas, Texas, United States
Strategic Clinical Research Group
🇺🇸Willow Park, Texas, United States
Moorfields Eye Hospital
🇬🇧London, United Kingdom
University of Campania Luigi Vanvitelli Eye Clinic
🇮🇹Naples, Italy
Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche
🇮🇹Rome, Italy
Princess Alexandra Eye Pavillion
🇬🇧Edinburgh, United Kingdom
Azienda Ospedaliera Universitaria Careggi
🇮🇹Florence, Italy
Retina Center of NJ, LLC.
🇺🇸Bloomfield, New Jersey, United States
Palmetto Retina Center
🇺🇸West Columbia, South Carolina, United States
University of Debrecen DE KK Szemészeti Klinika
ðŸ‡ðŸ‡ºDebrecen, Hungary
Hopital de la Croix-Rousse
🇫🇷Lyon, Rhone-Alpes, France
Ganglion Medical Center
ðŸ‡ðŸ‡ºPécs, Hungary
Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika
ðŸ‡ðŸ‡ºSzeged, Hungary
Kaplan Medical Center
🇮🇱Reẖovot, Israel
Budapest Retina Institute
ðŸ‡ðŸ‡ºBudapest, Hungary
Wills Eye Hospital/Mid Atlantic Retina
🇺🇸Philadelphia, Pennsylvania, United States
Augenklinik der LMU München
🇩🇪München, Germany
Tel-Aviv Sourasky Medical Center, Ichilov Hospital
🇮🇱Tel Aviv, Israel
Semmelweis Egyetem
ðŸ‡ðŸ‡ºBudapest, Hungary
University of Bonn
🇩🇪Bonn, Germany
University of Tuebingen
🇩🇪Tübingen, Germany
Rambam Health Care Campus
🇮🇱Haifa, Israel
Hadassah University Hospital
🇮🇱Jerusalem, Israel
Institut de la Macula
🇪🇸Barcelona, Spain
Retinal Research Institute
🇺🇸Phoenix, Arizona, United States
VitreoRetinal Associates
🇺🇸Gainesville, Florida, United States
University of Michigan/Kellogg Eye Center
🇺🇸Ann Arbor, Michigan, United States
The Retina Center
🇺🇸Minneapolis, Minnesota, United States
Ophthalmic Consultants of Boston
🇺🇸Boston, Massachusetts, United States
Casey Eye Institute/Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Austin Retina Associates
🇺🇸Austin, Texas, United States
University of Utah John A. Moran Eye Center
🇺🇸Salt Lake City, Utah, United States