Stargardt's Macular Dystrophy Clinical Trial
Official title:
A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt Disease
Verified date | June 2024 |
Source | Astellas Pharma Inc |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
Status | Active, not recruiting |
Enrollment | 121 |
Est. completion date | April 30, 2025 |
Est. primary completion date | April 30, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 60 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
France | Creteil University Eye Clinic University Paris EST | Créteil | |
France | Hopital de la Croix-Rousse | Lyon | Rhone-Alpes |
France | Centre ophtalmologique des Quinzes Vingts | Paris | |
Germany | University of Bonn | Bonn | |
Germany | Augenklinik der LMU München | München | |
Germany | University of Tuebingen | Tübingen | |
Hungary | Budapest Retina Institute | Budapest | |
Hungary | Semmelweis Egyetem | Budapest | |
Hungary | University of Debrecen DE KK Szemészeti Klinika | Debrecen | |
Hungary | Ganglion Medical Center | Pécs | |
Hungary | Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika | Szeged | |
Israel | Rambam Health Care Campus | Haifa | |
Israel | Hadassah University Hospital | Jerusalem | |
Israel | Rabin Medical Center, Beilinson campus | Petah tikva | |
Israel | Kaplan Medical Center | Re?ovot | |
Israel | Tel-Aviv Sourasky Medical Center, Ichilov Hospital | Tel Aviv | |
Italy | AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia, | Bologna | |
Italy | Azienda Ospedaliera Universitaria Careggi | Florence | |
Italy | Ospedale San Raffaele | Milano | |
Italy | University of Campania Luigi Vanvitelli Eye Clinic | Naples | |
Italy | Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche | Rome | |
Spain | Institut de la Macula | Barcelona | |
United Kingdom | Princess Alexandra Eye Pavillion | Edinburgh | |
United Kingdom | Moorfields Eye Hospital | London | |
United States | University of Michigan/Kellogg Eye Center | Ann Arbor | Michigan |
United States | Austin Retina Associates | Austin | Texas |
United States | Wilmer Eye Institute, Johns Hopkins | Baltimore | Maryland |
United States | Retina Center of NJ, LLC. | Bloomfield | New Jersey |
United States | Ophthalmic Consultants of Boston | Boston | Massachusetts |
United States | Retina Foundation of the Southwest | Dallas | Texas |
United States | VitreoRetinal Associates | Gainesville | Florida |
United States | Jules Stein Eye Institute/ David Geffen School of Medicine | Los Angeles | California |
United States | The Retina Center | Minneapolis | Minnesota |
United States | Retina Specialty Institute | Pensacola | Florida |
United States | Wills Eye Hospital/Mid Atlantic Retina | Philadelphia | Pennsylvania |
United States | Retinal Research Institute | Phoenix | Arizona |
United States | UPMC Eye Center | Pittsburgh | Pennsylvania |
United States | Casey Eye Institute/Oregon Health & Science University | Portland | Oregon |
United States | University of Utah John A. Moran Eye Center | Salt Lake City | Utah |
United States | Palmetto Retina Center | West Columbia | South Carolina |
United States | Strategic Clinical Research Group | Willow Park | Texas |
Lead Sponsor | Collaborator |
---|---|
Astellas Pharma Global Development, Inc. |
United States, France, Germany, Hungary, Israel, Italy, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Mean rate of change in the area of ellipsoid zone defect | The ellipsoid zone will be measured through the foveal center with an en face Spectral Domain-Optical Coherence Tomography (SD-OCT). | up to 18 Months | |
Secondary | Mean rate of change in the horizontal width of undetectable ellipsoid zone | The ellipsoid zone will be measured by a horizontal scan through the foveal center with SD-OCT. | Baseline up to 18 Months | |
Secondary | Mean rate of change in the area of atrophic lesion (definite decrease in autofluorescence) | The definite decrease in autofluorescence (DDAF) will be measured by fundus autofluorescence (FAF). | Baseline up to 18 Months | |
Secondary | Mean change in photopic sensitivity | Photopic sensitivity will be measured by microperimetry. | Baseline up to 18 Months | |
Secondary | Mean change in mesopic macular sensitivity | Mesopic macular sensitivity will be measured by microperimetry. | Baseline up to 18 Months | |
Secondary | Mean rate of change in the thickness of the outer nuclear layer | 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. | Baseline up to 18 Months | |
Secondary | Mean change in best corrected visual acuity | Best corrected visual acuity (BCVA) will be measured by Early Treatment Diabetic Retinopathy Study [ETDRS] letters chart. | Baseline up to 18 Months | |
Secondary | Time to persistent vision loss | Vision loss is defined as BCVA loss >/= 10, 15 or 20 letters from Baseline at two or more consecutive visits through Month 18. | Baseline up to 18 Months | |
Secondary | Emergence of at least one new atrophic lesion (DDAF) | The DDAF will be measured by FAF. | Up to 18 Months | |
Secondary | Number of participants with Adverse Events (AEs) | 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. |
Up to 18 Months | |
Secondary | Number of participants with vital sign abnormalities and/or AEs | Number of participants with potentially clinically significant vital sign values. | Up to 18 Months | |
Secondary | Number of participants with ophthalmic abnormalities and/or AEs | Number of participants with potentially clinically significant ophthalmic variables. | Up to 18 Months | |
Secondary | Number of participants with 12-Lead electrocardiogram (ECG) abnormalities and/or AEs | Number of participants with potentially clinically significant 12-Lead ECG values. | Up to 18 Months | |
Secondary | Number of participants with laboratory value abnormalities and/or AEs | Number of participants with potentially clinically significant laboratory values. | Up to 18 Months |
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