Clinical trial NCT03364153: Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Stargardt's Macular Dystrophy
Keywords: Zimura (previous name)
ARC1905
complement factor C5 inhibitor
avacincaptad pegol
STGD1
Izervay
Stargardt Disease 1
Stargardt's Macular Dystrophy
Lead sponsor: Astellas Pharma Inc
Sponsor class: INDUSTRY
First posted: 2017-12-06
Enrollment: 121
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Rate of change in the area of ellipsoid zone defect
timeFrame: Baseline up to 18 Months
Secondary outcomes:
measure: Change in best corrected visual acuity
timeFrame: Baseline up to 18 Months
measure: Change in photopic and/or mesopic macular sensitivity
timeFrame: Baseline up to 18 Months
measure: Rate of change in the area of atrophic lesion (definite decrease in autofluorescence)
timeFrame: Baseline up to 18 Months
measure: Rate of change in the thickness of the outer nuclear layer
timeFrame: Baseline up to 18 Months
measure: Time to persistent vision loss
timeFrame: Baseline up to 18 Months
measure: Emergence of at least one new atrophic lesion (DDAF)
timeFrame: Up to 18 Months
measure: Number of participants with Adverse Events (AEs)
timeFrame: Up to 18 Months
measure: Number of participants with vital sign abnormalities and/or AEs
timeFrame: Up to 18 Months
measure: Number of participants with ophthalmic abnormalities and/or AEs
timeFrame: Up to 18 Months
measure: Number of participants with 12-Lead electrocardiogram (ECG) abnormalities and/or AEs
timeFrame: Up to 18 Months
measure: Number of participants with laboratory value abnormalities and/or AEs
timeFrame: Up to 18 Months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Retinal Research Institute
state: Arizona
city: Los Angeles
country: United States
facility: Jules Stein Eye Institute/ David Geffen School of Medicine
state: California
city: Gainesville
country: United States
facility: VitreoRetinal Associates
state: Florida
city: Pensacola
country: United States
facility: Retina Specialty Institute
state: Florida
city: Baltimore
country: United States
facility: Wilmer Eye Institute, Johns Hopkins
state: Maryland
city: Boston
country: United States
facility: Ophthalmic Consultants of Boston
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan/Kellogg Eye Center
state: Michigan
city: Minneapolis
country: United States
facility: The Retina Center
state: Minnesota
city: Bloomfield
country: United States
facility: Retina Center of NJ, LLC.
state: New Jersey
city: Portland
country: United States
facility: Casey Eye Institute/Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: Wills Eye Hospital/Mid Atlantic Retina
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Eye Center
state: Pennsylvania
city: West Columbia
country: United States
facility: Palmetto Retina Center
state: South Carolina
city: Austin
country: United States
facility: Austin Retina Associates
state: Texas
city: Dallas
country: United States
facility: Retina Foundation of the Southwest
state: Texas
city: Willow Park
country: United States
facility: Strategic Clinical Research Group
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah John A. Moran Eye Center
state: Utah
city: Lyon
country: France
facility: Hopital de la Croix-Rousse
state: Rhone-Alpes
city: Créteil
country: France
facility: Creteil University Eye Clinic University Paris EST
city: Paris
country: France
facility: Centre ophtalmologique des Quinzes Vingts
city: Bonn
country: Germany
facility: University of Bonn
city: München
country: Germany
facility: Augenklinik der LMU München
city: Tübingen
country: Germany
facility: University of Tuebingen
city: Budapest
country: Hungary
facility: Budapest Retina Institute
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Debrecen
country: Hungary
facility: University of Debrecen DE KK Szemészeti Klinika
city: Pécs
country: Hungary
facility: Ganglion Medical Center
city: Szeged
country: Hungary
facility: Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika
city: Haifa
country: Israel
facility: Rambam Health Care Campus
city: Jerusalem
country: Israel
facility: Hadassah University Hospital
city: Petah tikva
country: Israel
facility: Rabin Medical Center, Beilinson campus
city: Reẖovot
country: Israel
facility: Kaplan Medical Center
city: Tel Aviv
country: Israel
facility: Tel-Aviv Sourasky Medical Center, Ichilov Hospital
city: Bologna
country: Italy
facility: AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia,
city: Florence
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi
city: Milano
country: Italy
facility: Ospedale San Raffaele
city: Naples
country: Italy
facility: University of Campania Luigi Vanvitelli Eye Clinic
city: Rome
country: Italy
facility: Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche
city: Barcelona
country: Spain
facility: Institut de la Macula
city: Edinburgh
country: United Kingdom
facility: Princess Alexandra Eye Pavillion
city: London
country: United Kingdom
facility: Moorfields Eye Hospital
Study officials:
affiliation: Astellas Pharma Global Development, Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: