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

Primary Purpose

Stargardt Disease 1

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Zimura
Sham
Sponsored by
IVERIC bio, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Stargardt Disease 1 focused on measuring Zimura, ARC1905, complement factor C5 inhibitor, avacincaptad pegol, STGD1

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • At least two pathogenic mutations of ABCA4 gene confirmed by a 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
  • 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 Zimura formulation

Sites / Locations

  • Retinal Research Institute
  • Jules Stein Eye Institute/ David Geffen School of Medicine
  • VitreoRetinal Associates
  • Retina Specialty Institute
  • Wilmer Eye Institute, Johns Hopkins
  • Ophthalmic Consultants of Boston
  • University of Michigan/Kellogg Eye Center
  • The Retina Center
  • Envision Ocular, LLC
  • Casey Eye Institute/Oregon Health & Science University
  • Wills Eye Hospital/Mid Atlantic Retina
  • UPMC Eye Center
  • Palmetto Retina Center
  • Austin Retina Associates
  • Retina Consultants of Texas
  • Retina Foundation of the Southwest
  • Strategic Clinical Research Group
  • University of Utah John A. Moran Eye Center
  • Hopital de la Croix-Rousse
  • Creteil University Eye Clinic University Paris EST
  • Centre ophtalmologique des Quinzes Vingts
  • University of Bonn
  • Augenklinik der LMU München
  • University of Tuebingen
  • Budapest Retina Institute
  • Semmelweis Egyetem
  • University of Debrecen DE KK Szemészeti Klinika
  • Ganglion Medical Center
  • Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika
  • Rambam Health Care Campus
  • Hadassah University Hospital
  • Rabin Medical Center, Beilinson campus
  • Kaplan Medical Center
  • Tel-Aviv Sourasky Medical Center, Ichilov Hospital
  • AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia,
  • Azienda Ospedaliera Universitaria Careggi
  • Ospedale San Raffaele
  • University of Campania Luigi Vanvitelli Eye Clinic
  • Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche
  • Institut de la Macula
  • Princess Alexandra Eye Pavillion
  • Moorfields Eye Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Sham Comparator

Arm Label

Cohort 1

Cohort 2

Arm Description

Zimura dose group

Sham dose group

Outcomes

Primary Outcome Measures

Spectral Domain-Optical Coherence Tomography (SD-OCT)
Mean rate of change in the area of ellipsoid zone defect measured by en face SD-OCT

Secondary Outcome Measures

Full Information

First Posted
December 1, 2017
Last Updated
October 3, 2023
Sponsor
IVERIC bio, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03364153
Brief Title
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

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
January 12, 2018 (Actual)
Primary Completion Date
April 2025 (Anticipated)
Study Completion Date
April 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
IVERIC bio, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
To evaluate the safety and efficacy of Zimura™ (complement factor C5 inhibitor) compared to Sham in subjects with autosomal recessive Stargardt disease 1 (STGD1).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Stargardt Disease 1
Keywords
Zimura, ARC1905, complement factor C5 inhibitor, avacincaptad pegol, STGD1

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
120 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
Zimura dose group
Arm Title
Cohort 2
Arm Type
Sham Comparator
Arm Description
Sham dose group
Intervention Type
Drug
Intervention Name(s)
Zimura
Other Intervention Name(s)
avacincaptad pegol
Intervention Description
Zimura Intravitreal Injection
Intervention Type
Other
Intervention Name(s)
Sham
Intervention Description
Sham Intravitreal Injection
Primary Outcome Measure Information:
Title
Spectral Domain-Optical Coherence Tomography (SD-OCT)
Description
Mean rate of change in the area of ellipsoid zone defect measured by en face SD-OCT
Time Frame
18 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: At least two pathogenic mutations of ABCA4 gene confirmed by a 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 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 Zimura formulation
Facility Information:
Facility Name
Retinal Research Institute
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85053
Country
United States
Facility Name
Jules Stein Eye Institute/ David Geffen School of Medicine
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
VitreoRetinal Associates
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32607
Country
United States
Facility Name
Retina Specialty Institute
City
Pensacola
State/Province
Florida
ZIP/Postal Code
32503
Country
United States
Facility Name
Wilmer Eye Institute, Johns Hopkins
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Ophthalmic Consultants of Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
University of Michigan/Kellogg Eye Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Facility Name
The Retina Center
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
Envision Ocular, LLC
City
Bloomfield
State/Province
New Jersey
ZIP/Postal Code
07003
Country
United States
Facility Name
Casey Eye Institute/Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Wills Eye Hospital/Mid Atlantic Retina
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
UPMC Eye Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Palmetto Retina Center
City
West Columbia
State/Province
South Carolina
ZIP/Postal Code
29169
Country
United States
Facility Name
Austin Retina Associates
City
Austin
State/Province
Texas
ZIP/Postal Code
78705
Country
United States
Facility Name
Retina Consultants of Texas
City
Bellaire
State/Province
Texas
ZIP/Postal Code
77401
Country
United States
Facility Name
Retina Foundation of the Southwest
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
Strategic Clinical Research Group
City
Willow Park
State/Province
Texas
ZIP/Postal Code
76087
Country
United States
Facility Name
University of Utah John A. Moran Eye Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Hopital de la Croix-Rousse
City
Lyon
State/Province
Rhone-Alpes
ZIP/Postal Code
69004
Country
France
Facility Name
Creteil University Eye Clinic University Paris EST
City
Créteil
ZIP/Postal Code
94010
Country
France
Facility Name
Centre ophtalmologique des Quinzes Vingts
City
Paris
ZIP/Postal Code
75012
Country
France
Facility Name
University of Bonn
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
Augenklinik der LMU München
City
München
ZIP/Postal Code
80336
Country
Germany
Facility Name
University of Tuebingen
City
Tübingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
Budapest Retina Institute
City
Budapest
ZIP/Postal Code
1133
Country
Hungary
Facility Name
Semmelweis Egyetem
City
Budapest
ZIP/Postal Code
H-1083
Country
Hungary
Facility Name
University of Debrecen DE KK Szemészeti Klinika
City
Debrecen
ZIP/Postal Code
4032
Country
Hungary
Facility Name
Ganglion Medical Center
City
Pécs
ZIP/Postal Code
7621
Country
Hungary
Facility Name
Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika
City
Szeged
ZIP/Postal Code
6720
Country
Hungary
Facility Name
Rambam Health Care Campus
City
Haifa
ZIP/Postal Code
3109601
Country
Israel
Facility Name
Hadassah University Hospital
City
Jerusalem
ZIP/Postal Code
9112001
Country
Israel
Facility Name
Rabin Medical Center, Beilinson campus
City
Petah tikva
ZIP/Postal Code
4941492
Country
Israel
Facility Name
Kaplan Medical Center
City
Reẖovot
ZIP/Postal Code
7610001
Country
Israel
Facility Name
Tel-Aviv Sourasky Medical Center, Ichilov Hospital
City
Tel Aviv
ZIP/Postal Code
6423906
Country
Israel
Facility Name
AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia,
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Careggi
City
Florence
ZIP/Postal Code
50121
Country
Italy
Facility Name
Ospedale San Raffaele
City
Milano
ZIP/Postal Code
20132
Country
Italy
Facility Name
University of Campania Luigi Vanvitelli Eye Clinic
City
Naples
ZIP/Postal Code
80131
Country
Italy
Facility Name
Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche
City
Rome
ZIP/Postal Code
00133
Country
Italy
Facility Name
Institut de la Macula
City
Barcelona
ZIP/Postal Code
08022
Country
Spain
Facility Name
Princess Alexandra Eye Pavillion
City
Edinburgh
ZIP/Postal Code
EH3 9HA
Country
United Kingdom
Facility Name
Moorfields Eye Hospital
City
London
ZIP/Postal Code
EC1V 2PD
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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