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
Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
Sponsors
Source
Ophthotech Corporation
Oversight Info
Is Fda Regulated Drug
Yes
Is Fda Regulated Device
No
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).
Overall Status
Recruiting
Start Date
2018-01-12
Completion Date
2020-09-01
Primary Completion Date
2020-09-01
Phase
Phase 2
Study Type
Interventional
Primary Outcome
Measure |
Time Frame |
|
Spectral Domain-Optical Coherence Tomography (SD-OCT) |
18 months |
Enrollment
120
Condition
Intervention
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
Gender
All
Minimum Age
18 Years
Maximum Age
50 Years
Healthy Volunteers
No
Overall Contact
Location
Facility |
Status |
|
Retinal Consultants of Arizona Phoenix Arizona 85053 United States |
Recruiting |
|
Jules Stein Eye Institute/ David Geffen School of Medicine Los Angeles California 90095 United States |
Recruiting |
|
VitreoRetinal Associates Gainesville Florida 32607 United States |
Recruiting |
|
Ophthalmic Consultants of Boston Boston Massachusetts 02114 United States |
Recruiting |
|
Kellogg Eye Center Ann Arbor Michigan 48105 United States |
Recruiting |
|
Oregon Health & Science University Portland Oregon 97239 United States |
Recruiting |
|
Mid Atlantic Retina Philadelphia Pennsylvania 19107 United States |
Recruiting |
|
Palmetto Retina Center West Columbia South Carolina 29169 United States |
Recruiting |
|
Austin Retina Associates Austin Texas 78705 United States |
Recruiting |
|
Retina Foundation of the Southwest Dallas Texas 75231 United States |
Recruiting |
|
Strategic Clinical Research Group Willow Park Texas 76087 United States |
Recruiting |
|
University of Utah John A. Moran Eye Center Salt Lake City Utah 84132 United States |
Recruiting |
|
Hopital de la Croix-Rousse Lyon Rhone-Alpes 69317 France |
Recruiting |
|
Hopital Intercommunal de Verdun, University Paris Est Créteil 94000 France |
Recruiting |
|
Budapest Retina Associates Budapest 1133 Hungary |
Recruiting |
|
Semmelweis University, Ophthalmology Clinics Budapest H-1085 Hungary |
Recruiting |
|
Debreceni Egyetem Klinikai Kozpont Szemklinika Debrecen 4032 Hungary |
Recruiting |
|
Ganglion Medical Center Pécs 7621 Hungary |
Recruiting |
|
Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika Szeged 6720 Hungary |
Recruiting |
|
Rambam Health Care Campus Haifa 310960 Israel |
Recruiting |
|
Hadassah Medical Center Jerusalem 91120 Israel |
Recruiting |
|
Rabin Medical Center, Beilinson campus Petah tikva 4941492 Israel |
Recruiting |
|
Kaplan Medical Center Reẖovot 7610001 Israel |
Recruiting |
|
Tel-Aviv Sourasky Medical Center, Ichilov Hospital Tel Aviv 64239 Israel |
Recruiting |
|
AOU Careggi Florence 50134 Italy |
Recruiting |
|
AOU Universita degli studi della Campania Luigi Vanvitelli - UOC Oculistica Naples 80131 Italy |
Recruiting |
|
Foundation Policlinico Tor Vergata, UOSD Patologie Retiniche Rome 00133 Italy |
Recruiting |
|
Institut de la Macula Barcelona 08022 Spain |
Recruiting |
Location Countries
Country
France
Hungary
Israel
Italy
Spain
United States
Verification Date
2018-08-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Keywords
Has Expanded Access
No
Condition Browse
Number Of Arms
2
Intervention Browse
Mesh Term
Complement System Proteins
Arm Group
Arm Group Label
Cohort 1
Arm Group Type
Experimental
Description
Zimura dose group
Arm Group Label
Cohort 2
Arm Group Type
Sham Comparator
Description
Sham dose group
Firstreceived Results Date
N/A
Firstreceived Results Disposition Date
N/A
Study Design Info
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Single (Investigator)
Study First Submitted
December 1, 2017
Study First Submitted Qc
December 1, 2017
Study First Posted
December 6, 2017
Last Update Submitted
August 8, 2018
Last Update Submitted Qc
August 8, 2018
Last Update Posted
August 9, 2018
ClinicalTrials.gov processed this data on August 24, 2018
Conditions
Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov,
conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.