Clinical trial NCT02341560: Phase 2/3, Randomized, Double-Masked, Sham-Controlled Trial of QPI-1007 in Subjects With Acute Nonarteritic Anterior Ischemic Optic Neuropathy (NAION)
Official title: A Phase 2/3, Randomized, Double-Masked, Sham-Controlled Trial of QPI-1007 Delivered By Single or Multi-Dose Intravitreal Injection(s) to Subjects With Acute Nonarteritic Anterior Ischemic Optic Neuropathy (NAION)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Non Arteritic Anterior Ischemic Optic Neuropathy
Keywords: NAION
Lead sponsor: Quark Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2015-01-19
Enrollment: 732
Sex: ALL
Minimum age (years): 50
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will determine the effect of QPI-1007 on visual function in subjects with recent-onset NAION and assess the safety and tolerability of intravitreal injections of QPI-1007 in this population. This study will also evaluate the structural changes in the retina following administration of QPI-1007.

Detailed description: This is a double masked, randomized, sham-controlled efficacy and safety study that will enroll approximately 800 subjects with recent-onset NAION. Subjects will be randomized into one of 3 groups in a 1:1:1 ratio, and assigned to receive QPI-1007 and/or a sham procedure. Subjects will have a two in three (66%) chance of receiving active treatment (no sham procedure) and a one in three (33%) chance of receiving sham procedure (no active treatment). Total study time involvement is approximately 12 months.

Eligibility criteria: Key Inclusion Criteria:

* Positive diagnosis of first episode of NAION in the study eye with symptom onset within 14 days prior to planned study drug administration/sham procedure
* Best corrected visual acuity score in the study eye is better than or equal to 15 letter score, measured using the ETDRS visual acuity protocol at Day 1 prior to study drug administration/sham procedure.
* Clear ocular media and able to undergo adequate pupil dilation to allow a good fundus examination

Key Exclusion Criteria:

* Present use or history of any treatment for the current episode of NAION, including systemic steroids, brimonidine, or traditional Chinese herbal medicine
* Prior episode of NAION in the study eye only
* Present use of drugs known to cause optic nerve or retinal toxicity at Day 1/Randomization, such as: chloroquine or hydroxychloroquine, ethambutol, Vigabatrin. Subjects who need to be prescribed any of these drugs during the course of the study will be discontinued from the trial.
* Any medical condition, concomitant therapy, or previous incisional or laser surgery that, in the opinion of the Investigator, would preclude IVT injection in the study eye only
* Clinical evidence of temporal arteritis

Primary outcomes:
measure: Effect of QPI-1007 as assessed by Best Corrected Visual Acuity (BCVA)
timeFrame: Baseline through Month 12
measure: Safety and tolerability of QPI-1007 as assessed by adverse events (AE), laboratory evaluations, ECGs, ophthalmic evaluations, slit lamp anterior/posterior examinations, intraocular pressure measurements, presence/absence of vitreous inflammation
timeFrame: Baseline through Month 12
Secondary outcomes:
measure: Mean change in BCVA score, as measured by ETDRS visual acuity protocol in the study eye
timeFrame: Day 1 through Month 12
measure: Mean change of Visual Fields, as assessed by Humphrey standard automated perimetry
timeFrame: Day 1 through Month 12
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: University of Arkansas Medical Center, Jones Eye Institute
state: Arkansas
city: Loma Linda
country: United States
facility: Loma Linda University Eye Institute
state: California
city: Los Angeles
country: United States
facility: University of Southern California
state: California
city: Los Angeles
country: United States
facility: NeuroEyeOrbit Institute
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Pasadena
country: United States
facility: Unversity of California, Los Angeles (UCLA)
state: California
city: Aurora
country: United States
facility: University of Colorado Denver, Department of Neurology
state: Colorado
city: Colorado Springs
country: United States
facility: Retina Consultants of Southern Colorado, PC
state: Colorado
city: Waterbury
country: United States
facility: The Eye Care Group
state: Connecticut
city: Cape Coral
country: United States
facility: Cape Coral Eye Center
state: Florida
city: Miami
country: United States
facility: University of Miami Miller School of Medicine/Bascom Palmer Eye Institute
state: Florida
city: Sarasota
country: United States
facility: Sarasota Retina Institute
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Chicago
country: United States
facility: University of Illinois at Chicago, Department of Ophthalmology (MC 648)
state: Illinois
city: Glenview
country: United States
facility: NorthShore University Healthsystem
state: Illinois
city: Lexington
country: United States
facility: University of Kentucky, Department of Neurology
state: Kentucky
city: Baltimore
country: United States
facility: Greater Baltimore Medical Center
state: Maryland
city: Bethesda
country: United States
facility: Bethesda Neurology, LLC
state: Maryland
city: Boston
country: United States
facility: Massachusetts Eye and Ear Infirmary
state: Massachusetts
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University in St. Louis
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center and Truhlsen Eye Institute
state: Nebraska
city: Newark
country: United States
facility: Rutgers, New Jersey Medical School
state: New Jersey
city: New York
country: United States
facility: New York Eye and Ear Infirmary of Mount Sinai
state: New York
city: New York
country: United States
facility: Cornell University School of Medicine
state: New York
city: Rochester
country: United States
facility: University of Rochester, Flaum Eye Institute
state: New York
city: Stony Brook
country: United States
facility: State University of New York at Stony Brook
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University
state: New York
city: Durham
country: United States
facility: Duke University Eye Center
state: North Carolina
city: Raleigh
country: United States
facility: Raleigh Neurology Associates
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic, Cole Eye Institute
state: Ohio
city: Oklahoma City
country: United States
facility: Dean McGee Eye Institute
state: Oklahoma
city: Philadelphia
country: United States
facility: University of Pennsylvania, Scheie Eye Institute
state: Pennsylvania
city: Charleston
country: United States
facility: Retina Consultants of Charleston
state: South Carolina
city: Houston
country: United States
facility: University of Houston, Neuro-Ophth of Texas
state: Texas
city: Houston
country: United States
facility: Houston Methodist Hospital, Blanton Eye Institute
state: Texas
city: San Antonio
country: United States
facility: Foresight Studies
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah John A. Moran Eye Center
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia Health Sciences Center
state: Virginia
city: Seattle
country: United States
facility: Swedish Medical Center, CO/NOCNW
state: Washington
city: Seattle
country: United States
facility: University of Washington Department of Ophthalmology
state: Washington
city: Sydney
country: Australia
facility: Sydney Eye Hospital/Save Sight Institute
state: New South Wales
city: Kent Town
country: Australia
facility: Vision South Australia
state: South Australia
city: Fitzroy
country: Australia
facility: The Royal Victorian Eye and Ear Hospital
state: Victoria
city: Perth
country: Australia
facility: Royal Perth Hospital
state: Western Australia
city: Changsha
country: China
facility: The Second Xiangya Hospital of Central South University
state: Hunan
city: Beijing
country: China
facility: Peking University Eye Center, The Third Hospital Peking University.
city: Beijing
country: China
facility: Beijing Tongren Hospital, Capital Medical University
city: Beijing
country: China
facility: Peking Union Medical College Hospital
city: Tianjin
country: China
facility: Tianjin Eye Hospital
city: Mainz
country: Germany
facility: Universitätsmedizin d. Johannes Gutenberg-Universität Mainz
state: Rheinland-Pfalz
city: Aachen
country: Germany
facility: Universitätsklinikum Aachen-Augenklinik
city: Dresden
country: Germany
facility: Universitätsklinikum Carl Gustav Carus-Dresden-Klinik und Poliklinik für Augenheilkunde
city: Münster
country: Germany
facility: Universitätsklinikum Münster
city: Regensburg
country: Germany
facility: Universitätsklinikum Regensburg-Klinik und Poliklinik für Augenheilkunde
city: Tubingen
country: Germany
facility: Universitäts-Augenklinik Tübingen
city: Hyderabad
country: India
facility: L V Prasad Eye Institute
state: Andhra Pradesh
city: Visakhapatnam
country: India
facility: L V Prasad Eye Institute
state: Andhra Pradesh
city: Guwahati
country: India
facility: Sri Sankaradeva Nethralaya
state: Assam
city: Bangalore
country: India
facility: Narayana Nethralaya
state: Karnataka,
city: Pune
country: India
facility: PBMA'S H. V. Desai Eye Hospital
state: Maharashtra
city: Bhubaneswar
country: India
facility: L V Prasad Eye Institute
state: Orissa
city: Chennai
country: India
facility: Sankara Nethralaya (Vision Research Foundation)
state: Tami Nadu
city: Madurai
country: India
facility: Aravind Eye Hospital
state: Tamil Nadu
city: Lucknow
country: India
facility: Sanjay Gandhi Postgraduate Institute of Medical Sciences
state: Uttar Pradesh
city: Noida
country: India
facility: ICARE Research Centre and Post Graduate Institute
state: Uttar Pradesh
city: New Delhi
country: India
facility: Dr. Rajendra Prasad Centre for Ophthalmic Sciences, A.I.I.M.S.
city: Wardha
country: India
facility: Datta Meghe Institute of Medical Sciences
city: Jerusalem
country: Israel
facility: Hadassah Medical Center
state: Yerushalayim
city: Beer-Sheva
country: Israel
facility: Soroka University Medical Center
city: Haifa
country: Israel
facility: Bnai Zion Medical Center
city: Petach-Tikva
country: Israel
facility: Rabin Medical Center, Belinson Campus
city: Rehovot
country: Israel
facility: Kaplan Medical Center
city: Tel-Aviv
country: Israel
facility: The Tel-Aviv Sourasky Medical Center
city: Tel-Hashomer
country: Israel
facility: The Chaim Sheba Medical Center
city: Bologna
country: Italy
facility: AOU Policlinico S. Orsola-Malpighi
city: Firenze
country: Italy
facility: AOU Careggi
city: Milano
country: Italy
facility: Scientific Institute Auxologico Italiano
city: Milano
country: Italy
facility: Scientific institute San Raffaele
city: Parma
country: Italy
facility: University Eye Clinic
city: Singapore
country: Singapore
facility: Singapore National Eye Centre
city: Singapore
country: Singapore
facility: Tan Tock Seng Hospital, Department of Ophthalmology
Study officials:
affiliation: Quark Pharmaceuticals
name: Sharon Klier, M.D.
role: STUDY_DIRECTOR
References:
citation: Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5.