Clinical trial NCT01064505: Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients
Official title: A Phase I Open-Label, Dose Escalation Trial of QPI-1007 Delivered by a Single Intravitreal Injection to Patients With Optic Nerve Atrophy (Stratum I) and Acute Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION) (Stratum II)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Optic Atrophy
Non-arteritic Anterior Ischemic Optic Neuropathy
Keywords: Ocular Neuroprotection
ocular neuroprotectant
NAION
NAAION
Non-arteritic ischemic optic neuropathy
Non-arteritic anterior ischemic optic neuropathy
chronic optic nerve atrophy
optic nerve atrophy
retinal degeneration
optic neuritis
end stage glaucoma
Leber's hereditary optic neuropathy
Lead sponsor: Quark Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2010-02-08
Enrollment: 48
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP

Brief summary: This is an open-label, dose escalation, safety, tolerability and pharmacokinetic study, where active study drug (QPI-1007) will be given to all patients who participate.

This study will determine whether QPI-1007 is safe when it is injected into the eye. The study will also reveal if there are any side effects of the drug and how long it takes for the body to clear the drug.

Detailed description: Patients will be enrolled according to one of two sets of criteria designated as Stratum I and Stratum II.

1. Stratum I will enroll Optic Nerve Atrophy patients who meet necessary criteria. Stratum I will consist of a maximum of 6 cohorts and each cohort will enroll 3 to 6 evaluable patients. Enrollment in Stratum I is now closed.
2. Stratum II will enroll patients diagnosed with acute Non Arteritic Anterior Ischemic Optic Neuropathy (NAION) within 28 days of symptom onset who meet necessary criteria. Stratum II will consist of a maximum of 3 cohorts and each cohort will enroll up to 10 evaluable patients. Enrollment in Stratum II is still open.

Eligibility criteria: Key Stratum I (Chronic Optic Nerve Atrophy) Inclusion Criteria:

* "Legally blind" in the study eye as the result of an irreversible condition affecting the posterior segment of the eye.
* Clear ocular media and able to undergo adequate pupil dilation.
* Visual acuity and visual field in the non-study eye are better than or equal to the study eye
* At least 21 years old.

Key Stratum I (Chronic Optic Nerve Atrophy) Exclusion Criteria:

* For the study eye only: history of any IVT injection or vitrectomy, vitreous hemorrhage, retinal detachment, or active inflammatory condition (e.g. conjunctivitis).
* For either eye: history of uveitis.

Enrollment is now closed in Stratum I.

Key Stratum II (Acute NAION) Inclusion Criteria:

* Positive diagnosis of NAION with symptom onset within 28 days prior to planned dosing with QPI-1007.
* Visual acuity in the study eye is between 20/40 and light perception.
* Clear ocular media and able to undergo adequate pupil dilation.
* At least 50 years old.

Key Stratum II (Acute NAION) Exclusion Criteria:

* For the study eye only: Macular disease, retinopathy, or other eye disease limiting visual acuity; prior intraocular surgery (other than Lasik) and cataract surgery within 3 months prior to dosing; glaucoma laser surgery within 1 month prior to dosing; pain on or aggravated by eye movement; history of vitreous hemorrhage; history of retinal detachment; any active inflammatory condition (e.g. conjunctivitis); glaucoma or ocular hypertension; or intraocular pressure \> 26 mmHg.
* For either eye: History of optic neuritis; or history of uveitis.
* Received any treatment for NAION prior to dosing.
* Any other abnormality which in the opinion of the investigator is suggestive of a disease other than NAION in the study eye only.
* Clinical evidence of temporal arteritis.
* History of collagen vascular disease or other inflammatory disease, or history of multiple sclerosis.

Enrollment in Stratum II is still open.

Primary outcomes:
measure: To determine the safety, tolerability and the dose-limiting toxicities (DLTs) of QPI 1007 when administered as a single intravitreal (IVT) injection.
timeFrame: 12 Months Post-injection
measure: To assess the pharmacokinetics (PK) of QPI-1007 when administered as a single IVT injection.
timeFrame: 12 Months Post-Injection
Secondary outcomes:
measure: To determine the presence of and to describe any anatomical changes in the optic nerve head and retina observed following the administration of a single IVT injection of QPI 1007.
timeFrame: 12 Months Post-Injection
measure: To assess any changes in visual acuity and visual field observed following the administration of a single IVT injection of QPI-1007.
timeFrame: 12 Months Post-Injection
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Retinal Consultants of Arizona
state: Arizona
city: Beverly Hills
country: United States
facility: Retina-Vitreous Associates Medical Group
state: California
city: Los Angeles
country: United States
facility: Jules Stein Eye Institute, University of California, Los Angeles
state: California
city: Aurora
country: United States
facility: Rocky Mountain Multiple Sclerosis Center at Anschutz Medical Campus, University of Colorado
state: Colorado
city: Miami
country: United States
facility: Bascom Palmer at the University of Miami
state: Florida
city: Sarasota
country: United States
facility: Sarasota Retina Institute
state: Florida
city: Chicago
country: United States
facility: University of Illinois at Chicago, Eye and Ear Infirmary
state: Illinois
city: Kansas City
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky, Department of Ophthalmology
state: Kentucky
city: Minneapolis
country: United States
facility: University of Minnesota, Department of Ophthalmology
state: Minnesota
city: Columbia
country: United States
facility: Mason Eye Institute, University of Missouri
state: Missouri
city: New York
country: United States
facility: New York Eye and Ear Infirmary
state: New York
city: Rochester
country: United States
facility: Flaum Eye Institute, University of Rochester Medical Center
state: New York
city: Asheville
country: United States
facility: Western Carolina Retinal Associates
state: North Carolina
city: Charlotte
country: United States
facility: Charlotte Eye, Ear, Nose and Throat Associates, PA
state: North Carolina
city: Durham
country: United States
facility: Duke Eye Center, Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Cole Eye Institute, Cleveland Clinic Foundation
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania, Department of Ophthalmology
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Allegheny Ophthalmic and Orbital Associates
state: Pennsylvania
city: Houston
country: United States
facility: Alkek Eye Center Baylor College of Medicine
state: Texas
city: Salt Lake City
country: United States
facility: John A. Moran Eye Center at the University of Utah
state: Utah
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Beer-Sheva
country: Israel
facility: Soroka University Medical Center, Dept of Ophthalmology
city: Haifa
country: Israel
facility: Bnai Zion Medical Center
city: Petach Tikva
country: Israel
facility: Rabin Medical Center, Belinson Campus, Dept of Ophthalmology
city: Rehovot
country: Israel
facility: Kaplan Medical Center, Department of Ophthalmology
city: Tel-Aviv
country: Israel
facility: The Tel-Aviv Sourasky Medical Center, Dept of Ophthalmology
city: Tel-Hashomer
country: Israel
facility: The Chaim Sheba Medical Center, Dept of Ophthalmology
Study officials:
affiliation: Quark Pharmaceuticals
name: Rabia Ozden, MD
role: STUDY_DIRECTOR
References: