Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients

Sponsor

Quark Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT01064505

Collaborator

(none)

Enrollment

Locations

Arm

37.9

Duration (Months)

1.7

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Optic Atrophy Non-arteritic Anterior Ischemic Optic Neuropathy	Drug: QPI-1007 at various doses	Phase 1

Detailed Description

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

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.
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.

Study Design

Study Type:

Interventional

Actual Enrollment :

48 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

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 Start Date :

Feb 1, 2010

Actual Primary Completion Date :

Apr 1, 2013

Actual Study Completion Date :

Apr 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: QPI-1007	Drug: QPI-1007 at various doses Single Intravitreal Injection Other Names: QPI-1007 siRNA small interfering RNA short interfering RNA

Arm

Intervention/Treatment

Experimental: QPI-1007

Drug: QPI-1007 at various doses

Single Intravitreal Injection

Other Names:

QPI-1007

siRNA

small interfering RNA

short interfering RNA

Outcome Measures

Primary Outcome Measures

To determine the safety, tolerability and the dose-limiting toxicities (DLTs) of QPI 1007 when administered as a single intravitreal (IVT) injection. [12 Months Post-injection]
To assess the pharmacokinetics (PK) of QPI-1007 when administered as a single IVT injection. [12 Months Post-Injection]

Secondary Outcome Measures

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. [12 Months Post-Injection]
To assess any changes in visual acuity and visual field observed following the administration of a single IVT injection of QPI-1007. [12 Months Post-Injection]

Eligibility Criteria

Criteria

Ages Eligible for Study:

50 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Retinal Consultants of Arizona	Phoenix	Arizona	United States	85014
2	Retina-Vitreous Associates Medical Group	Beverly Hills	California	United States	90211
3	Jules Stein Eye Institute, University of California, Los Angeles	Los Angeles	California	United States	90049
4	Rocky Mountain Multiple Sclerosis Center at Anschutz Medical Campus, University of Colorado	Aurora	Colorado	United States	80045
5	Bascom Palmer at the University of Miami	Miami	Florida	United States	33136
6	Sarasota Retina Institute	Sarasota	Florida	United States	34239
7	University of Illinois at Chicago, Eye and Ear Infirmary	Chicago	Illinois	United States	60612
8	Kansas University Medical Center	Kansas City	Kansas	United States	66208
9	University of Kentucky, Department of Ophthalmology	Lexington	Kentucky	United States	40536
10	University of Minnesota, Department of Ophthalmology	Minneapolis	Minnesota	United States	55455
11	Mason Eye Institute, University of Missouri	Columbia	Missouri	United States	65212
12	New York Eye and Ear Infirmary	New York	New York	United States	10003
13	Flaum Eye Institute, University of Rochester Medical Center	Rochester	New York	United States	14642
14	Western Carolina Retinal Associates	Asheville	North Carolina	United States	28803
15	Charlotte Eye, Ear, Nose and Throat Associates, PA	Charlotte	North Carolina	United States	28210
16	Duke Eye Center, Duke University Medical Center	Durham	North Carolina	United States	27705
17	Cole Eye Institute, Cleveland Clinic Foundation	Cleveland	Ohio	United States	44195
18	University of Pennsylvania, Department of Ophthalmology	Philadelphia	Pennsylvania	United States	19104
19	Allegheny Ophthalmic and Orbital Associates	Pittsburgh	Pennsylvania	United States	15212
20	Alkek Eye Center Baylor College of Medicine	Houston	Texas	United States	77030
21	John A. Moran Eye Center at the University of Utah	Salt Lake City	Utah	United States	84132
22	Virginia Commonwealth University	Richmond	Virginia	United States	23298
23	Soroka University Medical Center, Dept of Ophthalmology	Beer-Sheva		Israel	84101
24	Bnai Zion Medical Center	Haifa		Israel	33394
25	Rabin Medical Center, Belinson Campus, Dept of Ophthalmology	Petach Tikva		Israel	49100
26	Kaplan Medical Center, Department of Ophthalmology	Rehovot		Israel	76100
27	The Tel-Aviv Sourasky Medical Center, Dept of Ophthalmology	Tel-Aviv		Israel	64239
28	The Chaim Sheba Medical Center, Dept of Ophthalmology	Tel-Hashomer		Israel	52621