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

May 9, 2013 updated by: Quark Pharmaceuticals

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)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

48

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

50 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
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.
Time Frame: 12 Months Post-Injection
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.
Time Frame: 12 Months Post-Injection
12 Months Post-Injection

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Quark Pharmaceuticals

Investigators

  • Study Director: Rabia Ozden, MD, Quark Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2010

Primary Completion (Actual)

April 1, 2013

Study Completion (Actual)

April 1, 2013

Study Registration Dates

First Submitted

February 4, 2010

First Submitted That Met QC Criteria

February 4, 2010

First Posted (Estimate)

February 8, 2010

Study Record Updates

Last Update Posted (Estimate)

May 13, 2013

Last Update Submitted That Met QC Criteria

May 9, 2013

Last Verified

May 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • QRK.007

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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