NCT01064505

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
48

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Feb 2010

Typical duration for phase_1

Geographic Reach
2 countries

28 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2010

Completed
3 days until next milestone

First Submitted

Initial submission to the registry

February 4, 2010

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 8, 2010

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2013

Completed
Last Updated

May 13, 2013

Status Verified

May 1, 2013

Enrollment Period

3.2 years

First QC Date

February 4, 2010

Last Update Submit

May 9, 2013

Conditions

Keywords

Ocular Neuroprotectionocular neuroprotectantNAIONNAAIONNon-arteritic ischemic optic neuropathyNon-arteritic anterior ischemic optic neuropathychronic optic nerve atrophyoptic nerve atrophyretinal degenerationoptic neuritisend stage glaucomaLeber's hereditary optic neuropathy

Outcome Measures

Primary Outcomes (2)

  • 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 Outcomes (2)

  • 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

Study Arms (1)

QPI-1007

EXPERIMENTAL
Drug: QPI-1007 at various doses

Interventions

Single Intravitreal Injection

Also known as: QPI-1007, siRNA, small interfering RNA, short interfering RNA
QPI-1007

Eligibility Criteria

Age50 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • "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.

You may not qualify if:

  • 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.
  • 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.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (28)

Retinal Consultants of Arizona

Phoenix, Arizona, 85014, United States

Location

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

Location

Jules Stein Eye Institute, University of California, Los Angeles

Los Angeles, California, 90049, United States

Location

Rocky Mountain Multiple Sclerosis Center at Anschutz Medical Campus, University of Colorado

Aurora, Colorado, 80045, United States

Location

Bascom Palmer at the University of Miami

Miami, Florida, 33136, United States

Location

Sarasota Retina Institute

Sarasota, Florida, 34239, United States

Location

University of Illinois at Chicago, Eye and Ear Infirmary

Chicago, Illinois, 60612, United States

Location

Kansas University Medical Center

Kansas City, Kansas, 66208, United States

Location

University of Kentucky, Department of Ophthalmology

Lexington, Kentucky, 40536, United States

Location

University of Minnesota, Department of Ophthalmology

Minneapolis, Minnesota, 55455, United States

Location

Mason Eye Institute, University of Missouri

Columbia, Missouri, 65212, United States

Location

New York Eye and Ear Infirmary

New York, New York, 10003, United States

Location

Flaum Eye Institute, University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Western Carolina Retinal Associates

Asheville, North Carolina, 28803, United States

Location

Charlotte Eye, Ear, Nose and Throat Associates, PA

Charlotte, North Carolina, 28210, United States

Location

Duke Eye Center, Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Cole Eye Institute, Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

University of Pennsylvania, Department of Ophthalmology

Philadelphia, Pennsylvania, 19104, United States

Location

Allegheny Ophthalmic and Orbital Associates

Pittsburgh, Pennsylvania, 15212, United States

Location

Alkek Eye Center Baylor College of Medicine

Houston, Texas, 77030, United States

Location

John A. Moran Eye Center at the University of Utah

Salt Lake City, Utah, 84132, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

Soroka University Medical Center, Dept of Ophthalmology

Beersheba, 84101, Israel

Location

Bnai Zion Medical Center

Haifa, 33394, Israel

Location

Rabin Medical Center, Belinson Campus, Dept of Ophthalmology

Petah Tikva, 49100, Israel

Location

Kaplan Medical Center, Department of Ophthalmology

Rehovot, 76100, Israel

Location

The Tel-Aviv Sourasky Medical Center, Dept of Ophthalmology

Tel Aviv, 64239, Israel

Location

The Chaim Sheba Medical Center, Dept of Ophthalmology

Tel Litwinsky, 52621, Israel

Location

MeSH Terms

Conditions

Optic AtrophyOptic Neuropathy, IschemicRetinal DegenerationOptic NeuritisOptic Atrophy, Hereditary, Leber

Interventions

RNA, Small Interfering

Condition Hierarchy (Ancestors)

Optic Nerve DiseasesCranial Nerve DiseasesNervous System DiseasesEye DiseasesVascular DiseasesCardiovascular DiseasesEye Diseases, HereditaryRetinal DiseasesOptic Atrophies, HereditaryHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

RNA, AntisenseAntisense Elements (Genetics)Nucleic Acids, Nucleotides, and NucleosidesRNANucleic AcidsRNA, Small UntranslatedRNA, Untranslated

Study Officials

  • Rabia Ozden, MD

    Quark Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 4, 2010

First Posted

February 8, 2010

Study Start

February 1, 2010

Primary Completion

April 1, 2013

Study Completion

April 1, 2013

Last Updated

May 13, 2013

Record last verified: 2013-05

Locations