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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Quark Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01064505
First received: February 4, 2010
Last updated: May 9, 2013
Last verified: May 2013
  Purpose

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 Intervention Phase
Optic Atrophy
Non-arteritic Anterior Ischemic Optic Neuropathy
Drug: QPI-1007 at various doses
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: 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)

Resource links provided by NLM:


Further study details as provided by Quark Pharmaceuticals:

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. [ Time Frame: 12 Months Post-injection ] [ Designated as safety issue: No ]
  • To assess the pharmacokinetics (PK) of QPI-1007 when administered as a single IVT injection. [ Time Frame: 12 Months Post-Injection ] [ Designated as safety issue: No ]

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. [ Time Frame: 12 Months Post-Injection ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 48
Study Start Date: February 2010
Study Completion Date: April 2013
Primary Completion Date: April 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: QPI-1007 Drug: QPI-1007 at various doses
Single Intravitreal Injection
Other Names:
  • QPI-1007
  • siRNA
  • small interfering RNA
  • short interfering RNA

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

Ages Eligible for Study:   50 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01064505

  Show 28 Study Locations
Sponsors and Collaborators
Quark Pharmaceuticals
Investigators
Study Director: Rabia Ozden, MD Quark Pharmaceuticals
  More Information

No publications provided

Responsible Party: Quark Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01064505     History of Changes
Other Study ID Numbers: QRK.007
Study First Received: February 4, 2010
Last Updated: May 9, 2013
Health Authority: United States: Food and Drug Administration
Israel: Ministry of Health

Keywords provided by Quark Pharmaceuticals:
Non-arteritic ischemic optic neuropathy
Non-arteritic anterior ischemic optic neuropathy
Leber's hereditary optic neuropathy
Ocular Neuroprotection
ocular neuroprotectant
NAION
NAAION
chronic optic nerve atrophy
optic nerve atrophy
retinal degeneration
optic neuritis
end stage glaucoma

Additional relevant MeSH terms:
Optic Neuropathy, Ischemic
Ischemia
Atrophy
Optic Nerve Diseases
Optic Atrophy
Pathologic Processes
Pathological Conditions, Anatomical
Cranial Nerve Diseases
Nervous System Diseases
Eye Diseases
Vascular Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on September 18, 2014