Optic AtrophyPhase 1NCT01064505
Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients
A Phase 1 study testing QPI-1007 at various doses in people with Optic Atrophy and Non-arteritic Anterior Ischemic Optic Neuropathy, run by Quark Pharmaceuticals at 28 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Optic Atrophy, Non-arteritic Anterior Ischemic Optic Neuropathy
- What is being tested?
- QPI-1007 at various doses
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 28 sitesin 2 countries
- Study dates
- Feb 2010to Apr 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Trial locations and contacts
28 sites in 2 countries.
28 sites in 2 countries
United States22 sites
Western Carolina Retinal Associates
Asheville, North Carolina, 28803
Rocky Mountain Multiple Sclerosis Center at Anschutz Medical Campus, University of Colorado
Aurora, Colorado, 80045
Retina-Vitreous Associates Medical Group
Beverly Hills, California, 90211
Charlotte Eye, Ear, Nose and Throat Associates, PA
Charlotte, North Carolina, 28210
University of Illinois at Chicago, Eye and Ear Infirmary
Chicago, Illinois, 60612
Cole Eye Institute, Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Mason Eye Institute, University of Missouri
Columbia, Missouri, 65212
Duke Eye Center, Duke University Medical Center
Durham, North Carolina, 27705
Alkek Eye Center Baylor College of Medicine
Houston, Texas, 77030
Kansas University Medical Center
Kansas City, Kansas, 66208
University of Kentucky, Department of Ophthalmology
Lexington, Kentucky, 40536
Jules Stein Eye Institute, University of California, Los Angeles
Los Angeles, California, 90049
Bascom Palmer at the University of Miami
Miami, Florida, 33136
University of Minnesota, Department of Ophthalmology
Minneapolis, Minnesota, 55455
New York Eye and Ear Infirmary
New York, New York, 10003
University of Pennsylvania, Department of Ophthalmology
Philadelphia, Pennsylvania, 19104
Retinal Consultants of Arizona
Phoenix, Arizona, 85014
Allegheny Ophthalmic and Orbital Associates
Pittsburgh, Pennsylvania, 15212
Virginia Commonwealth University
Richmond, Virginia, 23298
Flaum Eye Institute, University of Rochester Medical Center
Rochester, New York, 14642
John A. Moran Eye Center at the University of Utah
Salt Lake City, Utah, 84132
Sarasota Retina Institute
Sarasota, Florida, 34239
Israel6 sites
Soroka University Medical Center, Dept of Ophthalmology
Beersheba, 84101
Bnai Zion Medical Center
Haifa, 33394
Rabin Medical Center, Belinson Campus, Dept of Ophthalmology
Petah Tikva, 49100
Kaplan Medical Center, Department of Ophthalmology
Rehovot, 76100
The Tel-Aviv Sourasky Medical Center, Dept of Ophthalmology
Tel Aviv, 64239
The Chaim Sheba Medical Center, Dept of Ophthalmology
Tel HaShomer, 52621
About this study
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)
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.
Read the 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.
- Being tested
- QPI-1007 at various doses
- 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
- Medical subject headings
- Optic Atrophy, Optic Neuropathy, Ischemic, Retinal Degeneration, Optic Neuritis, Optic Atrophy, Hereditary, Leber
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QPI-1007 at various dosesDrug
Also called QPI-1007, siRNA, small interfering RNA, short interfering RNA
Single Intravitreal Injection
| Study group | Role | What they receive |
|---|---|---|
| QPI-1007 | Experimental | Not described Receives: Drug: QPI-1007 at various doses |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the safety, tolerability and the dose-limiting toxicities (DLTs) of QPI 1007 when administered as a single intravitreal (IVT) injection.
Measured: 12 Months Post-injection
To assess the pharmacokinetics (PK) of QPI-1007 when administered as a single IVT injection.
Measured: 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.
Measured: 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.
Measured: 12 Months Post-Injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2010 (estimated)
- Study startFeb 2010
- Primary completionApr 2013
- Study completionApr 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Optic AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Quark Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rabia Ozden, MD, Study director, Quark Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01064505
- Sponsor's protocol ID
- QRK.007
- Status last verified
- May 2013
- First submitted
- 4 Feb 2010
- Last updated
- 13 May 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT01064505
- Acronym / sponsor ID
- QRK.007
- 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
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Quark PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Feb 2010
- Primary completion
- Apr 2013 (actual)
- Study completion
- Apr 2013 (actual)
- First posted
- 8 Feb 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
- 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
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| QPI-1007 | Experimental | Drug: QPI-1007 at various doses |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| QPI-1007 at various dosesAlso: QPI-1007, siRNA, small interfering RNA, short interfering RNASingle Intravitreal Injection | Drug | QPI-1007 |
Primary outcome
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
To assess the pharmacokinetics (PK) of QPI-1007 when administered as a single IVT injection.
Time frame: 12 Months Post-Injection
2 secondary outcomes
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2013.
Related trials for Optic Atrophy
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
Active, not recruitingPhase 1/2
Phase 1/2 Study of VOY-101 in Patients With Advanced Non-Neovascular Age-Related Macular Degeneration
Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 1/2
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Dry Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 3
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
Age-related Macular Degeneration (AMD), Geographic Atrophy (GA)
RecruitingPhase 1/2
Tributyrin for Progressive Supranuclear Palsy (PSP): Virtual Trial
Parkinson Disease, Multiple System Atrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rabia Ozden, MD study director
Quark Pharmaceuticals
28 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing QPI-1007 at various doses in people with Optic Atrophy and Non-arteritic Anterior Ischemic Optic Neuropathy, run by Quark Pharmaceuticals at 28 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 May 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2013.