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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-003500-40 | EudraCT Number |
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Terminated [Study prematurely terminated due to sponsor decision for reasons unrelated to safety]
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| Name | Class |
|---|---|
| Sepul Bio | INDUSTRY |
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Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject.
Subjects will be given the opportunity to enroll into this extension study for continued dosing if available data support current and/or future benefits for the subject. The Investigator, in consultation and agreement with the Medical Monitor, will decide on enrollment of each individual subject, as well as on dosing of the first treated eye and treatment initiation of the contralateral eye. Continued subject treatment in this study is desirable, but cannot be guaranteed, since it will depend on the risks and benefit of further treatment on a case-by-case basis, as discussed and agreed upon with the Medical Monitor.
The contralateral eye and the first treated eye will be injected 3 months apart. The injection interval of 3 months between both eyes will limit burden for the subjects, with a 3 month-visit frequency during the course of the study. This between-eye interval could be adapted if safety data are supportive, and for logistic reasons, and in agreement with the Medical Monitor.
The same safety monitoring protocol and efficacy assessments will apply to both eyes.
QR-110 will be administered via intravitreal (IVT) injection.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Drug QR-110 | Experimental | First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| QR-110 | Drug | First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration |
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of Ocular AEs | Frequency of ocular adverse events (AEs) | 24 months |
| Frequency of Non-ocular AEs | Frequency of non-ocular AEs | 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in BCVA in First Treated Eye | Change in Best Corrected Visual Acuity (BCVA) in First Treated Eye | 24 months |
| Change in Mobility Course Score | Change in Mobility course score |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Sepul Bio Chief Medical Officer | Sepul Bio | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Iowa | Iowa City | Iowa | 52242 | United States | ||
| Scheie Eye Institute, University of Pennsylvania |
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| ID | Title | Description |
|---|---|---|
| FG000 | Drug QR-110 | First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 17, 2022 | Nov 10, 2022 |
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| 24 months |
| Change in Photoreceptor Outer Segment Layer Thickness | Change in photoreceptor outer segment layer thickness by Optical Coherence Tomography (OCT) | 24 months |
| Change in OCI | Change in Oculomotor Instability (OCI) | 24 months |
| Change in FST Blue | Change in Full-Field Stimulus Testing (FST) - blue stimuli | 24 months |
| Change in FST Red | Change in Full-Field Stimulus Testing (FST) - red stimuli | 24 months |
| Change in VFQ-25 | Change in Visual Function Questionnaire-25 (VFQ-25) score (adult subjects) | 24 months |
| Change in CVAQ | Change in Cardiff Visual Ability Questionnaire for Children (CVAQC) score (pediatric subjects) | 24 months |
| Change in PLR | Change in Pupillary Light Reflex (PLR) (latency and amplitude) | 24 months |
| Change in NIRAF | Change in Near Infrared AutoFluorescence (NIRAF) | 24 months |
| Change in BCVA in Treated Contralateral Eye | Change in Best Corrected Visual Acuity (BCVA) in Treated Contralateral Eye | 24 months |
| Change in BCVA in Non-Treated Contralateral Eye | Change in Best Corrected Visual Acuity (BCVA) in Non-Treated Contralateral Eye | 24 months |
| Philadelphia |
| Pennsylvania |
| 19104 |
| United States |
| Ghent University Hospital and Ghent University | Ghent | B-9000 | Belgium |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Drug QR-110 | First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Mean | Standard Deviation | Years |
| ||||||||||||||||||||||
| Age, Customized | Median | Full Range | Years |
| ||||||||||||||||||||||
| Age, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Weight (Mean) | Mean | Standard Deviation | kg |
| ||||||||||||||||||||||
| Weight (Median) | Median | Full Range | kg |
| ||||||||||||||||||||||
| Height (Mean) | Mean | Standard Deviation | cm |
| ||||||||||||||||||||||
| Height (Median) | Median | Full Range | cm |
| ||||||||||||||||||||||
| BMI (Mean) | Mean | Standard Deviation | kg/m2 |
| ||||||||||||||||||||||
| BMI (Median) | Median | Full Range | kg/m2 |
| ||||||||||||||||||||||
| Genotype | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Frequency of Ocular AEs | Frequency of ocular adverse events (AEs) | Posted | Count of Participants | Participants | 24 months |
|
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| |||||||||||||||||||||||||||
| Primary | Frequency of Non-ocular AEs | Frequency of non-ocular AEs | Posted | Count of Participants | Participants | 24 months |
|
| ||||||||||||||||||||||||||||
| Secondary | Change in BCVA in First Treated Eye | Change in Best Corrected Visual Acuity (BCVA) in First Treated Eye | Posted | Mean | Standard Deviation | logMAR | 24 months |
|
| |||||||||||||||||||||||||||
| Secondary | Change in Mobility Course Score | Change in Mobility course score | Not Posted | 24 months | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change in Photoreceptor Outer Segment Layer Thickness | Change in photoreceptor outer segment layer thickness by Optical Coherence Tomography (OCT) | Not Posted | 24 months | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change in OCI | Change in Oculomotor Instability (OCI) | Not Posted | 24 months | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change in FST Blue | Change in Full-Field Stimulus Testing (FST) - blue stimuli | Not Posted | 24 months | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change in FST Red | Change in Full-Field Stimulus Testing (FST) - red stimuli | Not Posted | 24 months | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change in VFQ-25 | Change in Visual Function Questionnaire-25 (VFQ-25) score (adult subjects) | Not Posted | 24 months | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change in CVAQ | Change in Cardiff Visual Ability Questionnaire for Children (CVAQC) score (pediatric subjects) | Not Posted | 24 months | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change in PLR | Change in Pupillary Light Reflex (PLR) (latency and amplitude) | Not Posted | 24 months | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change in NIRAF | Change in Near Infrared AutoFluorescence (NIRAF) | Not Posted | 24 months | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change in BCVA in Treated Contralateral Eye | Change in Best Corrected Visual Acuity (BCVA) in Treated Contralateral Eye | Posted | Mean | Standard Deviation | logMAR | 24 months |
|
| |||||||||||||||||||||||||||
| Secondary | Change in BCVA in Non-Treated Contralateral Eye | Change in Best Corrected Visual Acuity (BCVA) in Non-Treated Contralateral Eye | Posted | Mean | Standard Deviation | logMAR | 24 months |
|
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Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Drug QR-110 | First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration | 0 | 9 | 1 | 9 | 8 | 9 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lumbar vertebral fracture | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cataract | Eye disorders | Systematic Assessment |
| ||
| Cataract cortical | Eye disorders | Systematic Assessment |
| ||
| Cataract subcapsular | Eye disorders | Systematic Assessment |
| ||
| Conjunctival haemorrhage | Eye disorders | Systematic Assessment |
| ||
| Cystoid macular oedema | Eye disorders | Systematic Assessment |
| ||
| Dry eye | Eye disorders | Systematic Assessment |
| ||
| Eye pain | Eye disorders | Systematic Assessment |
| ||
| Eye pruritus | Eye disorders | Systematic Assessment |
| ||
| Lacrimation increased | Eye disorders | Systematic Assessment |
| ||
| Lenticular opacities | Eye disorders | Systematic Assessment |
| ||
| Posterior capsule opacification | Eye disorders | Systematic Assessment |
| ||
| Retinal cyst | Eye disorders | Systematic Assessment |
| ||
| Retinal degeneration | Eye disorders | Systematic Assessment |
| ||
| Visual acuity reduced | Eye disorders | Systematic Assessment |
| ||
| Infections and infestations | Eye disorders | Systematic Assessment |
| ||
| Blebitis | Infections and infestations | Systematic Assessment |
| ||
| Posterior lens capsulotomy | Surgical and medical procedures | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Seasonal allergy | Immune system disorders | Systematic Assessment |
| ||
| Cystitis | Infections and infestations | Systematic Assessment |
| ||
| Gastroenteritis | Infections and infestations | Systematic Assessment |
| ||
| Influenza | Infections and infestations | Systematic Assessment |
| ||
| Paronychia | Infections and infestations | Systematic Assessment |
| ||
| Contusion | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Coronavirus test positive | Investigations | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Petit mal epilepsy | Nervous system disorders | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Dermabrasion | Surgical and medical procedures | Systematic Assessment |
| ||
| Wisdom teeth removal | Surgical and medical procedures | Systematic Assessment |
| ||
| Diplopia | Eye disorders | Systematic Assessment |
|
Institution shall be free to publish, present, or use any Data and results arising out of its performance of the protocol. At least 30 days prior to submission for publication, institution shall submit to Sponsor for review and comment any proposed oral or written publication. Institution will consider any such comments in good faith but is under no obligation to incorporate Sponsor's suggestions. The review period for abstracts or poster presentations shall be 30 days.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Zuhal Butuner - Chief Medical Officer | Sepul Bio | (905) 599 7887 | contact@sepulbio.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 4, 2022 | Nov 10, 2022 | SAP_001.pdf |
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| ID | Term |
|---|---|
| C565720 | Leber Congenital Amaurosis 10 |
| D001766 | Blindness |
| D057130 | Leber Congenital Amaurosis |
| D014786 | Vision Disorders |
| D012678 | Sensation Disorders |
| D009461 | Neurologic Manifestations |
| D005128 | Eye Diseases |
| D015785 | Eye Diseases, Hereditary |
| D005124 | Eye Abnormalities |
| D012164 | Retinal Diseases |
| C567003 | Meckel Syndrome, Type 4 |
| D029242 | Optic Atrophy, Hereditary, Leber |
| ID | Term |
|---|---|
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000013 | Congenital Abnormalities |
| D015418 | Optic Atrophies, Hereditary |
| D009896 | Optic Atrophy |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
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| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
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