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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-518378-14-00 | EU Trial (CTIS) Number |
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| Name | Class |
|---|---|
| Sepul Bio | INDUSTRY |
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The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290.
This is a double-masked, randomized, placebo-controlled, paired-eye study in which one eye of each subject will serve as a control.
At the start of the study the two eyes of each subject will be randomized such that one eye receives sepofarsen and the other eye receives placebo for the first year. In the second year, for all subjects, the eye that was randomized to receive sepofarsen will continue to receive sepofarsen. For the eye that was randomized to placebo in the first year, treatment in the second year will be allocated, as follows: 50% of the eyes will continue to receive placebo, and 50% of the eyes will receive sepofarsen.
Sepofarsen and placebo will be administered via intravitreal injection every 6 months.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sepofarsen - Treatment Eye - up to Month 12 | Active Comparator | Subjects to receive Sepofarsen in one eye (160µg first then 40µg) and Placebo in the fellow eye at baseline and at month 6. |
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| Placebo - Fellow Eye - up to Month 12 | Placebo Comparator | Subjects to receive Sepofarsen in one eye (160µg first then 40µg) and Placebo in the fellow eye at baseline and at month 6. |
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| Continued - Treatment Eye - up to Month 24 | Active Comparator | Subjects to receive Sepofarsen (40µg) in one eye and Placebo in the fellow eye at Month 12 and Month 18. |
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| Continued - Fellow Eye - up to Month 24 | Placebo Comparator | Subjects to receive Sepofarsen (40µg) in one eye and Placebo in the fellow eye at Month 12 and Month 18. |
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| Mixed - Treatment Eye - up to Month 24 | Active Comparator | Subjects to receive Sepofarsen (40µg) in one eye and Sepofarsen in the fellow eye (160µg first then 40µg) at Month 12 and at Month 18. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| sepofarsen | Drug | RNA antisense oligonucleotide for intravitreal injection |
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| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in Best-Corrected Visual Acuity (BCVA) | Change from baseline in BCVA based on the Freiburg Acuity and Contrast Test (FrACT) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) | 12 Months |
| Measure | Description | Time Frame |
|---|---|---|
| Eye-specific Patient Global Impression of Change (PGI-C) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) | PGI-C (PATIENT GLOBAL IMPRESSION OF CHANGE ) assesses a patient's perception of overall change in their vision in the respective eye over time. The minimum score (best outcome) is 1 ("Very Much Better") and the maximum score (worst outcome) is 7 ("Very Much Worse"). Higher scores indicate a worse health outcome (greater worsening). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Sepul Bio Patient Advocacy Director | Contact | +31 617060791 | contact@sepulbio.com | |
| Sepul Bio Chief Medical Officer | Contact |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Wayne and Gladys Valley Center for Vision | Recruiting | San Francisco | California | 94158 | United States | |
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Paired-eye design.
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| Mixed - Fellow Eye - Month 12 to Month 24 | Active Comparator | Subjects to receive Sepofarsen (40µg) in one eye and Sepofarsen in the fellow eye (160µg first then 40µg) at Month 12 and at Month 18. |
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| Mixed - Fellow Eye - up to Month 12 | Placebo Comparator | Subjects to receive Sepofarsen (40µg) in one eye and Sepofarsen in the fellow eye (160µg first then 40µg) at Month 12 and at Month 18 Note: up to Month 12 these subjects receive placebo in the Fellow Eye, as all subjects do. |
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| Placebo IVT | Other | Placebo with identical appearance to sepofarsen |
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| Month 12 |
| Change from baseline in Low Luminance Visual Acuity (LLVA) based on FrACT between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) | Month 12 |
| Change from baseline in retinal sensitivity as measured by dark-adapted Full-Field Stimulus Test (FST) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) | Month 12 |
| Change from baseline in Contrast Sensitivity (CS) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) based on qCSF | Month 12 |
| Change from baseline in retinal sensitivity as measured by light-adapted Full-Field Stimulus Test (FST) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) | Month 12 |
| Response in BCVA (FrACT), defined as an improvement of at least 0.2 logMAR from baseline at 12 months and compared between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs), evaluated by the percentage of eyes that achieve this improvement. | Month 12 |
| Response in BCVA (FrACT), defined as an improvement of at least 0.3 logMAR from baseline at 12 months and compared between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs), evaluated by the percentage of eyes that achieve this improvement. | Month 12 |
| Response in Low Luminance Visual Acuity (LLVA) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement. | Month 12 |
| Response in Full-Field Stimulus Test (FST) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement. | Month 12 |
| Response in Patient Global Impression of Change (PGI-C) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement. | Month 12 |
| Response in Contrast Sensitivity (CS) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement. | Month 12 |
| Response defined as a clinically relevant improvement according to a multimodal assessment in TEs compared to the corresponding response in PCEs at 12 months | The multimodal assessment is based on a combination of visual acuity, retinal sensitivity, CS, functional vision, and/or PRO | Month 12 |
| Change from baseline in the Michigan Retinal Degeneration Questionnaire (MRDQ) score for subjects ≥ 13 years of age | Month 12 |
| Change from baseline in the Michigan Vision-Related Anxiety Questionnaire (MVAQ) score for subjects ≥ 13 years of age | Month 12 |
| Change from baseline in the Pediatric Eye Questionnaire (PedEyeQ) score for subjects < 13 years of age | Month 12 |
| Change from baseline in eye-specific Patient Global Impression of Severity (PGI-S) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) | PGI-S (PATIENT GLOBAL IMPRESSION OF SEVERITY ) assesses a patient's perception of severity of their eye condition in the respective eye over the past week. The minimum score (best outcome) is 1 ("None") and the maximum score (worst outcome) is 5 ("Very Severe"). Higher scores indicate a worse health outcome (greater severity). | Month 12 |
| Change from baseline in Best-Corrected Visual Acuity (BCVA) based on the ETDRS and/or the BRVT between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) | Month 12 |
| Change from baseline in Low Luminance Visual Acuity (LLVA) based on the ETDRS between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) | Month 12 |
| University of Miami - Bascom Palmer Eye Institute |
| Recruiting |
| Miami |
| Florida |
| 33156 |
| United States |
| University of Iowa | Recruiting | Iowa City | Iowa | 52242 | United States |
| University of Minnesota Medical School | Recruiting | Minneapolis | Minnesota | 55455 | United States |
| University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| Universitair Ziekenhuis Gent (UZ) | Recruiting | Ghent | 9000 | Belgium |
| INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte | Recruiting | Belo Horizonte | Minas Gerais | 30150270 | Brazil |
| Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP) | Recruiting | São Paulo | São Paulo | 04023-062 | Brazil |
| University of Alberta | Recruiting | Edmonton | Alberta | T6G 2C8 | Canada |
| The Hospital for Sick Children - SickKids | Recruiting | Toronto | Ontario | M5G 2L3 | Canada |
| Centre de maladies rares CHNO des Quinze Vingt | Recruiting | Paris | 75012 | France |
| Justus-Liebig Universität - Department of Ophthalmology | Recruiting | Giessen | 35392 | Germany |
| Klinikum der Ludwig-Maximilian Universität München | Recruiting | München | 81377 | Germany |
| University of Tuebingen - Inst. for Ophthalmic Research | Recruiting | Tübingen | 72076 | Germany |
| Radboud Universitair Medisch Centrum | Recruiting | Nijmegen | 6525 GA | Netherlands |
| Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital) | Recruiting | Barcelona | 08950 | Spain |
| Moorfields Eye Hospital NHS Foundation Trust | Recruiting | London | EC1V 2PD | United Kingdom |
| ID | Term |
|---|---|
| C565720 | Leber Congenital Amaurosis 10 |
| D001766 | Blindness |
| D057130 | Leber Congenital Amaurosis |
| D012678 | Sensation Disorders |
| D014786 | Vision Disorders |
| D009461 | Neurologic Manifestations |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| 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 |
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