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NCT03913143 · ClinicalTrials.gov registry record · Phase 2
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
A Phase 2 study of Eye Diseases and Retinal Disease, sponsored by ProQR Therapeutics.
- Active
- Registry status
- Phase 2
- Development phase
- 36
- Enrollment target
- 14
- Study locations
NCT03913143: Active Phase 2 study of Eye Diseases and Retinal Disease, sponsored by ProQR Therapeutics.
NCT03913143 is a Phase 2 study of Eye Diseases and Retinal Disease that is active but no longer recruiting, run by ProQR Therapeutics. The registered enrollment target is 36 participants, below the 741,301-participant average among 27 other Eye Diseases trials with a reported enrollment target (100% lower). The trial reports 14 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03913143, a Phase 2 study of Eye Diseases and Retinal Disease, is active but no longer recruiting, sponsored by ProQR Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 36 participants
- Enrollment target
- 14
- Study locations
Study Summary
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment
Primary Outcome
Change in Best-corrected visual acuity (BCVA) relative to baseline after 12 months of treatment versus sham-procedure
Conditions Studied
Interventions
- DRUG sepofarsen
- OTHER Sham
Study Locations (14)
Other
- Universitair Ziekenhuis Gent (UZ) - Ghent
- Centre de maladies rares CHNO des Quinze Vingt - Paris
- Hospital Civil de Strasbourg - Strasbourg
- Justus-Liebig Universität - Department of Ophthalmology - Giessen
- University of Tuebingen - Inst. for Ophthalmic Research - Tübingen
- Eye Clinic University of Campania Luigi Vanvitelli - Naples
- Amsterdam University Medica Center - Locatie AMC - Amsterdam
- Het Oogziekenhuis Rotterdam - Rotterdam
- Moorfields Eye Hospital - NHS Foundation Trust - London
Iowa
- University of Iowa - Iowa City
Minas Gerais
- INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte - Belo Horizonte
São Paulo
- Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP) - São Paulo
Ontario
- The Hospital for Sick Children - SickKids - Toronto
Quebec
- McGill University Health Centre - Centre for Innovative Medicine - Montreal
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2019-04-04 |
| Est. Completion | 2023-03 |
| Phase | Phase 2 |
What the registry record for NCT03913143 still lists
NCT03913143 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 36 participants, a relatively small participant target, below the 741,301-participant average among 27 other Eye Diseases trials with a reported enrollment target (100% lower).
The record links to 10 conditions, with Eye Diseases appearing as the primary indexed condition, and to 2 interventions - of which sepofarsen is the first listed.
NCT03913143 lists 14 locations in 6 states (Other, Iowa, Minas Gerais).
Frequently Asked Questions
What is clinical trial NCT03913143 about?
NCT03913143 is a clinical study titled "A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)". The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutati...
What is the current status of trial NCT03913143?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 36 participants. The study started on 2019-04-04. Estimated completion is 2023-03.
What conditions does trial NCT03913143 study?
This clinical trial studies the following conditions: Eye Diseases, Retinal Disease, Eye Diseases, Hereditary, Blindness, Neurologic Manifestations.
What interventions are being tested in trial NCT03913143?
The interventions under investigation include: sepofarsen (DRUG), Sham (OTHER).
Who is sponsoring clinical trial NCT03913143?
This trial is sponsored by ProQR Therapeutics, which has 5 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03913143 being conducted?
This trial has 14 study locations across Iowa, Minas Gerais, São Paulo, Ontario, Quebec. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Where NCT03913143's enrollment target sits among peer trials
36 20th of 27 higher than 8 of 27 other Eye Diseases trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Eye Diseases trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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