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A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
NCT03913143 · View on ClinicalTrials.gov ↗
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
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03913143
The ClinicalTrials.gov registry entry for NCT03913143 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 36 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is ProQR Therapeutics, which has 2 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03913143 reports 14 study locations spanning 6 distinct geographic areas — top geographies include Other, Iowa, Minas Gerais. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03913143?
The interventions under investigation include: sepofarsen (DRUG), Sham (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03913143?
This trial is sponsored by ProQR Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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