# An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

> **NCT04855045** · PHASE2,PHASE3 · UNKNOWN · sponsor: **ProQR Therapeutics** · enrollment: 15 (estimated)

## Conditions studied

- Leber Congenital Amaurosis 10
- Blindness
- Leber Congenital Amaurosis
- Vision Disorders
- Sensation Disorders
- Neurologic Manifestations
- Eye Diseases
- Eye Disorders Congenital
- Retinal Disease
- Retinal Degeneration
- Retinal Dystrophies

## Interventions

- **DRUG:** sepofarsen

## Key facts

- **NCT ID:** NCT04855045
- **Lead sponsor:** ProQR Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2,PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** UNKNOWN
- **Start date:** 2021-03-23
- **Primary completion:** 2023-12
- **Final completion:** 2023-12
- **Target enrollment:** 15 (ESTIMATED)
- **Last updated:** 2022-03-25


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04855045

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04855045, "An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.". Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/clinical/NCT04855045. Licensed CC0.

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