Leber Congenital Amaurosis 10 — Clinical Trials

MONDO:00127232 active·1 recruitingLast updated: Mar 19, 2026, 11:37 PM

Trial Landscape

Leber Congenital Amaurosis 10 has an emerging clinical trial program, driven by drug treatment.

Recruiting

Active Trials

By Approach

Drug Treatment2

2 additional trials involve behavioral, dietary, diagnostic, or observational approaches.

By Phase

Phase 2

Phase 3

Sponsor Types

Industry2

Top Sponsors

Laboratoires Thea

ProQR Therapeutics

Trial Locations

United States2 trials

Belgium2 trials

Canada2 trials

France2 trials

Germany2 trials

Netherlands2 trials

United Kingdom2 trials

Brazil1 trial

Italy1 trial

Trials often recruit across multiple countries, so totals exceed the trial count.

2 Trials

Phase 3Recruiting

NCT06891443

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Sponsor:Laboratoires Thea

United StatesStarted: Jun 2025Est. completion: Oct 2028Updated: Mar 2026

Phase 2Active, Not Recruiting

NCT03913143

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Sponsor:ProQR Therapeutics

United StatesStarted: Apr 2019Est. completion: Mar 2023Updated: Mar 2022

Last updated over 18 months ago

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Data sourced from ClinicalTrials.gov via Kisho's automated pipeline. Trials refreshed every 4 hours for monitored diseases.

Last sync: March 19, 2026 at 11:37 PM