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Leber Congenital Amaurosis 10 clinical trials

View clinical trials related to Leber Congenital Amaurosis 10.

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NCT ID: NCT04855045 Recruiting - Eye Diseases Clinical Trials

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.

BRIGHTEN
Start date: March 23, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (<8 years of age) with LCA10 due to the c.2991+1655A>G mutation over 24 months of treatment.

NCT ID: NCT03913143 Active, not recruiting - Eye Diseases Clinical Trials

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

ILLUMINATE
Start date: April 4, 2019
Phase: Phase 2/Phase 3
Study type: Interventional

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

NCT ID: NCT03913130 Terminated - Eye Diseases Clinical Trials

Extension Study to Study PQ-110-001 (NCT03140969)

INSIGHT
Start date: May 13, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.

NCT ID: NCT03872479 Active, not recruiting - Retinal Disease Clinical Trials

Single Ascending Dose Study in Participants With LCA10

Start date: September 26, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in intron 26 of the CEP290 gene ("LCA10-IVS26").

NCT ID: NCT03396042 Completed - Eye Diseases Clinical Trials

Natural History Study of CEP290-Related Retinal Degeneration

Start date: December 17, 2017
Phase:
Study type: Observational

A prospective natural history study with systematic assessments and uniform follow-up to provide a high-quality dataset for assisting in the design of future clinical treatment trials involving patients with CEP290-related retinal degeneration caused by the common intron 26 mutation.