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Eye Disorders Congenital clinical trials

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NCT ID: NCT05176717 Terminated - Clinical trials for Retinitis Pigmentosa

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

Start date: December 15, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with early to moderate vision loss.

NCT ID: NCT05158296 Active, not recruiting - Clinical trials for Retinitis Pigmentosa

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

Start date: December 8, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.

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: NCT03780257 Completed - Clinical trials for Retinitis Pigmentosa

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

Stellar
Start date: March 6, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to evaluate the safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.

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.