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

2019-04-18 12:14:31 | BioPortfolio

Published on BioPortfolio: 2019-04-18T12:14:31-0400

Clinical Trials [376 Associated Clinical Trials listed on BioPortfolio]

Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the ...

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

This study will deliver AAV2-hRPE65v2 vector to twelve subjects, age three or older; subjects will receive the vector via subretinal injection during surgery. The purpose of this research ...

A Safety/Proof of Concept Study to Evaluate the Effects of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) Due to RPE65 or LRAT Mutations

The purpose of this study is to evaluate whether 7-day treatment with oral QLT091001 is safe, tolerable and can improve visual function in subjects with Leber Congenital Amaurosis (LCA) du...

Study of Subretinally Injected SAR439483 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

Primary Objective: To evaluate the safety and tolerability of ascending doses of SAR439483 administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (L...

Multi-dose Study for Efficacy, Safety, Tolerability, Exposure of QR-110 in LCA10

The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of QR-110 administered via intravitre...

PubMed Articles [1249 Associated PubMed Articles listed on BioPortfolio]

A high prevalence of biallelic RPE65 mutations in Costa Rican children with Leber congenital amaurosis and early-onset retinal dystrophy.

Leber congenital amaurosis (LCA) and early-onset retinal dystrophy (EORD), are primary causes of inherited childhood blindness. Both are autosomal recessive diseases, with mutations in more than 25 ge...

Generation of a human iPSC line from a patient with Leber congenital amaurosis caused by mutation in AIPL1.

The human induced pluripotent stem cell (hiPSC) line, derived from dermal fibroblasts from Leber congenital amaurosis patient with homozygous mutation c.265 T > C, p.Cys89Arg in aryl hydrocarbon...

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.

Leber congenital amaurosis type 10 is a severe retinal dystrophy caused by mutations in the CEP290 gene. We developed EDIT-101, a candidate genome-editing therapeutic, to remove the aberrant splice do...

Novel truncating mutation in CACNA1F in a young male patient diagnosed with optic atrophy.

Low vision in children can be accompanied by pallor of the optic disc with little or no characteristic morphologic changes of the retina. A variety of diseases can be the underlying cause, including h...

The findings of optical coherence tomography of retinal degeneration in relation to the morphological and electroretinographic features in RPE65-/- mice.

Mutations of the gene encoding RPE65 cause Leber congenital amaurosis (LCA) retinitis pigmentosa (RP). The optical coherence tomography (OCT) is increasingly utilized to noninvasively evaluate various...

Medical and Biotech [MESH] Definitions

A rare degenerative inherited eye disease that appears at birth or in the first few months of life that results in a loss of vision. Not to be confused with LEBER HEREDITARY OPTIC NEUROPATHY, the disease is thought to be caused by abnormal development of PHOTORECEPTOR CELLS in the RETINA, or by the extremely premature degeneration of retinal cells.

Hereditary conditions that feature progressive visual loss in association with optic atrophy. Relatively common forms include autosomal dominant optic atrophy (OPTIC ATROPHY, AUTOSOMAL DOMINANT) and Leber hereditary optic atrophy (OPTIC ATROPHY, HEREDITARY, LEBER).

Blood clot formation in any part of the CAROTID ARTERIES. This may produce CAROTID STENOSIS or occlusion of the vessel, leading to TRANSIENT ISCHEMIC ATTACK; CEREBRAL INFARCTION; or AMAUROSIS FUGAX.

Transient complete or partial monocular blindness due to retinal ischemia. This may be caused by emboli from the CAROTID ARTERY (usually in association with CAROTID STENOSIS) and other locations that enter the central RETINAL ARTERY. (From Adams et al., Principles of Neurology, 6th ed, p245)

Congenital structural deformities of the upper and lower extremities collectively or unspecified.

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