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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

2014-08-27 03:17:57 | BioPortfolio

Summary

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) due to RPE65 or LRAT mutations.

Study Design

Allocation: Non-Randomized, Control: Uncontrolled, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Visual Impairment Due to Leber Congenital Amaurosis (LCA)

Intervention

QLT091001

Location

Montreal Children's Hospital, McGill University Health Centre
Montreal
Quebec
Canada

Status

Recruiting

Source

QLT Inc

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:17:57-0400

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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 ...

Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT

To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT ge...

Study to Evaluate QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to the c.2991+1665A>G Mutation (p.Cys998X) in the CEP290 Gene

The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.

Treatment of RP and LCA by Primary RPE Transplantation

Early Phase I Study of the Safety and Preliminary Efficacy of Human primary Retinal Pigment Epithelial (HuRPE) Cells Subretinal Transplantation in Retinitis Pigmentosa (RP) and Leber Conge...

PubMed Articles [6148 Associated PubMed Articles listed on BioPortfolio]

A Cross-Sectional and Longitudinal Study of Retinal Sensitivity in RPE65-Associated Leber Congenital Amaurosis.

RPE65-associated Leber congenital amaurosis (RPE65-LCA) is an early-onset severe retinal dystrophy associated with progressive visual field loss. Phase I/II and III gene therapy trials have identified...

Parapapillary Optic Nerve Head Drusen in Leber Congenital Amaurosis.

A 20-year-old male who was known to have Leber congenital amaurosis was assessed. Best-corrected visual acuity was counting fingers in both eyes (OU). Anterior segment OU revealed poorly reacting pupi...

Visual impairment evaluation in 119 children with congenital Zika syndrome.

To assess visual impairment in a large sample of infants with congenital Zika syndrome (CZS) and to compare with a control group using the same assessment protocol.

Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.

Ocular gene therapy with recombinant adeno-associated virus (AAV) has shown vector-mediated gene augmentation to be safe and efficacious in the retina in one set of diseases (retinitis pigmentosa and ...

Deficiency of type 2 iodothyronine deiodinase reduces necroptosis activity and oxidative stress responses in retinas of Leber congenital amaurosis model mice.

Thyroid hormone (TH) signaling has been shown to regulate cone photoreceptor viability. Suppression of TH signaling with antithyroid drug treatment or by targeting iodothyronine deiodinases and TH rec...

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).

An ocular disease, occurring in many forms, having as its primary characteristics an unstable or a sustained increase in the intraocular pressure which the eye cannot withstand without damage to its structure or impairment of its function. The consequences of the increased pressure may be manifested in a variety of symptoms, depending upon type and severity, such as excavation of the optic disk, hardness of the eyeball, corneal anesthesia, reduced visual acuity, seeing of colored halos around lights, disturbed dark adaptation, visual field defects, and headaches. (Dictionary of Visual Science, 4th ed)

Vascular diseases characterized by thickening and hardening of the walls of ARTERIES inside the SKULL. There are three subtypes: (1) atherosclerosis with fatty deposits in the ARTERIAL INTIMA; (2) Monckeberg's sclerosis with calcium deposits in the media and (3) arteriolosclerosis involving the small caliber arteries. Clinical signs include HEADACHE; CONFUSION; transient blindness (AMAUROSIS FUGAX); speech impairment; and HEMIPARESIS.

Impairment of the vestibular function of both inner ears which can cause difficulties with balance, gait, VERTIGO, and visual blurring.

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