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Universal Rare Gene Study: A Registry and Natural History
Study of Retinal Dystrophies Associated with Rare Disease-Causing Genetic Variants

IRDs are genetically diverse (280 causative genes have been identified to date) (https://sph.uth.edu/retnet/) and have vastly different clinical manifestations, including age of onset, severity of disease, rate of progression, and structural and…

Study on two new methods for testing visual field in children with brain disease

To compare different methods for visual field testing in young and/or neurologically impaired children, to determine which test is most useful in detecting visual field defects.

NEW characterizing rate of progression in USHer syndrome (NEW-USH) study
a natural history study in patients with Usher syndrome

The study will help us to determine 1. the necessary type of (combined) examinations and 2. the sample size that is essential to evaluate (future) genetic therapy in Usher syndrome type 2 and USH2A associated nsRP.

Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability,
and Efficacy of EDIT-101 in Adult and Pediatric Participants with Leber Congenital Amaurosis
Type 10 (LCA10), with Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration
Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in
Intron 26 (IVS26) of the CEP290 Gene (*LCA10-IVS26*)

Primary-To evaluate the safety and tolerability of a single dose of EDIT-101 when administered to participants with LCA10-IVS26 mutationSecondary-To evaluate different doses of EDIT-101 for subsequent clinical evaluation-To evaluate the efficacy of…