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Active clinical trials for "Choroideremia"

Results 21-26 of 26

The Dark-Adapted Retinal Function Response in Choroideremia (DARC) Study

Choroideremia

Choroideremia (CHM) is an inherited retinal disorder that causes progressive vision loss, ultimately leading to complete blindness. The first symptom is generally night blindness, although, to date, little is known about the extent, type, pattern, and progression of dark-adapted visual function measures in CHM patients. We hypothesize that one of the key events causing night blindness in CHM is deficiency in the chromophore of the rod visual pigment, rhodopsin. We propose that this deficiency is at least in part due to inadequate delivery of vitamin A (all-trans-retinol) to the photoreceptors (PRs) from the ailing retinal pigment epithelium (RPE), characteristic of CHM. We hypothesize that increased availability of vitamin A would potentiate its entry into the RPE-mediated visual cycle, ultimately enabling delivery to the PRs. This would in turn allow rods to perform better by partially overcoming the RPE damage and the impaired chromophore recycling that we postulate exists in CHM. The goals of this proposal are: (1) to test the hypothesis that oral vitamin A supplementation can improve night time and peripheral vision in CHM patients, and (2) to provide detailed characterization of dark-adapted visual function outcome measures to guide interventional CHM trials.

Withdrawn2 enrollment criteria

IRIS PILOT - Extended Pilot Study With a Retinal Implant System

Retinitis PigmentosaCone-Rod Dystrophy1 more

Investigate whether blind subjects that fulfil the patient criteria provided with a Retinal Implant are able to differentiate between simple patterns like horizontal bar, vertical bar and cross.

Unknown status25 enrollment criteria

Natural Disease Progression in Participants With Choroideremia

Choroideremia

The primary objective of this study is to quantify disease progression measured by best corrected visual acuity (BCVA) in the real-world setting among Choroideremia (CHM) participants. The secondary objectives are to describe demographic and baseline clinical characteristics among CHM participants and to match CHM participants in the Intelligent Research in Sight (IRIS®) Registry to Biogen's investigator sponsored trial (IST) study population using propensity score matching.

Completed14 enrollment criteria

A Study to Assess Choroideremia (CHM) Health Outcomes

Choroideremia

The primary objectives of this study are to describe disease progression and severity by age in participants with CHM, to assess health-related quality-of-life, resource utilization and work productivity, and to assess quality-of-life, work productivity, and impact on daily activities in caregivers of participants with CHM at different stages of disease progression.

Completed14 enrollment criteria

"Natural History" Study of Choroideremia

Choroideremia

The purpose of this study is to understand the rate of progression of all stages of choroideremia using a variety of assessments performed in the clinic including visual field measures, specialized photography of the eye and participant-reported visual problems

Terminated6 enrollment criteria

Natural History of the Progression of Choroideremia Study

Choroideremia

The objective of this natural history study is to gain a better understanding of the progression of choroideremia (CHM) and add to the knowledge base for this rare disease.

Completed11 enrollment criteria

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