Active clinical trials for "Atrophy"

This study will determine whether a drug called sirolimus is safe to give to people with geographic atrophy (GA) and if it can help preserve vision in patients. GA is an advanced form of dry age-related macular degeneration (AMD). AMD affects the macula, the central part of the retina at the back of the eye needed for sharp, clear vision. There are two types of AMD, wet and dry. In dry AMD, cells in the macula die. GA may be partially caused by inflammation. Sirolimus helps prevent inflammation and therefore may help people with GA. Researchers want to see whether sirolimus can help prevent vision loss in people with GA. People at least 56 years of age who have GA related to AMD in both eyes may be eligible for this study. This study requires at least 8 visits to the National Eye Institute over 1 year. Study visits will be every 2 months for 1 year. Participants will undergo the following procedures: Participants will be screened with a medical history and physical exam. They will also have blood and urine tests, and eye exams. One eye will be selected as the study eye to receive the sirolimus injections. Participants will have a sirolimus injection into the study eye at the first visit and every 2 months thereafter unless contraindicated. There will be a follow-up eye exam 1 month after the first injection.

To test the clinical effect of rasagiline on subjects with MSA of the parkinsonian subtype.

This study will evaluate the safety and effectiveness of gene therapy for patients with gyrate atrophy, an inherited condition in which areas of the retina-the inner lining of the wall of the eye-become thin. Over several decades, this degeneration of the retina causes tunnel vision, night blindness, and other vision problems. Gyrate atrophy is caused by a defect in the gene responsible for producing an enzyme, ornithine aminotransferase (OAT), that breaks down an amino acid called ornithine. As a result, excessive ornithine buildup causes the retinal thinning. Currently, this condition can only be treated with amino acid tablets and a very low-protein diet with limited fruits and vegetables and more than 2,000 calories a day from carbohydrates and fats. Some patients cannot maintain this diet, and they need another treatment. One possible alternative is to replace the defective gene with one that functions normally. Patients who have been followed in NEI's Ocular Genetics service may be eligible to participate in this study. Study patients will undergo the following gene therapy procedure: Skin biopsy-A small piece of skin is surgically removed from the patient's thigh. Gene transfer-Skin cells called keratinocytes are taken from the biopsied tissue and grown in the laboratory. The normal gene that produces OAT is inserted into the cells, causing them to produce more of the enzyme. Skin graft-Under local anesthesia, a patch of skin about 2 1/4 inches x 2 1/4 inches is surgically removed from the upper thigh and some of the cells with increased OAT are grafted back onto this area. Patients will be followed at 1 week and 2 weeks after the procedure, then monthly for 6 months, again at 9 months and 1 year. Follow-up will continue at 1-year intervals in patients in whom the treatment is successful. During each follow-up visit patients will have 2 to 3 tablespoons of blood drawn for tests. A small biopsy (about 1/4 inch) of transplanted cells will also be done at 1 week, 1 month, 3 months, 6 months, 1 year, and each year or so thereafter. These tests will evaluate whether the treated skin cells are producing the deficient OAT enzyme and, if so, how much and for how long. They will also indicate whether the enzyme produced is sufficient to lower ornithine blood levels. Patients will also undergo various eye examinations before grafting and at scheduled follow-up visits. These tests may include electrophysiologic (ERG) testing, fundus photographs, scanning laser ophthalmoscope, visual field test, fluorescein angiogram, visual acuity, and manifest reaction.

The purpose of this study is to determine the safety and tolerability of multiple doses of RN6G in subjects with advanced dry, age-related macular degeneration including geographic atrophy.

In this single-center trial, we will evaluate the effects of NaPB on presymptomatic Spinal Muscular Atrophy (SMA) type I (cohort 1)and presymptomatic SMA type II (cohort 2) infants. A variety of outcome measures will be performed at each study visit to follow the course of the disease. Total duration of the study for type I infants will be 18 months, for type II infants, 24 months.

The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III.

The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the clinical efficacy of HU treatment in children with Type I SMA.

The purpose of this study is to determine the safety and efficacy of ospemifene in postmenopausal women experiencing moderate to severe vaginal dryness and vaginal pain associated with sexual activity.

The effect of whole body vibration (WBV) on bone strength and fall frequency in older adults is still vague. Although there is some evidence that WBV may impact Bone Mineral Density in very frail elderly, there is a lack of data concerning the effect of WBV in community-living elderly woman. We hypothesize that WBV significantly affects bone parameters as assessed by DXA, QCT, US and bone markers with parallel positive changes of fall related risk factors (power, strength, balance, and reaction time). We further hypothesize that the effects of WBV were superior to conventional resistance exercise.

Othera Pharmaceuticals' Othera (OT)-551 antioxidant eye drop has the potential for chronic treatment of the dry form of age-related macular degeneration. This pilot study of up to 10 eye drop tolerant participants with bilateral geographic atrophy is designed to characterize the effect of 0.45% concentration of OT-551 eye drops given 3 times a day on the progression of geographic atrophy area over a two-year period. Participants will have one eye randomized to receive the eye drop and the fellow eye will be observed only.