Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
Spinal Muscular AtrophyThis is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
Effect of Exercise on Risk-factors of Elderly Women
AtrophyThe purpose of this study is to determine whether exercise training may impact relevant risk factors and health costs of community living women older 65 years.
Efficacy of Therapeutic Interventions for Orthostatic Hypotension in Parkinson's Disease and Multiple...
Parkinson's DiseaseMultiple System Atrophy1 morePatients with Parkinson's Disease or Multiple System Atrophy (MSA), and symptoms of orthostatic hypotension, are eligible for the study. Each patient will have three weeks of conservative therapy, three weeks of therapy with fludrocortisone, and three weeks of therapy with domperidone. Autonomic testing, a symptom questionnaire, bedside blood pressure testing, and Unified Parkinson Disease Rating Scale (UPDRS) will be performed after each intervention.
Radiofrequency and Hybrid Fractional Laser for Vaginal Rejuvenation
Vaginal AtrophyThis is a single-center, randomized, prospective study designed to evaluate the efficacy of radiofrequency and hybrid fractional laser for vaginal rejuvenation. 120 subjects will be screened with 100 undergoing a three-part treatment of the vulvovaginal area IntraGen RF unit, IntraGen RF unit placebo, DiVa HFL unit, or DiVa HFL unit placebo. These treatments will be spaced one month apart and last about 25 minutes each. Each subject will be screened, undergo testing at baseline, and will be followed conservatively with no further therapy until they reach 6 months after the initiation of the designated treatment. At that time, all subjects will undergo subjective and objective testing. Those in the treatment group will be followed to 9 and 12 months after the initiation of treatment with appropriate analysis. Those in the placebo group will be provided a three-part treatment of the vulvovaginal area with the IntraGen RF unit and DiVA HFL unit. These treatments will be spaced one month apart and last about 25 minutes each. Six months after receiving three treatments of the dual therapy these patients will undergo subjective and objective testing. The primary outcome measure is improvement in vulvovaginal symptoms measured by the validated Vulvovaginal Symptoms Questionnaire. Data obtained from each investigation will be recorded in a password-protected digital spreadsheet, REDCap database and descriptive statistics will be obtained.
Safety and Tolerability Study of RO7171009 in Participants With Geographic Atrophy (GA) Secondary...
Geographic AtrophyThis Phase 1, open-label, multicenter study will investigate the safety and tolerability of RO7171009 following single and multiple intravitreal (ITV) administrations in patients with GA secondary to AMD. The study consists of two stages: Single Dose-Escalation (SAD) and Multiple-Dose (MD) stages.
Safinamide for Multiple System Atrophy (MSA)
Multiple System AtrophyThe study is a placebo controlled study, with two parallel arms, in which participants will be randomly assigned in a 2:1 ratio to receive either active (200 mg safinamide) or placebo in a double blind manner. Study population is patients diagnosed, with possible or probable parkinsonian variant of Multiple System Atrophy who are on a stable treatment of levodopa
Serial Platelet-Rich Plasma Injections for Vocal Fold Atrophy, Scar, and/or Sulcus Vocalis
PresbylarynxAtrophy; Larynx5 moreThis study will investigate the safety and efficacy of four serial monthly vocal fold injections of platelet-rich plasma to treat dysphonia secondary to vocal fold atrophy, scar, and/or sulcus vocalis with glottal insufficiency
Use of AAM for Correction of Age Related Volume Deficits in the Face
Age-Related AtrophyThe purpose of this study is to evaluate Allograft Adipose Matrix (AAM) in the face of people with age-related volume deficit - fat loss. AAM is a tissue used to correct fat loss. Participation in this study requires injections for placing AAM in the bilateral pre-jowl and malar areas of the face. The research will tell whether AAM is effective in maintaining mid-face fullness and whether the appearance of the skin improves after injection.
A Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in...
Geographic AtrophyThis is a 24-month, Phase III, multicenter, randomized, double-masked, sham-injection controlled study to assess the efficacy and safety of multiple IVT injections of APL-2 in subjects with GA secondary to AMD.
Investigation of Non-inferiority of Treatment of Vulvovaginal Dryness With WO2085 Moisturising Cream...
Vulvovaginal AtrophyThe aim of the study is to gain further experience with regard to the performance of the medical device WO2085 Moisturising Cream in comparison to a cream with 0.1% estriol.