A Study to Test How Well Different Doses of BI 754132 Are Tolerated in Patients With an Advanced...
Macular DegenerationThis is a study in adults with geographic atrophy, an advanced form of age-related macular degeneration. The purpose of this study is to find out how well different doses of BI 754132 are tolerated. The participants are in the study for about 4 months. During this time, they visit the study site about 10 times. Participants receive 1 injection of BI 754132 directly into one of the eyes affected by geographic atrophy. In this study, BI 754132 is given to humans for the first time. The doctors compare how well participants tolerate the different doses of BI 754132. The doctors also regularly check the general health of the participants.
Assessment of NfL and GFAP Levels, Atrophy of the Macula GCC by OCT and Whole Brain Atrophy by MRI...
Multiple SclerosisThe investigators hypothesize that serum neurofilament-light chain (NfL) levels at baseline and decrease of the macular ganglion cell complex (GCC) thickness at one year vs. baseline are as good as progression of whole brain atrophy at one year vs. baseline to predict later evolution of neurological disability in multiple sclerosis patients.
FOCUS: A Phase I/II First in Human Study to Evaluate the Safety and Efficacy of GT005 Administered...
Dry Age-related Macular DegenerationMacular Degeneration5 moreThis is an open label first in human Phase I/II multicentre study of GT005 in subjects with Macular Atrophy due to AMD
A Futility Trial of Sirolimus in Multiple System Atrophy
Multiple System AtrophySingle-center, randomized, placebo-controlled, phase-II, futility clinical trial to determine if oral sirolimus is of sufficient promise to slow disease progression in MSA, prior to embarking on a large-scale and costly phase III study to assess its efficacy. A futility design under the null hypothesis assumes that sirolimus will slow the progression of the disease, whereas the alternative hypothesis assumes no benefit of sirolimus. If the null hypothesis is rejected (i.e., futility of sirolimus to slow progression of MSA), a major phase III study will be discouraged, whereas non-futility will offer strong support for a phase III trial to detect clinical efficacy.
A Safety and Efficacy Study of Brimonidine Intravitreal Implant in Geographic Atrophy Secondary...
Geographic AtrophyMacular DegenerationThis study will assess the safety and efficacy of the brimonidine intravitreal implant in participants with geographic atrophy due to age-related macular degeneration.
A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
Muscular AtrophySpinalThis multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
Spinal Muscular Atrophy 1This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.
Long-Term Safety of Lampalizumab Intravitreal (ITV) Injections in Participants With Geographic Atrophy...
Geographic AtrophyThis multicenter open-label extension study is designed to evaluate the safety and tolerability of lampalizumab intravitreal injections in participants with GA secondary to age-related macular degeneration (AMD) who completed 96 weeks of treatment in Studies GX29176 (NCT02247479) or GX29185 (NCT02247531). The extension will enroll participants from the parent studies who received investigational lampalizumab, as well as lampalizumab-naive participants exposed to sham comparator. All participants will receive open-label lampalizumab in the present study.
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
Muscle AtrophyA long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
New Non-Hormonal Treatment by Radiofrequency for Vulvo-Vaginal Atrophy
Vulvo-vaginal AtrophyGenitourinary Syndrome of MenopauseVulvo-Vaginal Atrophy (VVA) or Genitourinary Syndrome of Menopause (GSM) is a common and under-reported condition associated with decreased estrogenization of the vaginal tissue The aim of this study is to evaluate safety and efficacy of " Dynamic Quadripolar Radio-frequency" thermal treatment with Vaginal Dynamic Radio-frequency (VDR™) and Radio-frequency Safety System (RSS™) for the treatment of VVA and GSM in postmenopausal women who either present contra-indication for menopause hormone therapy, or are not willing to use Menopause Hormone Therapy (MHT) or have failed to be helped using MHT.