Efficacy, Safety And Tolerability Study Of RN6G In Subjects With Geographic Atrophy Secondary to...
Age-Related MaculopathyThe purpose of this study is to determine the efficacy, safety and tolerability of multiple doses of RN6G in subjects with Geographic Atrophy Secondary to Age-related Macular Degeneration.
Intravitreal Injections of Sirolimus in the Treatment of Geographic Atrophy
Geographic AtrophyThis study will seek to enroll 50 persons who have central or non-central geographic atrophy (GA) associated with age-related macular degeneration (AMD). GA in treated eye must be between 0.75 disk areas (DA) and 8 DA. Eligible participants will be randomly chosen to receive one of the following treatments in the study eye: A 20 μL (440 μg) intravitreal (IVT) injection of sirolimus, or A sham treatment (subconjunctival injection of lidocaine) Participants with two (2) eligible eyes will have one eye randomly assigned to receive intravitreal sirolimus and no sham in the fellow eye. The first injection will begin at Day 0, which may occur on the date of screening/enrollment or up to two weeks following screening/enrollment visit, and every month thereafter. The visit schedule is as follows: A clinical evaluation, including safety measures, will occur monthly. Vision will be measured at the screening/enrollment visit and at 2, 3, 6, 9, 12, 18 and 24 months after the first injection has occurred. Fundus autofluorescence will occur at screening/enrollment and at 2, 6, 12, 18 and 24 months after the first injection has occurred. Fundus color photography and optical coherence tomography will occur at screening/enrollment and at 6, 12, 18 and 24 months after the first injection has occurred. The primary goal is to evaluate whether the persons receiving the sirolimus injections show a slower worsening of geographic atrophy compared to the persons receiving the sham injections. A secondary goal is to evaluate the impact of sirolimus on vision compared to the sham. NOTE: As of May 30, 2014, study injections were discontinued due to safety concerns. No further enrollments will occur and follow-up will continue on all active study participants on a quarterly basis. That is, on visits coinciding with 3 month intervals from date of enrollment.
YF476 and Type I Gastric Carcinoids (Norway)
Chronic Atrophic GastritisHypergastrinemia1 moreThe aim of the study is to find out if the experimental medicine, YF476, can make gastric carcinoids, a rare type of stomach tumour, shrink and disappear. Gastric carcinoids occur mainly in patients with chronic atrophic gastritis (CAG), a condition in which the acid-producing cells in the lining of the stomach can't make acid. Acid production is controlled by gastrin, a hormone (chemical messenger) that's released into the bloodstream. If the stomach can't make acid, blood levels of gastrin rise. High blood levels of gastrin in patients with CAG can cause other cells (ECL cells) in the lining of the stomach to grow and, over the years, to give rise to gastric carcinoids. Gastric carcinoids are usually benign, but they can become malignant. Therefore, patients with CAG and gastric carcinoids have the inside of their stomach checked regularly, by gastroscopy, to see if the gastric carcinoids need removing surgically. A gastroscope is a thin (1 cm), flexible tube at end of which is a mini video camera, which enables the user to inspect the lining of the stomach and a 'snare' to take samples of tissue (biopsies). YF476 (netazepide) is a gastrin receptor antagonist (blocks the effects of gastrin), so it's a potential new medical treatment for gastric carcinoids in patients with CAG. Up to 10 of these patients will take YF476 daily for up to 12 weeks. They'll make up to seven outpatient visits for tests, including checks on the safety of YF476 and up to four gastroscopies. At each gastroscopy, the gastric carcinoids will be measured and biopsies taken for laboratory tests. Patients will take up to 24 weeks to finish the study.
Benefits of the Use of Botox in the Treatment of Empty Nose Syndrome Syndrome
Empty Nose SyndromeAtrophic RhinitisEmpty Nose Syndrome patients suffer from disabling physical symptoms and considerable distress. To date there is no definitive cure for these symptoms. Established treatment modalities include saline irrigation, surgical implantation of materials or simply use of cotton wads/ silicon cones to simulate the resistive action to airflow of the resected turbinates. This study will research the effectiveness of a new treatment modality in the treatment of Empty Nose Syndrome. This novel treatment method involves the use of botulinum toxin type A (Botox).
An Observational Study of Parkinson's Disease and Other Synucleinopathies (EASE-PD)
Parkinson's DiseaseMultiple System AtrophyThe study is a multi-center, observational study to evaluate the feasibility of α-synuclein-related biomarkers and imaging data in the disease diagnosis and prognosis evaluation in Synucleinopathies and healthy subjects.
Study of Rifampicin in Multiple System Atrophy
Multiple System AtrophyThe purpose of this study was to determine whether Rifampicin was effective in slowing or reversing the progression of multiple system atrophy (MSA). Research studies indicate that there is an abnormality in protein synthesis and structure in parts of the brain responsible for MSA (protein misfolding) and the drug Rifampicin could potentially prevent or reverse this protein alteration. The study was done on participants with early MSA. The study consisted of taking the drug 2 times a day for 12 months. Participants underwent an evaluation of symptoms and function and will underwent a neurologic examination at the beginning of the study, at 6 months and at 12 months. They were also be contacted at 3 and 9 months by telephone. Studies were done at 10 participating sites.
A Trial to Evaluate the Efficacy of Low Dose Vaginal Estrogens in the Treatment of Atrophic Vaginitis...
Atrophic VaginitisAtrophic vaginitis is a common problem that occurs in up to 40% of postmenopausal women. The main symptoms are dryness, itching and burning in and around the vagina. These symptoms in turn can cause pain with intercourse. Other symptoms can include urinary frequency and urinary urgency. These symptoms are caused by the decreasing levels of estrogens in postmenopausal women. The current treatment options include hormone replacement therapy by mouth and patch and vaginal creams, tablets and rings. The current guideline recommend using hormone therapy at the lowest dose for the shortest time. We are trying to find the lowest effective dose of estrogen cream that helps with symptoms without causing an increase in estrogens in the blood stream. We want to compare two estrogen creams, estradiol and estriol, each at very low doses, with a placebo cream. Estradiol cream is available on prescription but we want to see how effective it is at one tenth the commonly prescribed dose. Estriol cream is not commonly prescribed and is only available at compounding pharmacies. We want to see if either of these estrogens is effective at one tenth the usual dose. We also want to see if these creams are effective when massaged onto the outer part of the vagina, rather than the usual method of treatment, which is inserting them into the vagina.
Fluocinolone Acetonide Intravitreal Inserts in Geographic Atrophy
Age-Related Macular DegenerationThis study will compare the safety and efficacy of Medidur FA treatment in one eye to the sham-treated fellow eye of subjects with geographic atrophy secondary to AMD.
Neuroprotection and Natural History in Parkinson's Plus Syndromes (NNIPPS)
Multiple System AtrophyProgressive Supranuclear PalsyNNIPPS is a clinical trial of riluzole (a drug previously shown to slow down the rate of progression og amyotrophic lateral sclerosis-ALS; Lou Gehrig's disease) involving nearly 800 people diagnosed with the 'parkinson plus' syndromes of multiple system atrophy (MSA) and progressive supranuclear plasy (PSP). In addition to showing whether riluzole is helpful in MSA and PSP, NNIPPS will improve criteria for making an accurate and early diagnosis, for assessing the rate of progression, and will advance understanding of the biology of these disabling and progressive neurodegenerative diseases.
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal...
Spinal Muscular AtrophyThe primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS 396443-CS4 (NCT02292537). The secondary objective of Part 1 of this study is to examine the pharmacokinetics (PK) of Nusinersen in participants with SMA. The primary objective of Part 2 of this study is to assess the long-term safety and tolerability of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments. The secondary objective of Part 2 of this study is to examine the PK of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments.