Study Evaluating the Efficacy and Safety of Three Formulations of Ultra-low Dose Estriol Vaginal...
Vaginal AtrophyA Phase 2, Dose-ranging, 12-week Randomized, Double-blind, Placebo controlled, Parallel-group Study Evaluating the Efficacy and Safety of Three Formulations of Ultra-low Dose Estriol Vaginal Gel (0.005% Estriol Vaginal Gel, 0.002% Estriol Vaginal Gel, 0.0008% Estriol Vaginal Gel) for the Treatment of Vaginal Dryness in Postmenopausal Women with Vulvovaginal Atrophy. Vulvovaginal atrophy is a natural consequence of the progressive estrogen deficiency that occurs in menopause. Epidemiological data have indicated that about 50% of otherwise healthy women over 60 years of age experience symptoms related to urogenital atrophy such as vaginal dryness, dyspareunia, burning, itching, as well as urinary complaints or infections of the lower urinary tract. As these alterations frequently affect the quality of life of postmenopausal women, it is important for doctors to detect their presence and offer treatment options. Estrogen therapy is the most effective treatment of moderate to severe symptoms of vulvar and vaginal atrophy. One advantage of local treatment with estrogen is avoidance of first-pass liver metabolism, making it possible to use lower doses of estrogen compared with oral therapy; the local route also minimize systemic adverse effects. The search for therapeutic alternatives which may present improvements in relation to the current products has been encouraged.
Comparison Between Microneedling Daycare Procedure and Tazarotene 0.1% Gel Local Application in...
Atrophic Post Acne ScarringPost acne scarring is a common complication of acne. Cosmetic appearance of the post acne facial scarring can be improved by various methods. Among the procedural methods microneedling (1) is a novel and a promising option. It is a minimally invasive day care procedure for the management of atrophic acne scars. Topical tazarotene 0.1% gel is an effective medical method in the management of acne vulgaris and macular acne scars (2, 3). Based on its mechanism of action and role in collagen synthesis, topical tazarotene is a logical choice to investigate for the management of atrophic post acne scars. This is a pilot study comparing microneedling and topical tazarotene for the treatment of atrophic post acne scarring in regard to extent and rapidity of improvement, patient satisfaction and any adverse events if any. Thirty six subjects with grade 2 to grade 4 atrophic post acne scars, classified on the basis of Goodman's Qualitative classification (4) criteria will be recruited. Goodman's qualitative and quantitative acne scarring grading system scoring will be performed for the assessment of severity of acne scarring at baseline. The face of each patient will be randomized for monthly microneedling on one side and topical tazarotene 0.1 % gel once a day local application on opposite side, using computer generated random number table. Follow ups will be done at every month until treatment completion (3 months) and 3 months after the last treatment session. Goodman's qualitative and quantitative acne scarring grading system scoring will be performed at 3rd and 6th month follow up visits. An improvement by two grades will be considered as excellent, one grade will be rated as good and no up gradation will be labelled as poor response. Patients will be also assessed by a blinded observer for clinical improvement and scored on a scale of 0 (no improvement) to 10 (maximum) at 3rd and 6th month follow up visits with the help of serial photographs taken under consistent background, position and lighting. All patients will be instructed to assess themselves using Patients' Global Assessment Score 0 (no response) to 10 (maximum) at 3rd and 6th month follow up visits. The investigators hope the outcome of the present study may propose a newer medical modality for acne scarring, i.e topical tazarotene 0.1% gel, which can be used at home, obviating the need for physician dependant microneedling procedure.
Role of Stem Cells, Platelet Rich Plasma in Treatment of Scars
Atrophic ScarThe Objectives of this randomized controlled trial aiming to establishing a protocol of post scar revision care and to Study the effect of PRP and/or Adipose derived Mesenchymal Stem cell injection in improvement of atrophic scar after scar revision.
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
Spinal Muscular AtrophyAn open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1 spinal muscular atrophy. The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy after 13 weeks; and to estimate the Maximum Tolerated Dose (MTD) of orally administered branaplam; and to identify the dose that is safe for long term use as well as that can provide durable efficacy optimal dosing regimen in patients with Type 1 SMA.
Study of BHV-3241 in Participants With Multiple System Atrophy
Multiple System AtrophyThe purpose of this study is to compare the efficacy of verdiperstat (BHV-3241) versus placebo in participants with Multiple System Atrophy
Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic Atrophy...
Geographic AtrophySafety Assessment of APL-2 in Patients with Geographic Atrophy
A Phase 3 Safety and Efficacy Study of Intravitreal Administration of Zimura (Complement C5 Inhibitor)...
Geographic AtrophyMacular DegenerationThe objectives of this study are to evaluate the safety and efficacy of Zimura (avacincaptad pegol) intravitreal administration in patients with geographic atrophy secondary to age-related macular degeneration (AMD)
Study of NBMI Treatment in Patients With Atypical Parkinsons (PSP or MSA)
Progressive Supranuclear PalsyMultiple System AtrophyIn total 20 subjects will be enrolled at one participating site -UMC Ljubljana. The 20 subjects will be treated with placebo and NBMI 300 mg in a cross-over design. In case of subject drop-outs, additional subjects may be enrolled as decided by the Sponsor, to allow for expected number of evaluable subjects in each group.
Combined LASERs and PRP for Postacne Scars
Atrophic Acne ScarThis study evaluates a new combined technique using two different laser wavelengths and Platelets Rich Plasma (PRP) to treat post-atrophic acne scars. Within the available knowledge of laser-tissue interactions and effects of PRP on wound healing, we will explore the clinical effects of our new combination procedure on a histopathological and immunohistochemical basis for guiding future post acne scars clinical research.
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With...
Spinal Muscular Atrophy Type IThis is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic pathogenic mutation of the survival motor neuron 1 gene (SMN1) with one or two copies of survival motor neuron 2 gene (SMN2). The primary objective of the study is to evaluate the efficacy of onasemnogene abeparvovec-xioi by assessing the proportion of symptomatic SMA Type 1 participants who achieve the ability to sit unaided for at least 10 seconds up to and including the 18 months of age trial visit. At least 6 participants aged < 6 months (< 180 days) at the time of gene replacement therapy (Day 1) will be enrolled.