The purpose of this study is to assess the dose dependent safety, tolerability and potential efficacy of QRX003 lotion in subjects with Netherton Syndrome (NS). This is a multi-center, randomized, vehicle-controlled, double-blind, parallel group comparison study of QRX003 lotion in adult subjects 18 years of age or older with NS. Approximately 18 subjects will be enrolled at approximately 7 sites. Subjects will be randomized (1:1:1) to treatment as follows:
1. QRX003 (dipalmitoyl hydroxyproline) lotion, 2% (Low dose)
2. QRX003 (dipalmitoyl hydroxyproline) lotion, 4% (High dose)
3. Vehicle lotion
Subjects will apply the assigned test article once daily in the morning to a designated Treatment Area for 12 weeks. Subjects will attend 5 clinic visits for up to 20 weeks.
This study aims to evaluate the efficacy and safety of lebrikizumab when used in combination with topical corticosteroid (TCS) treatment, compared with placebo, in pediatric participants with moderate-to-severe atopic dermatitis. Participants found to be eligible according to all of the study entry criteria will be randomly assigned in a 2:1 ratio to receive either lebrikizumab or placebo. This study can last up to 32 weeks, with 4 study periods. Screening Period: up to 4 weeks (≤30 days), TCS Standardization Period: 2 weeks, Treatment Period: 16 weeks, Post-Treatment Safety Follow-up Period: 12 weeks.
This is a Phase 3 multicenter, single-arm, open-label extension (OLE) study designed to investigate the long-term safety, tolerability, and efficacy of risankizumab 150 mg or 55 mg by weight every 12 weeks (Q12W) in the treatment of moderate to severe plaque Ps in eligible subjects who have completed all assessments in Study M19-977 and elect to participate in Study M19-973. The study is comprised of a 216-week treatment period and a follow-up phone call for safety approximately 140 days (20 weeks) after the last dose of study drug.
This study aims to evaluate the safety and efficacy of isotretinoin ointment (TMB-001 0.05%) in treating subjects > or = to 6 years of age with lamellar ichthyosis, and aims to assess the bodily absorption of the cream across application frequencies. Subjects with ARCI/RXLI will be randomized 2:1, to either receive the TMB-001 0.05% isotretinoin ointment or a vehicle ointment - applied daily - for 3 weeks. Subsequently, dosing will be increased to twice daily for 9 weeks. If significant improvement is observed at the end of the 9 weeks, subjects will be randomized 1:1 to receive the TMB-001 0.05% ointment, either applied daily or twice-daily, for 12 weeks. Prior to the Phase III trial, subjects will have the option to participate in a 14 day treatment period with TMB-001 0.05% - twice daily - followed by continued treatment with TMB-001 0.05% twice daily for 10 weeks.
The purpose of this study is to find out how long ustekinumab stays in and acts in participant's who are diagnosed with pediatric psoriasis and juvenile psoriatic arthritis. This is measured by blood tests. Another purpose is to find out if ustekinumab can cause side effects, which are unexpected or unwanted reactions from taking a drug. About 75 children will take part in this study worldwide. Participants will be in the study for a maximum of about 16 weeks.
In current Dermatology practice, options for vitiligo remain limited. The purpose of this study is to determine if once daily dosed topical rapamycin is effective for the treatment of patients with vitiligo. Participants will apply either 0.1% topical rapamcyin or 0.001% topical rapamycin for six months to a lesion on one side of the body, and topical placebo to a corresponding lesion on the opposite side of the body. The study also aims to evaluate patient satisfaction and identify any adverse effects on these dosing regimens.
The purpose of this study is to test whether a drug called PRA023 (the study drug) is a good treatment for patients with Systemic Sclerosis associated with Interstitial Lung Disease (SSc-ILD). The study drug PRA023 is an investigational drug that is given by infusion every 4 weeks. An investigational drug is not approved by The US Food and Drug Administration. It can only be used in a research study like this one. In this study, PRA023 will be compared with a placebo (dummy drug). The placebo will be a saline solution that does not have any study drug in it. The comparison with the placebo helps to determine whether the effects seen in your body is because of the PRA023 or not. This is a randomized study meaning that you will be assigned by chance (like flipping a coin) to receive either the study drug or placebo. This will be done with the help of a computer-based program and you will have 50% chance of receiving either the study drug or placebo. The study is double-blinded study and 50 weeks long, meaning you and your study doctor will not know what you are receiving, the study drug or placebo.
The study is sponsored by Prometheus Biosciences, Inc. The study is being done at approximately 25 sites across the United States. The main portion of the study will require 15 visits to the MUSC main campus and will have the following procedures completed over the course of your participation: blood draw, physician-led assessments of your disease (for example physical exam and skin thickness testing), tests to assess your pulmonary function and health (Pulmonary Function Test (PFT) and High Resolution Computed Tomography (HRCT)), electrocardiogram, as well as asked to complete surveys. Compensation is available for participation.
The purpose of this research study is to develop a better understanding of the cause and natural history of vascular anomalies and related syndromes. This study is being done in order to develop a better understanding of the cause of vascular anomalies in order to to improve care for people who are affected by these anomalies and related syndromes.
This study is being done at the University of Wisconsin-Madison (UW-Madison) and other sites in North America and Europe. A total of about 1000 people will participate in this study. About 20 – 30 people will take part in the study here at the Medical University of South Carolina.
The purpose of this research study is to determine if an investigational cream, ruxolitinib cream (0.75% and 1.5% strengths), is safe and effective to treat Atopic Dermatitis. In this study, ruxolitinib cream will be compared to a "vehicle cream." The vehicle cream looks like the ruxolitinib cream but contains no ruxolitinib. This study can last up to 55 weeks. For the first 8 weeks participants will be randomly assigned to receive either the ruxolitinib cream or vehicle cream. For the following 44 weeks participants will receive the ruxolitinib cream.
The purpose of this study is to obtain information on the safety, efficacy, and tolerability of an oral drug called HZN-825 for the treatment of diffuse cutaneous systemic sclerosis (dcSSc) in participants 18-75. The study drug is taken as a tablet twice a day for 52 weeks. This study has 12 planned visits over a 60 week period. The study will measure the improvement, stabilization or worsening of your symptoms, such as changes in your fatigue and pain levels, lung function, skin thickness and other participant reported outcomes.