Dance is a complex activity involving the integration of rhythm, spatial patterns, synchronization to external stimuli and whole-body coordination. While regular exercise is often recommended for people with Huntington's Disease (HD), there are no studies examining the use of dance to improve balance and mobility and positively affect the quality of…
The DIMENSION Study is evaluating the safety and effect on cognitive symptoms of an investigational oral drug in adults with early Huntington's disease. Cognitive symptoms may include difficulty paying attention or trouble thinking through steps of an activity or complex problems. Symptoms may also include difficulty planning, remembering, and staying…
The main purpose of this study is to look at the effectiveness, safety, and tolerability of study drug CTX-4430 when compared to a placebo (sugar pill) taken once-daily for 48 Weeks in adult patients with CysticFibrosis (CF).
Patients who have been diagnosed with decompensated nonalcoholic steatohepatitis (NASH) cirrhosis may be eligible to participate in this clinical trial. NASH is liver inflammation and damage caused by a buildup of fat in the liver. The main purpose of the study is to test the safety and efficacy of a drug called Emricasan (IDN-6556) in reducing the risk…
This study is for patients who have been prescribed tobramycin inhalation powder (TOBI Podhaler) or another inhaled medication used to treat cystic fibrosis patients whose lungs contain bacteria called Pseudomonas aeruginosa. The purpose of this research study is to assess how well TOBI Podhaler works on treating Pseudomonas aeruginosa in the lungs of…
The purpose of this research study is to find out whether a new investigational drug called SRX246, which might be useful to Huntington's Disease (HD) patients who sometimes feel irritable, angry, or even aggressive, is well tolerated and safe when it is given two times a day by mouth at doses as high as 160 mg. As part of this study, we will use a…
This study is being done to learn more about the safety and effectiveness of study drug VX-661 in combination with ivacaftor in patients aged 12 years and older with Cystic Fibrosis (CF). In this Study there are two treatment groups. If the patient meets all the rules for being in the Study, they willbe in one of the two treatment groups. The patient will…
The purpose of this study is to assess how effective the study drug Pulmaquin is in the management of chronic lung infections with Pseudomonas aeruginosa in subjects with non cystic fibrosis (nonCF) bronchiectasis by evaluating the time to first pulmonary exacerbation. A pulmonary exacerbation is defined as the new appearance or worsening in 4 or more of…
The purpose of this study is to find out whether the study drug altaluren can help lung function and can reduce the symptoms associated with cystic fibrosis (CF), and decrease the number of hospitalizations and the use of antibiotics for CF-related lung infections. The study will also investigate whether the drug can help improve overall patient quality of…
This study is being done to learn more about the safety and effectiveness of VX-440 used in combination with tezacaftor and ivacaftor in patients with cystic fibrosis. VX-440 given in combination with tezacaftor and ivacaftor is an investigational drug; investigational means the drug is not approved by the Food and Drug Administration (FDA), and is still…
The Sponsor is conducting this study to find out how safe and effective the investigational drug (alsocalled a study drug) obeticholic acid (also known as OCA) may be in improving compensated cirrhosis caused by NASH. Currently, there are no therapies approved for the treatment of NASH. OCA (the brand name is Ocaliva) has been approved in several…
The purpose of the ELEVATE CF study is to evaluate an oral investigational medication called VX-440 tosee if it is safe, effective, and well-tolerated when taken alone or with two other oral medications(VX-661 tezacaftor and ivacaftor) in people with cystic fibrosis (CF). This triple combination(TC) is being tested to see if it improves chloride transport (…
This is a 5-year, longitudinal, observational study of patients with NAFL or NASH designed to specifically address important clinical questions that remain incompletely answered from registration trials. The main purpose of this research study is to collect and study information on patients with NAFLD/NASH.The goal of this study is to observe any treatment…