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Displaying 1 - 11 of 11
Condition: Liver Disease / Liver Failure
Investigator: Steven Lobritto, MD
Status: Currently Recruiting
TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three-arm clinical trial of immunosuppressive therapy for children with acute liver failure. This is a double-blind study, so the study doctors and participants will not know to which arm the participants are assigned. The study will determine if corticosteroids or equine anti-…
Condition: Liver Disease / Liver Failure
Investigator: Elizabeth Verna, MD
Status: Currently Recruiting
The purpose of this research study is to determine if an investigational drug, VS-01, will help in the treatment of acute-on-chronic liver failure (ACLF) compared to supportive care (standard of therapy). ACLF is a potentially life-threatening condition characterized by a group of signs that occur together (syndrome) in patients with a late stage of…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Claire Keating, MD
Status: Closed
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…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Claire Keating, MD
Status: Closed
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 (…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Emily DiMango, MD
Status: Closed
This study is being done to learn more about the safety and effects of the combination of VX-661 and ivacaftor in subjects with Cystic Fibrosis (CF).
Condition: Lung Disease / Cystic Fibrosis
Investigator: Emily DiMango, MD
Status: Closed
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).
Condition: Lung Disease / Cystic Fibrosis
Investigator: Hossein Sadeghi, MD
Status: Closed
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…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Claire Keating, MD
Status: Closed
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…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Emily DiMango, MD
Status: Closed
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…
Condition: Cancer / Thyroid Cancer
Investigator: Antonio Fojo, MD, PhD
Status: Closed
This is a study designed for patients with medullary thyroid cancer. Treatment will consist of a drug called Ponatinib. Ponatinib is an investigational agent that blocks abnormal cancer proteins and therefore harms cancer cells. It was recently approved by the Food and Drug Administration for leukemia treatment, but is not approved for medullary thyroid…
Condition: Lung Disease / Cystic Fibrosis
Investigator: Emily DiMango, MD
Status: Closed
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…
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