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Displaying 1 - 24 of 24

A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma

Condition: Cancer / Liver Cancer
Investigator: Linda Wu, MD
Status: Currently Recruiting
The purpose of this study is to test the safety of an investigational drug called ALN-BCAT, and find out what effects, if any, ALN-BCAT has on people with your type of liver cancer. The effects being studied include: the frequency and severity of side effects (to determine if the drug is safe); whether ALN-BCAT may make tumors smaller; how ALN-BCAT may…
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Liquid biopsy and radiomics for liver cancer surveillance

Condition: Cancer / Liver Cancer
Investigator: Elizabeth Verna, MD
Status: Currently Recruiting
This study aims to use new technology involving molecular and imaging tests to help detect liver cancer early in patients. Participants enrolled will participate in it for approximately 5 years. Participation will last approximately 3 months. If you have any questions regarding this study, please contact the Transplant Clinical Research Center (TCRC).
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Study of Atezolizumab and Bevacizumab Alone or in Combination with Tiragolumab in Patients with Hepatocellular Carcinoma (HCC)

Condition: Cancer / Liver Cancer
Investigator: Linda Wu, MD
Status: Closed
The purpose of this study is to compare the effects, good or bad, of tiragolumab plus atezolizumab plus bevacizumab versus placebo plus atezolizumab plus bevacizumab on patients with HCC. In this study, participants will be randomized you will get either tiragolumab plus atezolizumab plus bevacizumab or placebo plus atezolizumab plus bevacizumab. A placebo…
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Study of EDIT-301 in Patients with Severe Sickle Cell Disease (SCD)

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the…
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Study of PF-07209326 in Patients with Sickle Cell Disease

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Andrew Eisenberger, MD
Status: Closed
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
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A Study of Atezolizumab Plus Bevacizumab Therapy in Patients With Hepatocellular Carcinoma at High Risk of Recurrence

Condition: Cancer / Liver Cancer
Investigator: Gulam Manji, MD, PhD
Status: Closed
The purpose of this study is to compare the effects, good or bad, of atezolizumab plus bevacizumab versus observation (not receiving any treatment) on patients with completely resected (surgically removed) or ablated (removed using heat) hepatocellular carcinoma (the most common type of liver cancer) who are at high risk for disease recurrence.…
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Study of stem cell transplant effects in patients with sickle cell disease

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle cell disease. Patients will receive busulfan…
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A study for patients with cystic fibrosis using study drug ataluren (PTC124)

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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TPST-1120 as Monotherapy and in Combination With Nivolumab in Subjects With Advanced Cancers

Condition: Cancer / Liver Cancer
Investigator: Yvonne Saenger, MD
Status: Closed
The purpose of this study is to learn more about TPST-1120. The aims of the study are to find out if TPST-1120 can be given safely to people, to determine a safe dose of the study drug, if it is safe when given in combination with other anti-cancer drugs, what effects the drug may have, and to learn more about how the body processes the study drug. This is…
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A study for patients with Cystic Fibrosis using study drug VX-440

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…
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A study for patients with advanced hepatocellular carcinoma using study drug pembrolizumab

Condition: Cancer / Liver Cancer
Investigator: Gulam Manji, MD, PhD
Status: Closed
The purpose of this study is to test the safety, tolerability, and anti-tumor activity of the research study drug, Pembrolizumab (also known as MK-3475) in subjects with advanced hepatocellular carcinoma (a type of liver cancer also referred to as HCC) who have taken sorafenib.
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A study for patients with Cystic Fibrosis using study drug VX-440

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 (…
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A study for patients with Cystic Fibrosis using study drug VX-661

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).
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A study for patients with liver cancer using study drug RO7070179

Condition: Cancer / Liver Cancer
Investigator: Gulam Manji, MD, PhD
Status: Closed
The purpose of this study is to assess the safety, tolerability, and effectiveness of RO7070179 given to patients with Hepatocellular Carcinoma (HCC), a primary type of liver cancer, with normal to moderately impaired liver function. RO7070179 is given intravenously to study its effects in the treatment of liver cancer. RO7070179 is an investigational anti-…
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A study for children with sickle cell using study drug Hydroxyurea

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Nancy Green, MD
Status: Closed
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…
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A Study of Atezolizumab in Combination with Cobimetinib Versus Atezolizumab Monotherapy in Participants with Unresectable Cholangiocarcinoma

Condition: Cancer / Liver Cancer
Investigator: Susan Bates, MD
Status: Closed
The purpose of this study is to compare any good and bad effects of using atezolizumab alone or with another drug called cobimetinib. About 82 people are expected to taking part in this study. Adding cobimetinib to atezolizumab could help to shrink your cancer, but it could also cause extra side effects. This study will allow the researchers to know whether…
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A study for patients with Cystic Fibrosis using oral study drug CTX-4430

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).
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A study for patients with biliary or gallbladder cancer using study drug DKN-01

Condition: Cancer / Liver Cancer
Investigator: Gulam Manji, MD, PhD
Status: Closed
This research study is being done to see how an investigational drug called DKN-01 combined with standard cancer drugs (gemcitabine and cisplatin) works in people with biliary or gallbladder cancer. DKN-01 is an antibody that blocks the function of another protein in the body called dickkopf-1 (Dkk-1). Too much (over expression) of the Dkk-1 protein has…
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Study of Drug in Patients with Previously Treated Bile Duct Cancer (Cholangiocarcinoma)

Condition: Cancer / Liver Cancer
Investigator: Susan Bates, MD
Status: Closed
This study is designed to evaluate the efficacy and safety of the study drug AG-120 as compared to placebo in patients with advanced cholangiocarcinoma. AG-120 blocks an abnormal form of a protein called IDH1, which is involved in cancer cell metabolism. Abnormal IDH1 causes too much of a substance called 2-HG to be produced. Researchers believe that having…
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An study for patients with cystic fibrosis (CF) treated with TOBI Podhaler or other FDA approved inhaled antipseudomonal antibacterial drug

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…
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A 5-Year Study of the Natural History and Management of Patients with Hepatocellular Carcioma (A Type of Liver Cancer)

Condition: Cancer / Liver Cancer
Investigator: Elizabeth Verna, MD
Status: Closed
Patients will be asked to participate in this research study because they have been diagnosed with hepatocellular carcinoma (HCC) which is a type of liver cancer. By doing this study, researchers hope to: Find out more about patients that have HCC including their age, race/ethnicity, and other medical conditions they may have; learn more about how…
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A study for patients with Cystic Fibrosis using study drug VX-661 in combination with Ivacaftor

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…
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Study of CTX001 in Patients with Severe Sickle Cell Disease (SCD)

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Markus Mapara, MD
Status: Closed
This study is being done to learn more about the safety and effects of CTX001 (the Study Product) in patients with severe Sickle Cell Disease (SCD). The Study Product is considered investigational; investigational means the Study Product is not approved by the United States Food and Drug Administration (FDA). The goal of this study is to see if a single…
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A study for subjects with non-cystic fibrosis bronchiectasis (NCFB) using study drug Pulmaquin to manage chronic lung infections

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…
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