This study is being done to determine if REGN2810 (anti-PD-1) in combination with ipilimumab (anti-CTLA-4) plus initial platinum-based doublet chemotherapy, or REGN2810 in combination with ipilimumab will prolong how long you live without your disease progressing compared to the standard of care pembrolizumab (anti-PD-1) monotherapy treatment. Neither…
We are conducting a survey to gather feedback about lung cancer patients experience with treatment and the challenges you faced. Your insights can help shape better care for future patients and explore how technology can be of help! The survey takes 30-40 minutes, and eligible participants who complete it will receive a gift card. Interested in…
We are doing this study because we want to find out if MRI brain scans alone is better or worse than MRI brain scans plus preventative brain radiation for patients with small lung cancer. The usual approach for patients with small cell lung cancer is prophylactic cranial irradiation. If you decide to take part in this study, you will either receive PCI for…
Are you undergoing a stem cell transplant? You may be at high risk for infections after the transplant. This study is looking at the safety and effectiveness of an investigational medication for those who have undergone a stem cell transplant. Recovery after a stem cell transplant can already be challenging without the added worry of life-threatening viral…
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 purpose of this study is to see whether or not the medicine, Abatacept, when given after hematopoietic stem cell transplantation (HSCT) is effective in treating GVHD in children and adolescents with sickle cell disease (SCD). We are also planning to gather additional information about the safety of treating sickle cell disease with a hematopoietic stem…
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 (…
The main purpose of the study is to find out about whether the study drug is safe in humans and aneffective treatment in pediatric patients with acute Graft versus Host Disease (GVHD). GVHD mayoccur after a bone marrow or stem cell transplant in which someone receives bone marrow tissue orcells from a donor (called an allogeneic transplant). The new,…
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).
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…
In this trial, we will evaluate whether partially-HLA matched allogeneic multivirus-specific VSTs, will be safe and produce anti-viral effects in immunodeficient recipients infected with one of more of the targeted viruses that are persistent despite conventional anti-viral therapy. This will be in patients who have received a hematopoietic stem cell…
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 primary objectives of this study are to measure the safety and effectiveness of bone marrow transplant (BMT) using specific chemotherapy in children with less severe SCD and to understand the long term effects of bone marrow transplant in children with SCD, by evaluating ovarian reserve, sickle cell related brain problems, sickle cell related kidney…
This purpose of this study is to determine whether the tumor cells of lung cancer patients contain changes to the DNA sequence of two proteins found on the cells surface- ALK and EGFR. If the tumor contains changes to the DNA of one of these proteins, the patient may be invited to participate on a trial that will look at drugs which may target tumors that…
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 test a new medicine to prevent liver venoocclusive disease (VOD) in patients undergoing hematopoietic stem cell transplant (HSCT) who are at high risk or very high risk for liver VOD following HSCT. Liver VOD can be a complication of bone marrow and/or stem cell transplant,which may be life threatening. The condition is a…