This is a phase 1 study for participants with relapsed/refractory acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or juvenile myelomonocytic leukemia (JMML) diagnosis. This study will observe the safety, side effects, and best dose of Imetelstat as an investigational drug, or drug that is not approved by the Food and Drug Administration (FDA…
This study will enroll with children, adolescents and young adults that are less than 22 years old that have returned or 'relapsed' acute myeloid leukemia (AML) diagnoses. Within this group, the study is also targeting those are also unable to receive additional chemotherapy treatments containing anthracycline, which is a type of antibiotic that…
This study is called a screening study and the purpose of this study to find better ways to diagnose and treat leukemia that has come back after treatment or difficult to treat. Bone marrow, blood, and medical information about cancer and treatment will be collected. The results from this screening study may give other information about leukemia that is…
This research study will look at a newer treatment option for Barrett's Esophagus (BE) which uses nitrous oxide to freeze unwanted tissue. The most commonly used treatment method is Radiofrequency Ablation (RFA) which uses heat to destroy the pre-cancerous tissue. Nitrous Oxide ablation using the C2 CryoBalloon Focal Ablation System, an FDA-cleared…
The purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with…
The purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs…
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 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 purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in young adults with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) that has either come back or has not responded to the standard therapy. Pevonedistat works by blocking some of the enzymes that are needed for cell growth.
The purpose of the study is to compare two types of endoscopic treatments in patients with Barretts esophagus (BE) with abnormal cells (dysplasia); radiofrequency ablation (RFA) and hybrid argon plasma coagulation (H-APC). H-APC is a newer technique in the treatment of BE and has not been adequately studied and is not approved by the FDA for routine use.…
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…
The purpose of this study is to provide samples for biomarker testing for the different MPD-RC treatment studies. As part of the treatment study you currently participating in, this is an additional research study in which you we will ask for you to donate blood and/or bone marrow samples, two nail clippings, and buccal (cheek) swab. In order to…
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…
The purpose of this study is to compare two different combinations of treatment plans to a standard transplant procedure in order to see whether one or both of them are better at reducing the occurrence and severity of chronic GVHD. The research portion of this study involves manipulation of your stem cell product by removing T-cells, which cause chronic…
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…
This study is for children and teens who have been diagnosed with Acute Myeloid Leukemia (AML). AML is a cancer of the bone marrow, the spongy tissue inside the large bones of the body where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood cells called blasts. These blast cells crowd out the normal cells of the bone…
C2 Therapeutics is currently enrolling patients to participate in the Coldplay 3 clinical trial. Coldplay 3 is evaluating the effectiveness of the Coldplay CryoBalloon Focal Ablation System in patients with Barrett's esophagus who have not yet received treatment. Participants will undergo treatment with the Coldplay CryoBalloon Focal Ablation System…
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 this study is to evaluate a new technique called Wide Area Transepithelial Sample 3-Dimensional Esophageal Biopsy (WATS) with computer assisted method of analysis of brush biopsy. This study will assess whether performing standard biopsies in addition to the WATS brush biopsies identifies more patients with dysplasia than does performing…