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 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…
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…
The experimental drug being investigated in this study is called rucaparib. Rucaparib is being developed by Clovis Oncology, Inc. (the Sponsor), a biopharmaceutical company with headquarters in the United States (US). Rucaparib (trade name: Rubraca) is a medicine that is approved in the European Union (EU) and the US for use in certain types of ovarian…
This study plans to learn more about a device called the Cefaly and if it is effective for treating migraine attacks. This device is approved by the Food and Drug Administration (FDA) for the prevention of episodic migraines, but clinical data has not yet been collected on its effectiveness in treating migraine attacks. The Cefaly device works by generating…
The study drug (TSR-042) in this study is designed to stop cancer from growing by helping your immune system recognize and fight the cancer. The Study Drug is designed to help your immune system by attaching to a protein called PD-1 and stopping one of the signals that keeps your immune system from recognizing the cancer. This may help your immune system…
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 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…
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…
This study plans to learn more about a device called the Cefaly and if it is effective for treating migraine attacks. This device is approved by the Food and Drug Administration(FDA) for the prevention of episodic migraines, but clinical data has not yet been collected on its effectiveness in treating migraine attacks.
SY-1365 is an investigational drug that stops the function of a protein known as CDK7. Previous workhas shown that CDK7 is necessary for some cancers to grow. Therefore, SY-1365 could be used to slow down the growth of cancer. This research study is being done to understand the highest tolerated dose of SY-1365, and to assess the safety of SY-1365 in…
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.
This study is for patients who have severe migraine headaches that might be a result of a hole in the heart wall called a Patent Foreman Ovale, or PFO. It is known that as many as 40% of patients with migraine headaches also have a PFO. It is not well understood how the two are related, or how having a hole in the heart wall could cause headaches. In this…
This is a Phase 1/2 multicenter study of VX15/2503 in pediatric patients and young adults with solid tumors which have come back or have not responded to standard therapy. The study treatment is considered experimental because VX15/2503 is not approved by the United States (US) Food and drug Administration (FDA) for treating pediatric patients and young…