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…
Pain is a natural response to injury to let us know there may be damage to our bodies. Pain is the way our body tells us that we must take care of ourselves. Chronic pain is different and lasts for a long time. In some cases, chronic pain remains without any sign of body damage. Today, there is no way for a doctor to measure someone's chronic pain or…
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 purpose of this clinical study is to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in individuals with geographic atrophy secondary to dry age-related macular degeneration (AMD) who qualify, based on genetic testing.
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 patients with Glaucoma. The purpose of this study is to investigate the safety and effectiveness of a study drug called Bimatoprost Sustained Release (SR) compared to treatment with eye drug Timolol. The Bimatoprost SR is an implant that is very small, is biodegradable (dissolves naturally in the body), and contains the bimatoprost drug.
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…
Stargardt macular degeneration isa genetic condition, which in mostcases causes vision loss early in lifeand leads to legal blindness. Thereis currently no FDA-approvedtreatment.This trial will evaluate the safetyand effects of ALK-001, a newpotential drug treatment, on theprogression of Stargardt disease.ALK-001 is a capsule administereddaily by mouth.This…
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.
Glaucoma is the leading cause of irreversible blindness worldwide. The most important test to detect progression is visual field testing. However, this test is very subjective, often unreliable, and variable. One of the main causes of unreliable tests is the lack of attentiveness or concentration during the test. Previous studies have shown that listening…
Glaucoma is a given name of a group of diseases that affect the retinal ganglion cells in the eye and the optic nerve. Glaucoma cause degeneration of the retinal ganglion cells in the retina and their projections (axons) that form the optic nerve. Retinal ganglion cells and their axons transmit all of our vision information from the eye to the brain through…
The abicipar is an injectable medication stored as a liquid in a single-use glass vial for delivery into the back of the eye. The active component is a protein drug that was made using bioengineering technology. It was designed to bind to and inactivate a protein called Vascular Endothelial GrowthFactor (VEGF) that plays an important role in causing AMD.…
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…
Macular degeneration is a progressive eye disease that damages the macula. The macula is the part of the eye that is responsible for central vision, which is needed to perform straight-ahead activities, such as reading, driving, or watching TV. This research is being conducted to learn about the safety and efficacy (how effectively the study drug works) of…