362 Studies Now Enrolling
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Displaying 1 - 18 of 18
Condition: Neurological Disorders / Huntington's Disease
Investigator: Ashwini Rao, EdD, OTR
Status: Currently Recruiting
Child to Adult Neurodevelopment in Gene Expanded Huntingtons Disease (ChANGE HD) HD has long been considered an adult-onset disease. However, research has shown that brain changes are evident before motor symptoms begin. In fact, the gene responsible for HD plays a role in brain development and is present throughout the lifespan. With gene knockdown therapy…
Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
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…
Condition: Neurological Disorders / Huntington's Disease
Investigator: Ashwini Rao, EdD, OTR
Status: Currently Recruiting
Dance is a complex activity involving the integration of rhythm, spatial patterns, synchronization to external stimuli and whole-body coordination. While regular exercise is often recommended for people with Huntington's Disease (HD), there are no studies examining the use of dance to improve balance and mobility and positively affect the quality of…
Condition: Eye
Investigator: Brian Marr, MD
Status: Closed
In this research study we want to learn more about treatment of advanced or recurrent retinoblastoma. For children with retinoblastoma that have an advanced stage of presentation in one eye or if they have failed all conventional treatment, eye removal is considered. This study will investigate the utility of a chemoplaque(s) to salvage eyes involved with…
Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 study of a drug called uproleselan. We are testing new experimental drugs such as uproleselan in the hopes of finding a treatment that may be effective against acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that has come back or that has not responded to standard therapy. This study looks at how well…
Condition: Neurological Disorders / Huntington's Disease
Investigator: Karen Marder, MD, MPH
Status: Closed
The DIMENSION Study is evaluating the safety and effect on cognitive symptoms of an investigational oral drug in adults with early Huntington's disease. Cognitive symptoms may include difficulty paying attention or trouble thinking through steps of an activity or complex problems. Symptoms may also include difficulty planning, remembering, and staying…
Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
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…
Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
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…
Condition: Eye
Investigator: Tongalp Tezel, MD
Status: Closed
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.
Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of Decitabine (DEC) and Vorinostat (VOR) followed by the standard chemotherapy drugs (Fludarabine, Cytarabine and G-CSF (FLAG)). The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children with acute myeloid leukemia (AML)…
Condition: Eye / Glaucoma
Investigator: Jeffrey Liebmann, MD
Status: Closed
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…
Condition: Eye
Investigator: Tongalp Tezel, MD
Status: Closed
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.…
Research Study of New Treatment Called NT-501 (Encapsulated Cell Therapy (ECT) Implant) for Glaucoma
Condition: Eye / Glaucoma
Investigator: George Cioffi, MD
Status: Closed
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…
Condition: Eye
Investigator: Tongalp Tezel, MD
Status: Closed
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…
Condition: Neurological Disorders / Huntington's Disease
Investigator: Karen Marder, MD, MPH
Status: Closed
The purpose of this research study is to find out whether a new investigational drug called SRX246, which might be useful to Huntington's Disease (HD) patients who sometimes feel irritable, angry, or even aggressive, is well tolerated and safe when it is given two times a day by mouth at doses as high as 160 mg. As part of this study, we will use a…
Condition: Eye / Glaucoma
Investigator: Lama Al-Aswad, MD, MPH
Status: Closed
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.
Condition: Eye
Investigator: Stephen Tsang, MD, PhD
Status: Closed
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…
Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in children 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.
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