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Displaying 1 - 29 of 29

A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML

Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
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…
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Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)

Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
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…
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A Study to Test the Effects and Safety of Riliprubart in People with Chronic CIDP for Which the Usual Treatment Does Not Work

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Currently Recruiting
This is a global, multicenter, placebo-controlled, phase 3 study evaluating the efficacy and safety of riliprubart in adult participants with CIDP who are refractory or had an insufficient response to standard of care (SOC) therapies, defined as Ig administered IV (IVIg) or SC (SCIg), or corticosteroids. Treatment duration will be 48 weeks. Participant must…
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Pediatric Acute Leukemia (PedAL) Screening Trial - Developing New Therapies for Relapsed Leukemias (Children and Young Adults)

Condition: Cancer / 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…
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Study of People with First-Degree Relatives (Parents, Siblings, Children) with Multiple Sclerosis

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Philip De Jager, PhD
Status: Currently Recruiting
Genes and Environment in MS (GEMS) is a longitudinal cohort study investigating how genetics and environment can lead to the onset of Multiple Sclerosis. We are recruiting first-degree relatives (siblings, parents, and children) of people who have been diagnosed with MS. This study involves the collection of magnetic resonance imaging (MRI) of the brain,…
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Multiple Sclerosis (MS) Genetics

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Philip De Jager, PhD
Status: Currently Recruiting
MS Genetics aims to better understand the genetic component of demyelinating diseases by doing genetic testing on blood samples of participants. This study involves a one-time blood draw and completion of questionnaires spread out over a couple of years in hopes to better understand the genes that makes one predisposed to Multiple Sclerosis and various…
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MS Snapshot: A Study to Better Understand Multiple Sclerosis

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Philip De Jager, PhD
Status: Currently Recruiting
The Snapshot study aims to understand brain and spinal cord aging in healthy individuals and individuals with multiple sclerosis (MS). We aim to identify new tools that will help us to achieve successful brain aging in the general population and in people with an underlying autoimmune disease. A critical aspect of this study is that we are looking for…
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Study of OCR/RTX vs. Diroximel for Patients with Relapsing-Remitting Multiple Sclerosis (RRMS) or Clinically Isolated Syndrome (CIS)

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Rebecca Straus Farber, MD
Status: Closed
The purpose of this study is to evaluate the differences between immune profiles of individuals remaining on Ocrelizumab/Rituximab versus those transitioning to diroximel fumarate at two years. Baseline visits for subjects transitioning to diroximel fumarate (DRF) will take place 6 months after last Ocrelizumab (OCR) or Rituximab (RTX) dose, prior to…
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Study of Quizartinib in Combination with Chemotherapy for Children and Young Adults with Recurrent /Refractory Acute Myeloid Leukemia (AML)

Condition: Cancer / 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…
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ProChocolate Study - Patients with Multiple Sclerosis (MS) and Family Members of Patients with MS

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Rebecca Straus Farber, MD
Status: Closed
We are currently recruiting individuals with Multiple Sclerosis (MS), first-degree family members of persons with MS, and healthy individuals to take part in our research on the effects of probiotics. Our goal is to better understand how the health of the gut microbiome influences the immune system.
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Study of Nivolumab in Combination with 5-Azacytidine in Acute Myeloid Leukemia (AML) - Children and Adults Age 1 to 30

Condition: Cancer / 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…
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Prebiotic vs. Probiotic Treatment in Multiple Sclerosis (MS) or Clinically Isolated Syndrome (CIS)

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Rebecca Straus Farber, MD
Status: Closed
This study will be used to evaluate the immunologic effects of Prebiotics (soluble fiber required by beneficial bacteria), in comparison to Probiotics (beneficial bacteria), in individuals with Multiple Sclerosis (MS) and Clinically Isolated Syndrome (CIS).
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ASPIRE: Aspirin for Exercise in Multiple Sclerosis

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Victoria Leavitt, PhD
Status: Closed
ASPIRE is a clinical trial to investigate aspirin as a treatment to improve exercise performance in people with multiple sclerosis (MS) who experience overheating during exercise. Persons with multiple sclerosis benefit from exercise, but many avoid it because of exhaustion and overheating. This randomized controlled trial (RCT) tests aspirin as a method to…
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A Study to Assess the Clinical and Biological Predictors of Disease Course in Patients with Guillain-Barre Syndrome

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
International GBS Outcome Study (IGOS) is a study of the Inflammatory Neuropathy Consortium (INC) and Peripheral Nerve Society (PNS) on disease course and outcome in Guillain-Barr syndrome (GBS). Guillian Barre syndrome is an autoimmune disorder affecting the peripheral nervous system, leading to paralysis and potential fatality if it progresses to involve…
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A study for patients with Multiple Sclerosis (MS) using study drug ocrelizumab for disease-modifying treatment

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Claire Riley, MD
Status: Closed
The purpose of this study is to see if ocrelizumab (study drug) will stabilize, or potentiallyimprove the signs and symptoms of your MS. Ocrelizumab is a type of drug called amonoclonal antibody. Monoclonal antibodies act like your bodys immune system andattach to certain cells in order to attack germs and other illnesses in your body.Ocrelizumab attaches…
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A Study for Charcot-Marie-Tooth type 1A Patients to Compare 2 doses of PXT3003 vs. a Placebo

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
Charcot-Marie-Tooth type 1A (CMT1A) disease is a rare, slowly progressing and debilitating hereditary condition commonly causing weakness of muscles in the feet and lower parts of the legs. There is currently no approved drug to cure CMT1A disease, and the available treatments commonly target the pain and inflammation associated with the disease. In order…
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Biomarker Study of Philadelphia Chromosome Negative Myeloproliferative Neoplasms (MPN)

Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
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…
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A Study of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (MyCIDPchoice)

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
We are doing this study to find out if the research drug known as Rozanolixizumab can have additional benefit for people who are already receiving IVIG( Intravenous Immune Globulin) treatment for CIDP, and to have further safety evaluation of the research drug as well. If you choose to be part of this research study, the following procedures will occur:…
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A Study for Patients with Neuropathy Associated Pain to Compare study drugs Nortriptyline, Duloxetine, Pregabalin and Mexiletine

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
Peripheral Neuropathy is a result of nerve damage, which may cause weakness, numbness, tingling, and pain in your hands, feet, and other areas of your body. Over 20 million Americans are affected by peripheral neuropathy, most commonly caused by diabetes. In approximately 25-50% of all people with peripheral neuropathy, the cause is unknown. These cases…
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Study of Combination Treatment to Reduce Occurrence and Severity of Chronic Graft Versus Host Disease (GVHD)

Condition: Cancer / Leukemia
Investigator: Monica Bhatia, MD
Status: Closed
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…
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Top-CSPN Study

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
This study is looking at using topiramate as a potential therapy for patients with cryptogenic sensory peripheral neuropathy. Patients who are randomized into the study will return to the clinic for follow up every 16 weeks. The total length of the study is 96 weeks.
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Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and Treatment with Subcutaneous Immunoglobulin

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to see how effective two doses of an investigational new drug called IgPro20 is when given subcutaneously (a shot given into the fat layer between the skin and the muscle) to treat CIDP in patients that still require IVIG and also to look at how safe the IgPro20 is for patients.
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A study of adding study drug Sorafenib to the usual treatment in children and teens with AML (Acute Myeloid Leukemia)

Condition: Cancer / Leukemia
Investigator: Alice Lee, MD
Status: Closed
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…
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Study for patients with Amyloidsis (TTR) using an investigational drug, ALN-TTR02 for treatment

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to collect information about how patients with FAP respond to the study drug ALN-TTR02 over 18 months. This includes studying any potential improvements in FAP and any side effects that participants may have from the study drug. In addition, the study will examine how the body handles (distributes, break down, eliminates) the…
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Phase 3 Gene Therapy for Painful Diabetic Neuropathy

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
You are being considered to participate in this research study because you have type I or II diabetes with current treatment control and, you are experiencing painful diabetic peripheral neuropathy (DPN) in both lower extremities legs.The specific events that result in painful diabetic peripheral neuropathy are not well understood, but high blood sugar,…
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Study for treating patients with neuropathy using study drug fingolimod

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to evaluate the efficacy and safety of study drug, fingolimod taken daily compared with a placebo (does not contain active medication) on delaying disability progression in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The study will consist of 3 periods: a Screening Period (lasting for up to 45…
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A Study to Assess the Treatment-Related Fluctuations in CIDP Patients Using Daily Grip Strength Measurements

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
Subjects with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) will be enrolled in the study and Intravenous Immunoglobulin (IVIg) will be given to eligible subjects on the study. CIDP is a chronic inflammatory condition that causes the immune system to attack the nerves. IVIg is one common treatment for CIDP. IVIG is a blood product that is…
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MLN4924 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome (18 to 21 years old)

Condition: Cancer / 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 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.
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Study of patients with neuropathy using study drug, ISIS TTR Rx

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to evaluate the efficacy and safety of ISIS TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy (FAP). The purpose of this study is to determine if ISIS 420915 can help people with mild or moderate FAP. To be in this study you must have FAP and be able to walk without help or walk with using only one cane.
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