The reason for this study is to find out if the experimental study drug, tesevatinib, is effective as a possible treatment for recurrent glioblastoma. Certain genetic information about your tumor will also be collected, by drawing blood, to help better understand why tesevatinib did or did not work in treating your recurrent glioblastoma. Although…
The purpose of this research study is to learn about the effects of the study drug, PF-06840003,and to find the best dose for treating research subjects/participants with malignant glioblastoma or anaplastic glioma. PF-068400003 is a new investigational drug. A new investigational drug is one that is currently not approved for sale in this country. PF-…
This study aims to evaluate the efficacy of single event multi level chemoneurolysis with the a botulinum toxin A drug, Dysport, on walking energy expenditure and gait in children with spastic diplegia Cerebral Palsy.
The purpose of this research study is to obtain information on the safety and effectiveness of PBCAR20A to treat certain types of cancers. PBCAR20A is a new investigational treatment for certain types of cancer of the blood, such as leukemia and lymphoma. It is made from a type of blood cells known as T cells. The T cells in PBCAR20A came from healthy…
Pevonedistat is currently being studied in humans with advanced solid tumors and acute myelogenous leukemia (AML). The current study is designed to evaluate pevonedistat in combination with azacitidine in patients with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and low-blast AML. This study will assess whether taking…
The purpose of this research is to find out whether an investigational drug called CVT-301, is helpful to people with Parkinson's Disease (PD). The treatment involves a new method for taking levodopa (LD) through an inhaler (which is like an asthma inhaler) to provide relief of motor fluctuations (OFF episodes), which are experienced by many Parkinson…
This is a research study to test the effects of VB-111 combined with Bevacizumab versus Bevacizumab alone on glioblastoma . This study will enroll 252 subjects age 18 years and older who have recurrent glioblastoma. All subjects will be randomly assigned in a 1:1 ratio to receive either VB-111 combined with Bevacizumab or Bevacizumab alone. Survival, tumor…
The purpose of this study is to test the safety and effectiveness of avapritinib (also known as BLU-285) in individuals who have been diagnosed with aggressive systemic mastocytosis (ASM), Systemic mastocytosis with associated hematologic neoplasm (SM-AHN), or mast cell Leukemia (MCL). Avapritinib is an experimental drug means that is is not yet known if…
The purpose of this part of the study is to obtain further information about the safety and effectiveness(how well it works) of azacitidine plus birinapant (the investigational drug), in people with higher-risk myelodysplastic syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL). One of the standard drugs for these diseases is azacitidine which all…
The purpose of this study is to investigate the safety and effectiveness of tirasemtiv as a potential new therapy for the improvement of breathing, muscle weakness and muscle fatigue in patients with Amyotrophic Lateral Sclerosis (ALS). The effects of tirasemtiv on the performance of your elbow, wrist, knee, ankle, and hand muscles and how long you can…
THE PURPOSE OF THIS STUDY is to find out whether a type of brain imaging (called "Magnetic Resonance Spectroscopy Imaging" of "MRS") might be useful to help assess the presence of Alzheimer's disease (AD) and assess progression of the disease. MRS uses magnetic resonance imaging (MRI) methods to image the chemicals in the brain.…
The purpose of this study is to determine how well a drug called rigosertib works on people with MDS and to study the safety of rigosertib when it is given to people with MDS. Rigosertib is currently being tested in other clinical research studies.
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…
The main purpose of this study is to improve treatment of ocular side effects that result from depatuxizumab mafodotin (Depatux-M) use in patients with glioblastoma (GBM). Most people treated with Depatux-M will experience eye side effects. When these side effects are not tolerated, Depatux-M is usually interrupted until symptoms improve and then restarted…
The purpose of the study is to see if a medicine called ASP2215 is both effective and safe as atreatment for AML patients with mutations in FLT3 gene identified by an investigational FLT3Mutation Assay companion diagnostic (a specific way to test your blood or bone marrow, which is used to see if you have a mutation of the FLT3 gene), compared to salvage…
The purpose of this study is to find out whether an experimental drug, bevacizumab with temozolomide is safe when given to elderly patients with brain tumors and investigate the side effects of the drug combination and how often they occur. This study will also explore whether bevacizumab and temozolomide can be effective in shrinking tumors when given to…
The purpose of this study is to test the effectiveness of investigational drug APL-130277 for patients with Parkinson's disease. APL-130277 is an investigational drug, which means that the study drug is currently being tested. It is not approved by the U.S. Food and Drug Administration (FDA). The active ingredient in APL-130277 is apomorphine, a…
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.
This is a two-part research study of an investigational drug called ABBV-321 in subjects with glioblastoma (brain tumor). This study will be conducted in two-parts: 1) Dose Escalation Phase: The purpose of the first part, called a Dose Escalation Phase, is where we will increase the dose of ABBV-321 until we find the highest dose before unmanageable side…
The purpose of this research study is to test an investigational drug called NP001. The study will evaluate if NP001 has an effect on slowing the signs and symptoms of your ALS, if there are any side effects, and if there are any medical problems with giving the drug as a solution into your vein (intravenous IV administration). An investigational drug is…
The purpose of the research study is to see if selinexor has any effects against your cancer and toevaluate the side-effects of selinexor or selinexor in combination with temozolomide (TMZ ). One specific way cancer cells continue to grow is by getting rid of certain proteins called tumor suppressor proteins that would normally cause cancer cells to die.…
The purpose of the study is to evaluate the efficacy and safety of imetelstat for the treatment of patients with myelodysplastic syndrome (MDS). All patients participating in Part 1 of this study will get 7.5 mg/kg of imetelstat as a 2-hour intravenous infusion once every 4 weeks. The dose of imetelstat may be increased, held or decreased depending on how…
The purpose of this research study is to determine the effectiveness (how well something works) and safety of an investigational compound called alvocidib when used in a combination chemotherapy therapy regimen in patients with acute myeloid leukemia (AML) who test positive for NOXA priming.
This study is for patients who have been diagnosed with Multiple System Atrophy (MSA). The main goals of this study are to determine how safe and well tolerated AZD3241 is in patients who have MSA, and whether AZD3241 has certain effects in the brain of patients with MSA. These effects in the brain are measured with positron emission tomography (PET)…
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…
New data from the study of ABT-414 and ABBV-221 in patients with recurrent glioblastoma multiforme (GBM) have shown several examples where it seems evidence of disease progression (cancer that continues to grow and spread) was seen in MRI images, but examination of surgically removed tissue under the microscope did not agree with the MRI.
This is a randomized, double-blind, placebo-controlled, parallel-group study that will evaluate the efficacy and safety of gantenerumab versus placebo in participants with prodromal (very early form) to mild AD. Participants will have a chance of receiving either injections of gantenerumab or placebo as per the visit schedule. The primary efficacy…
The purpose of this study is to see if a medicine called ASP2215 is both safe and effective as atreatment for patients with Acute Myeloid Leukemia (AML). ASP2215 is an experimental drug that is being studied to treat AML. In this study, ASP2215 will be given in addition to a standard chemotherapy regimen that patients could be receiving even if they were…