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Displaying 1 - 59 of 59

Study of AZD9574 in Patients with Brain Cancer

Condition: Cancer / Brain Cancer
Investigator: Maria Diaz Ordonez, MD
Status: Currently Recruiting
The main purpose of this study is to determine a dose of AZD9574 that is safe and tolerable (does not cause side effects that could prevent people from taking the drug) when given alone or in combination with temozolomide. The study will also evaluate how much of the study drug is in your blood at various times. In addition, the action of the study drug…
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Study to Evaluate Multiple Treatments for Patients with Newly Diagnosed or Recurrent GBM

Condition: Cancer / Brain Cancer
Investigator: Andrew Lassman, MD
Status: Currently Recruiting
The purpose of this study is to evaluate multiple investigational treatments for either newly diagnosed or recurrent brain tumors to determine if any of these study treatment(s) improve overall survival as compared to standard treatments. Investigational means that these are new treatments sometimes called "study drugs" and are not approved by the…
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Study of Focused Ultrasound (FUS) with DEFINITY Microbubbles in Children and Young Adults with Progressive Diffuse Midline Glioma (DMG)

Condition: Cancer / Brain Cancer
Investigator: James Hinkley Garvin Jr, MD
Status: Currently Recruiting
Children and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine…
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Study of Lutathera in Patients with Glioblastoma (GB)

Condition: Cancer / Brain Cancer
Investigator: Mikhail Doubrovin, MD
Status: Currently Recruiting
The purpose of the study is to evaluate the safety of Lutathera alone in patients with recurrent glioblastoma and the combination of Lutathera with radiotherapy with temozolomide (TMZ) in patients with newly diagnosed glioblastoma (GB) and to establish the appropriate dose of this combination. Lutathera is a radioligand therapy, which is a targeted…
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Non-Invasive ultrasound treatment for low back and leg pain

Condition: Neurological Disorders / Neuromuscular
Investigator: Clark Smith, MD
Status: Currently Recruiting
The purpose of this research is to study whether Discogen's non-invasive, low-intensity pulsed ultrasound treatment can help reduce pain for people with leg pain and low back pain. Eligible participants will be randomized into either a Discogen treatment group or a sham control group. All participants will be asked to come to the Columbia University…
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Study of Chemotherapy Drug, Lomustine, in Addition to Temozolomide in Patients with Glioblastoma (GBM)

Condition: Cancer / Brain Cancer
Investigator: Fabio Iwamoto, MD
Status: Currently Recruiting
The purpose of this study is to compare the usual radiation therapy and usual chemotherapy, temozolomide, to using the usual radiation therapy and usual chemotherapy plus the chemotherapy drug lomustine (Gleostine). Lomustine (Gleostine) is already approved by the FDA for use in brain tumors but it is usually used alone and is not the first-line choice for…
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Study of Eflornithine & Temozolomide in Patients with Glioblastoma Multiforme (GBM)

Condition: Cancer / Brain Cancer
Investigator: Maria Diaz Ordonez, MD
Status: Currently Recruiting
The purpose of this study is to measure how safe eflornithine is in combination with temozolomide in patients with newly diagnosed glioblastoma multiforme (GBM) or astrocytoma following radiation therapy. Safety and tolerability will be determined on the basis of physical exams, laboratory tests, and questions about any problems you might experience during…
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Study of Sonocloud 9 in Patients with Glioblastoma (GBM)

Condition: Cancer / Brain Cancer
Investigator: Brian Gill, MD
Status: Currently Recruiting
The purpose of this study is to see if the implantable device, Sonocloud 9, is effective getting chemotherapy to better reach your tumor compared to receiving standard-of-care chemotherapy Temozolomide and Lomustine and to see how safe and tolerable the device is. Sonocloud 9 works by making the blood-brain barrier (BBB) more permeable (allowing liquids or…
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Study of PF-07799544 in Patients with Solid Brain Tumors

Condition: Cancer / Brain Cancer
Investigator: Aya Haggiagi, MD
Status: Currently Recruiting
The purpose of this study is to learn about the safety (the impact of the study drug on your body) and the effects of study drug PF-07799544 for specific types of cancer on the body and to find the best dose for treating certain cancers. For BRAF (B-raf proto-oncogene serine/threonine kinase) mutated solid tumors, PF-07799544 may be combined with…
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ALS Families Project

Condition: Neurological Disorders / Neuromuscular
Investigator: Matthew Harms, MD
Status: Currently Recruiting
This program provides family members of individuals with familial ALS the opportunity to contribute to research focused on learning more about why motor neuron degeneration begins and how or why it progresses. This study provides genetic counseling and testing to help participants understand and manage their risk and determine if they want to learn their…
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Study of Tirabrutinib in Patients with Primary Central Nervous System Lymphoma (PCNSL)

Condition: Cancer / Brain Cancer
Investigator: Fabio Iwamoto, MD
Status: Currently Recruiting
The purpose of this study is to learn the effects of the investigational new drug, tirabrutinib, when given for the treatment of PCNSL. Investigational new drug means a drug that has not been approved as a marketed product (that is, available to be prescribed or sold) by the United States (US) Food and Drug Administration (FDA). Tirabrutinib acts by…
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PTC124-GD-041-DMD (ALSO KNOWN AS STUDY 041) CLINICAL TRIAL OF ATALUREN IN NONSENSE MUTATION DUCHENNE MUSCULAR DYSTROPHY (NMDMD)

Condition: Neurological Disorders / Neuromuscular
Investigator: Darryl De Vivo, MD
Status: Closed
Columbia University is currently enrolling in an Ataluren Study from PTC Therapeutics, called PTC-041. We are looking for 5 year old boys, ambulating, confirmed nonsense mutation DMD and on steroids for at least 12 months. If you would like more information about this please reach out to Ameneh Onativia, Columbia Sr. Clinical Research Coordinator II at 212…
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Assess Treatments for Painful Diabetic Peripheral Neuropathy (PDPN)

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to Assess Treatments for Painful Diabetic Peripheral Neuropathy (PDPN). To be in this study you must have type II Diabetes Mellitus, PDPN, must be 18 years of age or older, and be able to stop taking some of your current pain medications including pills, creams, patches, marijuana, cannabidiol (CBD) oil, etc. Acetaminophen (…
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Study of AGEN1181 Alone or in Combination with Balstilimab in Patients with Advanced Sarcoma

Condition: Cancer / Sarcoma
Investigator: Brian Henick, MD
Status: Closed
This research study mainly aims to test the safety and tolerability of AGEN1181 and balstilimab and to determine the most appropriate dose of the study drug AGEN1181 alone and in combination with balstilimab. AGEN1181 targets cells that are a part of the body's immune system to block a protein called cytotoxic T-lymphocyte-associated protein-4 (CTLA-4…
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A Study of AL102 in Patients With Progressing Desmoid Tumors (RINGSIDE)

Condition: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
You are being asked to participate in this study because you have been diagnosed with a progressive desmoid tumor. The purpose of this study is to see if the study drug, called AL102 (referred to from now on as the study drug), is safe and effective in people with progressive desmoid tumors. The study will also look at how your body responds to the study…
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Study of Radiosurgery & Nivolumab Alone or in Combination with Ipilimumab in Patients with Meningioma

Condition: Cancer / Brain Cancer
Investigator: Aya Haggiagi, MD
Status: Closed
Phase 2, Treatment A: This study is being done to see if your recurrent brain tumor may respond to a combination of radiosurgery (a type of radiation therapy) with nivolumab (a type of immunotherapy drug). A small number of patients have already received this combination previously and have tolerated the treatment relatively well. However, we would like to…
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Study of Vismodegib and GSK2256098 to Shrink Tumors in Patients with Meningioma

Condition: Cancer / Brain Cancer
Investigator: Mary Welch, MD
Status: Closed
The purpose of this study is to test good and bad effects of these two different drugs against meningioma tumors with altered genes. Today, therapy for meningioma is the same for all patients and is not based on tumor genetic testing. This trial is trying to see if tumor genetic testing would be helpful at guiding treatment in patients with meningioma.…
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Study of REC-2282 in Patients with Progressive NF2 Mutated Meningiomas

Condition: Cancer / Brain Cancer
Investigator: Gurcharanjeet Kaur, MD
Status: Closed
The purpose of this study is to find out if REC-2282, an investigational new drug, is an effective and safe treatment in patients with progressive NF2 mutated meningiomas who have either NF2 disease-related meningioma or recurrent sporadic meningiomas that have NF2 mutation. Investigational new drug means a drug that has not been approved as a marketed…
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Study of Safusidenib in Patients with Brain Glioma

Condition: Cancer / Brain Cancer
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to see how safe and effective Safusidenib erbumine, the study drug, is in treating brain glioma and to find the recommended dose of the study drug for clinical studies. Safusidenib is not Food and Drug Administration (FDA) approved.
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Study of Immune Therapy Drugs in Addition to the Usual Radiation Therapy for Patients with Brain Cancer (Glioblastoma)

Condition: Cancer / Brain Cancer
Investigator: Fabio Iwamoto, MD
Status: Closed
This study is being done to answer the following question: Can we shrink or stabilize your brain cancer by adding immune therapy drugs to the usual radiation therapy? We are doing this study because we want to find out if this approach is better or worse than the usual approach for your recurrent glioblastoma. The usual approach is defined as care most…
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Study of Vaccine for Cytomegalovirus (CMV) in Patients with Brain Tumors

Condition: Cancer / Brain Cancer
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this study is to test the safety of a new vaccine, VBI-1901, and to know more about its ability to boost the response of the immune system against Cytomegalovirus. The reason for testing this vaccine in people with a brain tumor is that it might one day be used to boost the ability of the immune system to fight a certain type of brain tumor.…
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Study of DSP-0390 in Patients with Recurrent High-Grade Glioma

Condition: Cancer / Brain Cancer
Investigator: Mary Welch, MD
Status: Closed
The purpose of this study is to evaluate how safe the investigational drug, DSP-0390, is at different doses and to identify a recommended dose that is safe for further study. Investigational means that the study drug is currently being tested and has not been approved for use alone or in combination with any drug by the U.S. Food and Drug Administration (…
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ImmuneSense Study: IGV-001 in Newly Diagnosed Patients With Glioblastoma

Condition: Cancer / Brain Cancer
Investigator: Brian Gill, MD
Status: Closed
The purpose of this study is to see how safe the study medicine, IGV-001 plus standard of care (SOC), is and to compare IGV-001 with a placebo see if it works in slowing glioblastoma multiforme (GBM) tumor regrowth. SOC refers to the usual medicine(s) or procedures used to treat your cancer. A placebo looks like the study medicine but does not contain any…
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Study of Belinostat and Guadecitabine for Treatment of Chondrosarcoma

Condition: Cancer / Sarcoma
Investigator: Shaheer Khan, DO
Status: Closed
The purpose of this study is to test the good and bad effects of the drugs called belinostat and SGI-110 (guadecitabine). These drugs could shrink your cancer, but they could also cause side effects. The study doctors hope to learn if the study drugs will shrink the cancer more often than observed with other standard treatment options. We dont know if…
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Study of Pemigatinib in Patients with Glioblastoma (GBM) or Other CNS Tumors

Condition: Cancer / Brain Cancer
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this study is to evaluate and determine the efficacy of pemigatinib in participants with recurrent glioblastoma or other CNS tumors with changes in the FGFR1, FGFR2 or FGFR3 genes. Pemigatinib (also referred as INCB054828) is an investigational drug that is being studied by Incyte Corporation (Sponsor) for use in the treatment of previously…
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Study of Tumor Treating Fields (Optune, 200kHz) with Radiation Therapy and Temozolomide for the Treatment of Newly Diagnosed Glioblastoma

Condition: Cancer / Brain Cancer
Investigator: Fabio Iwamoto, MD
Status: Closed
Optune is a portable, battery-operated device that generates TTFields at a specific frequency. The TTFields are delivered to the brain through 4 sticky bandages (which hold ceramic discs) that are placed on the surface of the head (called arrays). The arrays create electric forces within the brain that disrupt cancer cell division. Optunes TTFields are a…
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Study of Unesbulin in Patients with Leiomyosarcoma (LMS)

Condition: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
The purpose of this study is to evaluate the effectiveness and safety of an investigational drug, unesbulin. This investigational drug is being studied in unresectable or metastatic, relapsed, or refractory leiomyosarcoma along with Dacarbazine. Dacarbazine is a drug that is FDA-approved for the treatment of other types of cancer and recommended by the…
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Study of Drug - CFT8634 in Patients with Synovial Sarcoma or SMARCB1-Null Tumors

Condition: Cancer / Sarcoma
Investigator: Shaheer Khan, DO
Status: Closed
The study drug, CFT8634, is an investigational therapy that has been shown in animal studies to help break down the BRD9 protein, an important protein in the body that helps SMARCB-null tumors, such as your own, to grow. CFT8634 is able to do so by using the body's own protein-recycling equipment. (In short, your cells are capable of breaking down…
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A Study for Patients with Malignant Peripheral Nerve Sheath Tumors Using Study Drugs PLX3397 and Sirolimus

Condition: Cancer / Sarcoma
Investigator: Gulam Manji, MD, PhD
Status: Closed
The purpose of this study is to determine if treatment with PLX3397 and Sirolimus will be tolerated and result in shrinking of the cancer or stopping the cancer from growing. In the phase I portion, the maximum tolerated dose of the study drug will be determined. In the phase II portion, progression-free survival will be assessed at the dose level found in…
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Study of Envafolimab in Patients with Sarcoma or Myxofibrosarcoma

Condition: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
The purpose of this research study is to determine the effectiveness and safety of envafolimab, an experimental drug that is not approved for use, when given alone or in combination with ipilimumab to stimulate your immune system to attack cancer cells. Envafolimab is an experimental (investigational) cancer drug that overrides the PD-1/PD-L1 checkpoint,…
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Study of INBRX 109 in Patients with Metastatic or Inoperable Chondrosarcoma

Condition: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
The experimental drug called INBRX 109 has been developed as a treatment for unresectable or metastatic conventional chondrosarcoma. The purpose of this study is to learn about the effects of the study drug, INBRX-109 on you, your immune system, and your cancer. This study is being done to understand if INBRX-109 is effective against your chondrosarcoma and…
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Study of ATX-101 in Patients with Dedifferentiated Liposarcoma (LPS) and Leiomyosarcoma (LMS)

Condition: Cancer / Sarcoma
Investigator: Benjamin Izar, MD
Status: Closed
The purpose of this study is to evaluate the effectiveness of a new investigational drug, ATX-101, for the treatment of dedifferentiated liposarcoma and leiomyosarcoma. ATX-101 is an intravenous (IV) drug which blocks the interaction of a protein called PCNA with a number of stress response proteins. These interactions are thought to be important for cancer…
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Study of Milademetan in Comparison to Trabectedin in Patients with Liposarcoma

Condition: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
The purpose of this study is to learn about the safety, tolerability, and effectiveness of an investigational drug called milademetan as compared to the use of trabectedin. In this study, subjects will be randomly assigned (like flipping a coin) to receive one of two study drugs: milademetan or trabectedin. This is an open-label study, meaning you will know…
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Study of MTX110 in Combination with Gadolinium in Children & Young Adults with Newly Diagnosed Brain Cancer (Diffuse Midline Gliomas)

Condition: Cancer / Brain Cancer
Investigator: James Hinkley Garvin Jr, MD
Status: Closed
The purpose of this study is to find the best dose of a drug called MTX110 and a contrast agent called gadolinium that can be given safely in children with newly diagnosed diffuse midline gliomas, which is a kind of brain tumor. All patients enrolled in the study will receive infusion of MTX110 and Gadolinium delivered with a pump directly into the tumor…
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Study of Paclitaxel With and Without Nivolumab and Nivolumab and Cabozantinib in Taxane Pretreated Subjects with Angiosarcoma

Condition: Cancer / Sarcoma
Investigator: Mark Stein, MD
Status: Closed
The purpose of this study is to: 1. Compare treatment with chemotherapy (paclitaxel) alone to using a combination of nivolumab plus chemotherapy (paclitaxel) treatment in patients with angiosarcoma who have not been treated with paclitaxel chemotherapy alone. 2. Evaluate the effect of nivolumab in combination with cabozantinib on angiosarcoma (cancer) in…
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Study of Drug - BI 907828 in Patients with Dedifferentiated Liposarcoma (DDLPS)

Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to test the study drug, BI 907828 for safety, effectiveness, and side effects when compared to receiving the standard treatment, doxorubicin, in subjects with dedifferentiated liposarcoma (DDLPS). BI 907828 has proven to minimize a critical protein interaction between MDM2 and p53, two proteins in your body, which when their…
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A Study of Selinexor in Combination With Standard of Care Therapy for Newly Diagnosed or Recurrent Glioblastoma

Condition: Cancer / Brain Cancer
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this research study is to see if the combination of medications approved for the treatment of glioblastoma, together with the study drug, selinexor (also known as XPOVIO), has any effects on your cancer. Selinexor works by trapping tumor suppressor proteins within the cell nucleus. This is expected to cause the cancer cells to die or stop…
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A Study of PTC596 in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)

Condition: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The purpose of this study is to evaluate the effectiveness of a new investigational drug, PTC596, used in combination with dacarbazine, for the treatment of locally recurrent, unresectable, or metastatic relapsed/refractory leiomyosarcoma, a type of soft tissue sarcoma. The purpose of this study is to evaluate whether adding PTC596 to dacarbazine is safe…
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Study of AG-881 in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)

Condition: Cancer / Brain Cancer
Investigator: Andrew Lassman, MD
Status: Closed
This study is being done to evaluate the safety and usefulness of the study drug, AG-881, as compared to placebo (a medically inactive substance) in residual or recurrent non-enhancing Grade 2 glioma (a type of brain cancer) that has a mutation in the IDH1 or IDH2 metabolic enzyme (a substance produced by the body to assist in breaking down chemicals).…
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Study of ONC201 in Adults With Recurrent H3 K27M-mutant Glioma

Condition: Cancer / Brain Cancer
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this research study is to test how safe and how well an investigational drug known as ONC201 works in treating high grade glioma (a type of brain cancer). The FDA (the U.S. Food and Drug Administration) has not approved ONC201 as a treatment for this condition.
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Spearhead 1 Study in Subjects With Advanced Synovial Sarcoma or Myxoid/Round Cell Liposarcoma

Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to find out if an investigational drug, called ADP-A2M4, is safe and how well it works in attacking Synovial Sarcoma or Myxoid/Round Cell Liposarcoma (MRCLS) cancer cells. The study involves taking certain types of immune cells from the blood, called T cells, and changing them in a laboratory before putting them back into the…
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Study for Patients with Anaplastic Astrocytoma That Progress/Recur After Radiation and Chemotherapy

Condition: Cancer / Brain Cancer
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this study is to measure how well and how safe eflornithine is in combination with lomustine, compared to lomustine taken alone, in treating patients whose anaplastic astrocytoma has come back after radiation and chemotherapy. Safety and how well you can tolerate the drug will be determined on the basis of physical exams, laboratory tests,…
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Study of AMG-232 with Preoperative Radiotherapy in Subjects with Soft Tissue Sarcoma (STS)

Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to test the safety of a study drug called AMG 232 given with radiation in patients with soft tissue sarcoma (STS) prior to surgery. The AMG 232 is investigational. It has been tested in other cancers but not yet in sarcoma. AMG 232 has not been tested using the dosing schedule in this study and it has not been tested with…
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Study to Evaluate AL102 in Patients with Progressing Desmoid Tumors - DUPLICATE

Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
You are being asked to participate in this study because you have been diagnosed with a progressive desmoid tumor. The purpose of this study is to see if the study drug, called AL102 (referred to from now on as the study drug), is safe and effective in people with progressive desmoid tumors. The study will also look at how your body responds to the study…
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Genomic Translation for ALS Care

Condition: Neurological Disorders / Neuromuscular
Investigator: Matthew Harms, MD
Status: Closed
The purpose of this study is to look for abnormal genes and gene expression profiles that help determine why a person develops amyotrophic lateral sclerosis (ALS) and related motor neuron diseases (MND) and why their symptoms present and progress with a particular pattern. For up to three years, subjects will be followed every 3 months at the Columbia ALS…
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A Study for Patients With Generalized Myasthenia Gravis Using Intravenous Human Immunoglobulin Concentrate, IGIV-C.

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves your muscle weakness associated with MG symptoms. A placebo is a medication that looks like the study medication but has no active ingredient. Immunoglobulin is an antibody produced by white blood cells that is used by the immune…
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Study of Talimogene Laherparepvec (T-VEC) and Radiation in Soft Tissue Sarcoma

Condition: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The purpose of this study is to evaluate the effectiveness of preoperative radiation therapy used in combination with the immunostimulatory agent talimogene laherparepvec (T-VEC) to improve outcomes for patients with resectable soft tissue sarcomas, including soft tissue sarcoma (liposarcoma (excluding myxoid liposarcoma), leiomyosarcoma and…
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Study of Drug in Patients with Progressive Supranuclear Palsy

Condition: Neurological Disorders / Neuromuscular
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a study for persons with a diagnosis of Progressive Supranuclear Palsy, involving an experimental treatment.
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ADI-PEG 20 in Combination With Gemcitabine and Docetaxel for the Treatment of Bone and Soft Tissue Sarcoma

Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this research study is to learn more about adding the drug ADI-PEG 20 to a standard treatment regimen called gem-tax (the chemotherapy drugs gemcitabine and docetaxel) for subjects with bone and soft tissue sarcoma, and how the addition of ADI-PEG 20 affects the tumors response to treatment and the side effects you experience.
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Efficacy and Safety of IGIV-C in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves the ability to taper participant's dose of corticosteroids without causing significant increase in your muscle weakness and worsening MG symptoms. A placebo is a medication that looks like the study medication but has no…
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Selinexor in Combination With Ixazomib for the Treatment of Advanced Sarcoma

Condition: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The purpose of this study is to evaluate the safety and effectiveness of a new combination treatment involving two drugs: selinexor and ixazomib, for the treatment of dedifferentiated liposarcoma, malignant peripheral nerve sheath tumor, alveolar soft part sarcoma and Ewing sarcoma. The primary objective of the study is to determine the maximum tolerated…
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A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
Small fiber neuropathy is a painful condition caused by damage to the small nerves in your arms andlegs. This damage causes burning, sharp-stabbing and aching pain, amongst other types of pain. It can be sometimes difficult to know the cause of the damage to the small nerves in your arms and legs. In most cases, treatment for small fiber neuropathy is…
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Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (Soft Tissue Sarcoma)

Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to determine the safety and effectiveness of the study drug nirogacestat for the treatment of desmoid tumors/aggressive fibromatosis (DT/AF). One group will receive the study medication, the other group will receive a placebo. After the initial phase of the study, participants may elect to participate in another phase of the…
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A study for patients with ALS using study drug NP001

Condition: Neurological Disorders / Neuromuscular
Investigator: Jinsy Andrews, MD, MSc
Status: Closed
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…
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Tazemetostat in Combination With Doxorubicin as Therapy for Advanced Epithelioid Sarcoma

Condition: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The purpose of this study is to learn about the effects, safety, and proper dosage of tazemetostat in combination with doxorubicin for the treatment of soft tissue sarcoma. Doxorubicin is a common therapy for soft tissue sarcoma, but is not FDA approved in combination with tazemetostat. This study treatment combination may suppress or prevent tumor growth…
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A Study of AL3818 (Anlotinib) in Patients with Metastatic or Advanced Alveolar Soft Part Sarcoma, Leiomyosarcoma and Synovial Sarcoma

Condition: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The researchers hope to study the effectiveness of an investigational drug called AL3818 in people who have metastatic (meaning their disease has spread) or advanced ), specifically alveolar soft partsarcoma (ASPS), leiomyosarcoma (LMS) and synovial sarcoma (SS). The main purpose of the study isto learn the percentage of patients that respond to treatment…
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Study of HuMax-AXL-ADC in Patients with Sarcoma

Condition: Cancer / Sarcoma
Investigator: Brian Henick, MD
Status: Closed
The purpose of this study is to establish the safety profile of the study drug HuMax-AXL-ADC in a mixed population of patients with specified solid tumors. HuMax-AXL-ADC binds to a specific antigen called Axl, which is expressed on the tumor cells in the cancer types treated in this trial. When the trial drug binds to the antigen on the tumor cell, the…
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Study of Drug (INCB059872) in Participants with Relapsed or Refractory Ewing Sarcoma

Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The main purpose of this research study is to test different doses of INCB059872 and see which doses are safe and tolerable in participants that have Ewing sarcoma. This study will research the effect that INCB059872 has on your cancer. Researchers will study the way INCB059872 come into and leave your body over time (pharmacokinetics). INCB059872 is a…
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Safety, Tolerability and Pharmacokinetics of an Anti-PD-1 Monoclonal Antibody in Subjects With Advanced Malignancies

Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to determine if the study drug toripalimab is safe, to determine future doses to give to patients with cancer, and to measure the amount of toripalimab in the blood. Up to approximately 240 subjects will be enrolled in Part B. The dose given in Part B will be 240 mg. The dose was chosen based upon safety data obtained from…
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