Research Studies and Clinical Trials Search
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Acute Leukemia or Myelodysplastic Syndrome: A study comparing matched unrelated donor versus haploidentical related myeloablative hematopoietic cell transplantation
This study is for children and young adults: -Ages 6 months to 21 years old -With a diagnosis of acute leukemia or myelodysplastic syndrome (MDS)...
This study is for children and young adults: -Ages 6 months to 21 years old -With a diagnosis of acute leukemia or myelodysplastic syndrome (MDS) This study is being done to learn more about hematopoietic cell transplantation (HCT) that use mismatched related donor (haploidentical or haplo) versus a matched unrelated donor (MUD). The research team will use bone marrow or stem cells from a mismatched related family donor or from a MUD and then compare the transplant results.
Acute Myeloid Leukemia (AML): A clinical study of enasidenib to treat relapsed/refractory AML with IDH-2 mutations
This study is for: • Patients who are between the age of 2 and 18 years old • Have been diagnosed with Acute Myeloid Leukemia (AML) that has relapsed (has come back) or is refractory (has not responded to earlier treatment)...
This study is for: • Patients who are between the age of 2 and 18 years old • Have been diagnosed with Acute Myeloid Leukemia (AML) that has relapsed (has come back) or is refractory (has not responded to earlier treatment) • The AML also must have a specific genetic change, also called a mutation, in a protein called IDH2 The overall goal of this study is to find out what effects, good and/or bad, the drug enasidenib has on children and adolescents with AML with IDH2 mutations.
Acute Myeloid Leukemia (AML): A study of chemotherapy with or without venetoclax in children with relapsed AML without FLT3/ITD mutation
This study is for: • Patients who have enrolled in APAL2020SC • Who are greater than 28 days old to less than 22 years old • Who have been diagnosed with acute myeloid leukemia without FLT3/ITD mutation that has relapsed (has come back)...
This study is for: • Patients who have enrolled in APAL2020SC • Who are greater than 28 days old to less than 22 years old • Who have been diagnosed with acute myeloid leukemia without FLT3/ITD mutation that has relapsed (has come back) This is a randomized trial (like the flip of a coin) to find what effects, good and/or bad, chemotherapy with venetoclax verus without venetoclax has on children and young adults with AML.
B-ALL: Eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults
This study is being done children and adults. • Between 1-30 years old for one of the groups • Diagnosed with B-cell acute lymphoblastic leukemia (B-ALL) There are two groups in this study. For the treatment group, patients age 1 through 30 are eligible. In this group...
This study is being done children and adults. • Between 1-30 years old for one of the groups • Diagnosed with B-cell acute lymphoblastic leukemia (B-ALL) There are two groups in this study. For the treatment group, patients age 1 through 30 are eligible. In this group, researchers would like to know if not giving a child total body irradiation (also called TBI) therapy before a child's transplant is as good as giving a child radiation before transplant and a child's growth and development will not be affected by radiation. For the observation group, all patients are eligible. The purpose of this part of the study is to use the NGS-MRD test to see if the patient would qualify for the Non-TBI Group, and to gather more research data.
B-Cell Acute Lymphoblastic Leukemia (B-ALL): A clinical trial to study inotuzumab ozogamicin (InO) in combination with chemotherapy in children and young adults with relapsed or refractory B-ALL
This study is for: • Patients who are between the ages of 1 year and 22 years old • Who have been diagnosed with B-lymphoblastic leukemia (B-ALL) that is refractory (has not responded to earlier treatment) or is in second or greater relapse (has come back)...
This study is for: • Patients who are between the ages of 1 year and 22 years old • Who have been diagnosed with B-lymphoblastic leukemia (B-ALL) that is refractory (has not responded to earlier treatment) or is in second or greater relapse (has come back) • The ALL also must have a specific protein present called CD22 The overall goal of this study is to find out what effect, good and/or bad, the study drug inotuzumab ozogamicin (InO) in combination with chemotherapy has on children and young adults with relapsed or refractory B-ALL. The treatment used in this study involves cancer fighting medicine called chemotherapy plus the medicine InO. The treatment on this study takes about 2 months, divided into 2 phases of therapy.
B-Cell Acute Lymphoblastic Leukemia (B-ALL): A study eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults
There are two groups involved in this study, Observational and Treatment. Patients of any age diagnosed with B-Cell acute lymphoblastic leukemia (B-ALL), T-cell acute lymphoblastic leukemia (T-ALL), or Mixed phenotype acute leukemia (MPAL) may join the Observational group. B-ALL...
There are two groups involved in this study, Observational and Treatment. Patients of any age diagnosed with B-Cell acute lymphoblastic leukemia (B-ALL), T-cell acute lymphoblastic leukemia (T-ALL), or Mixed phenotype acute leukemia (MPAL) may join the Observational group. B-ALL patients 1 to 31 years of age requiring hematopoietic cell transplantation and NGS-MRD negative may join the Treatment group. In this study, researchers would like to see if not giving patients total body irradiation (also called TBI) therapy before transplant is as good as giving patients radiation before transplant.
B-Cell Non-Hodgkin Lymphoma (B-NHL): A study of glofitumab with or without chemotherapy in children with relapsed or refractory mature B-NHL
This study is for: • Part 1: Patients who are greater than 6 months old to less than 18 years old • Part 2: Patients who are greater than 6 months old to less than 31 years old • Who have been diagnosed with mature B-Cell non-Hodgkin lymphoma that has relapsed (has...
This study is for: • Part 1: Patients who are greater than 6 months old to less than 18 years old • Part 2: Patients who are greater than 6 months old to less than 31 years old • Who have been diagnosed with mature B-Cell non-Hodgkin lymphoma that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The goal of this study are to find what effects, good and/or bad, the drug glofitumab with or without chemotherapy has on children and young adults with relapsed or refractory mature B-NHL. Part 1 is looking learn more about the pharmacology (for example, the best dose) of the medication. Part 2 increases the number of participants based on what is learned from Part 1.
Bone Cancer: A study comparing two surgery methods to treat cancer in the lung
This study is for: • Patients less than 50 years old • Who have osteosarcoma (OST), a type of bone cancer, that has spread into the lungs...
This study is for: • Patients less than 50 years old • Who have osteosarcoma (OST), a type of bone cancer, that has spread into the lungs The overall goal of this study is to compare the effects, good and/or bad, of two different surgery methods to find out which is better. The two types of surgeries are called open thoracic surgery and thoracoscopy. Study patients will be randomly assigned to one of the two types of surgery.
Brain Tumors: A study to see if memantine protects the brain during radiation therapy treatment in children
This study is for: • Patients ages 4 to 18 • Who have been diagnosed with a brain tumor...
This study is for: • Patients ages 4 to 18 • Who have been diagnosed with a brain tumor The goal of this study is to see if the drug memantine makes a difference in changes to attention, memory, or other thought processes (called cognitive function changes) in children and adolescents treated with radiation therapy for a brain tumor. In this study participants will get 1 of 2 treatments: memantine (active drug) or placebo (no active drug). The treatment (memantine or placebo) participants receive is decided by a process called randomization, which means that the treatment is assigned based on chance like flipping a coin. Participants will also complete questionnaires and short computer testing sessions.
Cancer or Cancer Related Illness: Blood and/or tissue samples collected and stored for future research
Patients with a suspected or confirmed oncology or bone marrow failure diagnosis are eligible to participate. If enrolled, blood and/or tissue samples will be collected, deidentified, and stored for use in future research projects. De-identified clinical data will also be...
Patients with a suspected or confirmed oncology or bone marrow failure diagnosis are eligible to participate. If enrolled, blood and/or tissue samples will be collected, deidentified, and stored for use in future research projects. De-identified clinical data will also be collected for future research.
Cancer with ALK or ROS1 mutations: A study of ensartinib as treatment for tumors
This study is for: • Patients who have enrolled in APEC1621SC • Who are between the ages of 12 months old and 21 years old • Have been diagnosed with cancer that is relapsed (has come back) or is refractory (has not responded to earlier...
This study is for: • Patients who have enrolled in APEC1621SC • Who are between the ages of 12 months old and 21 years old • Have been diagnosed with cancer that is relapsed (has come back) or is refractory (has not responded to earlier treatment) • The cancer also must have a specific genetic change, also called a mutation, in a gene called ALK or ROS1 • Are able to swallow pills The medicine, ensartinib, has been shown to block the growth of cancer cells with ALK or ROS1 in laboratory experiments. Early studies with ensartinib have been conducted in adults. The study drug is taken by mouth for up to 24 months. The goal of this study is to find what effects ensartinib has on a child's tumor, any side effects it may cause, and to learn more about the pharmacology of the medication.
Cancer: A study screening for genetic changes (mutations) that may respond to specific drug therapies for tumors
This study is for: • Patients who are between the ages of 12 months and 21 years old • Who have been diagnosed with solid tumors that have relapsed (have come back) or are refractory (have not responded to earlier treatment)...
This study is for: • Patients who are between the ages of 12 months and 21 years old • Who have been diagnosed with solid tumors that have relapsed (have come back) or are refractory (have not responded to earlier treatment) The goal of this study is to find better ways to diagnose and treat relapsed or refractory tumors in children, adolescents, and young adults. The study team will collect some information about each participant's cancer and treatment. The study team will also collect extra tumor, blood, and/or other tissue from procedures done by doctors to help make a diagnosis and learn more about different cancers now and in the future. If the study team finds out that the leukemia has a specific genetic change (mutation) that may respond to a drug that "targets" those changes, they may present a treatment study using that specific drug to the participant. This combination of a tumor with a mutation and a drug that aims at that mutation is called a "match."
Cancer: An informational registry and tissue/blood storage bank for future research
This study is available for those who are 25 years old or younger. The goal of this study is to find better ways to diagnose, treat and prevent cancers in children, adolescents and young adults. We will collect some information about each participant's cancer and treatment. We...
This study is available for those who are 25 years old or younger. The goal of this study is to find better ways to diagnose, treat and prevent cancers in children, adolescents and young adults. We will collect some information about each participant's cancer and treatment. We will also collect extra tumor, blood, bone marrow, or other tissue from procedures done by doctors to help make a diagnosis and learn more about different cancers now and in the future.
Chemotherapy-induced vomiting: A study of Olanzapine in children receiving chemotherapy for Hematopoietic Stem Cell Transplant conditioning
Patients 2.5 to 18 years of age and are about to receive chemotherapy before a blood or bone marrow transplant may join this study. Chemotherapy drugs are used to prepare children for blood or bone marrow transplant. A few of the side effects of these drugs are nausea and...
Patients 2.5 to 18 years of age and are about to receive chemotherapy before a blood or bone marrow transplant may join this study. Chemotherapy drugs are used to prepare children for blood or bone marrow transplant. A few of the side effects of these drugs are nausea and vomiting. The purpose of this research study is to find out whether adding the drug olanzapine to standard antiemetics (drugs that help prevent vomiting) will be helpful in controlling chemotherapy-induced vomiting (CINV). To find this out, some of the participants in this study will receive olanzapine and others will receive a placebo (a substance that looks like olanzapine but does not have any active or medicinal ingredients). This will allow us to compare treatment of CINV with and without olanzapine.
Craniopharyngioma: A Study Identifying Clinically Relevant Biological Targets for Medical Therapy
This study is being done for children and adults: • 1 to 21 years old. • Diagnosed with craniopharyngioma...
This study is being done for children and adults: • 1 to 21 years old. • Diagnosed with craniopharyngioma. This study wants to learn what might be the best treatment for children with a tumor called craniopharyngioma. The main goal of this study is to collect extra tissue at the time of surgery and medical information after surgery. Researchers will also collect and test a small amount of blood.
Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG): A study of Selinexor with Radiation Therapy
This study is for children and young adults: -Ages 12 months to 21 years old -With a new diagnosis of high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG)...
This study is for children and young adults: -Ages 12 months to 21 years old -With a new diagnosis of high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG). This study is being done to find the highest and safest dose of selinexor with radiation therapy.
Germ Cell Tumors (GCT): A study comparing accelerated versus standard chemotherapy
Patients who are ages 11 to 45 years with germ cell tumors (GCT) may be eligible to participate in this study. In this study, researchers want to determine whether accelerated BEP (standard chemotherapy drugs Bleomycin, Etoposide, and cisPlatin) will be effective and well...
Patients who are ages 11 to 45 years with germ cell tumors (GCT) may be eligible to participate in this study. In this study, researchers want to determine whether accelerated BEP (standard chemotherapy drugs Bleomycin, Etoposide, and cisPlatin) will be effective and well-tolerated for patients with advanced GCTs. Researchers don't know yet if accelerated treatment is helpful in advanced GCTs and are hoping this study will answer that question.
Germ Cell Tumors (GCT): Monitoring patients with low risk GCT and using the drug carboplatin instead of cisplatin for patients with standard risk GCT.
Eligible patients must be diagnosed with having a germ cell tumor (GCT). Patients who are diagnosed with Stage I GCT may be eligible no matter the age. Those ages 0-25 who are diagnosed with Stage II-IV may be eligible depending on the location of the GCT. Researchers want to see...
Eligible patients must be diagnosed with having a germ cell tumor (GCT). Patients who are diagnosed with Stage I GCT may be eligible no matter the age. Those ages 0-25 who are diagnosed with Stage II-IV may be eligible depending on the location of the GCT. Researchers want to see if monitoring patients with low risk germ cell tumors after their tumor is removed is as effective as following surgery with chemotherapy and/or more surgery. Researchers also want to compare the effects of carboplatin with cisplatin on patients with standard risk germ cell tumors to find out which is better.
High Risk Medulloblastoma: A clinical study of elfornithine/DFMO as maintenance therapy after standard treatment
This study is for: • Patients who are between the ages of 0 and 21 years old • Who have been diagnosed with molecular high risk medulloblastoma, very high risk medulloblastoma, or medulloblastoma that has relapsed (has come back) or is refractory (has not responded to earlier...
This study is for: • Patients who are between the ages of 0 and 21 years old • Who have been diagnosed with molecular high risk medulloblastoma, very high risk medulloblastoma, or medulloblastoma that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The goal of this research study is to find the effects, good and/or bad, the investigational drug eflornithine HCl (DFMO) has on high risk and very high risk medulloblastoma. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to either keep the tumors in remission or, if for active tumors, for the tumors to respond to the treatment. It will also look at the safety and tolerability of DFMO.
High-grade Glioma (HGG) with BRAFV600-mutation: A clinical trial of dabrafenib and trametinib after radiation therapy
This study is for: • People who are ages 3 through 21 • Who are newly diagnosed with high-grade glioma...
This study is for: • People who are ages 3 through 21 • Who are newly diagnosed with high-grade glioma (HGG) with BRAFV600-mutation High-grade gliomas (HGGs) are hard to treat successfully. Two drugs that will be used in this study are dabrafenib and trametinib. This study will look at how well the combination of dabrafenib and trametinib works when given to children and young adults with HGGs after they receive radiation therapy. The use of this combination to treat pediatric brain tumors is experimental.
High-Risk Neuroblastoma (HRNBL): A Study of the Addition of Dinutuximab to Therapy
This study is being done for children and adults: • 30 years old or younger. • Diagnosed with High-Risk Neuroblastoma (HRNBL)...
This study is being done for children and adults: • 30 years old or younger. • Diagnosed with High-Risk Neuroblastoma (HRNBL) The overall goal of this study is to compare the outcomes of dinutuximab given early with Induction therapy to the effects of standard Induction therapy with the addition of dinutuximab later in therapy in people with HRNBL to find out which is better.
Hodgkin Lymphoma: A study to compare standard therapy to the use of two drugs, Brentuximab vedotin and Nivolumab
This study is being done for children and adults: • 5 to 60 years old. • Diagnosed with Hodgkin lymphoma (HL)...
This study is being done for children and adults: • 5 to 60 years old. • Diagnosed with Hodgkin lymphoma (HL) The overall goal of this study is to compare the effects, good and/or bad, of brentuximab vedotin and nivolumab against standard therapy for people with HL to find out which is better.
Infant Leukemia: A study of revumenib (SNDX-5613) in combination with chemotherapy in infants with relapsed or refractory KMT2A-rearranged infant leukemia
This study is for: • Patients ages 1 month to less than 6 years old • Who have been diagnosed with KMT2A-rearranged infant leukemia that has relapsed (has come back) or is refractory (has not responded to earlier treatment)...
This study is for: • Patients ages 1 month to less than 6 years old • Who have been diagnosed with KMT2A-rearranged infant leukemia that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The medicine, revumenib (SNDX-5613), has been shown to specifically kill these cancer cells with KMT2A rearrangement in laboratory experiments. Early studies with revumenib have been conducted in adults and pediatrics. The goals of this study are to find what effects, good and/or bad, the drug revumenib in combination with chemotherapy has on infants with relapsed or refractory leukemia and to learn more about the pharmacology (for example, the best dose) of the medication.
Langerhans Cell Histiocytosis (LCH): Treatment study for Children and Adolescents
This study is being done for children and adolescents: • age 17 or younger. • Diagnosed with Langerhans Cell Histiocytosis (LCH)...
This study is being done for children and adolescents: • age 17 or younger. • Diagnosed with Langerhans Cell Histiocytosis (LCH) The purpose of this research study is to find out if extending the treatment even further (from 12 months to 24 months) and adding another drug called 6-mercaptopurine will result in fewer LCH relapses (when the disease comes back) and fewer long-term disease-related problems. Study patients will receive study treatment for about 12 or 24 months. After treatment, study patients will then be followed for at least 5 years.
Leukemia & Lymphoma: A study of tagraxofusp with or without chemotherapy in pediatric patients with relapsed or refractory CD123 expressing hematologic malignancies
This study is for: • Patients who are between the ages of 1 and 21 years old • Who have been diagnosed with a hematologic maligancy (including, but not limited to acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia...
This study is for: • Patients who are between the ages of 1 and 21 years old • Who have been diagnosed with a hematologic maligancy (including, but not limited to acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia, acute undifferentiated leukemia, blastic plasmacytoid dendritic cell neoplasm, Hodgkin lymphoma, and non-Hodgkin lymphoma) that has relapsed (has come back) or is refractory (has not responded to earlier treatment). • The cancer also must have a specific protein present called CD123 The goal of this study is to improve survival rates in children and young adults with relapsed hematological malignancies, determine the recommended phase 2 dose (RP2D) of tagraxofusp given alone and in combination with chemotherapy, as well as to find the effects, both good and/or bad, tagraxofusp has in pediatric patients.
Leukemia or Lymphoma: A study to see if levocarnitine protects the liver from chemotherapy
This study is being done for children and adults: • Ages 15-40. • Diagnosed with B-ALL, T-ALL, Lymphoblastic Lymphoma (LLy), or Mixed-Phenotype Acute Leukemia/Lymphoma (MPAL)...
This study is being done for children and adults: • Ages 15-40. • Diagnosed with B-ALL, T-ALL, Lymphoblastic Lymphoma (LLy), or Mixed-Phenotype Acute Leukemia/Lymphoma (MPAL). The study involves taking the drug levocarnitine during standard of care chemotherapy. The overall goal of this study is to determine whether taking levocarnitine will reduce the rate of severe liver damage caused by asparaginase chemotherapy during the first month of treatment.
Leukemia: An expanded access program of revumenib (SNDX-5613) in patients with relapsed or refractory leukemia with genetic alterations associated with HOXA overexpression
This expanded access program is for: • Patients who are older than 6 months old • Who have been diagnosed with acute leukemia with a gene alternation that has been associated with HOXA overexpression that has relapsed (has come back) or is refractory (has not responded to...
This expanded access program is for: • Patients who are older than 6 months old • Who have been diagnosed with acute leukemia with a gene alternation that has been associated with HOXA overexpression that has relapsed (has come back) or is refractory (has not responded to earlier treatment). • Who are not otherwise eligible to participate in other Syndax-sponsored clinical studies and have no approved treatment options. This expanded access program will provide revumenib in a controlled clinical setting.
Leukemia: An informational registry & study screening for genetic changes (mutations) that may respond to specific drug therapies for relapsed or refractory leukemia
This study is for: • Patients less than 22 years old • Who have a known or suspected diagnosis of relapsed (has come back) or refractory (has not responded to earlier treatment) leukemia...
This study is for: • Patients less than 22 years old • Who have a known or suspected diagnosis of relapsed (has come back) or refractory (has not responded to earlier treatment) leukemia The goal of this study is to find better ways to diagnose and treat relapsed or refractory leukemia in children, adolescents and young adults. The Study Team will collect some information about each participant's cancer and treatment. The Study Team will also collect extra blood and/or bone marrow from procedures done by doctors to help make a diagnosis and learn more about different cancers now and in the future. If the Study Team finds out that the leukemia has a specific genetic change (mutation) that may respond to a drug that "targets" those changes, they may present a treatment study using that specific drug to the participant.
Low-Grade Glioma: A Study of the Drugs Selumetinib vs. Carboplatin/Vincristine
Study participants must be ages 2 through 21 and newly diagnosed or Previously Untreated Low-Grade Glioma (LGG). In this study, researchers want to if selumetinib works just as well as the standard treatment using carboplatin and vincristine (called CV) for patients with LGG...
Study participants must be ages 2 through 21 and newly diagnosed or Previously Untreated Low-Grade Glioma (LGG). In this study, researchers want to if selumetinib works just as well as the standard treatment using carboplatin and vincristine (called CV) for patients with LGG. Selumetinib is a drug that works by blocking a protein (a basic building block of the human body) that lets cancer cells grow without stopping. Early studies have shown that selumetinib was successful in treating patients with LGG that came back after a first attempt at treatment. However, the use of selumetinib in patients with previously-untreated LGG is considered experimental because selumetinib has not yet been FDA approved for this type of cancer.
Neuroblastoma: A clinical trial of elfornithine/DFMO and etoposide for relapsed/refractory neuroblastoma
This study is for: • Patients who are between the ages of 0 and 31 years old • Who have been diagnosed with neuroblastoma that has relapsed (has come back) or is refractory (has not responded to earlier treatment)...
This study is for: • Patients who are between the ages of 0 and 31 years old • Who have been diagnosed with neuroblastoma that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The goal of this study is to find what effects, good and/or bad, the investigational drug eflornithine HCl (difluoromethylornithine DFMO) along with etoposide has on neuroblastoma. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). Currently there are no standard of care treatments for patients with neuroblastoma in remission or with relapsed/refractory neuroblastoma. This study will look at the ability of this study drug to either keep the tumors in remission or, if for active tumors, for the tumors to respond to the treatment. The study will also look at the safety and tolerability of DFMO.
Neuroblastoma: A study of DFMO as maintenance therapy
This study is for: • Patients who are between the ages of 0 and 21 years old • Who have been diagnosed with high-risk neuroblastoma (HRNB) that is now in remission and no longer seen as active disease on scans and in bone marrow samples...
This study is for: • Patients who are between the ages of 0 and 21 years old • Who have been diagnosed with high-risk neuroblastoma (HRNB) that is now in remission and no longer seen as active disease on scans and in bone marrow samples. • Who have completed prior treatment for HRNB The goal this study is to find what effects, good and/or bad, the investigational drug eflornithine HCl (DFMO) has on neuroblastoma that is in remission (cured according to medical tests). An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to keep tumors in remission and will also look at the safety and tolerability of DFMO.
Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG) in Children: A study of selumetinib versus standard treatment
Study participants must be ages 2 through 21, and have been diagnosed with Neurofibromatosis type 1 (NF1) and a cancer called low-grade glioma (LGG). In this study, researchers would like to see if a drug called Selumetinib works just as well as the standard treatment for NF1...
Study participants must be ages 2 through 21, and have been diagnosed with Neurofibromatosis type 1 (NF1) and a cancer called low-grade glioma (LGG). In this study, researchers would like to see if a drug called Selumetinib works just as well as the standard treatment for NF1-associated LGG. Researchers would also like to see if selumetinib is better than the standard treatment in improving vision. Selumetinib is a drug that works by blocking proteins (a basic building block of the human body) needed for cell growth and killing cancer cells. The use of selumetinib in the treatment of previously untreated NF1-associated LGG is considered experimental because selumetinib has not yet been FDA approved for this type of cancer.
NGGCT Brain Cancer (Non-Germinomatous Central Nervous System Germ Cell Tumor): A clinical trial of radiation therapy compared to standard treatment
This clinical study is available for those ages 3 to 30 who have been diagnosed with a type of brain cancer called NGGCT and your tumor is "localized". The term localized means that the tumor has not spread to other parts of your brain or body. One goal for this study is to see...
This clinical study is available for those ages 3 to 30 who have been diagnosed with a type of brain cancer called NGGCT and your tumor is "localized". The term localized means that the tumor has not spread to other parts of your brain or body. One goal for this study is to see if treatment with RT to the spine and a portion of the brain works just as well as the standard treatment for people with localized NGGCT whose disease responds well to induction chemotherapy, or who have no signs of disease following surgery. The other goal is to see if we can lower the number of relapses in the spine for patients who receive RT to the spine and a portion of the brain, as compared to patients on previous studies who received RT to a portion of the brain only. Treatment is divided into 2 phases of therapy. In the first phase, participants will receive induction chemotherapy. In the second phase, participants will receive RT or additional chemotherapy plus RT. Some people on this study will also have surgery before the second phase of treatment to remove any tumor that remains after the first phase of treatment.
Primary Mediastinal B-cell lymphoma (PMBCL): A study combining nivolumab and chemo-immunotherapy as a treatment option
This study is for children: • 2 years old and older • Received a diagnosis of primary mediastinal B-cell lymphoma (PMBCL)...
This study is for children: • 2 years old and older • Received a diagnosis of primary mediastinal B-cell lymphoma (PMBCL) The goal of this study is to compare the effects, good and/or bad, of nivolumab with chemo-immunotherapy versus chemo-immunotherapy alone. In this study children will get 1 of 2 treatment plans.
Recurrent or Progressive Low-Grade Glioma: A Study to Compare Treatment with the Drug Selumetinib Alone vs. Selumetinib and Vinblastine
Study participants must be ages 2 through 25 and are suspected of having progressive or recurrent low-grade glioma (LGG). This study looks at how well selumetinib and vinblastine work when given together to children and young adults with recurrent or progressive LGGs. Vinblastine...
Study participants must be ages 2 through 25 and are suspected of having progressive or recurrent low-grade glioma (LGG). This study looks at how well selumetinib and vinblastine work when given together to children and young adults with recurrent or progressive LGGs. Vinblastine is often given to children with progressive or recurrent LGGs. Selumetinib is a drug that works by blocking a protein (a basic building block of the human body) that lets cancer cells grow without stopping. Early studies with selumetinib have had some success in treating patients with LGG that came back after a first attempt at treatment. However, the use of selumetinib in children and young adults with recurrent or progressive LGG is considered experimental because selumetinib has not yet been FDA approved for this type of cancer.
Severe combined immunodeficiency (SCID): A study to see how different amounts of a drug called busulfan help improve the immune system of infants after a stem cell transplant
This study is being done for infants: •Diagnosed with severe combined immunodeficiency (SCID). Researchers want to find out if lower doses of a chemotherapy drug called busulfan before stem cell transplant can help...
This study is being done for infants: •Diagnosed with severe combined immunodeficiency (SCID). Researchers want to find out if lower doses of a chemotherapy drug called busulfan before stem cell transplant can help patients with Severe Combined Immune Deficiency (SCID).
Sickle Cell Disease and Other Hemoglobinopathies: A Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow
This study is for children and adults: • 1-70 years old • Undergoing transplant...
This study is for children and adults: • 1-70 years old • Undergoing transplant. This research study is done to collect data on a type of bone marrow stem cell transplant for patients with sickle cell anemia or severe hemoglobinopathy such as thalassemia. Being in this study includes a review of patient medical history and completing questionnaires.
Solid and CNS Tumors: A study of tiragolumab and atezolizumab in patients with relapsed or refractory SMARCB1 or SMARCA4 deficient tumors
This study is for: • Patients who are either older than or equal to 1 year old • Who have been diagnosed a tumor that is SMARCB1 (INI1) or SMARCA4 deficient that has relapsed (has come back) or is refractory (has not responded to earlier treatment)...
This study is for: • Patients who are either older than or equal to 1 year old • Who have been diagnosed a tumor that is SMARCB1 (INI1) or SMARCA4 deficient that has relapsed (has come back) or is refractory (has not responded to earlier treatment) • The specific types of tumors for this study include: - Renal medullary carcinoma - Malignant rhabdoid tumor (extra-CNS) - Atypical teratoid rhabdoid tumor (CNS) - Poorly differentiated chordoma - Epithelioid sarcoma - Other SMARCB1 or SMARCA4 deficient tumors The goal of this study is to find what effects, good and/or bad, the drugs tiragolumab and atezolizumab have on patients with relapsed or refractory solid or CNS tumors. Note: Children's Mercy is not participating in Part A of this study.
Solid Tumors: A study of CBL0137 for relapsed or refractory solid tumors
This study is for: • Part B1: - Patients who are between the ages of 1 and 21 years old - Who have been diagnosed with progressive (growing) or recurrent (has come back) diffuse intrinsic pontine...
This study is for: • Part B1: - Patients who are between the ages of 1 and 21 years old - Who have been diagnosed with progressive (growing) or recurrent (has come back) diffuse intrinsic pontine glioma (DIPG) or other diffuse midline gliomas that has a mutation in the H3 K27M gene • Part B2: - Patients who are between the ages of 1 and 30 years old - Who have been diagnosed with osteoscaroma that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The goal of this study is to find what effects, good and/or bad, the drug CBL0137 has on children and young adults with relapsed or refractory solid tumors. Note: Children's Mercy is not participating in Part A of this study.
Wilms Tumors: A study of Combination Chemotherapy
This study is being done for children and adults: • 30 years old or younger. • Diagnosed with Diffuse Anaplastic Wilms Tumor (DAWT) or Relapsed Favorable Histology Wilms Tumor (FHWT)...
This study is being done for children and adults: • 30 years old or younger. • Diagnosed with Diffuse Anaplastic Wilms Tumor (DAWT) or Relapsed Favorable Histology Wilms Tumor (FHWT) In this study, we want to find out if we can improve treatment for patients with newly diagnosed DAWT and those with high-risk or very high-risk relapsed FHWT. Patients on study will be given a combination of chemotherapy drugs plus radiation therapy.