159 matching studies

Sponsor Condition of Interest
Nanoparticle Albumin-Bound Rapamycin, Temozolomide, and Irinotecan Hydrochloride in Treating Pediatric...
Children's Oncology Group Childhood Solid Neoplasm Recurrent Central Nervous System Neoplasm Recurrent Malignant Solid Neoplasm Refractory Central Nervous System Neoplasm Refractory Malignant Solid Neoplasm
This phase I trial studies the side effects and best dose of nanoparticle albumin-bound rapamycin when given together with temozolomide and irinotecan hydrochloride in treating pediatric patients with solid tumors that have come back after a period of time during which the tumor... expand

This phase I trial studies the side effects and best dose of nanoparticle albumin-bound rapamycin when given together with temozolomide and irinotecan hydrochloride in treating pediatric patients with solid tumors that have come back after a period of time during which the tumor could not be detected or has not responded to treatment. Nanoparticle albumin-bound rapamycin may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as temozolomide and irinotecan hydrochloride, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving nanoparticle albumin-bound rapamycin, temozolomide, and irinotecan hydrochloride may work better in treating pediatric patients with solid tumors.

Type: Interventional

Start Date: Jul 2017

open study

Azacitidine and Combination Chemotherapy in Treating Infants With Acute Lymphoblastic Leukemia and KMT2A...
National Cancer Institute (NCI) Acute Leukemia of Ambiguous Lineage B Acute Lymphoblastic Leukemia KMT2A Gene Rearrangement Mixed Phenotype Acute Leukemia
This pilot phase II trial studies the side effects of azacitidine and combination chemotherapy in infants with acute lymphoblastic leukemia and KMT2A gene rearrangement. Drugs used in chemotherapy, such as methotrexate, prednisolone, daunorubicin hydrochloride, cytarabine, dexamethasone,... expand

This pilot phase II trial studies the side effects of azacitidine and combination chemotherapy in infants with acute lymphoblastic leukemia and KMT2A gene rearrangement. Drugs used in chemotherapy, such as methotrexate, prednisolone, daunorubicin hydrochloride, cytarabine, dexamethasone, vincristine sulfate, pegaspargase, hydrocortisone sodium succinate, azacitidine, cyclophosphamide, mercaptopurine, leucovorin calcium, and thioguanine work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug may kill more cancer cells.

Type: Interventional

Start Date: Mar 2017

open study

HIV-1 Specific T -Cells (HST-NEETs) for HIV-Infected Individuals
Catherine Bollard HIV Infections
This is a phase I, multi-site, study of the safety, immunologic and virologic responses of ex vivo expanded HIV-1 multi-antigen specific T-cell therapy (HST-NEET) as a therapeutic strategy in HIV-infected individuals suppressed on antiretroviral therapy (ART). expand

This is a phase I, multi-site, study of the safety, immunologic and virologic responses of ex vivo expanded HIV-1 multi-antigen specific T-cell therapy (HST-NEET) as a therapeutic strategy in HIV-infected individuals suppressed on antiretroviral therapy (ART).

Type: Interventional

Start Date: Mar 2019

open study

Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin...
National Cancer Institute (NCI) Advanced Malignant Solid Neoplasm RB1 Positive Recurrent Childhood Ependymoma Recurrent Ewing Sarcoma Recurrent Glioma
This phase II Pediatric MATCH trial studies how well palbociclib works in treating patients with Rb positive solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with activating alterations (mutations) in cell cycle genes that have spread to other places in the body and... expand

This phase II Pediatric MATCH trial studies how well palbociclib works in treating patients with Rb positive solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with activating alterations (mutations) in cell cycle genes that have spread to other places in the body and have come back or do not respond to treatment. Palbociclib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Jun 2018

open study

Iobenguane I-131 or Crizotinib and Standard Therapy in Treating Younger Patients With Newly-Diagnosed...
Children's Oncology Group Ganglioneuroblastoma INRG Stage L2 INRG Stage M INRG Stage MS Neuroblastoma
This phase III trial studies iobenguane I-131 or crizotinib and standard therapy in treating younger patients with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma. Radioactive drugs, such as iobenguane I-131, may carry radiation directly to tumor cells and not... expand

This phase III trial studies iobenguane I-131 or crizotinib and standard therapy in treating younger patients with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma. Radioactive drugs, such as iobenguane I-131, may carry radiation directly to tumor cells and not harm normal cells. Crizotinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving iobenguane I-131 or crizotinib and standard therapy may work better in treating younger patients with neuroblastoma or ganglioneuroblastoma.

Type: Interventional

Start Date: May 2018

open study

Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma,...
National Cancer Institute (NCI) Advanced Malignant Solid Neoplasm Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma Deleterious ATM Gene Mutation Deleterious BRCA1 Gene Mutation
This phase II Pediatric MATCH trial studies how well olaparib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with defects in deoxyribonucleic acid (DNA) damage repair genes that have spread to other places in the body (advanced) and... expand

This phase II Pediatric MATCH trial studies how well olaparib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with defects in deoxyribonucleic acid (DNA) damage repair genes that have spread to other places in the body (advanced) and have come back (relapsed) or do not respond to treatment (refractory). Olaparib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Jul 2017

open study

Cockroach Immunotherapy in Children and Adolescents
National Institute of Allergy and Infectious Diseases (NIAID) Persistent Asthma
Scientific evidence has shown that, over the past two decades, the combination of cockroach allergy and cockroach exposure is one of the most important factors contributing to the dramatic increase in asthma morbidity seen in inner city children with asthma. Therefore, a major... expand

Scientific evidence has shown that, over the past two decades, the combination of cockroach allergy and cockroach exposure is one of the most important factors contributing to the dramatic increase in asthma morbidity seen in inner city children with asthma. Therefore, a major goal of the Inner City Asthma Consortium (ICAC) is to evaluate the efficacy of cockroach immunotherapy in inner city asthma. The primary objective of the study is to determine if asthma severity can be improved by cockroach subcutaneous immunotherapy (SCIT) treatment.

Type: Interventional

Start Date: Jul 2018

open study

A Study to Determine the Outcomes of Patients With Localized B Cell Lymphoblastic Lymphoma (B-LLy) When...
National Cancer Institute (NCI) B Acute Lymphoblastic Leukemia B Lymphoblastic Lymphoma Blasts More Than 25 Percent of Bone Marrow Nucleated Cells Down Syndrome
This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab,... expand

This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab, may induce changes in body's immune system and may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as vincristine, dexamethasone, prednisone, prednisolone, pegaspargase, methotrexate, cytarabine, mercaptopurine, doxorubicin, cyclophosphamide, and thioguanine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Leucovorin decreases the toxic effects of methotrexate. Giving monoclonal antibody therapy with chemotherapy may kill more cancer cells. Giving blinatumomab and combination chemotherapy may work better then combination chemotherapy alone in treating patients with B-ALL. This trial also assigns patients into different chemotherapy treatment regimens based on risk (the chance of cancer returning after treatment). Treating patients with chemotherapy based on risk may help doctors decide which patients can best benefit from which chemotherapy treatment regimens.

Type: Interventional

Start Date: Jun 2019

open study

PI3K/mTOR Inhibitor LY3023414 in Treating Patients With Relapsed or Refractory Advanced Solid Tumors,...
National Cancer Institute (NCI) Advanced Malignant Solid Neoplasm Ann Arbor Stage III Non-Hodgkin Lymphoma Ann Arbor Stage IV Non-Hodgkin Lymphoma Malignant Glioma Recurrent Central Nervous System Neoplasm
This phase II Pediatric MATCH trial studies how well PI3K/mTOR inhibitor LY3023414 works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with TSC or PI3K/MTOR mutations that have spread to other places in the body (metastatic) and have come... expand

This phase II Pediatric MATCH trial studies how well PI3K/mTOR inhibitor LY3023414 works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with TSC or PI3K/MTOR mutations that have spread to other places in the body (metastatic) and have come back (recurrent) or do not respond to treatment (refractory). PI3K/mTOR inhibitor LY3023414 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Jul 2017

open study

Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related...
Dana-Farber Cancer Institute Langerhans Cell Histiocytosis
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders. expand

This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.

Type: Interventional

Start Date: May 2015

open study

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory...
National Cancer Institute (NCI) Advanced Malignant Solid Neoplasm Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma Childhood Langerhans Cell Histiocytosis Histiocytic Sarcoma
This Pediatric MATCH screening and multi-sub-study phase II trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors, non-Hodgkin lymphomas, or histiocytic disorders that have progressed following at least one line of... expand

This Pediatric MATCH screening and multi-sub-study phase II trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors, non-Hodgkin lymphomas, or histiocytic disorders that have progressed following at least one line of standard systemic therapy and/or for which no standard treatment exists that has been shown to prolong survival. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with genetic changes or abnormalities (mutations) may benefit more from treatment which targets their tumor's particular genetic mutation, and may help doctors plan better treatment for patients with solid tumors or non-Hodgkin lymphomas.

Type: Interventional

Start Date: Jul 2017

open study

COMPASSION XT PAS - Post-approval Study of the SAPIEN XT THV in Patients With Pulmonary Valve Dysfunction
Edwards Lifesciences Pulmonary Valve Insufficiency Pulmonary Valve Stenosis Heart Defects, Congenital Congenital Abnormalities Cardiovascular Diseases
This study will confirm the safety and effectiveness of the Edwards Lifesciences SAPIEN XT Transcatheter Heart Valve (THV) System in patients with a dysfunctional right ventricular outflow tract (RVOT) conduit with a clinical indication for intervention in a post-market setting.... expand

This study will confirm the safety and effectiveness of the Edwards Lifesciences SAPIEN XT Transcatheter Heart Valve (THV) System in patients with a dysfunctional right ventricular outflow tract (RVOT) conduit with a clinical indication for intervention in a post-market setting.

Type: Interventional

Start Date: Jul 2017

open study

Axitinib and Nivolumab in Treating Patients With Unresectable or Metastatic TFE/Translocation Renal Cell...
National Cancer Institute (NCI) Metastatic Renal Cell Carcinoma Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions Stage III Renal Cell Cancer AJCC v8 Stage IV Renal Cell Cancer AJCC v8 TFE3 Gene Translocation
This phase II trial studies how well axitinib and nivolumab works in treating patients with TFE/translocation renal cell carcinoma that cannot be removed by surgery or has spread to other places in the body. Axitinib may stop the growth of tumor cells by blocking some of the... expand

This phase II trial studies how well axitinib and nivolumab works in treating patients with TFE/translocation renal cell carcinoma that cannot be removed by surgery or has spread to other places in the body. Axitinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving axitinib and nivolumab may work better in treating patients with TFE/translocation renal cell carcinoma compared to standard treatment, including surgery, chemotherapy, or immunotherapy.

Type: Interventional

Start Date: Oct 2018

open study

Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
Global Blood Therapeutics Sickle Cell Disease
This study consists of three parts, Parts A, B, and C. Part A is a single dose PK study in pediatric participants with Sickle Cell Disease. Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent Sickle Cell Disease participants who were 12-17 years... expand

This study consists of three parts, Parts A, B, and C. Part A is a single dose PK study in pediatric participants with Sickle Cell Disease. Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent Sickle Cell Disease participants who were 12-17 years of age. Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of GBT440 is pediatric participants with Sickle Cell Disease who are 4 to 17 years of age.

Type: Interventional

Start Date: May 2016

open study

Pembrolizumab in Treating Younger Patients With Recurrent, Progressive, or Refractory High-Grade Gliomas,...
National Cancer Institute (NCI) Constitutional Mismatch Repair Deficiency Syndrome Lynch Syndrome Malignant Glioma Progressive Ependymoma Progressive Medulloblastoma
This phase I trial studies the side effects and best dose of pembrolizumab and to see how well it works in treating younger patients with high-grade gliomas (brain tumors that are generally expected to be fast growing and aggressive), diffuse intrinsic pontine gliomas (brain... expand

This phase I trial studies the side effects and best dose of pembrolizumab and to see how well it works in treating younger patients with high-grade gliomas (brain tumors that are generally expected to be fast growing and aggressive), diffuse intrinsic pontine gliomas (brain stem tumors), brain tumors with a high number of genetic mutations, ependymoma or medulloblastoma that have come back, progressed, or have not responded to previous treatment. Monoclonal antibodies, such as pembrolizumab, may block tumor growth in different ways by targeting certain cells.

Type: Interventional

Start Date: May 2015

open study

Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk...
National Cancer Institute (NCI) Ganglioneuroblastoma High-Risk Neuroblastoma NMYC Gene Amplification
This phase II trial studies the side effects and how well dinutuximab and sargramostim work with combination chemotherapy in patients with high-risk neuroblastoma undergoing stem cell transplant. Immunotherapy with monoclonal antibodies, such as dinutuximab, may induce changes... expand

This phase II trial studies the side effects and how well dinutuximab and sargramostim work with combination chemotherapy in patients with high-risk neuroblastoma undergoing stem cell transplant. Immunotherapy with monoclonal antibodies, such as dinutuximab, may induce changes in the body's immune system and may interfere with the ability of tumor cells to grow and spread. Sargramostim helps the body produce normal infection-fighting white blood cells. Giving chemotherapy before a stem cell transplant, with drugs such as cisplatin, etoposide, vincristine, doxorubicin, cyclophosphamide, thiotepa, melphalan, etoposide, carboplatin, topotecan, and isotretinoin, helps kill any cancer cells that are in the body and helps make room in a patient's bone marrow for new blood-forming cells (stem cells). Giving dinutuximab and sargramostim with combination chemotherapy may work better in treating patients with high-risk neuroblastoma undergoing stem cell transplant.

Type: Interventional

Start Date: Jan 2019

open study

A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma
St. Jude Children's Research Hospital Medulloblastoma
Historically, medulloblastoma treatment has been determined by the amount of leftover disease present after surgery, also known as clinical risk (standard vs. high risk). Recent studies have shown that medulloblastoma is made up of distinct molecular subgroups which respond differently... expand

Historically, medulloblastoma treatment has been determined by the amount of leftover disease present after surgery, also known as clinical risk (standard vs. high risk). Recent studies have shown that medulloblastoma is made up of distinct molecular subgroups which respond differently to treatment. This suggests that clinical risk alone is not adequate to identify actual risk of recurrence. In order to address this, we will stratify medulloblastoma treatment in this phase II clinical trial based on both clinical risk (low, standard, intermediate, or high risk) and molecular subtype (WNT, SHH, or Non-WNT Non-SHH). This stratified clinical and molecular treatment approach will be used to evaluate the following: - To find out if participants with low-risk WNT tumors can be treated with a lower dose of radiation to the brain and spine, and a lower dose of the chemotherapy drug cyclophosphamide while still achieving the same survival rate as past St. Jude studies with fewer side effects. - To find out if adding targeted chemotherapy after standard chemotherapy will benefit participants with SHH positive tumors. - To find out if adding new chemotherapy agents to the standard chemotherapy will improve the outcome for intermediate and high risk Non-WNT Non-SHH tumors. - To define the cure rate for standard risk Non-WNT Non-SHH tumors treated with reduced dose cyclophosphamide and compare this to participants from the past St. Jude study. All participants on this study will have surgery to remove as much of the primary tumor as safely possible, radiation therapy, and chemotherapy. The amount of radiation therapy and type of chemotherapy received will be determined by the participant's treatment stratum. Treatment stratum assignment will be based on the tumor's molecular subgroup assignment and clinical risk. The participant will be assigned to one of three medulloblastoma subgroups determined by analysis of the tumor tissue for tumor biomarkers: - WNT (Strata W): positive for WNT biomarkers - SHH (Strata S): positive for SHH biomarkers - Non-WNT Non-SHH, Failed, or Indeterminate (Strata N): negative for WNT and SHH biomarkers or results are indeterminable Participants will then be assigned to a clinical risk group (low, standard, intermediate, or high) based on assessment of: - How much tumor is left after surgery - If the cancer has spread to other sites outside the brain [i.e., to the spinal cord or within the fluid surrounding the spinal cord, called cerebrospinal fluid (CSF)] - The appearance of the tumor cells under the microscope - Whether or not there are chromosomal abnormalities in the tumor, and if present, what type (also called cytogenetics analysis)

Type: Interventional

Start Date: Jun 2013

open study

Blinatumomab in Treating Younger Patients With Relapsed B-cell Acute Lymphoblastic Leukemia
National Cancer Institute (NCI) B Acute Lymphoblastic Leukemia
This randomized phase III trial studies how well blinatumomab works compared with standard combination chemotherapy in treating patients with B-cell acute lymphoblastic leukemia that has returned after a period of improvement (relapsed). Immunotherapy with blinatumomab, may induce... expand

This randomized phase III trial studies how well blinatumomab works compared with standard combination chemotherapy in treating patients with B-cell acute lymphoblastic leukemia that has returned after a period of improvement (relapsed). Immunotherapy with blinatumomab, may induce changes in body's immune system and may interfere with the ability of tumor cells to grow and spread. It is not yet known whether standard combination chemotherapy is more effective than blinatumomab in treating relapsed B-cell acute lymphoblastic leukemia.

Type: Interventional

Start Date: Dec 2014

open study

Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1
Kristina Hardy Neurofibromatosis Type 1
The main objective of the study is to assess the efficacy of a home-based, computerized cognitive training (CT) program, called CogmedRM, targeted to improve working memory in children with NF1 and working memory difficulties.This is a Phase II randomized parallel group controlled... expand

The main objective of the study is to assess the efficacy of a home-based, computerized cognitive training (CT) program, called CogmedRM, targeted to improve working memory in children with NF1 and working memory difficulties.This is a Phase II randomized parallel group controlled clinical trial comparing two interventions on cognitive outcomes. Participants will be stratified by stimulant medication use and randomized equally between the two interventions within stratum. Participants will be in the study for to 11 weeks.

Type: Interventional

Start Date: May 2016

open study

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis...
National Cancer Institute (NCI) Neurofibromatosis 1 Plexiform Neurofibromas
Background: People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (PNs) can have pain that affects their daily lives. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning. Objectives: To examine... expand

Background: People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (PNs) can have pain that affects their daily lives. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning. Objectives: To examine and improve questionnaires about daily living for people with NF1 and PNs. Eligibility: People ages 5 and older with NF1 and a PN Design: Participants will be screened with medical history. This study will have 2 phases. Phase 1 participants will talk about existing pain assessment questionnaires and how PNs affect their life. They will have group discussions of up to 8 people of a similar age with NF1 and PNs, or the parents of children with it. These will last about 90 minutes. Children ages 5 to 7 and their parents will have one-on-one meetings instead. These will last about 45 minutes. Discussions will be audiotaped. After the questionnaires have been changed, individual interviews will discuss the new wording, instructions, questions, and electronic format of the new forms. Phase 1 participants may be invited to Phase 2. Phase 2 participants will complete the new questionnaires. These may be pen-and-paper or electronic. The questionnaires will take about 30 minutes for adults and teens. Children will work one-on-one with a staff member and may need up to 45 minutes. A small group of participants will be complete the forms twice in clinic and 1 month later at home. Also, a small group who start a new pain treatment or have a dose increase in their treatment will complete the forms twice before the treatment change and 1 month later.

Type: Observational

Start Date: Nov 2015

open study

Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma,...
National Cancer Institute (NCI) Advanced Malignant Solid Neoplasm Ann Arbor Stage III Hodgkin Lymphoma Ann Arbor Stage III Non-Hodgkin Lymphoma Ann Arbor Stage IV Hodgkin Lymphoma Ann Arbor Stage IV Non-Hodgkin Lymphoma
This phase II Pediatric MATCH trial studies how well tazemetostat works in treating patients with solid tumors, non-hodgkin lymphoma, or histiocytic disorders that have spread to other places in the body and have come back or do not respond to treatment and have EZH2, SMARCB1,... expand

This phase II Pediatric MATCH trial studies how well tazemetostat works in treating patients with solid tumors, non-hodgkin lymphoma, or histiocytic disorders that have spread to other places in the body and have come back or do not respond to treatment and have EZH2, SMARCB1, or SMARCA4 gene mutations. Tazemetostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Jul 2017

open study

Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
Children's Research Institute Duchenne Muscular Dystrophy
Canakinumab is an anti-interleukin 1 beta (IL1β) antibody approved for use in young children with familial Mediterranean fever, systemic onset juvenile idiopathic arthritis and TNF-receptor associated periodic fever syndrome. This study is a pilot trial to investigate the effects... expand

Canakinumab is an anti-interleukin 1 beta (IL1β) antibody approved for use in young children with familial Mediterranean fever, systemic onset juvenile idiopathic arthritis and TNF-receptor associated periodic fever syndrome. This study is a pilot trial to investigate the effects of canakinumab on clinical safety and potential clinical efficacy as demonstrated by short-term changes in select serum biomarkers in a sample of young boys with DMD who are most likely to have high levels of muscle inflammation. Steroid naive DMD subjects aged greater than or equal to 2 years old to less than 6 years old will receive a single subcutaneous dose of canakinumab and undergo safety and serum biomarker monitoring for 30 days. The first 3 subjects will receive 2 mg/kg and if well tolerated, the second 3 subjects will receive 4 mg/kg.

Type: Interventional

Start Date: May 2019

open study

Pevonedistat, Azacitidine, Fludarabine Phosphate, and Cytarabine in Treating Patients With Relapsed or...
National Cancer Institute (NCI) Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome Blasts 0.1 Percent or More of Bone Marrow Nucleated Cells Blasts 5 Percent or More of Bone Marrow Nucleated Cells Recurrent Acute Myeloid Leukemia Recurrent High Risk Myelodysplastic Syndrome
This phase I trial studies the side effects and how well pevonedistat, azacitidine, fludarabine phosphate, and cytarabine work in treating patients with acute myeloid leukemia that has come back or has not responded to treatment or high-risk myelodysplastic syndrome that has... expand

This phase I trial studies the side effects and how well pevonedistat, azacitidine, fludarabine phosphate, and cytarabine work in treating patients with acute myeloid leukemia that has come back or has not responded to treatment or high-risk myelodysplastic syndrome that has come back. Pevonedistat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as azacitidine, and fludarabine phosphate, and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug (combination chemotherapy) and pevonedistat may work better in treating patients with acute myeloid leukemia or myelodysplastic syndrome.

Type: Interventional

Start Date: May 2019

open study

Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas,...
National Cancer Institute (NCI) Adrenal Cortex Carcinoma Alveolar Soft Part Sarcoma Central Nervous System Neoplasm Childhood Clear Cell Sarcoma of Soft Parts Clear Cell Sarcoma of Soft Tissue
This phase II trial studies how well cabozantinib-s-malate works in treating younger patients with sarcomas, Wilms tumor, or other rare tumors that have come back, do not respond to therapy, or are newly diagnosed. Cabozantinib-s-malate may stop the growth of tumor cells by blocking... expand

This phase II trial studies how well cabozantinib-s-malate works in treating younger patients with sarcomas, Wilms tumor, or other rare tumors that have come back, do not respond to therapy, or are newly diagnosed. Cabozantinib-s-malate may stop the growth of tumor cells by blocking some of the enzymes needed for tumor growth and tumor blood vessel growth.

Type: Interventional

Start Date: May 2017

open study

Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease
Allistair Abraham, MD Sickle Cell Disease
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving... expand

This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).

Type: Interventional

Start Date: Apr 2018

open study