
Search Clinical Trials
Sponsor Condition of Interest |
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Study of Ribociclib and Everolimus in HGG and DIPG or Ribociclib and Temozolomide in DHG, H3G34-mut1
Nationwide Children's Hospital
High Grade Glioma
Diffuse Intrinsic Pontine Glioma
Anaplastic Astrocytoma
Glioblastoma
Glioblastoma Multiforme
The goal of this study is to determine the efficacy of the 1) ribociclib and everolimus
to treat pediatric and young adult patients newly diagnosed with a high-grade glioma
(HGG), including DIPG, that have genetic changes in pathways (cell cycle, PI3K/mTOR) that
these drugs target or 2) ribociclib1 expand
The goal of this study is to determine the efficacy of the 1) ribociclib and everolimus to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (cell cycle, PI3K/mTOR) that these drugs target or 2) ribociclib and temozolomide to treat pediatric and young adult patients newly diagnosed with diffuse hemispheric glioma (DHG), H3G34-mutant. The main question the study aims to answer is whether the combinations of ribociclib and everolimus or ribociclib and temozolomide can prolong the life of patients diagnosed with HGG/DIPG or DHG H3G34-mutant. Type: Interventional Start Date: Aug 2024 |
A Feasibility Safety Study of Benign Centrally-Located Intracranial Tumors in Pediatric and Young A1
InSightec
Benign Centrally-Located Intracranial Tumors
The goal of this prospective, non-randomized, single-arm, feasibility study is to develop
data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign
intracranial tumors which require clinical intervention in pediatric and young adult
subjects.
Indication of Use: Ablation of b1 expand
The goal of this prospective, non-randomized, single-arm, feasibility study is to develop data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial tumors which require clinical intervention in pediatric and young adult subjects. Indication of Use: Ablation of benign intracranial tumors in children and young adults which are ExAblate accessible. Type: Interventional Start Date: Feb 2017 |
A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (1
National Cancer Institute (NCI)
Stage II Nasopharyngeal Carcinoma AJCC v8
Stage III Nasopharyngeal Carcinoma AJCC v8
Stage IV Nasopharyngeal Carcinoma AJCC v8
This phase II trial tests effects of nivolumab in combination with chemotherapy drugs
prior to radiation therapy patients with nasopharyngeal carcinoma (NPC). Immunotherapy
with monoclonal antibodies, such as nivolumab, may help the body's immune system attack
the cancer, and may interfere with the1 expand
This phase II trial tests effects of nivolumab in combination with chemotherapy drugs prior to radiation therapy patients with nasopharyngeal carcinoma (NPC). 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. Chemotherapy drugs, such as gemcitabine and cisplatin, 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. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. Researchers want to find out what effects, good and/or bad, adding nivolumab to chemotherapy has on patients with newly diagnosed NPC. In addition, they want to find out if children with NPC may be treated with less radiation therapy and whether this decreases the side effects of therapy. Type: Interventional Start Date: Jun 2024 |
A Master Protocol (LY900023) That Includes Several Clinical Trials of Drugs for Children and Young1
Eli Lilly and Company
Neoplasms
Child
Adolescent
The main purpose of the master is to help the research sites and sponsor carry out
several clinical trials more efficiently by providing a common research protocol.
Individual clinical trials under this master protocol define drug/disease-specific
research goals and activities to test them. New stu1 expand
The main purpose of the master is to help the research sites and sponsor carry out several clinical trials more efficiently by providing a common research protocol. Individual clinical trials under this master protocol define drug/disease-specific research goals and activities to test them. New studies will be added as new drugs emerge against different cancers. Participation in the trial will depend on how long the benefit lasts. Type: Interventional Start Date: Jan 2020 |
T Cell Therapy Opposing Novel COVID-19 Infection in Immunocompromised Patients
Children's National Research Institute
SARS-CoV-2 Infection
This is an open label, phase I dose-escalation study to evaluate the safety of
coronavirus-specific T cell (CST) therapy for prevention of SARS-CoV-2 infection in
immunocompromised patients following hematopoietic stem cell transplantation (HSCT).
Participants will receive donor-derived CSTs for p1 expand
This is an open label, phase I dose-escalation study to evaluate the safety of coronavirus-specific T cell (CST) therapy for prevention of SARS-CoV-2 infection in immunocompromised patients following hematopoietic stem cell transplantation (HSCT). Participants will receive donor-derived CSTs for prevention of SARS-CoV-2 infection after HSCT (≥28 days and <4 months after HSCT). In this dose escalation trial, three doses (1x107/m2, 2x107/m2, and 4x107/m2) will be tested for safety, with study arms for adult (≥18 years of age and <80 years) HSCT recipients (Arm A) and two arms for pediatric (≥12 years of age and <18 years; ≥2 years and <12 years) HSCT recipients (Arm B and Arm C, respectively), and defined dose escalations in each study arm. The study agent will be assessed for safety (stopping rules defined) and antiviral activity. Type: Interventional Start Date: Oct 2021 |
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed D1
National Human Genome Research Institute (NHGRI)
Genetic Disease
Without an explanation for severe and sometimes life-threatening symptoms, patients and
their families are left in a state of unknown. Many individuals find themselves being
passed from physician to physician, undergoing countless and often repetitive tests in
the hopes of finding answers and insig1 expand
Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible. To date, the UDP has evaluated 3300 medical records and admitted 750 individuals with rare and undiagnosed conditions to the NIH Clinical Center. The NIH UDP has identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the Undiagnosed Diseases Network (UDN), for fiscal years 2013-2020. The clinical sites will perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN will further the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases.... Type: Observational Start Date: Sep 2015 |
A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopath1
George Washington University
MELAS Syndrome
Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
This is a parallel arm non-randomized dose-escalation, open-label basket exploratory
phase 1 clinical trial where Mitochondrial encephalopathy, lactic acidosis, stroke-like
episodes (MELAS) and Leber's hereditary optic neuropathy-Plus (LHON-Plus) participants
will undergo simultaneous enrollment in1 expand
This is a parallel arm non-randomized dose-escalation, open-label basket exploratory phase 1 clinical trial where Mitochondrial encephalopathy, lactic acidosis, stroke-like episodes (MELAS) and Leber's hereditary optic neuropathy-Plus (LHON-Plus) participants will undergo simultaneous enrollment in two disease-based arms and receive daily oral doses of glycerol tributyrate to assess its safety and potential for efficacy using clinical, biochemical, and molecular evidence. This study will utilize a two-month baseline lead-in phase to establish and document the clinical baseline for each participant in both arms in order to compare the molecular and clinical parameters. This is clinically relevant in light of the high clinical heterogeneity among subjects affected by the same mitochondrial disease (MELAS or LHON-Plus). For ethical concerns prompted by the lack of treatment for these two intractable and progressive mitochondrial diseases, there will not be a placebo control group. Thus, each participant will act as their own control and receive oral doses of glycerol tributyrate, eliminating the need for a placebo. Considering the high clinical heterogeneity among participants affected by MELAS or LHON-Plus and some clinical divergence between MELAS and LHON-Plus, this strategy is beneficial to every enrolled participants, as each will receive the investigational drug, glycerol tributyrate. In addition, this approach will determine the subject-specific maximal optimized dose in a personalized medicine-based approach. After approval of the IRB protocol from the Institutional Review Board Data and signed consent form from all participants, this investigational basket clinical trial has three phases spanning over 20 months: - A baseline lead-in phase (2 months) to collect participant-specific baseline for clinical, biochemical, molecular and metabolic biomarkers that will be monitored throughout the subsequent dose-escalation and clinical phases. - A dose-escalation phase (6 months) to determine the participant-specific maximum tolerated dose (MTD) during which participant-specific clinical and biochemical biomarkers are collected every month. - A clinical phase at a fixed subject-specific MTD dose (12 months) to collect participant-specific clinical, biochemical, molecular and metabolic biomarkers and to perform three scheduled skin biopsies: at the outset, mid-point, and the end of this clinucal phase. We have planned for a 12-month-long clinical phase at a fixed participant-specific MTD considering the absence of reliable predictors that makes idiosyncratic disease-specific symptoms for MELAS and LHON-Plus impossible to forecast among participant for assessing the potential efficacy of glycerol tributyrate by monitoring clinical symptoms specific for each disease. During the 12-month-long time-frame, disease-specific clinical symptoms will be collected as preliminary evidence of efficacy of glycerol tributyrate using disease-specific biomarkers. Finally, discharge procedure during which the clinical investigator will record non-serious adverse events or serious adverse events for 7 or 30 days, respectively, after the last day of study participation. Type: Interventional Start Date: Sep 2025 |
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofi1
Healx Limited
Neurofibromatosis Type 1
The trial will be an open label, single arm, phase 2 study in 20 participants. The study
will assess the tolerability and efficacy of HLX-1502 in participants with NF1 16 years
of age or older with progressive and/or symptomatic PN. expand
The trial will be an open label, single arm, phase 2 study in 20 participants. The study will assess the tolerability and efficacy of HLX-1502 in participants with NF1 16 years of age or older with progressive and/or symptomatic PN. Type: Interventional Start Date: Jan 2025 |
International Rare Brain Tumor Registry
Children's National Research Institute
Astroblastoma
BCOR ITD Sarcoma
CNS Sarcoma
Unclassified Tumor, Malignant
The objective of the International Rare Brain Tumor Registry (IRBTR) is to better
understand rare brain tumors through the collection of biospecimens and matched clinical
data of children, adolescents, and young adult patients diagnosed with rare brain tumors. expand
The objective of the International Rare Brain Tumor Registry (IRBTR) is to better understand rare brain tumors through the collection of biospecimens and matched clinical data of children, adolescents, and young adult patients diagnosed with rare brain tumors. Type: Observational [Patient Registry] Start Date: Jan 2023 |
Combination Therapy for the Treatment of Diffuse Midline Gliomas
University of California, San Francisco
Diffuse Intrinsic Pontine Glioma
Diffuse Midline Glioma, H3 K27M-Mutant
Recurrent Diffuse Intrinsic Pontine Glioma
Recurrent Diffuse Midline Glioma, H3 K27M-Mutant
Recurrent WHO Grade III Glioma
This phase II trial determines if the combination of ONC201 with different drugs,
panobinostat or paxalisib, is effective for treating participants with diffuse midline
gliomas (DMGs). Despite years of research, little to no progress has been made to improve
outcomes for participants with DMGs, and1 expand
This phase II trial determines if the combination of ONC201 with different drugs, panobinostat or paxalisib, is effective for treating participants with diffuse midline gliomas (DMGs). Despite years of research, little to no progress has been made to improve outcomes for participants with DMGs, and there are few treatment options. ONC201, panobinostat, and paxalisib are all enzyme inhibitors that may stop the growth of tumor cells by clocking some of the enzymes needed for cell growth. This phase II trial assesses different combinations of these drugs for the treatment of DMGs. Type: Interventional Start Date: Oct 2021 |
A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT
Children's Oncology Group
Central Nervous System Nongerminomatous Germ Cell Tumor
Choriocarcinoma
Embryonal Carcinoma
Immature Teratoma
Malignant Teratoma
This phase II trial studies the best approach to combine chemotherapy and radiation
therapy (RT) based on the patient's response to induction chemotherapy in patients with
non-germinomatous germ cell tumors (NGGCT) that have not spread to other parts of the
brain or body (localized). This study has1 expand
This phase II trial studies the best approach to combine chemotherapy and radiation therapy (RT) based on the patient's response to induction chemotherapy in patients with non-germinomatous germ cell tumors (NGGCT) that have not spread to other parts of the brain or body (localized). This study has 2 goals: 1) optimizing radiation for patients who respond well to induction chemotherapy to diminish spinal cord relapses, 2) utilizing higher dose chemotherapy followed by conventional RT in patients who did not respond to induction chemotherapy. Chemotherapy drugs, such as carboplatin, etoposide, ifosfamide, and thiotepa, 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. Radiation therapy uses high energy x-rays or high-energy protons to kill tumor cells and shrink tumors. Studies have shown that patients with newly-diagnosed localized NGGCT, whose disease responds well to chemotherapy before receiving radiation therapy, are more likely to be free of the disease for a longer time than are patients for whom the chemotherapy does not efficiently eliminate or reduce the size of the tumor. The purpose of this study is to see how well the tumors respond to induction chemotherapy to decide what treatment to give next. Some patients will be given RT to the spine and a portion of the brain. Others will be given high dose chemotherapy and a stem cell transplant before RT to the whole brain and spine. Giving treatment based on the response to induction chemotherapy may lower the side effects of radiation in some patients and adjust the therapy to a more efficient one for other patients with localized NGGCT. Type: Interventional Start Date: Jul 2021 |
Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Ref1
Children's Oncology Group
Recurrent B Acute Lymphoblastic Leukemia
Recurrent B Lymphoblastic Lymphoma
Refractory B Acute Lymphoblastic Leukemia
Refractory B Lymphoblastic Lymphoma
This phase II trial studies how well inotuzumab ozogamicin works in treating younger
patients with B-lymphoblastic lymphoma or CD22 positive B acute lymphoblastic leukemia
that has come back (relapsed) or does not respond to treatment (refractory). Inotuzumab
ozogamicin is a monoclonal antibody, ca1 expand
This phase II trial studies how well inotuzumab ozogamicin works in treating younger patients with B-lymphoblastic lymphoma or CD22 positive B acute lymphoblastic leukemia that has come back (relapsed) or does not respond to treatment (refractory). Inotuzumab ozogamicin is a monoclonal antibody, called inotuzumab, linked to a toxic agent called ozogamicin. Inotuzumab attaches to CD22 positive cancer cells in a targeted way and delivers ozogamicin to kill them. Type: Interventional Start Date: Jun 2017 |
Testing the Addition of an Anti-cancer Drug, DT2216, to the Usual Chemotherapy Treatment for Relaps1
Children's Oncology Group
Childhood Fibrolamellar Carcinoma
Recurrent Childhood Fibrolamellar Carcinoma
Recurrent Childhood Malignant Solid Neoplasm
Recurrent Fibrolamellar Carcinoma
Recurrent Malignant Solid Neoplasm
This phase I/II trial tests the safety, side effects and best dose of DT2216 in
combination with irinotecan and how well it works in treating children, adolescents and
young adults with solid tumors and fibrolamellar cancer that has come back after a period
of improvement (relapsed) or that has not1 expand
This phase I/II trial tests the safety, side effects and best dose of DT2216 in combination with irinotecan and how well it works in treating children, adolescents and young adults with solid tumors and fibrolamellar cancer that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). DT2216 is an anti-apoptotic protein B-cell lymphoma-extra large targeted protein degrader. It may stop the growth of tumor cells by blocking Bcl-xL, a protein needed for tumor cell survival. Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and deoxyribonucleic acid repair and may kill tumor cells. Giving DT2216 in combination with irinotecan may be safe, tolerable, and/or effective in treating children, adolescents and young adults with relapsed or refractory solid tumors or fibrolamellar cancer. Type: Interventional Start Date: Jun 2025 |
Pragmatic Clinic-Based Trial of a Mindfulness Based Intervention for Mood Concerns in Youth With Ty1
Children's National Research Institute
Type 1 Diabetes (T1D)
Type 1 diabetes (T1D) is a common chronic illness among children requiring a high degree
of self-management for good glycemic control. Adolescents are at risk for poor disease
management and health outcomes due to a number of factors, including high rates of
depression, anxiety, and stress. Accessi1 expand
Type 1 diabetes (T1D) is a common chronic illness among children requiring a high degree of self-management for good glycemic control. Adolescents are at risk for poor disease management and health outcomes due to a number of factors, including high rates of depression, anxiety, and stress. Accessing support for these challenges can be a barrier to care, so the current study, BRinging Empowerment and Attention to Teen HEalth-T1D, evaluates the efficacy of a virtual, group-based mindfulness based intervention and a virtual group-based diabetes education intervention on improving symptoms of depression and anxiety, and diabetes self management in teens with T1D. The study also aims to study how these interventions might be implemented in diabetes clinic settings. Type: Interventional Start Date: May 2025 |
Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols
Children's National Research Institute
Rhabdomyolysis
There is a significant unmet need for optimized treatment in rhabdomyolysis. There are
few prospective interventional studies on treatment for rhabdomyolysis, a condition which
affects diverse and underrepresented populations at a higher rate. While steroids are
often used off-label, a systematic s1 expand
There is a significant unmet need for optimized treatment in rhabdomyolysis. There are few prospective interventional studies on treatment for rhabdomyolysis, a condition which affects diverse and underrepresented populations at a higher rate. While steroids are often used off-label, a systematic study has not yet been initiated, and steroids have not been yet considered in as a consideration to standard care guidelines. The hypothesis is that patients who receive dexamethasone in addition to standard care versus placebo and standard care will have improvement in pain, length of hospital stay, and decrease in kidney complications. Type: Interventional Start Date: Oct 2024 |
Family Intervention for Black Teens With Type 1 Diabetes
Wayne State University
Type 1 Diabetes
Family Relations
The purpose of this study is to conduct a multicenter, randomized effectiveness trial of
The 3Ms 2.0 compared to an educational control condition for improving adolescent
glycemic control and diabetes-related family relationships and reducing primary caregiver
diabetes-related distress among Black1 expand
The purpose of this study is to conduct a multicenter, randomized effectiveness trial of The 3Ms 2.0 compared to an educational control condition for improving adolescent glycemic control and diabetes-related family relationships and reducing primary caregiver diabetes-related distress among Black adolescents with type 1 diabetes (T1D) and their primary caregivers. The proposed study would develop and test The 3Ms 2.0 adapted intervention when delivered using a mobile health approach (accessed via parents' cell phone). The intervention will also include new family intervention content (videoclips and text messages). Type: Interventional Start Date: Sep 2024 |
Individualized Nutrition to Optimize Preterm Infant Growth and Neurodevelopment
Children's National Research Institute
Very Preterm Maturity of Infant
Very Low Birth Weight Infant
Human milk has several well-established benefits but does not adequately meet the
increased nutritional demands of the growing preterm infant, necessitating additional
nutrient supplementation in a process known as fortification. In U.S. neonatal intensive
care units (NICUs), human milk is primaril1 expand
Human milk has several well-established benefits but does not adequately meet the increased nutritional demands of the growing preterm infant, necessitating additional nutrient supplementation in a process known as fortification. In U.S. neonatal intensive care units (NICUs), human milk is primarily supplemented using standardized fortification, in which a multicomponent fortifier is added to human milk to achieve assumed nutrient content based on standard milk reference values. However, this method does not account for the significant variability in human milk composition or in preterm infant metabolism, and up to half of all very premature infants experience poor growth and malnutrition using current nutritional practices. Poor postnatal growth has adverse implications for the developing preterm brain and long-term neurodevelopment. Recent advances allow for individualized methods of human milk fortification, including adjustable and targeted fortification. Adjustable fortification uses laboratory markers of protein metabolism (BUN level) to estimate an infant's protein requirements. In targeted fortification, a milk sample is analyzed to determine its specific macronutrient and energy content, with additional macronutrient supplementation provided as needed to achieve goal values. Emerging data suggest that both methods are safe and effective for improving growth, however information on their comparable efficacy and neurodevelopmental implications are lacking, particularly using advanced quantitative brain MRI (qMRI) techniques. Through this prospective, randomized-controlled trial, the investigators will compare the impact of individualized human milk fortification on somatic growth and neurodevelopment in preterm infants. Infants will be randomized to receive one of three nutritional interventions: standardized (control group), adjustable, or targeted human milk fortification. Infants will undergo their assigned nutritional intervention until term-equivalent age or discharge home, whichever is achieved first. Brain qMRI will be performed at term-corrected age, and neurodevelopmental follow-up will be performed through 5 years of age. Type: Interventional Start Date: Feb 2024 |
Evaluation of The Food Allergy Mastery Program
Children's National Research Institute
Food Allergy in Children
The proposed research project will evaluate a novel behavioral intervention that promotes
early adolescent food allergy self-management and adjustment through 1) food allergy
education, 2) problem-solving, communication, assertiveness, and anxiety management skill
building, and 3) peer support. expand
The proposed research project will evaluate a novel behavioral intervention that promotes early adolescent food allergy self-management and adjustment through 1) food allergy education, 2) problem-solving, communication, assertiveness, and anxiety management skill building, and 3) peer support. Type: Interventional Start Date: Jun 2023 |
Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependy1
University of California, San Francisco
Medulloblastoma
Medulloblastoma, Childhood
Medulloblastoma Recurrent
Ependymoma
Ependymoma Malignant
The current study will use a new treatment approach based on the molecular
characteristics of each participant's tumor. The study will test the feasibility in the
pilot phase of performing real-time drug screening on tissue taken during surgery in
patients with relapsed medulloblastoma or ependymom1 expand
The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility in the pilot phase of performing real-time drug screening on tissue taken during surgery in patients with relapsed medulloblastoma or ependymoma and of having a specialized tumor board assign a treatment plan based on the results of this screening and genomic sequencing. The aim of this trial is to allow every child and young adult with relapsed medulloblastoma and ependymoma to receive the most effective and least toxic therapies currently available and will pave the way for improved understanding and treatment of these tumors in the future. Moreover, if successful, it could serve as a paradigm for personalized medicine programs for other types of cancer. Type: Interventional Start Date: Feb 2022 |
Measuring Analgesic Interventions
Julia Finkel
Pain
It is generally recognized that pain assessment and management especially in newborns,
children and other nonverbal populations is an unmet need. According to the American
Medical Association, "the pediatric population is at risk of inadequate pain management,
with age-related factors affecting pai1 expand
It is generally recognized that pain assessment and management especially in newborns, children and other nonverbal populations is an unmet need. According to the American Medical Association, "the pediatric population is at risk of inadequate pain management, with age-related factors affecting pain management in children. Children are often given minimal or no analgesia for procedures that would routinely be treated aggressively in adults. Although much is now known about pain management in children, it has not been widely or effectively translated into routine clinical practice". These two factors combine to emphasize the necessity for an objective tool to quantify pain and monitor the effectiveness of analgesia, especially during treatments. Further, it is reported that many patients require a combination of treatments, and it is often necessary to test a variety of treatments before the personal match for treatment is found. The method in place to change the care on a subjective basis is difficult, time consuming, and not easily individualized. This pilot study is part of an ongoing effort to develop a method to objectively assess response to specific analgesic interventions. It specifically aims to discern the impact of analgesic interventions on sensory nerve fiber sensitivity in a diverse patient population. Type: Observational Start Date: Oct 2018 |
Childhood Cancer Survivor Study
St. Jude Children's Research Hospital
Cancer
The Childhood Cancer Survivor Study (CCSS) will investigate the long-term effects of
cancer and its associated therapies. A retrospective cohort study will be conducted
through a multi-institutional collaboration, which will involve the identification and
active follow-up of a cohort of approximate1 expand
The Childhood Cancer Survivor Study (CCSS) will investigate the long-term effects of cancer and its associated therapies. A retrospective cohort study will be conducted through a multi-institutional collaboration, which will involve the identification and active follow-up of a cohort of approximately 50,000 survivors of cancer, diagnosed before 21 years of age, between 1970 and 1999 and 10,000 sibling controls. This project will study children and young adults exposed to specific therapeutic modalities, including radiation, chemotherapy, and/or surgery, who are at increased risk of late-occurring adverse health outcomes. A group of sibling controls will be identified and data collected for comparison purposes. Type: Observational Start Date: Jan 1995 |
Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Dis1
University of Rochester
Sickle Cell Disease (SCD)
The WeDecide study is a large observational study comparing the long-term effects of
matched related donor hematopoietic stem cell transplantation (MRD HCT) and
non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle
cell disease (SCD). The study aims to assess health1 expand
The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention. With 160 children in the MRD HCT group and 320 in the NT-DMT group, aged 3-20.9 years, the study will follow participants for three years, examining factors like disease severity, treatment history, and social determinants of health. By providing a comprehensive comparison, the study seeks to inform clinical decisions and improve understanding of SCD treatment outcomes, ultimately supporting families and healthcare providers in choosing the best treatment options. Type: Observational Start Date: Jun 2024 |
Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mi1
Children's Oncology Group
B Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Central Nervous System Leukemia
Mixed Phenotype Acute Leukemia
Testicular Leukemia
This phase III trial studies whether inotuzumab ozogamicin added to post-induction
chemotherapy for patients with High-Risk B-cell Acute Lymphoblastic Leukemia (B-ALL)
improves outcomes. This trial also studies the outcomes of patients with mixed phenotype
acute leukemia (MPAL), and B-lymphoblastic1 expand
This phase III trial studies whether inotuzumab ozogamicin added to post-induction chemotherapy for patients with High-Risk B-cell Acute Lymphoblastic Leukemia (B-ALL) improves outcomes. This trial also studies the outcomes of patients with mixed phenotype acute leukemia (MPAL), and B-lymphoblastic lymphoma (B-LLy) when treated with ALL therapy without inotuzumab ozogamicin. Inotuzumab ozogamicin is a monoclonal antibody, called inotuzumab, linked to a type of chemotherapy called calicheamicin. Inotuzumab attaches to cancer cells in a targeted way and delivers calicheamicin to kill them. Other drugs used in the chemotherapy regimen, such as cyclophosphamide, cytarabine, dexamethasone, doxorubicin, daunorubicin, methotrexate, leucovorin, mercaptopurine, prednisone, thioguanine, vincristine, and pegaspargase or calaspargase pegol 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. This trial will also study the outcomes of patients with mixed phenotype acute leukemia (MPAL) and disseminated B lymphoblastic lymphoma (B-LLy) when treated with high-risk ALL chemotherapy. The overall goal of this study is to understand if adding inotuzumab ozogamicin to standard of care chemotherapy maintains or improves outcomes in High Risk B-cell Acute Lymphoblastic Leukemia (HR B-ALL). The first part of the study includes the first two phases of therapy: Induction and Consolidation. This part will collect information on the leukemia, as well as the effects of the initial treatment, to classify patients into post-consolidation treatment groups. On the second part of this study, patients with HR B-ALL will receive the remainder of the chemotherapy cycles (interim maintenance I, delayed intensification, interim maintenance II, maintenance), with some patients randomized to receive inotuzumab. The patients that receive inotuzumab will not receive part of delayed intensification. Other aims of this study include investigating whether treating both males and females with the same duration of chemotherapy maintains outcomes for males who have previously been treated for an additional year compared to girls, as well as to evaluate the best ways to help patients adhere to oral chemotherapy regimens. Finally, this study will be the first to track the outcomes of subjects with disseminated B-cell Lymphoblastic Leukemia (B-LLy) or Mixed Phenotype Acute Leukemia (MPAL) when treated with B-ALL chemotherapy. Type: Interventional Start Date: Oct 2019 |
National Collaborative to Improve Care of Children With Complex Congenital Heart Disease
Children's Hospital Medical Center, Cincinnati
Hypoplastic Left Heart Syndrome (HLHS)
The purpose of this initiative is to improve care and outcomes for infants with HLHS by
expanding the NPC-QIC national registry to gather clinical care process, outcome, and
developmental data on infants with HLHS between diagnosis and 12 months of age, by
improving the use of standards into everyd1 expand
The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, by improving the use of standards into everyday practice across pediatric cardiology centers, and by engaging parents as partners in the process. Type: Observational [Patient Registry] Start Date: May 2016 |
The Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Chi1
PedAL BCU, LLC
Acute Lymphoblastic Leukemia
Acute Myeloid Leukemia
Acute Myeloid Leukemia Post Cytotoxic Therapy
Juvenile Myelomonocytic Leukemia
Mixed Phenotype Acute Leukemia
This study aims to use clinical and biological characteristics of acute leukemias to
screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone
marrow and blood from patients with leukemia that has come back after treatment or is
difficult to treat may provide informat1 expand
This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and blood from patients with leukemia that has come back after treatment or is difficult to treat may provide information about the patient's leukemia that is important when deciding how to best treat it, and may help doctors find better ways to diagnose and treat leukemia in children, adolescents, and young adults. Type: Interventional Start Date: Apr 2022 |
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