169 matching studies

Sponsor Condition of Interest
Clinical and Laboratory Study of Methylmalonic Acidemia
National Human Genome Research Institute (NHGRI) Organic Acidemia Methylmalonic Acidemia Inborn Errors of Metabolism
This study will evaluate patients with methylmalonic acidemia (MMA) to learn more about the genetic causes of the various types of this inherited metabolic disorder and the medical complications associated with it. People with MMA may have problems with learning and development... expand

This study will evaluate patients with methylmalonic acidemia (MMA) to learn more about the genetic causes of the various types of this inherited metabolic disorder and the medical complications associated with it. People with MMA may have problems with learning and development and kidney malfunctioning. They can become seriously ill, sometimes with little warning. There is no cure for any MMA, but special diets and vitamin therapies are used for treatment. Patients between 2 and 70 years of age with MMA may be eligible for this study. Participants are admitted to the NIH Clinical Center for 4-5 days each year for 5-10 years for the following tests and procedures: - Medical history, physical examination, eye examination - Consultations from dentists and specialists in the nervous system, digestive tract, endocrine, and kidney, as needed. - 24-hour urine collection to examine for methylmalonic acid, other acids, sugar, and proteins for measuring kidney function. - Blood test to assess liver and thyroid function, blood counts and blood chemistries, methylmalonic acid levels, and for genetic tests and basic research studies. - Blood test to measure growth hormone production. For this test, a very small amount of blood is collected overnight (every 20-30 minutes from 8:00 PM to 8:00 AM) through an intravenous catheter (plastic tube placed in a vein). The total amount of blood drawn is approximately 1 tablespoon. Patients who have stopped growing or whose weight does not permit collection of 1 tablespoon of blood do not undergo this procedure. - Frequent blood pressure measurements, including overnight monitoring - Skin biopsy for cell culture (cells to grow in the laboratory for future testing). For this procedure, an area of skin is numbed with an anesthetic such as lidocaine. A 4-mm diameter circular area is then removed using a sharp punch and scissors. The wound is dressed and usually heals within a week. - Photographs of the face and body (wearing underwear) to help track growth and appearance. - Ultrasound of the kidneys - Hand x-ray to determine bone age - Dual energy x-ray absorptionometry (DEXA) scan to assess bone density. For the DEXA scan, the patient lies still on a table while the spine and hip are scanned using a small amount of radiation. Any patient who becomes seriously ill during the evaluation may be cared for at the NIH or transferred to another hospital if it is deemed advisable.

Type: Observational

Start Date: Jun 2004

open study

Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients
Bayer Mixed Tumor, Malignant
This study is designed to investigate whether the use of copanlisib is safe, feasible and beneficial to pediatric patients with solid solid tumors or lymphoma that are recurrent or refractory to standard therapy. expand

This study is designed to investigate whether the use of copanlisib is safe, feasible and beneficial to pediatric patients with solid solid tumors or lymphoma that are recurrent or refractory to standard therapy.

Type: Interventional

Start Date: Apr 2018

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

Active Reference (Fluoxetine) Fixed-dose Study of Vortioxetine in Paediatric Patients Aged 7 to 11 Years...
H. Lundbeck A/S Depressive Disorder, Major
Investigation of the efficacy and safety of a new potential treatment of Major depressive disorder (MDD) in paediatric patients (age 7 to 11 years) expand

Investigation of the efficacy and safety of a new potential treatment of Major depressive disorder (MDD) in paediatric patients (age 7 to 11 years)

Type: Interventional

Start Date: May 2016

open study

Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN...
Medical College of Wisconsin Sickle Cell Disease
This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care. The primary outcome is 2-year overall survival. expand

This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care. The primary outcome is 2-year overall survival.

Type: Interventional

Start Date: Nov 2016

open study

A Study of Nivolumab Plus Brentuximab Vedotin in Patients Between 5 and 30 Years Old, With Hodgkin's...
Bristol-Myers Squibb Hodgkin Disease
The purpose of this study is to determine whether nivolumab plus brentuximab vedotin (followed by brentuximab vedotin plus bendamustine in patient with suboptimal response) is safe and effective in treating patients with Hodgkin's lymphoma (cHL). Eligible patients are children,... expand

The purpose of this study is to determine whether nivolumab plus brentuximab vedotin (followed by brentuximab vedotin plus bendamustine in patient with suboptimal response) is safe and effective in treating patients with Hodgkin's lymphoma (cHL). Eligible patients are children, adolescents, and young adults relapsed or refractory to first line.

Type: Interventional

Start Date: Mar 2017

open study

A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory...
Epizyme, Inc. Rhabdoid Tumors INI1-negative Tumors Synovial Sarcoma Malignant Rhabdoid Tumor of Ovary
This is a Phase I, open-label, dose escalation and dose expansion study with BID (suspension) and TID (tablet) oral dose of tazemetostat. Subjects will be screened for eligibility within 14 days of the planned first dose of tazemetostat. A treatment cycle will be 28 days. Response... expand

This is a Phase I, open-label, dose escalation and dose expansion study with BID (suspension) and TID (tablet) oral dose of tazemetostat. Subjects will be screened for eligibility within 14 days of the planned first dose of tazemetostat. A treatment cycle will be 28 days. Response assessment will be evaluated after 8 weeks of treatment and subsequently every 8 weeks while on study. The study has two parts: Dose Escalation and Dose Expansion. Dose escalation for subjects with the following relapsed/refractory malignancies: - Rhabdoid tumors: - Atypical teratoid rhabdoid tumor (ATRT) - Malignant rhabdoid tumor (MRT) - Rhabdoid tumor of kidney (RTK) - Selected tumors with rhabdoid features - INI1-negative tumors: - Epithelioid sarcoma - Epithelioid malignant peripheral nerve sheath tumor - Extraskeletal myxoid chondrosarcoma - Myoepithelial carcinoma - Renal medullary carcinoma - Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) (with Sponsor approval) - Synovial Sarcoma with a SS18-SSX rearrangement Dose Expansion at the MTD or the RP2D - Cohort 1 -(closed to enrollment) ATRT - Cohort 2 - MRT/RTK/selected tumors with rhabdoid features - Cohort 3 - INI-negative tumors: - Epithelioid sarcoma - Epithelioid malignant peripheral nerve sheath tumor - Extraskeletal myxoid chondrosarcoma - Myoepithelial carcinoma - Renal medullary carcinoma - Chordoma (poorly differentiated or de-differentiated) - Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) with Sponsor approval - Cohort 4 -(closed to enrollment) Tumor types eligible for Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement

Type: Interventional

Start Date: Jan 2016

open study

Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry...
Bayer Hemophilia A
This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A patients who have switched from an existing therapy to Kovaltry or... expand

This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A patients who have switched from an existing therapy to Kovaltry or Jivi. In doing so, real world evidence will be obtained from both patient and physician perspectives offering key insights for effective therapeutic management of patients with hemophilia A and to more fully understand what drives patient switching from a patient perspective and a physician perspective.

Type: Observational

Start Date: Aug 2018

open study

Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus...
Pharmacyclics LLC. Chronic Graft Versus Host Disease
Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD) expand

Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Type: Interventional

Start Date: Nov 2018

open study

MR-guided High Intensity Focused Ultrasound (HIFU) on Pediatric Solid Tumors
AeRang Kim Relapsed Pediatric Solid Tumors Refractory Pediatric Solid Tumors Rhabdomyosarcoma Ewing Sarcoma Osteosarcoma
The purpose of this study is to determine if Magnetic Resonance guided High Intensity Focused Ultrasound ablative therapy is safe and feasible for children, adolescents, and young adults with refractory or relapsed solid tumors. expand

The purpose of this study is to determine if Magnetic Resonance guided High Intensity Focused Ultrasound ablative therapy is safe and feasible for children, adolescents, and young adults with refractory or relapsed solid tumors.

Type: Interventional

Start Date: Apr 2014

open study

Neonatal Seizure Registry - Developmental Functional EValuation
University of California, San Francisco Neonatal Seizure Hypoxic-Ischemic Encephalopathy Stroke Intracranial Hemorrhages Epilepsy
The NSR-DEV study is a longitudinal cohort study of around 280 Neonatal Seizure Registry participants that aims to evaluate childhood outcomes after acute symptomatic neonatal seizures, as well as examine risk factors for developmental disabilities and whether these are modified... expand

The NSR-DEV study is a longitudinal cohort study of around 280 Neonatal Seizure Registry participants that aims to evaluate childhood outcomes after acute symptomatic neonatal seizures, as well as examine risk factors for developmental disabilities and whether these are modified by parent well-being.

Type: Observational [Patient Registry]

Start Date: Mar 2020

open study

Vemurafenib 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 Ependymoma Ewing Sarcoma
This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body and have come back or do not respond to treatment.... expand

This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body and have come back or do not respond to treatment. Vemurafenib 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

A Phase I Study of Lyso-thermosensitive Liposomal Doxorubicin and MR-HIFU for Pediatric Refractory Solid...
AeRang Kim Pediatric Cancer Solid Tumors Rhabdomyosarcoma Ewing Sarcoma Soft Tissue Sarcomas
This study is looking to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of lyso-thermosensitive liposomal doxorubicin (LTLD) administered in combination with MR-HIFU in children with relapsed/refractory solid tumors, which may include but are not... expand

This study is looking to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of lyso-thermosensitive liposomal doxorubicin (LTLD) administered in combination with MR-HIFU in children with relapsed/refractory solid tumors, which may include but are not limited to rhabdomyosarcoma and other soft tissue sarcomas, Ewing's sarcoma family of tumors, osteosarcoma, neuroblastoma, Wilms' tumor, hepatic tumors, and germ cell tumors.

Type: Interventional

Start Date: Oct 2016

open study

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMT CTN 1507)
Medical College of Wisconsin Sickle Cell Disease
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1)... expand

This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with SCD with strokes; and (2) adults with severe SCD.

Type: Interventional

Start Date: Oct 2017

open study

Preventing Asthma in High Risk Kids
Boston Children’s Hospital Asthma
This trial is a randomized, double-blind, placebo controlled trial designed to test whether two years treatment of preschool children aged 2-3 years of age at high risk for asthma with omalizumab (anti-IgE) for two years will prevent the progression to childhood asthma, as reflected... expand

This trial is a randomized, double-blind, placebo controlled trial designed to test whether two years treatment of preschool children aged 2-3 years of age at high risk for asthma with omalizumab (anti-IgE) for two years will prevent the progression to childhood asthma, as reflected by a reduction in the prevalence of active asthma in the Final 12 months during 2 year observation period off study drug.

Type: Interventional

Start Date: Nov 2018

open study

Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults
Laurent Pharmaceuticals Inc. Cystic Fibrosis
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF. expand

An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.

Type: Interventional

Start Date: Oct 2018

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 Recurrent Brain Neoplasm Recurrent Childhood Ependymoma
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 (recurrent), progressed, or have not responded to previous treatment (refractory). Immunotherapy with monoclonal antibodies, such as pembrolizumab, may induce changes in the body's immune system, and may interfere with the ability of tumor cells to grow and spread.

Type: Interventional

Start Date: May 2015

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

Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
Novartis Pharmaceuticals Sickle Cell Disease (SCD)
The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric patients ages 6 months to <18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and safety... expand

The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric patients ages 6 months to <18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and safety of crizanlizumab was already demonstrated in adults with sickle cell disease. The approach is to extrapolate from the PK/pharmacodynamics (PD) already established in the adult population. The study is designed as a Phase II, multicenter, open-label study.

Type: Interventional

Start Date: Oct 2018

open study

Rituximab and LMP-Specific T-Cells in Treating Pediatric Solid Organ Recipients With EBV-Positive, CD20-Positive...
Children's Oncology Group EBV-Related Post-Transplant Lymphoproliferative Disorder Monomorphic Post-Transplant Lymphoproliferative Disorder Polymorphic Post-Transplant Lymphoproliferative Disorder Recurrent Monomorphic Post-Transplant Lymphoproliferative Disorder Recurrent Polymorphic Post-Transplant Lymphoproliferative Disorder
This pilot phase II trial studies how well rituximab and latent membrane protein (LMP)-specific T-cells work in treating pediatric solid organ recipients with Epstein-Barr virus-positive, cluster of differentiation (CD)20-positive post-transplant lymphoproliferative disorder.... expand

This pilot phase II trial studies how well rituximab and latent membrane protein (LMP)-specific T-cells work in treating pediatric solid organ recipients with Epstein-Barr virus-positive, cluster of differentiation (CD)20-positive post-transplant lymphoproliferative disorder. Rituximab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. LMP-specific T-cells are special immune system cells trained to recognize proteins found on post-transplant lymphoproliferative disorder tumor cells if they are infected with Epstein-Barr virus. Giving rituximab and LMP-specific T-cells may work better in treating pediatric organ recipients with post-transplant lymphoproliferative disorder than rituximab alone.

Type: Interventional

Start Date: Mar 2017

open study

Hospital-Based Cluster Trial: Magnetically Controlled Growing Rods Using Distraction Intervals
Children's Spine Foundation Early-Onset Scoliosis Deformity of Spine
A hospital-based cluster stratified randomization control study will be conducted to investigate spinal growth in Early Onset Scoliosis patients between 5 and 9 years of age. Patients must have a major coronal curve measuring over 50 degrees and be undergoing Magnetically Controlled... expand

A hospital-based cluster stratified randomization control study will be conducted to investigate spinal growth in Early Onset Scoliosis patients between 5 and 9 years of age. Patients must have a major coronal curve measuring over 50 degrees and be undergoing Magnetically Controlled Growing Rod treatment. We will be studying 6-week lengthening intervals compared to 16-week lengthening intervals on spinal growth within 3 years.

Type: Interventional

Start Date: Nov 2019

open study

Safety and Effectiveness of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML),...
Daiichi Sankyo, Inc. Acute Myeloid Leukemia
Quizartinib is an experimental drug. It is not approved for regular use. It can only be used in medical research. Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able to join this study if it has come back after remission or is not responding... expand

Quizartinib is an experimental drug. It is not approved for regular use. It can only be used in medical research. Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able to join this study if it has come back after remission or is not responding to treatment.

Type: Interventional

Start Date: Aug 2018

open study

Pediatric cGVHD Symptom Scale
National Cancer Institute (NCI) Graft vs Host Disease
Background: Some children/adolescents who have had a stem cell transplant live with chronic graft-versus-host-disease (cGVHD). cGVHD is a side effect of the transplant that can cause multiple bothersome symptoms and negatively affect a child/adolescent squality of daily life.... expand

Background: Some children/adolescents who have had a stem cell transplant live with chronic graft-versus-host-disease (cGVHD). cGVHD is a side effect of the transplant that can cause multiple bothersome symptoms and negatively affect a child/adolescent squality of daily life. The questionnaires that measure thesymptoms caused by cGVHD are designed for adults. Children/adolescents may not describe their symptoms in the same way. The goal of this research is to improve the way we measure how bothersome these symptoms are for children/adolescents living with cGVHD. Objective: To develop a questionnaire (The Pediatric cGHVD Symptom Scale) for children/adolescents living with cGVHD to identify the symptoms they are experiencing and describe how bothersome those symptoms are to them. An additional goal is to design a parent/guardian companion questionnaire that can be used to capture the symptom experiences of very young children who may not be able to complete a questionnaire. Eligibility: Children/adolescents ages 5-17 who are receiving treatment for cGVHD after a stem cell transplant, and their parent/guardian.. Design: This study consists of 2 projects. Children/adolescents with cGVHD and their parent/guardianparticipants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17. In project 1, participants will complete an age-appropriate questionnaire about cGVHD symptoms. The questionnaire will ask about the child/adolescent s physical functioning and emotional well-being. The parent/guardian will out fill out a companion questionnaire online. The child/adolescent will then review their completed questionnaire during an interview with a researcher and will be asked whether the questions about their symptoms were difficult to understand. The parent/guardian and child/adolescent will then be interviewed together to further explore their responses to the questionnaires. Interviews will be done in person, by phone, and online. . Based on what is learned through these interviews, the wording of the questionnaire will be improved for better comprehension and ease of response. In project 2, participants will complete this revised questionnairefor their age group along with some other questionnaires that ask about quality of life. Both the child/adolescent and parent/guardian will fill out the questionnaires online at three separate time points. In both projects, children/adolescents with cGVHD and their parent/guardian participants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17. ...

Type: Observational

Start Date: Oct 2019

open study

Larotrectinib in Treating Patients With Previously Untreated TRK Fusion Solid Tumors and TRK Fusion Relapsed...
Children's Oncology Group Central Nervous System Neoplasm Infantile Fibrosarcoma Recurrent Acute Leukemia Refractory Acute Leukemia Solid Neoplasm
This phase II trial studies the side effects and how well larotrectinib works in treating patients with previously untreated TRK fusion solid tumors and TRK fusion acute leukemia that has come back. Larotrectinib may stop the growth of cancer cells with TRK fusions by blocking... expand

This phase II trial studies the side effects and how well larotrectinib works in treating patients with previously untreated TRK fusion solid tumors and TRK fusion acute leukemia that has come back. Larotrectinib may stop the growth of cancer cells with TRK fusions by blocking the TRK enzymes needed for cell growth.

Type: Interventional

Start Date: Sep 2019

open study

BBD Longitudinal Study of Osteogenesis Imperfecta
Baylor College of Medicine Osteogenesis Imperfecta
Osteogenesis Imperfecta (OI) is a rare disorder that causes bones to break easily. People with OI may have broken bones with little or no trauma, dentinogenesis imperfecta (DI), and, in adult years, hearing loss. It is seen in both genders and all races. OI can range from very... expand

Osteogenesis Imperfecta (OI) is a rare disorder that causes bones to break easily. People with OI may have broken bones with little or no trauma, dentinogenesis imperfecta (DI), and, in adult years, hearing loss. It is seen in both genders and all races. OI can range from very severe to very mild. Individuals with the most severe type of OI may die at birth. People with severe OI who survive may have bowed arms and legs, very short stature and be unable to walk. People with the mildest form of OI may only break bones occasionally and have normal height and lifespan. People with OI also often have problems with the spine. The spine problems include compression fractures and scoliosis (a curvature of the spine). DI is characterized by grey or brown teeth that may chip and wear down and break easily. Before the genetic cause of OI was known, OI was classified into four types. Each type was based upon the symptoms and severity of OI. In most people with OI, the cause is a change in one of the genes that makes a protein called type 1 collagen. In the past decade, it was discovered that in about 5% of people with OI it is in another gene. Some doctors now classify OI both on how severe it is as well as which gene is causing OI. Our research aims are: 1. Perform DNA testing and collect natural history data on all individuals enrolled in this longitudinal study. The genetic cause of the brittle bone disease will be compared with things like severity, various features and response to treatments. 2. We will see how often people with type I OI have vertebral compression fractures of the spine. We will do x-rays to see how often they get compression fractures of the vertebrae, what happens over time and any risk factors that increase the risk of these compression fractures. 3. We will follow people with all forms of OI to see how often they develop scoliosis (curvature of the spine). We will look at the effects of scoliosis on lung function, ability to walk and quality of life. We will also look at the effects of various treatments (bracing, surgery, etc.) on scoliosis and lung function. 4. We will look at dental health in people with OI. We will see how often people with OI have problems with teeth alignment. Importantly, we will see how dental health impacts a person's quality of life.

Type: Observational

Start Date: Jun 2015

open study