159 matching studies

Sponsor Condition of Interest
The Mechanistic Biology of Primary Immunodeficiency Disorders
National Institute of Allergy and Infectious Diseases (NIAID) Primary Immunodeficiency Disorders
Background: Primary immunodeficiency disorders, or PIDs, are diseases that weaken the immune system. This makes it easier for a person to get sick. Some PIDs are mild and may not be diagnosed until later in life. Other kinds are severe and can be identified shortly after birth.... expand

Background: Primary immunodeficiency disorders, or PIDs, are diseases that weaken the immune system. This makes it easier for a person to get sick. Some PIDs are mild and may not be diagnosed until later in life. Other kinds are severe and can be identified shortly after birth. Researchers want to learn more about PIDs by comparing data from relatives and healthy volunteers to people with a PID. Objective: To learn more about PIDs, including their genetic causes. Eligibility: People ages 0 75 with a PID or their healthy biological relatives the same ages Healthy volunteers ages 18 75 Design: Participants will be screened with a medical history, physical exam, and HIV blood test. They may have a pregnancy test. Participants may repeat the screening tests. Blood taken at screening will be used for genetic tests and research tests. Participants will be told test results that affect their health. Some blood will be stored for future research. Adult participants with a PID may have a small piece of skin removed. The area will be numbed. A small tool will take a piece of skin about the size of a pencil eraser. Researchers may collect fluid or tissue samples from PID participants regular medical care. They will use them for research tests. Participants with a PID will have 3 follow-up visits over 10 years (for infants, 2 years). Visits will include a physical exam, medical history, and blood draw. Participants with a PID and their relatives will be called once a year for 10 years. They will talk about how they are feeling and if they have developed any new symptoms or illnesses. ...

Type: Observational

Start Date: May 2018

open study

A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A...
Bioverativ Therapeutics Inc. Hemophilia A With Inhibitors
The primary purpose of this study is to describe the time to tolerization (i.e., ITI success) with rFVIIIFc in participants within a maximum of 48 weeks (12 months) of ITI treatment. expand

The primary purpose of this study is to describe the time to tolerization (i.e., ITI success) with rFVIIIFc in participants within a maximum of 48 weeks (12 months) of ITI treatment.

Type: Interventional

Start Date: Jun 2017

open study

The EVARREST® Pediatric Mild or Moderate Liver and Soft Tissue Bleeding US Study
Ethicon, Inc. Controlling Mild to Moderate Bleeding During Surgery
The objective of this study is to evaluate the safety and hemostatic effectiveness of EVARREST as an adjunct to controlling mild to moderate soft hepatic parenchyma or soft tissue bleeding during open hepatic, abdominal, pelvic, retroperitoneal, and thoracic (non-cardiac) surgery... expand

The objective of this study is to evaluate the safety and hemostatic effectiveness of EVARREST as an adjunct to controlling mild to moderate soft hepatic parenchyma or soft tissue bleeding during open hepatic, abdominal, pelvic, retroperitoneal, and thoracic (non-cardiac) surgery in pediatric population.

Type: Interventional

Start Date: Mar 2018

open study

An Efficacy and Safety Study of Palovarotene for the Treatment of MO
Clementia Pharmaceuticals Inc. Exostoses, Multiple Hereditary
This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple osteochondromas (MO). expand

This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple osteochondromas (MO).

Type: Interventional

Start Date: Apr 2018

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 Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension...
Actelion Pulmonary Arterial Hypertension
This is a prospective, multicenter, open-label, randomized, controlled, parallel group, group-sequential, event-driven Phase 3 study to evaluate efficacy, safety and pharmacokinetics (PK) of macitentan in children. expand

This is a prospective, multicenter, open-label, randomized, controlled, parallel group, group-sequential, event-driven Phase 3 study to evaluate efficacy, safety and pharmacokinetics (PK) of macitentan in children.

Type: Interventional

Start Date: Nov 2017

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

The Pediatric Anesthesia Quality Improvement Project
The Society for Pediatric Anesthesia Surgery Anesthesia Children
The Study is designed to collect information about adverse events that occur in children undergoing anesthesia in participating hospitals. Demographic information will be collected on all anesthetics. An analysis of each adverse event will be performed and entered into the database.... expand

The Study is designed to collect information about adverse events that occur in children undergoing anesthesia in participating hospitals. Demographic information will be collected on all anesthetics. An analysis of each adverse event will be performed and entered into the database. From this information we will devise strategies to prevent these adverse events.

Type: Observational [Patient Registry]

Start Date: Feb 2008

open study

Phase II Pediatric Study With Dabrafenib in Combination With Trametinib in Patients With HGG and LGG
Novartis Pharmaceuticals Diffuse Astrocytoma Anaplastic Astrocytoma Astrocytoma Oligodendroglioma, Childhood Anaplastic Oligodendroglioma
The purpose of this study is to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma or relapsed or refractory high grade glioma. expand

The purpose of this study is to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma or relapsed or refractory high grade glioma.

Type: Interventional

Start Date: Dec 2017

open study

Natural History Study of SCID Disorders
National Institute of Allergy and Infectious Diseases (NIAID) Severe Combined Immunodeficiency (SCID) Leaky SCID Omenn Syndrome Reticular Dysgenesis ADA SCID
This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine the patient, recipient and transplant-related variables that are most important... expand

This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine the patient, recipient and transplant-related variables that are most important in determining outcome, study investigators will uniformly collect pre-, post- and peri-transplant (or other treatment) information on all children enrolled into this study. Children will be divided into three strata: - Stratum A: Typical SCID with virtual absence of autologous T cells and poor T cell function - Stratum B: Atypical SCID (leaky SCID, Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function), and - Stratum C: ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy. Each Group/Cohort Stratum will be analyzed separately.

Type: Observational

Start Date: Sep 2010

open study

Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
Association for Pediatric Pulmonary Hypertension Hypertension, Pulmonary
The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the disease course and long-term outcome of PH in childhood. Patients will undergo... expand

The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the disease course and long-term outcome of PH in childhood. Patients will undergo clinical assessments and receive standard medical care, as determined by treating physicians in their daily clinical practice. The TOPP-2 registry is specifically designed to capture the variables that have been proposed as treatment goals in PePH and the reasons for changes in treatment strategy. The TOPP-2 registry uses the new clinical classification of PH as outlined at the 5th World Symposium for Pulmonary Hypertension (WSPH) in Nice 2013 and includes new characterizations for children with PH. The registry is planned and implemented under the scientific leadership of the Association for Pediatric Pulmonary Hypertension (PePH), independently from the financial sponsors. At least 200 of the enrolled patients will have a follow-up period of 3 years.

Type: Observational [Patient Registry]

Start Date: Aug 2015

open study

Dyslipidemia of Obesity Intervention in Teens
New England Research Institutes Dyslipidemia Obesity
This trial of pitavastatin will determine efficacy and safety in this high risk population and provide evidence for clinicians to target this treatable risk factor to achieve an impact on early atherosclerosis, and potentially achieve primary prevention of adult cardiovascular... expand

This trial of pitavastatin will determine efficacy and safety in this high risk population and provide evidence for clinicians to target this treatable risk factor to achieve an impact on early atherosclerosis, and potentially achieve primary prevention of adult cardiovascular disease.

Type: Interventional

Start Date: May 2018

open study

Health Equity: Advance Care Planning for Spanish Speaking Teens With Cancer-1st Resubmission
Maureen Lyon Cancer
Pediatric ACP (Advance Care Planning) (pACP) (1) Supports communication with children/adolescents at any stage of a serious illness in understanding their illness, complications, fears, and hopes, as well as treatment preferences regarding future medical care with their family;... expand

Pediatric ACP (Advance Care Planning) (pACP) (1) Supports communication with children/adolescents at any stage of a serious illness in understanding their illness, complications, fears, and hopes, as well as treatment preferences regarding future medical care with their family; (2) Communicates these goals of care and treatment preferences with their physician; and (3) Documents these goals of care and end-of-life treatment preferences. Among adolescents, cancer is the leading cause of disease-related deaths. Survival has improved far less for 15 to 24 year-olds than it has for older patients. About one-fourth will die prematurely. No pediatric Advance Care Planning (pACP) model exists to serve Spanish speaking adolescents with cancer. Objective: To take first steps to implement pACP as a routine, structured intervention in pediatric hospitals with underserved Spanish speaking adolescents living with cancer through timely conversations with their families to relieve suffering (physical, psychological, spiritual) and maximize the quality of life

Type: Interventional

Start Date: Sep 2019

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

Pharmacokinetics, Safety, and Antiviral Activity of the Elvitegravir/Cobicistat/Emtricitabine/Tenofovir...
Gilead Sciences Acquired Immune Deficiency Syndrome (AIDS) HIV Infections
There will be 3 cohorts in this study. The primary objectives of Cohort 1 are to evaluate the steady state pharmacokinetics (PK) for elvitegravir (EVG) and tenofovir alafenamide (TAF) and confirm the dose of the elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide (E/C/F/TAF)... expand

There will be 3 cohorts in this study. The primary objectives of Cohort 1 are to evaluate the steady state pharmacokinetics (PK) for elvitegravir (EVG) and tenofovir alafenamide (TAF) and confirm the dose of the elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide (E/C/F/TAF) single tablet regimen (STR) (Part A) and to evaluate the safety and tolerability of E/C/F/TAF STR through Week 24 (Part B) in HIV-1 infected, antiretroviral (ARV) treatment-naive adolescents. The primary objectives of Cohort 2 are to evaluate the PK of EVG and TAF in virologically suppressed HIV-1 infected children 6 to < 12 years of age weighing ≥ 25 kg administered E/C/F/TAF STR (Part A) and to evaluate the safety and tolerability of E/C/F/TAF STR through Week 24 in virologically suppressed HIV-1 infected children 6 to < 12 years of age weighing ≥ 25 kg (Part B). The primary objectives of cohort 3 are to evaluate the PK of EVG and TAF and confirm the dose of the STR, and to evaluate the safety and tolerability of E/C/F/TAF low dose (LD) STR in virologically suppressed HIV-1 infected children ≥ 2 years of age and weighing ≥ 14 to < 25 kg.

Type: Interventional

Start Date: May 2013

open study

Long-Term Safety Study Of Tofacinib In Patients With Juvenile Idiopathic Arthritis
Pfizer Juvenile Idiopathic Arthritis
Evaluate long-term safety and tolerability of tofacitinib in patients with JIA, who have previously participated in tofacitinib JIA studies. expand

Evaluate long-term safety and tolerability of tofacitinib in patients with JIA, who have previously participated in tofacitinib JIA studies.

Type: Interventional

Start Date: Mar 2013

open study

LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell...
North American Consortium for Histiocytosis Langerhans Cell Histiocytosis
The LCH-IV is an international, multicenter, prospective clinical study for pediatric Langerhans Cell Histiocytosis LCH (age < 18 years). expand

The LCH-IV is an international, multicenter, prospective clinical study for pediatric Langerhans Cell Histiocytosis LCH (age < 18 years).

Type: Interventional

Start Date: Jul 2014

open study

Cisplatin and Combination Chemotherapy in Treating Children and Young Adults With Hepatoblastoma or Liver...
Children's Oncology Group Childhood Hepatocellular Carcinoma Childhood Malignant Liver Neoplasm Elevated Alpha-Fetoprotein Hepatoblastoma Hepatocellular Malignant Neoplasm, Not Otherwise Specified
This partially randomized phase II/III trial studies how well, in combination with surgery, cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or hepatocellular carcinoma. Drugs used in chemotherapy, such as cisplatin, doxorubicin,... expand

This partially randomized phase II/III trial studies how well, in combination with surgery, cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or hepatocellular carcinoma. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan, sorafenib, gemcitabine and oxaliplatin, 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 combination chemotherapy may kill more tumor cells than one type of chemotherapy alone.

Type: Interventional

Start Date: May 2018

open study

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Martina Bebin Tuberous Sclerosis Complex
Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to the onset of their first seizure expand

Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to the onset of their first seizure

Type: Interventional

Start Date: Dec 2016

open study

Pevonedistat, Irinotecan Hydrochloride, and Temozolomide in Treating Patients With Recurrent or Refractory...
Children's Oncology Group Recurrent Central Nervous System Neoplasm Recurrent Lymphoma Recurrent Malignant Solid Neoplasm Refractory Central Nervous System Neoplasm Refractory Lymphoma
This phase I trial studies the side effects and best dose of pevonedistat when given together with irinotecan hydrochloride and temozolomide in treating patients with solid tumors or lymphoma that have come back after a period of improvement or that do not respond to treatment.... expand

This phase I trial studies the side effects and best dose of pevonedistat when given together with irinotecan hydrochloride and temozolomide in treating patients with solid tumors or lymphoma that have come back after a period of improvement or that do not respond to treatment. Pevonedistat and irinotecan hydrochloride may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as temozolomide, 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 pevonedistat, irinotecan hydrochloride, and temozolomide may work better in treating patients with solid tumors or lymphoma.

Type: Interventional

Start Date: Nov 2017

open study

ATHN 2: Factor Switching Study
American Thrombosis and Hemostasis Network Hemophilia
This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched factor products. The study will follow each patient for up to 1 year. Patients will... expand

This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched factor products. The study will follow each patient for up to 1 year. Patients will be recruited at Hemophilia Treatment Centers (HTC) which are ATHN-affiliates. The primary outcome being studied is the development of inhibitor (i.e., antibodies to factor) at 1 year or 50 exposure days, whichever comes first. The study will be conducted at approximately 30 HTCs, with a planned enrollment of 600 patients.The entire study duration is projected to be approximately 6 years. In addition, optional substudies will be included for some products, as "Product-Specific Modules". These will be questionnaires to collect data for subjects receiving selected Factor products. For example, subjects receiving Kovaltry will be approached to participate in the 'Kovaltry Product-Specific Module'; subjects receiving Adynovate will be approached to participate in the 'Adynovate Product-Specific Module'. Questions will be related to product use, perceptions of product use, and other post-marketing consumer data.

Type: Observational

Start Date: Sep 2015

open study

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Columbia University Mitochondrial Disorders Mitochondrial Genetic Disorders Mitochondrial Diseases Disorder of Mitochondrial Respiratory Chain Complexes Deletion and Duplication of Mitochondrial DNA
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders. expand

The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.

Type: Observational

Start Date: Dec 2010

open study

Multi-antigen T Cell Infusion Against Neuro-oncologic Disease
Catherine Bollard Brain Tumor
This Phase I dose-escalation trial is designed to determine the safety and feasibility of rapidly generated tumor multi-antigen associated specific cytotoxic T lymphocytes (TAA-T) in patients with newly diagnosed diffuse intrinsic pontine gliomas DIPGs (Group A) or recurrent,... expand

This Phase I dose-escalation trial is designed to determine the safety and feasibility of rapidly generated tumor multi-antigen associated specific cytotoxic T lymphocytes (TAA-T) in patients with newly diagnosed diffuse intrinsic pontine gliomas DIPGs (Group A) or recurrent, progressive, or refractory non-brainstem CNS malignancies (Group B). Pediatric and adult patients who have high-risk CNS tumors with known positivity for one or more Tumor Associated Antigens (TAA) (WT1, PRAME and/or survivin) will be eligible to receive Tumor Antigen Associated Cytotoxic T Lymphocytes (TAA-T). Patients will be enrolled in one of two groups: Group A includes patients with newly diagnosed diffuse intrinsic pontine gliomas (DIPGs) who will undergo irradiation as part of their upfront therapy and Group B includes patients with recurrent, progressive or refractory CNS tumors including medulloblastoma, non-brainstem high-grade glioma, and ependymoma, among others. TAA-T will be generated from patient's peripheral blood mononuclear cells (PBMCs) or by apheresis. This protocol is designed as a phase I dose-escalation study. Group A patients (DIPG patients): TAA-T will be infused any time > 2 week after completion of radiotherapy. Group B patients (other recurrent/progressive/refractory CNS tumors): TAA-T will be infused any time > 2 weeks after completing most recent course of conventional (non-investigational) therapy for their disease AND after appropriate washout periods as detailed in eligibility criteria.

Type: Interventional

Start Date: Dec 2018

open study

Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma,...
National Cancer Institute (NCI) Advanced Malignant Solid Neoplasm ALK Fusion Protein Expression ALK Gene Mutation ALK Gene Translocation Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
This phase II Pediatric MATCH trial studies how well ensartinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with ALK or ROS1 genomic alterations that have come back or do not respond to treatment and have spread to other places... expand

This phase II Pediatric MATCH trial studies how well ensartinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with ALK or ROS1 genomic alterations that have come back or do not respond to treatment and have spread to other places in the body. Ensartinib 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

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
UniQure Biopharma B.V. Hemophilia B
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype... expand

This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10^13 gc/kg.

Type: Interventional

Start Date: Jun 2018

open study