Clinical trials: Lymphatic Insufficiency
Nephrotic Syndrome Steroid-Dependent, Nephrotic Syndrome Steroid-Resistant
Biomarkers and Outcome Predictors of Pediatric Nephrotic Syndrome: A Genetic, Transcriptomic, and Secretome Multiomics Study
Idiopathic Nephrotic Syndrome is a rare disease of the kidneys, which typically affects children. For most affected children there is the need of a prolonged treatment with drugs reducing the activity of the immune system, also…
- Conditions
- Nephrotic Syndrome Steroid-Dependent, Nephrotic Syndrome Steroid-Resistant, Nephrotic Syndrome in Children, Glomerulonephritis
- Trial name
- PRECISE (NCT06792448)
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Anemia, Chronic Kidney Disease
Study to Assess Darbepoetin Alfa Dosing for the Correction of Anemia in Pediatric Patients With Chronic Kidney Disease
The primary objectives of this study are the following: 1. To test if the proportion of participants achieving a hemoglobin value greater than or equal to 10.0 g/dL at any time point after the first dose during the study is…
- Conditions
- Anemia, Chronic Kidney Disease, Kidney Disease
- Trial name
- NCT00436748
- Testing
- Darbepoetin Alfa
- Where
- 67 sites in United States, Belgium and 8 more
- Volunteers
- 116 people
- Sponsor
- Amgen
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Adrenoleukodystrophy, Batten Disease
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 in patients who are undergoing standard treatment with umbilical cord blood transplant (UCBT) for inborn…
- Conditions
- Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell
- Trial name
- DUOC-01 (NCT02254863)
- Testing
- DUOC-01
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Joanne Kurtzberg, MD
Vitamin D Deficiency, Stem Cell Transplant Complications
A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT
Research has suggested that children with sufficient vitamin D levels undergoing hematopoietic stem cell transplant (HSCT) have improved outcomes, including lower incidences of infection and graft-versus-host disease (GVHD), as…
- Conditions
- Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder
- Trial name
- NCT03176849
- Testing
- Vitamin D3, Standard Vitamin D3 Supplementation
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- Phoenix Children's Hospital
Osteosarcoma, Leukemia
Open-label Leucovorin Pharmacokinetic Study in Patients Receiving High Dose Methotrexate With or Without Voraxaze
The purpose of this study is to investigate whether the administration of Voraxaze reduces exposure to leucovorin and its active metabolite to below the level achieved in patients who have not received Voraxaze.
- Conditions
- Osteosarcoma, Leukemia, Lymphoma
- Trial name
- LVPK (NCT00634504)
- Testing
- glucarpidase, high-dose methotrexate, leucovorin, high-dose methotrexate, leucovorin
- Where
- 25 sites in United States
- Volunteers
- 20 people
- Sponsor
- BTG International Inc.
I Cell Disease, Fucosidosis
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory…
- Conditions
- I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy
- Trial name
- NCT00005900
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Fairview University Medical Center
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Chronic Kidney Disease, Stage 3 (Moderate)
Short-term Effect of a New Nordic Renal Diet on Phosphorus Homeostasis in CKD Stage 3-4
Hyperphosphatemia is a severe complication to chronic kidney disease (CKD) and is associated with increased risk of vascular calcification, cardiovascular morbidity and mortality.
- Trial name
- NCT03472105
- Testing
- New Nordic Renal Diet
- Where
- Denmark
- Volunteers
- 18 people
- Sponsor
- University of Copenhagen
Chronic Kidney Disease; Iron Deficiency Anemia
A Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)
Primary Objectives: To evaluate the safety (compared to iron sucrose) and efficacy of ferumoxytol in pediatric CKD subjects with iron deficiency anemia (IDA) or who are at risk of development of IDA Secondary Objective: To…
- Trial name
- NCT03619850
- Testing
- Ferumoxytol, Iron sucrose
- Where
- 21 sites in United States, Hungary and 3 more
- Volunteers
- 129 people
- Sponsor
- AMAG Pharmaceuticals, Inc.
Iron Deficiency Anemia
A Trial of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease
Study AMAG-FER-CKD-253 is an extension study of the combined AMAG-FER-CKD-251 (NCT01155375) and AMAG-FER-CKD-252 (NCT01155388) studies to evaluate the efficacy and safety of episodic treatment of iron deficiency anemia (IDA) with…
- Trial name
- NCT01264679
- Testing
- Ferumoxytol
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- AMAG Pharmaceuticals, Inc.
Anemia
Efficacy and Safety of Spironolactone in Pediatric Hemodialysis Patients With Anemia.
The goal of this clinical trial is to learn if Spironolactone drug works to treat anemia in hemodialysis pediatric patients. It will also learn about the safety of Spironolactone drug.
- Trial name
- NCT07715318
- Testing
- Spironolactone (drug), Iron Supplement, Erythropoietin stimulating agent
- Volunteers
- 40 people
- Sponsor
- Ain Shams University
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Chronic Venous Insufficiency, Pediatric ALL
Venous Disease in Pediatric Population
Chronic venous disease (CVD) is widespread in adult subjects of western countries and is responsible of important morbidity and healthcare costs.
- Trial name
- VEDIPEDI (NCT04424095)
- Testing
- In-office vascular visit, Echo Duplex of lower limbs
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- University of Catanzaro
Respiratory Failure, Shock
Comparing Post Cardiac Surgery Outcomes in ESRD Patient's With Early Dialysis Versus Standard Care
End-stage renal disease (ESRD) is an important risk factor for coronary and valvular cardiac disease leading to cardiac surgery.
- Conditions
- Respiratory Failure, Shock, Coagulopathy
- Trial name
- NCT04705701
- Testing
- Hemodialysis
- Sponsor
- Northwell Health
Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose…
- Conditions
- Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension
- Trial name
- POPS or POP02 (NCT04278404)
- Testing
- The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:
- Where
- 52 sites in United States, Canada
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Duke University
Multiple Myeloma
Study of PD-1Ab21-BCMA CAR-T Therapy for Consolidation of Multiple Myelomawith Renal Dysfunction
The purpose of this study is to determine the efficacy and safety of targeted BCMA CART cells secreting PD1 and interleukin 21 fusion protein immunotherapy for first-line consolidation therapy of multiple myeloma with renal…
- Trial name
- NCT07489534
- Testing
- PD-1Ab21-BCMA CAR-T cell immunotherapy
- Where
- China
- Volunteers
- 25 people
- Sponsor
- Daihong Liu
ECMO Outcomes, Respiratory Failure (Pediatric Patients)
ECMO Use in Pediatric Oncology and HSCT Patients: A Descriptive and Comparative Analysis
This was an observational study, with a descriptive and comparative portion. The descriptive analysis, a large single center experience, examined bleeding complications, infectious rates, and survival outcomes in patients under…
- Conditions
- ECMO Outcomes, Respiratory Failure (Pediatric Patients), Malignancies, Hematologic, Solid Tumor Malignancies
- Trial name
- NCT07712835
- Testing
- Extracorporeal membrane oxygenation (ECMO)
- Volunteers
- 22 people
- Sponsor
- Children's Mercy Hospital Kansas City