Clinical trials: Immune Defects
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
Arrhythmia, Long QT Syndrome
AI-powered ECG Analysis for Deadly Arrhythmias and ICI Myocarditis
ELDORA is a non-interventional observational data-science study aiming to develop and validate clinical-grade artificial intelligence tools applied to electrocardiogram (ECG) data.
- Conditions
- Arrhythmia, Long QT Syndrome, Immune Checkpoint Inhibitor-Related Myocarditis
- Trial name
- ELDORA (NCT07644715)
- Where
- France
- Volunteers
- 127,000 people, healthy volunteers welcome
- Sponsor
- Groupe Hospitalier Pitie-Salpetriere
Crohn's Disease, Rheumatoid Arthritis
OTIS Humira Pregnancy Registry
The purpose of the OTIS Autoimmune disease in pregnancy study is to monitor planned and unplanned pregnancies exposed to certain medications, to evaluate the possible teratogenic effect of these medications and to follow live…
- Trial name
- NCT01086059
- Where
- United States
- Volunteers
- 944 people, healthy volunteers welcome
- Sponsor
- University of California, San Diego
Primary Ciliary Dyskinesia, Primary Immune Deficiency
Defining the Genetic Etiology of Suppurative Lung Disease in Children and Adults
The investigators will utilize a systematic approach for the diagnostic evaluation of patients to identify characteristics which may distinguish between Primary Immunodeficiency (PID) disorders versus Primary Ciliary Dyskinesia…
- Conditions
- Primary Ciliary Dyskinesia, Primary Immune Deficiency, Kartagener Syndrome
- Trial name
- NCT04702243
- Testing
- Genetic Testing for PCD or PID, Unaffected Family Member Genetic Testing
- Where
- 8 sites in United States, Canada
- Volunteers
- 436 people
- Sponsor
- University of North Carolina, Chapel Hill
Long QT Syndrome, Congenital Long Qt Syndrome
Cardiac Rehabilitation of Children and Adolescent With Long QT Syndrome
Children and adolescents with inherited cardiac arrhythmia su ch Long QT Syndrome (LQTS) have lower physical and quality of life than their healthy peers.
- Conditions
- Long QT Syndrome, Congenital Long Qt Syndrome, Inherited Cardiac Conduction Disorder, Pediatric ALL
- Trial name
- RYTHMO'FIT (NCT05964322)
- Where
- 2 sites in France
- Volunteers
- 8 people
- Sponsor
- University Hospital, Montpellier
Molar Incisor Hypomineralization
Evaluation of the Effectiveness of Various Desensitizing Treatment Protocols
The objective of this research was to evaluate the clinical success of desensitization treatments using four different materials in the first permanent molars (FPMs) affected by MIH.
- Trial name
- NCT07085689
- Testing
- Sodium Fluoride Varnish (5%), Silver diamine fluoride- potassium iodide, Aqueous Silver Fluoride
- Where
- Turkey (Türkiye)
- Volunteers
- 52 people
- Sponsor
- Hacettepe University
Candidiasis, Chronic Mucocutaneous
CAMB/MAT2203 in Patients With Mucocutaneous Candidiasis
This is an open-label, dose-titration trial to study the efficacy, safety, and pharmacokinetics of oral cochleate amphotericin B (CAMB) in the treatment of mucocutaneous candidiasis infections in patients who are refractory or…
- Trial name
- CAMB (NCT02629419)
- Testing
- Amphotericin B
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Matinas BioPharma Nanotechnologies, Inc.
EBV, HSV
The Natural History of Severe Viral Infections and Characterization of Immune Defects in Patients Without Known Immunocompromise
Background: Infections caused by viruses are common causes of illnesses: the common cold, many ear infections, sore throats, chicken pox, and the flu are caused by different viruses.
- Conditions
- EBV, HSV, VZV, HPV
- Trial name
- NCT01011712
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Osteoarthritis, Avascular Necrosis
Post-market Surveillance Study With the HYPERION Hip Endoprosthesis System in Defect Reconstruction
The study is a multi-center, prospective, non-controlled, consecutive cohort post market surveillance study. The objective of this study is to obtain survival and clinical outcome data on the Hyperion® system in primary and…
- Conditions
- Osteoarthritis, Avascular Necrosis, Rheumatoid Arthritis, Functional Deformities
- Trial name
- NCT03382665
- Where
- 3 sites in Germany
- Volunteers
- 70 people
- Sponsor
- Zimmer Biomet
Non-ischemic Cardiomyopathy, Cardiomyopathies
Cardiovascular Multimodality Imaging Study
Determining the etiology of cardiomyopathy is of high clinical importance for optimal treatment strategy and prediction of prognosis.
- Conditions
- Non-ischemic Cardiomyopathy, Cardiomyopathies, Hypertrophic Cardiomyopathy, Right Ventricular Arrhythmogenic Cardiomyopathy
- Trial name
- NCT06610019
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- Montefiore Medical Center
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency…
- Conditions
- Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome
- Trial name
- CVID/PIRD (NCT07284641)
- Testing
- Hematopoietic stem cell transplant (HSCT)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Paul Szabolcs
DiGeorge Syndrome, DiGeorge Anomaly
Thymus Transplantation With Immunosuppression
The research purpose is to determine if thymus transplantation with immunosuppression is a safe and effective treatment for complete DiGeorge anomaly.
- Conditions
- DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- 884 (NCT00579709)
- Testing
- Thymus Tissue for Transplantation
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Rheumatologic Conditions (JRA,Lupus), Epilepsy
Coping Skills Training (CST) for Children With Chronic Health Conditions
Purpose of the study The purpose of this study is to pilot an adapted Coping Skills Training (CST) intervention for feasibility and preliminary efficacy with a sample of children 8 to 12 years of age and their parents.
- Conditions
- Rheumatologic Conditions (JRA,Lupus), Epilepsy, Spina Bifida, Asthma
- Trial name
- NCT00359775
- Testing
- Coping Skills Training
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Children's Hospital and Health System Foundation, Wisconsin
ERAS, Pediatric ALL
Pediatric Enhanced Recovery After Cardiac Surgery
Identify our institution's current perioperative management strategies for pediatric cardiac surgery patients. Identify predictive factors for receiving perioperative care following P-ERACS strategies.
- Conditions
- ERAS, Pediatric ALL, Congenital Heart Disease
- Trial name
- PERCS (NCT05665712)
- Testing
- enhanced recovery
- Where
- Canada
- Volunteers
- 483 people, healthy volunteers welcome
- Sponsor
- University of Alberta
Poliomyelitis, Postpoliomyelitis Syndrome
Study of "Post-Polio Syndrome"
Polio or poliomyelitis is the disease caused by the poliovirus. The virus attacks cells in the spinal cord and causes symptoms of fever, sore throat, headache, vomiting, and stiffness of the neck.
- Trial name
- NCT00001185
- Where
- United States
- Volunteers
- 360 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Dentin Sensitivity, Molar Incisor Hypomineralization
MIH and Hypersensitivity in Children
The aim of this study was to evaluate the efficacy of the association of casein phosphopeptide plus amorphous calcium phosphate (CPP-ACPF) mousse and photo-bio-modulation therapy (PMBT) (diode laser, RAFFAELLO 980 BIO - Dental…
- Trial name
- NCT05705037
- Testing
- CPP-ACPF, PBMT, CPP-ACPF+PBMT
- Where
- Italy
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Università degli Studi di Brescia
Congenital Heart Defects, Thymectomy
Impact of Thymectomy on Immunity in Infants After Cardiac Surgery
Heart surgery in infants typically involves complete removal of the thymus gland to improve access to the heart. However, the thymus plays a key role in developing the immune system in early childhood, especially in the…
- Conditions
- Congenital Heart Defects, Thymectomy, Immunological Deficiency Syndrome
- Trial name
- THYMIC (NCT07019857)
- Testing
- Blood sample collection at surgery time and 12 months later
- Where
- 2 sites in France
- Volunteers
- 70 people
- Sponsor
- Nantes University Hospital
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Cystic Fibrosis, Immunologic Deficiency Syndrome
Natural History of Noncirrhotic Portal Hypertension
Background: Noncirrhotic Portal Hypertension (NCPH) is caused by liver diseases that increase pressure in the blood vessels of the liver. It seems to start slowly and not have many warning signs.
- Conditions
- Cystic Fibrosis, Immunologic Deficiency Syndrome, Turner Syndrome, Congenital Hepatic Fibrosis
- Trial name
- NCT02417740
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)