Clinical trials: Immune Defects
Sickle Cell Disease, Thalassemia
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma
- Trial name
- NCT00730314
- Testing
- Hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital Los Angeles
Molar Incisor Hypomineralization, Hypersensitivity
Intraosseous vs Infiltration Anesthesia in Molar-Incisor Hypomineralization (MIH) Children
This study aims to compare the effectiveness of intraosseous and infiltration anesthesia techniques administered using a computer-assisted anesthesia device (SleeperOne® 5; Dental HiTec, France) during restorative treatments of…
- Trial name
- MIH (NCT07352813)
- Testing
- Intraosseous anaesthesia using a computerized system, Infiltration anaesthesia using a computerized system
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Ankara Yildirim Beyazıt University
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Spinal Cord Injuries, Spina Bifida
Examining Capacity Building of Youth With Physical Disabilities to Pursue Participation Following the PREP Intervention.
The goal of this clinical trial is to learn about how a participation-based intervention builds capacity of youth with physical disabilities to pursue activities of their choice in the community.
- Conditions
- Spinal Cord Injuries, Spina Bifida, Musculoskeletal Disorder, Juvenile Arthritis
- Trial name
- NCT06007885
- Testing
- Pathways and Resources to Engagement and Participation (PREP)
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- McGill University
Arrhythmogenic Right Ventricular Cardiomyopathy, Cardiomyopathies
Mayo AVC Registry and Biobank
Arrhythmogenic ventricular cardiomyopathy (AVC) is a genetic condition which affects the heart and can lead to heart failure and rhythm problems, of which, sudden cardiac arrest or death is the most tragic and dangerous.
- Conditions
- Arrhythmogenic Right Ventricular Cardiomyopathy, Cardiomyopathies, Heart Diseases, Cardiovascular Diseases
- Trial name
- NCT03049254
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Hypogammaglobulinemia, Congenital Heart Disease
Post-bypass Prophylactic IVIG in Infants and Neonates
The purpose of this study protocol is to determine if administering Intravenous Immunoglobulin (IVIG) for treatment of cardiopulmonary bypass (CPB) induced hypogammaglobulinemia in the early post-operative period can impact…
- Trial name
- NCT02043379
- Testing
- IVIG
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Alabama at Birmingham
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well olaparib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with defects in deoxyribonucleic acid (DNA) damage repair genes that have…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Low Grade Glioma
- Trial name
- NCT03233204
- Testing
- Olaparib
- Where
- 113 sites in United States, Puerto Rico
- Volunteers
- 6 people
- Sponsor
- National Cancer Institute (NCI)
Molar Incisor Hypomineralization, Oral Health
Prevalence, Etiology and Effects on Oral Health and Life Quality of Molar Incisor Hypomineralization
Molar incisor hypomineralization (MIH) can lead to many clinical conditions and affect oral health-related quality of life (OHRQoL).
- Conditions
- Molar Incisor Hypomineralization, Oral Health, Child, Only, Pediatric ALL
- Trial name
- NCT05779332
- Testing
- survey application
- Where
- Turkey (Türkiye)
- Volunteers
- 655 people, healthy volunteers welcome
- Sponsor
- Recep Tayyip Erdogan University Training and Research Hospital
Bleeding, Cardiac Defect
ZEPLAST- PED: ZEro_PLASma Trial in Small Infants Undergoing Cardiac Surgery
In pediatric patients (newborns and infants weighing less than 10 kg) undergoing cardiac surgery with extracorporeal circulation postoperative bleeding represents a known complication with a significant impact on outcome.
- Conditions
- Bleeding, Cardiac Defect, Surgery--Complications, Fibrinogenopenia; Acquired
- Trial name
- ZEPLAST-PED (NCT04434001)
- Testing
- Fibrinogen Concentrate Human, Prothrombin Complex Concentrate, Fresh Frozen Plasma
- Where
- Italy
- Volunteers
- 40 people
- Sponsor
- IRCCS Policlinico S. Donato
Complete DiGeorge Anomaly, DiGeorge Syndrome
Thymus Transplantation Safety-Efficacy
Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no thymus function, bone marrow stem cells do not develop into educated T cells, which fight infection.
- Conditions
- Complete DiGeorge Anomaly, DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- NCT01220531
- Testing
- Cultured Thymus Tissue
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
DiGeorge Anomaly, Complete DiGeorge Anomaly
Phase I/II Thymus Transplantation With Immunosuppression #950
The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and…
- Conditions
- DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- #950 (NCT00579527)
- Testing
- Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
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
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)
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