Clinical trials: Immune Defects, open to volunteers
Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism
Growing up With the Young Endocrine Support System (YESS!)
Transition from paediatric to adult endocrinology is a challenge for adolescents, families and doctors. Up to 25% of young adults with chronic endocrine disorders are lost to follow-up ('drop-out') once the young adult moves out…
- Conditions
- Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism, Growth Hormone Deficiency, Combined Pituitary Hormone Deficiency
- Trial name
- YESS (NCT04252001)
- Testing
- YESS! game, Snake '97, Transition-toolkit
- Where
- 7 sites in Belgium, Netherlands and 2 more
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Hematologic Diseases
The Interplay Between Inborn Error of Immunity and Blood Disorders: Unravelling Immune Defects Behind Common Haematological Diseases
The universe of Inborn errors of Immunity (IEI) is rapidly expanding: their clinical spectrum is not only characterised by infections but often includes haematological complications.
- Trial name
- NCT06857604
- Testing
- Biological samples
- Where
- 7 sites in Canada, France and 3 more
- Volunteers
- 700 people
- Sponsor
- Meyer Children's Hospital IRCCS
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
Immune Defects
Detection and Characterization of Host Defense Defects
This protocol is designed to evaluate selected patients with documented recurrent or unusual infections and their family members for clinical and laboratory correlates of immune abnormalities.
- Trial name
- NCT00001355
- Where
- United States
- Volunteers
- 3,600 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
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
Immune Deficiencies
Detection and Characterization of Infections and Infection Susceptibility
This screening study will examine the causes of immune disorders affecting white blood cells, which defend against infections and will try to develop better means of diagnosis and treatment of these immune disorders.
- Trial name
- NCT00404560
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
Acute Leukemia, Adenomatous Polyposis
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll.
- Conditions
- Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML
- Trial name
- SJFAMILY (NCT03050268)
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- St. Jude Children's Research Hospital
Adenovirus, Cytomegalovirus Infections
Anti-viral T-cell Therapy by Gamma Capture
The primary purpose of this phase I/II study is to evaluate whether partially matched, ≥1/6 Human Leukocyte Antigens (HLA) -matched, viral specific T cells have efficacy against adenovirus, Cytomegalovirus (CMV), and Epstein Barr…
- Conditions
- Adenovirus, Cytomegalovirus Infections, Epstein-Barr Virus Infections
- Trial name
- Gamma Capture (NCT06027879)
- Testing
- Specific T- Lymphocytes
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Paul Szabolcs
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)
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
Neutropenia
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are…
- Trial name
- NCT06056297
- Testing
- Mavorixafor
- Where
- 114 sites in United States, Argentina and 23 more
- Volunteers
- 176 people
- Sponsor
- X4 Pharmaceuticals
Kidney Transplant, Familial Hypercholesterolaemia
Cardiovascular Risk in Children With Chronic Conditions Study
Children living with chronic health conditions face a higher risk of developing cardiovascular diseases than their peers, largely due to the accelerated aging of the heart and blood vessels.
- Conditions
- Kidney Transplant, Familial Hypercholesterolaemia, Type 1 Diabetes Mellitus (T1DM), Type 2 Diabetes Mellitus (T2DM)
- Trial name
- CR3C (NCT07086989)
- Where
- 3 sites in Hungary
- Volunteers
- 300 people
- Sponsor
- Semmelweis University
Spinal Cord Injuries, Cerebral Palsy
Mobile Health Self-Management and Support System for Chronic and Complex Health Conditions
This study will assess the benefits of using mobile health system designed for individuals with chronic and complex health conditions (such as those with Spinal Cord Injury,Cerebral Palsy, Spina Bifida, and Traumatic Brain…
- Conditions
- Spinal Cord Injuries, Cerebral Palsy, Spinal Dysraphism, Brain Injuries
- Trial name
- NCT02592291
- Testing
- MHealth
- Where
- United States
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
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
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
Congenital Heart Disease, Pediatric ALL
Digital Health Promotion for Children With Congenital Heart Disease and Their Guardians
Congenital heart disease (CHD) affects approximately 1% of live births, and children with CHD often fail to meet WHO physical activity guidelines. Digital interventions to promote physical activity in CHD patients are limited.
- Trial name
- NCT06626672
- Testing
- Digital Health Intervention
- Where
- Germany
- Volunteers
- 60 people
- Sponsor
- Deutsches Herzzentrum Muenchen
Pediatric ALL, Congenital Heart Disease
Exercise MRI to Evaluate Cardiorespiratory Fitness in Children With Heart Disease
There are many barriers to heart-healthy lifestyles in pediatric patients with acquired and congenital heart disease. Investigators want to further understand how participants heart and skeletal muscles work together during…
- Conditions
- Pediatric ALL, Congenital Heart Disease, Heart Transplantation
- Trial name
- NCT06325280
- Testing
- Exercise cardiac MRI Assessment, Cardiopulmonary Exercise Test
- Where
- Canada
- Volunteers
- 20 people
- Sponsor
- University of Alberta
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health