Clinical trials: Immune Defects, open to volunteers
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
Molar Incisor Hypomineralisation
Correlation Between Colour and Hypersensitivity of First Permanent Molars With Molar Incisor Hypomineralisation (MIH)
Cheese molars (MIH: molar incisor hypomineralisation) can be recognized not only by their colour abnormalities but also by their hypersensitivity.
- Trial name
- NCT07727044
- Where
- Switzerland
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- University of Basel
Chronic Pain, Depression Disorders
Analysis of the Therapeutic and Clinical Profile of Cannabinoid Treatments: A Real-World Study
What is the purpose of this study? The use of medicinal cannabis has grown significantly in Brazil. However, because many patients use these products through "compassionate use" (when traditional treatments haven't worked), there…
- Conditions
- Chronic Pain, Depression Disorders, Anxiety Disorders, Quality of Life
- Trial name
- CANAREAL (NCT07362407)
- Testing
- Participants are followed while using cannabis-based products (such as CBD or THC oils, extracts, or isolates) as prescribed by their own physicians for therapeutic purposes.
- Where
- Brazil
- Volunteers
- 384 people
- Sponsor
- Santa Catarina Federal University
Unspecified Adult Solid Tumor, Protocol Specific
Phase 1/2 Clinical Trial of CP-506 (HAP) in Monotherapy or With Carboplatin or ICI
A modular, first time in human, open label, multiple dose, accelerated escalation with cohort expansion study of the safety and pharmacokinetics of intravenous infusion of CP-506, a tumor agnostic Hypoxia Activated Prodrug in…
- Trial name
- NCT04954599
- Testing
- CP-506, Carboplatin, Immune checkpoint inhibitor
- Where
- 5 sites in Belgium, Netherlands and 1 more
- Volunteers
- 126 people
- Sponsor
- Maastricht University Medical Center
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
Neurogenic Detrusor Overactivity
Open-label, Long-term Safety, Efficacy, and Pharmacokinetics Study of Vibegron in Pediatric Subjects 2 Years to < 18 Years of Age With NDO and on CIC
The purpose of this study is to evaluate the safety, efficacy, and PK of Vibegron in pediatric participants with NDO who are regularly using CIC
- Trial name
- KANGUROO (NCT05491525)
- Testing
- Vibegron
- Where
- 38 sites in United States, Belgium and 14 more
- Volunteers
- 71 people
- Sponsor
- Urovant Sciences GmbH
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
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
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
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)
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
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
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
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
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
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)
Severe Asthma, COPD
Evaluation of Ventilation Defects Downstream of Mucus Plugs in Patients With Muco-Obstructive Lung Disease
In this study, xenon MRI will be used to evaluate regional functional consequences of mucus plugs in the lungs of patients with muco-obstructive pulmonary disease.
- Conditions
- Severe Asthma, COPD, Non-CF Bronchiectasis, Cystic Fibrosis (CF)
- Trial name
- NCT06985225
- Testing
- Hyperpolarized 129 Xenon
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Kansas Medical Center
Spinal Cord Injury, Neurogenic Bladder (NB)
Coupled Gentamicin-Lactobacillus Rhamnosus in NLUTD
The main objective of the proposed research study is to determine in men and women with spinal cord injury/disease and neurogenic bladder whether the dose of coupled gentamicin \& Lactobacillus rhamnosus GG affects the…
- Conditions
- Spinal Cord Injury, Neurogenic Bladder (NB), Neurogenic Lower Urinary Tract Dysfunction, Multiple Sclerosis
- Trial name
- NCT07643974
- Testing
- Gentamicin, Lactobacillus Rhamnosus GG
- Where
- 2 sites in United States
- Volunteers
- 48 people
- Sponsor
- Medstar Health Research Institute
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