Clinical trials: Immune Defect, open to volunteers
Pediatric ALL, Cardiac Defect
Evaluating the Relationship Between Skin Color and Pulse Oximeter Accuracy in Children
This multisite study seeks to understand whether and how pulse oximeter accuracy varies across children with different skin colors in real world clinical settings.
- Trial name
- PACH (NCT06529575)
- Where
- 3 sites in United States
- Volunteers
- 650 people
- Sponsor
- University of Pennsylvania
Psoriasis, Pregnancy
Stelara and Tremfya Pregnancy Exposure Registry OTIS Autoimmune Diseases in Pregnancy Project
The purpose of the OTIS Autoimmune Diseases 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…
- Conditions
- Psoriasis, Pregnancy, Psoriatic Arthritis, Crohn Disease
- Trial name
- NCT02103361
- Testing
- Ustekinumab, Guselkumab
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- University of California, San Diego
Chediak-Higashi Syndrome
Study of Chediak-Higashi Syndrome
Chediak-Higashi syndrome (CHS) is a rare autosomal recessive disorder characterized in its classical form by oculocutaneous albinism, a bleeding diathesis, recurrent infection due to abnormal neutrophil and natural killer cell…
- Trial name
- NCT00005917
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched hematopoietic stem cell transplantation (HSCT) is safe and effective for patients aged…
- Conditions
- Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD)
- Trial name
- BOLT+BMT (NCT01852370)
- Testing
- CD3/CD19 negative allogeneic hematopoietic stem cells
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Paul Szabolcs
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
This is a prospective longitudinal natural history study with a retrospective cross-sectional arm aimed at determining the natural history of sphingosine phosphate lyase insufficiency syndrome (SPLIS), a recently recognized…
- Trial name
- SPLIS-HIS (NCT06669949)
- Testing
- no intervention
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- University of California, San Francisco
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
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
Sphingolipidoses, Enzyme Deficiency
SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)
This protocol aims to gather information about sphingosine phosphate lyase insufficiency syndrome (SPLIS), also known as NPHS14, and to create a SPLIS patient registry.
- Trial name
- SPLIS-OSPRI (NCT04885179)
- Testing
- no intervention
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
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