Clinical trials: Primary Immune Deficiency Disorders
Cannabinoid Hyperemesis Syndrome
Haloperidol For Outpatient Symptom Management in Patients Diagnosed With Cannabinoid Hyperemesis Syndrome in the Emergrency Department
Patients diagnosed with cannabinoid hyperemesis syndrome that are safe for discharge from the emergency department will be sent an outpatient prescription for haloperidol to be used as needed at home.
- Trial name
- NCT07726069
- Testing
- Haloperidol
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University of Illinois Chicago
Hereditary Angioedema (HAE)
Open-label, Single-arm Study to Assess the Pharmacokinetics, Safety, and Tolerability of a Single Subcutaneous Dose of Icatibant in Healthy Japanese Volunteers
This is a single-dose study to evaluate the pharmacokinetics, safety, and tolerability of icatibant administered to adult Japanese subjects.
- Trial name
- NCT02045264
- Testing
- Icatibant (30 mg)
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Shire
Primary T-cell Immunodeficiency Disorders, Common Variable Immunodeficiency
Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies
Background: Allogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems.
- Conditions
- Primary T-cell Immunodeficiency Disorders, Common Variable Immunodeficiency, Immune System Diseases, Autoimmune Lymphoproliferative
- Trial name
- NCT02579967
- Testing
- Immunosuppression Only Conditioning -Closed with amendment L, Reduced Intensity Conditioning, Myeloablative Conditioning-Closed with amendment L
- Where
- 2 sites in United States
- Volunteers
- 354 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Hereditary Angioedema
Autonomic Nervous System Profile in Hereditary Angioedema
The goal of this study is to compare the autonomic nervous system control of patients with hereditary angioedema to healthy individuals.
- Trial name
- ANS-HAE (NCT06408805)
- Where
- Italy
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Istituti Clinici Scientifici Maugeri SpA
Fungal Infections, Primary Immune Deficiencies
Studies of Disorders With Increased Susceptibility to Fungal Infections
Background: Researchers are interested in studying disorders that make individuals more susceptible to fungal infections, specifically infections with the Candida yeast.
- Trial name
- NCT01222741
- Where
- United States
- Volunteers
- 850 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID)
Evaluation of Patients With Immune Function Abnormalities
This study will evaluate patients with abnormal immune function that results in recurrent or unusual infections or chronic inflammation.
- Conditions
- Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID), Leukocyte Adhesion Deficiency 1 (LAD), Graft Versus Host Disease (cGvHD)
- Trial name
- NCT00128973
- Where
- United States
- Volunteers
- 3,500 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Nervous System Disease, Immune System Disease
Detection and Characterization of Neurologic Manifestations of Inborn and Acquired Errors of Immunity
Background: Immune system and nervous system have significant interaction so that People with immunity diseases can have complications that affect the nervous system and people with some neurological disease may have defects in…
- Trial name
- NCT06169150
- Where
- United States
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hypercholesterolemia, Hyperbilirubinemia
Study of Efficacy and Safety of the Plasmapheresis Method With Albumin Compensation Compared With the Plasmapheresis Method Without Albumin Compensation for Aging Biomarkers Correction in Men and Women Aged 40 to 55 Years Old
Reaching active aging makes it important to implement new methods affecting the biological age of a person. Biochemical parameters of a blood test are aging biomarkers that are ones of the most accessible for testing.
- Conditions
- Hypercholesterolemia, Hyperbilirubinemia, Hyper IgE Syndrome, Hyper Beta Lipoproteinemia
- Trial name
- NCT04897113
- Testing
- plasma exchange with albumin, plasma exchange without albumin
- Where
- 2 sites in Russia
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- National Medical Research Center for Rehabilitation and Balneology
Hereditary Angioedema Type I and II
Global Registry to Gather Data on Natural History of Patients With Hereditary Angioedema Type I and II
The objective of this international hereditary angioedema (HAE) register is to collect homogeneous clinical and laboratory data on patients with HAE type I and II gathering better information on the natural course of the disease…
- Trial name
- HGR (NCT03828279)
- Testing
- functional and antigenic C1 inhibitor
- Where
- Italy
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- HAE Global Registry Foundation
Lymphoma, Leukemia
Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation
RATIONALE: Following stem cell transplantation, a major risk is graft-versus-host disease (GVHD). This occurs when donor immune cells that have been infused recognise the host's cells as 'foreign' and attack these cells.
- Conditions
- Lymphoma, Leukemia, Myeloma, Myelodysplastic Syndromes
- Trial name
- ToTem (NCT03836690)
- Testing
- CD62L- Tem
- Where
- United Kingdom
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University College, London
Ataxia Telangiectasia
Biomarkers for Babies and Young Children With Ataxia Telangiectasia
The goal of this observational study is to identify progressive changes of quantitative brain and lung imaging, serum and movement-related biomarkers reflecting disease progression in pre-symptomatic infants and very young…
- Trial name
- BOBCAT (NCT07709728)
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
Hereditary Angioedema
A Drug-Drug Interaction Study to Evaluate Drug Transporter Interactions
This is an open-label, three part study to evaluate the effect of BCX7353 on drug transporters as well as the effect of an inhibitor of drug transport on BCX7353.
- Trial name
- NCT03136237
- Testing
- BCX7353, Digoxin, BCX7353 + digoxin
- Where
- United Kingdom
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema
Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations
This is a 3 part, phase 1 crossover study in healthy subjects to evaluate the pharmacokinetic profile of KVD824 following single and multiple doses of novel KVD824 modified-release formulations compared with a reference KVD824…
- Trial name
- NCT05118958
- Testing
- KVD824 Prototype 1 modified-release tablet, KVD824 Prototype 2 modified-release tablet, KVD824 Immediate-Release Capsule
- Where
- United Kingdom
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Wiskott-Aldrich Syndrome, Thrombocytopenia
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
- Conditions
- Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding
- Trial name
- WAS (NCT00909363)
- Testing
- Promacta, blood drawing in patients with WAS, blood drawing in healthy controls
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Angioedema, Angioedemas, Hereditary
Angioedema Biomarker Research Study
This clinical trial aims to evaluate and compare novel and commercially available diagnostic assays through blood tests for the differential diagnosis and comprehensive assessment of patients experiencing recurrent angioedema…
- Conditions
- Angioedema, Angioedemas, Hereditary, Urticaria, Mastocytosis
- Trial name
- NCT06210698
- Testing
- Venipuncture
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Foundation For Rare Disease Research
Hereditary Angioedema
Safety and Pharmacokinetics of GNR-038 in Healthy Volunteers
It is an open-label dose-escalating study in sequential cohorts to assess safety and pharmacokinetics of GNR-038.
- Trial name
- NCT04557319
- Testing
- GNR-038, 25 МЕ/kg, GNR-038, 50 МЕ/kg, GNR-038, 100 МЕ/kg
- Where
- Russia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- AO GENERIUM