Clinical trials: Primary Immune Deficiency Disorders
Primary Immunodeficiency Diseases (PID)
Ex Vivo Evaluation of JAK-inhibitor and Gene Therapeutical Approach in JAK-STAT Related Disorders
The investigators want to study the JAK-inhibitors and their impact on the immune system and evaluate the potential of a gene-therapeutic strategy
- Trial name
- JAKarta (NCT07261891)
- Testing
- blood sampling
- Where
- Belgium
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- prof. dr. Rik Schrijvers
Lymphoproliferative Disorders, Autoimmune Lymphoproliferative
Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation
Background: Blood stem cells in the bone marrow make all the cells to normally defend a body against disease. Allogeneic blood or marrow transplant is when these stem cells are transferred from one person to another.
- Conditions
- Lymphoproliferative Disorders, Autoimmune Lymphoproliferative, Primary T-cell Immunodeficiency Disorders, Immune System Diseases
- Trial name
- NCT03663933
- Testing
- e-ATG, Immunosuppression Only Conditioning, Reduced Intensity Conditioning
- Where
- 2 sites in United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Hereditary Angioedema
Study to Determine How BCX4161 is Metabolized and Eliminated by the Body
The purpose of the study is to assess the extent that radioactive dose of BCX4161 taken by mouth ends up in the urine, feces and expired air.
- Trial name
- NCT02218294
- Testing
- BCX4161
- Where
- United Kingdom
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
HIV Infections, Men
Improving HIV Testing, Linkage, and Retention in Care for Men Through U=U Messaging
This study will evaluate the impact of U=U messaging and counseling on gaps in the HIV care cascade for men, including testing uptake and ART initiation (Aim 1), achieving viral suppression and retention in care (Aim 2) in two…
- Conditions
- HIV Infections, Men, Treatment Adherence
- Trial name
- NCT05602376
- Testing
- U=U testing messaging scripts, U=U adherence messaging scripts
- Where
- 2 sites in South Africa
- Volunteers
- 3,642 people, healthy volunteers welcome
- Sponsor
- Desmond Tutu HIV Foundation
HIV/AIDS, Tuberculosis
Comparison of Door-to-door Versus Community Gathering to Provide HIV Counseling and Testing Services in Rural Lesotho
The purpose of this trial is to determine if door-to-door is more effective than community gathering in providing voluntary HIV counseling and testing (VCT) in communities in rural Lesotho.
- Conditions
- HIV/AIDS, Tuberculosis, Diabetes Mellitus, Arterial Hypertension
- Trial name
- DoDoPi (NCT01459120)
- Testing
- Door-to-door, Pitso
- Where
- 2 sites in Lesotho
- Volunteers
- 1,800 people, healthy volunteers welcome
- Sponsor
- SolidarMed
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
Chronic Liver Disease
An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis
Hepatic steatosis may cause inflammation and fibrosis within the liver potentially leading to end-stage liver disease cirrhosis, liver failure and death.
- Trial name
- FALL (NCT05335603)
- Where
- Denmark
- Volunteers
- 335 people, healthy volunteers welcome
- Sponsor
- University of Copenhagen
Neurodegenerative Disorders
Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases
Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5).
- Trial name
- NCT00874783
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization
Infections, Streptococcal
Primary and Booster Vaccination Study With a Pneumococcal Vaccine in HIV Infected, HIV Exposed Uninfected and HIV Uninfected Children 6 to 10 Weeks of Age.
The purposes of this study: To evaluate the immunogenicity, safety and reactogenicity of pneumococcal vaccine GSK1024850A in HIV infected infants, HIV exposed uninfected infants and HIV unexposed uninfected infants following a…
- Trial name
- NCT00829010
- Testing
- Pneumococcal vaccine GSK1024850A, Tritanrix-HepB/Hib, measles
- Where
- South Africa
- Volunteers
- 489 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Hiv
Identifying Efficient Linkage Strategies for Men
HIV self-testing (HIVST) has been found to be a highly acceptable approach for men to learn of their HIV status and has resulted in increased testing uptake.
- Trial name
- IDEaL (NCT05137210)
- Testing
- Male Counseling and Facility Navigation, Home-Based ART Initiation and Facility Navigation, Stepped Intervention
- Where
- Malawi
- Volunteers
- 569 people, healthy volunteers welcome
- Sponsor
- University of California, Los Angeles
Job Syndrome
NFIL3-induced Pathological Enhancement of IgE Class Switch Recombination in Hyper-IgE Syndrome
Hyper IgE syndrome (HIES) is a rare and complex primary immunodeficiency that affects multiple systems. It is characterized by elevated Immunoglobulin E(IgE), recurrent skin and pulmonary infections and eczematoid…
- Trial name
- NCT02228941
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Shanghai Children's Medical Center
Common Variable Immunodeficiency
Usage of Spirometry in Managing IgG Therapy in CVID With Airway Disease
Although there is evidence in the literature that gammaglobulin replacement therapy can lead to a reduction in the prevalence of pulmonary infection and improved lung function, there is no published study to guide immunologists…
- Trial name
- NCT05193552
- Testing
- Hizentra
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
HIV Infections, Sexually Transmitted Diseases
Parents Matter!: Interventions to Promote Effective Parent-Child Communication
Parents Matter is a community-based project whose goal it is to develop and test an intervention designed to promote effective parent-child communication about sexuality in order to promote sexual health among adolescents.
- Conditions
- HIV Infections, Sexually Transmitted Diseases, Pregnancy
- Trial name
- NCT00137943
- Testing
- Full Parent communication intervention, Brief Parent Communication Intervention, Control Parent Intervention
- Where
- 3 sites in United States
- Volunteers
- 2,210 people, healthy volunteers welcome
- Sponsor
- Centers for Disease Control and Prevention
Meningococcal Disease
Safety and Immunogenicity of Novartis Meningococcal B Vaccine When Administered to Immunocompromised Children and Adolescents Compared to Healthy Subjects.
The study aims at evaluating the safety and immunogenicity of rMenB+OMV NZ when administered to subjects from 2 to 17 years of age with increased risk of meningococcal disease because either of primary or secondary complement…
- Trial name
- NCT02141516
- Testing
- rMenB+OMV
- Where
- 20 sites in Italy, Poland and 3 more
- Volunteers
- 239 people, healthy volunteers welcome
- Sponsor
- Novartis
Hereditary Angioedema
A Single Dose Safety, Tolerability, Pharmacokinetic and Food Effect Study of KVD900 (Sebetralstat) in Healthy Volunteers
A safety, tolerability, pharmacokinetic and food effect study of KVD900 in healthy volunteers.
- Trial name
- NCT04349800
- Testing
- KVD900
- Where
- United Kingdom
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Primary Immune Deficiency, Exercise Capacity
Evaluation of Adult Patients With Immunodeficiency Within the Scope of the ICF
Primary immunodeficiency is a clinically, immunologically, and genetically heterogeneous group of diseases that occur as a result of quantitative and/or qualitative deficiency of one or more cellular and molecular components…
- Conditions
- Primary Immune Deficiency, Exercise Capacity, Physical Activity, Quality of Life
- Trial name
- NCT06014463
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
THC, Cannabis
THC Crossover Study
This is a randomized, crossover study enrolling experienced dual cannabis-tobacco smokers (N=18) to describe the differences in THC and toxicant exposure, examining pharmacokinetic, subjective, and cardiovascular effects from…
- Conditions
- THC, Cannabis, Cannabis Smoking, Cannabis Use, Unspecified
- Trial name
- TRDRP (NCT04429568)
- Testing
- Smoked Cannabis, Vaped Cannabis, Tobacco Cigarette
- Where
- 2 sites in United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Chronic Granulomatous Disease, Inflammatory Bowel Disease
Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations
Background: PIDD stands for primary immune dysregulation. It is a general term that includes many different inherited immune system disorders. The immune system is the part of the body that helps fight disease and infection.
- Trial name
- NCT03278912
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Ebola Virus Disease, Marburg Virus Disease
Evaluation of Safety, Tolerability and Immune Responses of Ebola-S and Marburg Vaccines in Healthy Adults
Primary Objective: To evaluate the safety and tolerability of cAd3-EBO-S and cAd3 Marburg vaccines when administered Intramuscular (IM) at a dose of 1 x 10^11 particle units (PU) to healthy adults.
- Trial name
- NCT04723602
- Testing
- cAd3-Marburg, cAd3-EBO-S
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Albert B. Sabin Vaccine Institute
Primary Immune Deficiency Disorder
Pathophysiology of Inborn Immunodeficiencies
The pathophysiology of primary immunodeficiencies (PID), which encompass a broad range of different diseases with susceptibility to infection and/or a deregulated inflammatory response, is poorly understood.
- Trial name
- NCT03422614
- Testing
- Diagnostic Test
- Where
- Switzerland
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- University of Zurich