Clinical trials: Immunologic Deficiency Syndromes, active
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)
DOCK8 Deficiency
Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency
Background: -DOCK8 deficiency is a genetic disorder that affects the immune system and can lead to severe recurrent infections and possible death from infections or certain types of cancers, including blood cancers.
- Trial name
- NCT01176006
- Testing
- Reduced-intensity hematopoietic stem cell, Fludarabine(Fludara, Berlex Laboratories), Cyclophosphamide(CTX, Cytoxan)
- Where
- United States
- Volunteers
- 76 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
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)
WHIM, Warts
Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM
Background: Warts, hypogammaglobulinemia, infections and myelokathexis syndrome (WHIMs) is a rare disorder that affects the immune system. People with WHIMs can have severe infections all over their body.
- Conditions
- WHIM, Warts, Hypogammaglobulinemia, Immunodeficiency
- Trial name
- NCT07775313
- Testing
- Busulfan, Palifermin, Plerixafor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
COVID-19, Immunodeficiency
OPtimisation of Antiviral Therapy in Immunocompromised COVID-19 Patients: a Randomized Factorial Controlled Strategy Trial
The overall purpose of the trial is to evaluate the efficacy and safety of possible combination antiviral therapy DAA (remdesivir + nirmatrelvir/r)∞ versus the reference monotherapy (nirmatrelvir/r alone) and to assess the…
- Trial name
- OPTICOV (NCT05587894)
- Testing
- Paxlovid 5 days, Paxlovid 10 days, Veklury
- Where
- 18 sites in France, Italy and 1 more
- Volunteers
- 256 people
- Sponsor
- ANRS, Emerging Infectious Diseases
Down Syndrome
Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy
Background: Down syndrome is a genetic disorder that can cause heart defects and other problems in the body. People with Down syndrome are more likely to have infections, autoimmunity, and blood diseases.
- Trial name
- NCT07416201
- Where
- United States
- Volunteers
- 700 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
DOK 8, STAT1
Genetic Analysis of Immune Disorders
The purposes of this study are to 1) identify the genes responsible for certain immune disorders, 2) learn about the medical problems they cause, and 3) learn how to predict who is likely to develop these disorders and what the…
- Conditions
- DOK 8, STAT1, GATA2, Immunodeficiency
- Trial name
- NCT00001467
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Idiopathic CD4+ Lymphocytopenia, Cryptococcal Meningitis
Etiology, Pathogenesis, and Natural History of Idiopathic CD4+ Lymphocytopenia
Background: Idiopathic CD4+ lymphocytopenia (ICL) is a condition in which there is a decreased level of CD4+ lymphocytes (a type of white blood cell), which can lead to opportunistic infections or autoimmune disorders and…
- Conditions
- Idiopathic CD4+ Lymphocytopenia, Cryptococcal Meningitis, Warts
- Trial name
- NCT00867269
- Where
- United States
- Volunteers
- 950 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
GATA2, Immunodeficiency
Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
Background: GATA2 deficiency is a disease caused by mutations in the GATA2 gene. It can cause different types of leukemia and other diseases. Researchers want to see if a stem cell transplant can be used to treat this condition.
- Conditions
- GATA2, Immunodeficiency, MDS
- Trial name
- NCT01861106
- Testing
- Allogeneic HSCT, Busulfan Test dose, Fludarabine (Fludara, Berlex Laboratories)
- Where
- United States
- Volunteers
- 144 people
- Sponsor
- National Cancer Institute (NCI)
Human Immunodeficiency Virus, Castleman's Disease
Molecular Characterization of Viral-associated Tumors, Tumors Occurring in the Setting of HIV or Other Immune Disorders and Castleman Disease
Background: A person s genome is the collection of all their genes. A gene instructs individual cells to make proteins. Proteins are involved in all of our body s chemical processes.
- Conditions
- Human Immunodeficiency Virus, Castleman's Disease, Kaposi's Sarcoma, Viral-Associated Cancer
- Trial name
- NCT03300830
- Where
- United States
- Volunteers
- 280 people
- Sponsor
- National Cancer Institute (NCI)
Immunodeficiency, Hematopoietic Stem Cell Transplantation
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
Background: Allogeneic hematopoietic stem cell transplant involves taking blood stem cells from a donor and giving them to a recipient. The transplants are used to treat certain diseases and cancers.
- Trial name
- NCT05027945
- Testing
- Allogeneic HSCT, Busulfan test dose, Mycophenolate mofetil (MMF)
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- National Cancer Institute (NCI)
HIV, Immunodeficiencies
Studies of the Pathogenesis of HIV Infection in Human Peripheral Blood Cells and/or Body Fluids in People Living With and Without HIV
We are studying virologic and/or immunologic parameters of HIV infection and other infectious or non-infectious immune deficiency diseases in order to better understand the pathogenesis of HIV.
- Conditions
- HIV, Immunodeficiencies, Infectious Diseases
- Trial name
- NCT00001281
- Where
- 2 sites in United States
- Volunteers
- 2,419 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Coccidioidomycosis
Pathogenesis and Genetics of Disseminated or Refractory Coccidioidomycosis
Background: Coccidioidomycosis is caused by a fungus that grows in the southwest United States and parts of Mexico and South America. This disease is caused by breathing dust containing the fungus.
- Trial name
- NCT02190266
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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)
HIV
Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients
The purpose of the study is to evaluate the safety and feasibility of administering SB-728mR-HSPC after conditioning with busulfan.
- Trial name
- NCT02500849
- Testing
- SB-728mR-HSPC Infusion 3 days following busulfan conditioning
- Where
- 5 sites in United States
- Volunteers
- 12 people
- Sponsor
- City of Hope Medical Center
Infections, Pneumonia
Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)
The Hyper IgE Syndromes (HIES) are primary immunodeficiencies resulting in eczema and recurrent skin and lung infections. Autosomal dominant Hyper IgE syndrome (AD-HIIES; Job's syndrome) is caused by STAT3 mutations, and is a…
- Conditions
- Infections, Pneumonia, Immune System Diseases, STAT3 Transcription Factor
- Trial name
- NCT00006150
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
LAD-1, DOCK8
Apheresis of Patients With Immunodeficiency
Background: Gene therapy is being investigated as a possible treatment for individuals with immunodeficiency diseases or other conditions that make it difficult to fight off infection.
- Conditions
- LAD-1, DOCK8, GATA2 Deficancy
- Trial name
- NCT01212055
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- National Cancer Institute (NCI)
JAK1 GOF, STAT1 GOF
Upadacitinib in Treatment of JAK/STAT Pathway Disorders With Activating Mutations
This study focuses on a genetic condition that affects the Janus Kinase-Signal Transducer and Activator of Transcription (JAK-STAT) immune signaling pathway.
- Conditions
- JAK1 GOF, STAT1 GOF, STAT3 GOF, STAT5B GOF
- Trial name
- SAFE-JAKi (NCT07670156)
- Testing
- Upadacitinib
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Lisa Satter
Non-Hodgkin Lymphoma, Lymphoma Non-Hodgkin
Toxin Exposure and Immune Dysregulation in Non-Hodgkin Lymphoma
The goal of this observational cohort study is to learn how toxin and occupational exposures, germline genetic variation, and immune dysregulation relate to B-cell non-Hodgkin lymphoma among active-duty service members and other…
- Conditions
- Non-Hodgkin Lymphoma, Lymphoma Non-Hodgkin, B-cell Lymphoma, Immune Dysregulation
- Trial name
- TOXNHL (NCT07652580)
- Where
- 3 sites in United States
- Volunteers
- 300 people
- Sponsor
- Henry M. Jackson Foundation for the Advancement of Military Medicine
GATA2, Immunodeficiency
Allogeneic Hematopoietic Stem Cell Transplantation With Briquilimab-Based Conditioning in Participants With GATA2 Deficiency
Background: People with GATA-binding factor 2 (GATA2) deficiency have a mutation on the GATA2 gene. This gene affects immune function.
- Trial name
- NCT05907746
- Testing
- Mycophenolate Mofetil, Tacrolimus, Post-Transplant Cyclophosphamide
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- National Cancer Institute (NCI)