Clinical trials: Immunologic Deficiency Syndromes
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
Immunodeficiency
Sample Collection for Systems Evaluation of Patients With Unknown or Incompletely Characterized Immune Defects
Background: The immune system defends the body against disease. It has many different parts spread out in the body, including in the blood and skin.
- Trial name
- NCT04408950
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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)
Pneumonia, Ventilator-Associated, Pneumonia, Hospital-Acquired
Impact of a Strategy Based on Bacterial DNA Detection to Optimize Antibiotics in Immunocompromised Patients With Hospital-acquired Pneumonia Requiring Mechanical Ventilation
RESPIRE is a randomized, unblinded, controlled study to measure the impact of a strategy based on a PCR test on the adjustment of antimicrobial therapy in immunocompromised patients suspected with ventilator-associated or…
- Conditions
- Pneumonia, Ventilator-Associated, Pneumonia, Hospital-Acquired, Immunodeficiency
- Trial name
- RESPIRE (NCT05405491)
- Testing
- PCR based strategy, Standard care
- Where
- France
- Volunteers
- 31 people
- Sponsor
- University Hospital, Lille
Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD)
A Study to Evaluate Tabelecleucel in Participants With Epstein Barr Virus (EBV) Associated Diseases
The purpose of this study is to assess the efficacy and safety of tabelecleucel in participants with EBV-associated diseases.
- Conditions
- Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD), EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (EBV+ AID LPD), EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (EBV+ CNS PTLD)
- Trial name
- NCT04554914
- Testing
- Tabelecleucel
- Where
- 40 sites in United States, Austria and 5 more
- Volunteers
- 190 people
- Sponsor
- Pierre Fabre Medicament
Epstein-Barr Virus, Cytomegalovirus Infections
R-MVST Cells for Treatment of Viral Infections in Children and Young Adults
The primary objective is to determine the safety and feasibility of administering R-MVST cells to patients with refractory viral reactivation and/or symptomatic disease caused by Epstein Barr Virus (EBV), cytomegalovirus (CMV)…
- Conditions
- Epstein-Barr Virus, Cytomegalovirus Infections, Adenovirus, BK Virus Infection
- Trial name
- NCT06926894
- Testing
- Rapidly generated virus specific T (R-MVST) cells
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Monica Bhatia
Leukemia, Bone Marrow Failure Syndrome
Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC
The primary objective of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT).
- Conditions
- Leukemia, Bone Marrow Failure Syndrome, Immunodeficiencies
- Trial name
- NCT03145545
- Testing
- Apha/beta T and CD19+ cell depletion using CliniMACS device
- Where
- United States
- Sponsor
- Children's Hospital of Philadelphia
Hematologic Disorders, Hemoglobinopathies
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) in patients with non-malignant or malignant diseases.
- Conditions
- Hematologic Disorders, Hemoglobinopathies, Immunodeficiencies
- Trial name
- NCT01666080
- Testing
- Busulfan, Fludarabine, Total body irradiation
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Myelokathexis, Infections
Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
Background: WHIMS (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis Syndrome) is a rare disease. It can cause cancers, infections, and warts. Researchers want to see if a drug called plerixafor can treat WHIMS.
- Conditions
- Myelokathexis, Infections, Neutropenia, Warts
- Trial name
- NCT02231879
- Testing
- Plerixafor, G-CSF
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Kidney Diseases
Assessment of T-cell Response and In-vitro Proof-of-concept of T-cell Engineering in Chronic ESKD Patients.
This is a comparative, prospective, non-interventional study to evaluate immune response in patients with chronic kidney disease.
- Trial name
- LYMPHOVIR-IRCT (NCT06474169)
- Testing
- 1 blood sample for each participant
- Where
- France
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Régional d'Orléans
SCID, Omenn's Syndrome
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
- Conditions
- SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome
- Trial name
- NCT01652092
- Testing
- Alemtuzumab 0.3 mg, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Hematologic Malignancy, Solid Tumor
Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular…
- Conditions
- Hematologic Malignancy, Solid Tumor, Sickle Cell Disease, Aplastic Anemia
- Trial name
- NCT06689800
- Testing
- Let's Get REAL
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Washington University School of Medicine
HIV, Acquired Immunodeficiency Syndrome
Safety, Immunogenicity, Efficacy of Ad26.Mos4.HIV, MVA-BN-HIV and PGT121, PGDM1400, and VRC07-523LS in HIV-1-Infected Adults
A multicenter, randomized, parallel-group, placebo-controlled, double-blind, Phase 1/2a clinical study to investigate the safety, tolerability, immunogenicity and exploratory efficacy of a vaccine regimen consisting of an…
- Conditions
- HIV, Acquired Immunodeficiency Syndrome, Immunologic Deficiency Syndrome, Acquired, Sexually Transmitted Diseases, Viral
- Trial name
- NCT04983030
- Testing
- Ad26.Mos4.HIV, MVA-BN-HIV, PGT121
- Where
- 6 sites in United States
- Volunteers
- 28 people
- Sponsor
- Boris Juelg, MD PhD
Immunologic Deficiency Syndromes
Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Adenosine deaminase (ADA) enzyme deficiency results in severe combined immunodeficiency (SCID), a fatal autosomal recessive inherited immune disorder.
- Trial name
- NCT03478670
- Testing
- Strimvelis
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Fondazione Telethon
Immunologic Deficiency Syndromes, Primary Immune Deficiency (PID)
Genetic Study of Immunodeficiency: Search for New Genetic Causes for Primary Immunodeficiencies
Individuals with suspected primary immunodeficiency will be studied and the results compared with healthy controls. Primary immunodeficiency may manifest as recurrent, severe or unusual infections as well as signs and symptoms of…
- Trial name
- GSI (NCT02735824)
- Testing
- Skin Biopsy, Mouth Swab or Saliva Collection, Blood Sampling
- Where
- Switzerland
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University Children's Hospital, Zurich
Immunodeficiency
Vaccine-induced Immunity in Immunocompromised Patients
Managing respiratory virus infections in immunocompromised patients requires a multidisciplinary approach, including vaccination, though its effectiveness is often suboptimal in these individuals.
- Trial name
- RESPONSE (NCT06612515)
- Where
- Germany
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- University of Cologne
Human Immunodeficiency Virus, Alcohol-Related Disorders
Project Khanya: Peer-Delivered Intervention to Improve HIV Medication Adherence and Substance Use in South Africa
The purpose of this study is to evaluate a stepped care behavioral intervention for HIV medication adherence and substance use ("Khanya") integrated into an HIV primary care setting in South Africa.
- Conditions
- Human Immunodeficiency Virus, Alcohol-Related Disorders, Drug Use, Substance Use
- Trial name
- NCT05933226
- Testing
- Khanya
- Where
- South Africa
- Volunteers
- 160 people
- Sponsor
- University of Maryland, College Park
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