Clinical trials: Immune Dysregulation Syndromes
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)
Immune Thrombocytopenia, Autoimmune Hemolytic Anemia
Severe Immune Cytopenia Registry Www.Sic-reg.Org
Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the…
- Conditions
- Immune Thrombocytopenia, Autoimmune Hemolytic Anemia, Evans Syndrome, Primary Immunodeficiency
- Trial name
- sic-reg (NCT03576742)
- Testing
- potential biomarkers
- Where
- Austria
- Volunteers
- 80 people
- Sponsor
- Medical University of Graz
Acute Aortic Syndrome, Aortic Dissection Type A
Protective Effect of Thymosin α1 Against Negative Immune Dysregulation and Organ Dysfunction After Acute Aortic Dissection Surgery (PANDA II)
Systemic inflammatory response syndrome (SIRS) and multiple organ dysfunction syndrome (MODS) are the major causes of death in patients with acute aortic syndrome (AAS).
- Trial name
- PANDA (NCT05339529)
- Testing
- Thymosin Alpha1, 28A-Glycine-28B-L-Arginine-28C-L-Glutamic Acid-28D-L-Alanine-28E-L-Proline-28F-L-Alanine-28G-L-Asparagine-, Blank control
- Where
- 15 sites in China
- Volunteers
- 600 people
- Sponsor
- Nanjing Medical University
IPEX
CD4^LVFOXP3 in Participants With IPEX
This first-in-human, Phase 1 clinical trial will test the feasibility of the manufacturing and the safety of the administration of CD4^LVFOXP3 in up to 30 evaluable human participants with IPEX and evaluate the impact of the…
- Trial name
- NCT05241444
- Testing
- CD4^LVFOXP3
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Bacchetta, Rosa, MD
Acute Respiratory Distress Syndrome (ARDS)
Immune Mechanisms and ECMO Strategies for Moderate-to-Severe ARDS
Acute respiratory distress syndrome (ARDS) poses a major threat to human health. Despite extracorporeal membrane oxygenation (ECMO) support, mortality among patients with ARDS remains high due to severe complications and…
- Trial name
- NCT07726940
- Where
- 39 sites in China
- Volunteers
- 1,776 people
- Sponsor
- Southeast University, China
Aortic Dissection, Aortic Dissection Aneurysm
Protective Effect of Ulinastatin and Thymosin α1 Against Negative Immune Dysregulation and Organ Dysfunction After Acute Aortic Dissection Surgery (PANDA XI)
Systemic inflammatory response syndrome (SIRS) and multiple organ dysfunction syndrome (MODS) are the major causes of death in patients with acute aortic syndrome (AAS).
- Trial name
- NCT06966687
- Testing
- Ulinastatin and Thymosin α1, Ulinastatin only
- Where
- 4 sites in China
- Volunteers
- 200 people
- Sponsor
- Nanjing Medical University
Chronic Myeloproliferative Disorders, Fanconi Anemia
Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma
RATIONALE: Analyzing tissue and blood samples from healthy volunteers or patients with Fanconi anemia, myelodysplasia, myeloproliferative disorders, or myeloma in the laboratory may help doctors learn more about the causes of…
- Conditions
- Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00900055
- Testing
- microarray analysis, polyacrylamide gel electrophoresis, polymerase chain reaction
- Where
- United States
- Volunteers
- 213 people, healthy volunteers welcome
- Sponsor
- OHSU Knight Cancer Institute
Lymphoproliferative Disorders, Autoimmune Lymphoproliferative
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
Background: During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies.
- Conditions
- Lymphoproliferative Disorders, Autoimmune Lymphoproliferative, Immune System Diseases, Common Variable Immunodeficiency
- Trial name
- NCT04339777
- Testing
- Busulfan test dose, Fludarabine, Busulfan
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- National Cancer Institute (NCI)
Sepsis, Septic Shock
Immune Dysregulation During Sepsis: A Natural History Cohort
Background: Sepsis is an illness that occurs when the body s immune system runs out of control. This can damage organs. Sepsis causes an estimated 1 in 5 deaths worldwide.
- Conditions
- Sepsis, Septic Shock, Sepsis-Associated Immune Dysregulation
- Trial name
- NCT07735741
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Sleep Apnea, Obstructive, Type 1 Diabetes (T1D)
Type 1 Diabetes and Obstructive Sleep Apnea
The purpose of the study is to investigate the role of sleep apnea in glycemic dysregulation in adults with Type 1 diabetes.
- Trial name
- T1D and OSA (NCT06915831)
- Testing
- Continuous positive airway pressure (CPAP)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of Chicago
Long COVID, Long Covid19
RECOVER-VITAL: Platform Protocol to Measure the Effects of Antiviral Therapies on Long COVID Symptoms
This study is a platform protocol designed to be flexible so that it is suitable for a wide range of settings within health care systems and in community settings where it can be integrated into COVID-19 programs and subsequent…
- Trial name
- RECOVER-VITAL (NCT05595369)
- Testing
- Experimental: Paxlovid 25 day dosing, Experimental: Paxlovid 15 day dosing
- Where
- 69 sites in United States, Puerto Rico
- Volunteers
- 963 people
- Sponsor
- Kanecia Obie Zimmerman
Immunodeficiencies, Immune Dysregulation Syndromes
Alpha/Beta T and CD19+ Depleted Peripheral Stem Cells for Patients With Primary Immunodeficiencies
This is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with alpha/beta T and CD19+ depleted peripheral stem cell grafts from unrelated or partially matched…
- Trial name
- NCT02990819
- Testing
- Apha/beta T and CD19+ cell depletion using CliniMACS device
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Children's Hospital of Philadelphia
Lymphohistiocytosis, Hemophagocytic, Secondary Hemophagocytic Lymphohistiocytosis
Hemophagocytic Lymphohistiocytosis (HLH) Evaluation and Research of Clinical, ImmUnoLogic and TranscriptomE Study
Background: Hemophagocytic lymphohistiocytosis (HLH) is a disease caused by disrupted immune function. People with HLH are prone to fevers and illnesses, which can be fatal.
- Conditions
- Lymphohistiocytosis, Hemophagocytic, Secondary Hemophagocytic Lymphohistiocytosis, Macrophage Activation Syndrome, Hyperinflammatory Syndromes
- Trial name
- NCT06339177
- Where
- 2 sites in United States
- Volunteers
- 300 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency…
- Conditions
- Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome
- Trial name
- CVID/PIRD (NCT07284641)
- Testing
- Hematopoietic stem cell transplant (HSCT)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Paul Szabolcs
Sjögren, Autoimmune Thyroid Disease
Prevalence of Pre-clinical Sjögren Disease and Other Immune Disturbances in Subjects With Autoimmune Thyroiditis Disease
The main study hypothesis is that Sjögren Disease (SD), usually considered a disorder typical of adult females, may occur not exceptionally in adolescence or even in childhood as a subclinical process.
- Trial name
- NCT06324578
- Testing
- AT Testing
- Where
- 2 sites in Italy
- Volunteers
- 150 people
- Sponsor
- IRCCS Burlo Garofolo
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
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
Primary Immunodeficiency Disorders (PIDs)
Leniolisib for Immune Dysregulation in PIDs
This study is an exploratory, non-randomized, open-label, within-patient dose escalation study. The primary objective is to assess safety and tolerability of leniolisib.
- Trial name
- NCT06549114
- Testing
- Leniolisib
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Pharming Technologies B.V.