Clinical trials: Immunologic Disease
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)
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)
Eosinophilic Disease, Immune Deficiency
Screening Protocol for Genetic Diseases of Allergic Inflammation
Background: Mast cells are responsible for most symptoms of allergic reactions. In some allergic diseases, it is unusually easy to cause mast cells to release their contents and cause allergic reactions.
- Conditions
- Eosinophilic Disease, Immune Deficiency, HaTS, Urticaria Anaphylaxis
- Trial name
- NCT00852943
- Where
- United States
- Volunteers
- 834 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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)
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)
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis
A Phase III Clinical Study to Evaluate the Efficacy, Safety, and Tolerability of Plonmarlimab in Subjects With Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis (Also Known as Macrophage Activation Syndrome [MAS])
A study designed to evaluate the efficacy, safety, immunogenicity, PK, and PD characteristics of Plonmarlimab in patients with relapsed/refractory rheumatic and immunologic disease-associated MAS, and to explore biomarkers…
- Trial name
- NCT07208058
- Testing
- Plonmarlimab
- Where
- 18 sites in China
- Volunteers
- 30 people
- Sponsor
- TJ Biopharma Co., Ltd.
Non Malignant Diseases, Immunodeficiencies
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
This Phase II pilot study evaluates the safety and effectiveness of reduced-intensity conditioning (RIC) regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric and young adult patients with…
- Conditions
- Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- RIC (NCT01050855)
- Testing
- Reduced-Intensity Conditioning
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Children's Hospital of Philadelphia
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
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
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
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
- Conditions
- Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders
- Trial name
- FAM BMT (NCT03128996)
- Testing
- RIC regimen, GVHD prophylaxis regimen
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Washington University School of Medicine
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
Chronic Granulomatous Disease-associated Colitis
Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis
Background: Chronic granulomatous disease (CGD) weakens the body's defense against germs. CGD can also damage the colon. It can cause inflammation (colitis) that disrupts the good bacteria.
- Trial name
- NCT05333471
- Testing
- MTP 101-LF
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Activated PI3Kdelta Syndrome (APDS); PASLI Disease
Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI
This study is designed to provide long-term CDZ173 treatment, a selective PI3Kδ inhibitor, to the patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI who participated in the CCDZ173X2201 study or who were…
- Trial name
- NCT02859727
- Testing
- CDZ173
- Where
- 8 sites in United States, Belarus and 5 more
- Volunteers
- 37 people
- Sponsor
- Pharming Technologies B.V.
Immunological Disease, Autoimmune Diseases
Biologics And Clinical Immunology Cohort at Sinai
The study team plans to establish a bioregistry of patients receiving biologic therapy as part of their standard treatment at the Mount Sinai Therapeutic Infusion Center and affiliated practices.
- Conditions
- Immunological Disease, Autoimmune Diseases, Primary Immune Deficiency
- Trial name
- BACICS (NCT04784364)
- Where
- United States
- Volunteers
- 800 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Immune Deficiency, Immune Dysregulation Disorder
Allergy and Immunology Natural History Study
This protocol is a natural history study designed to evaluate subjects (and some family members) with suspected or identified genetic diseases of allergic inflammation or Immune Dysregulation.
- Conditions
- Immune Deficiency, Immune Dysregulation Disorder, Allergic Inflammation
- Trial name
- NCT05584488
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Hematologic Malignancies, Inborn Errors of Metabolism Disorders
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants…
- Conditions
- Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
- Trial name
- NCT01881334
- Testing
- CliniMACS CD34 Reagent System
- Where
- United States
- Sponsor
- Joanne Kurtzberg, MD
Severe Combined Immunodeficiency Disease, X-linked
Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
SCID-X1 is a genetic disorder of blood cells caused by DNA changes in a gene that is required for the normal development of the human immune system.
- Trial name
- LVXSCID-ND (NCT01512888)
- Testing
- CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs
- Where
- 3 sites in United States
- Volunteers
- 28 people
- Sponsor
- St. Jude Children's Research Hospital