Clinical trials: Chronic Granulomatous Disease
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
Chronic Granulomatous Disease
Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)
In this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.
- Trial name
- NCT01338675
- Testing
- Busulfan
- Where
- South Korea
- Volunteers
- 5 people
- Sponsor
- Seoul National University Hospital
Chronic Granulomatous Disease
Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease
This study will investigate the safety and effectiveness of a modified stem cell transplant procedure for treating chronic granulomatous disease (CGD) in patients with active infection.
- Trial name
- NCT00001765
- Testing
- Nexell Isolex with T-cell Depletion, Baxter isolex 300i
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease, Crohn'S-like IBD
Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease
This study will determine if the drug infliximab is safe for treating inflammatory bowel disease (IBD) in patients with chronic granulomatous disease (CGD).
- Conditions
- Chronic Granulomatous Disease, Crohn'S-like IBD, Inflammatory Bowel Disease (IBD)
- Trial name
- NCT00325078
- Testing
- Infliximab
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease (CGD)
Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
Background: X-Linked Chronic Granulomatous Disease (X-CGD) is caused by a gene mutation that makes the immune system to not work properly.
- Trial name
- NCT07113743
- Testing
- Busulfan, Tocilizumab, Eltrombopag
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hyper-Immunoglobulin E. Syndrome (HEIS), Chronic Granulomatous Disease (CGD)
Skin Immunity Sample Collection Involving Blisters and Biopsies
Background: The way the body heals and protects itself from getting sick is called the immune response. Some people with weak immune systems get sick often or get rashes and skin infections.
- Trial name
- NCT03921515
- Testing
- Blister induction, Skin Biopsies
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Metabolism, Inborn Errors, Granulomatous Disease, Chronic
Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders
OBJECTIVES: I. Determine the safety of total body irradiation and fludarabine followed by allogeneic peripheral blood stem cell or bone marrow transplantation in combination with cyclosporine and mycophenolate mofetil for…
- Trial name
- NCT00010361
- Testing
- cyclosporine, fludarabine, mycophenolate mofetil
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Fred Hutchinson Cancer Center
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
Chediak Higashi Syndrome, Chronic Granulomatous Disease
Learning and Behavior Problems in Children With Chronic Granulomatous Disease and Related Disorders
This study will try to determine what causes learning, behavioral and emotional problems in children with chronic granulomatous disease (GCD) and other phagocyte disorders.
- Conditions
- Chediak Higashi Syndrome, Chronic Granulomatous Disease, Job's Syndrome, Leukocyte Disorder
- Trial name
- NCT00005933
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease
Generation of Powerful Biological Tools for Understanding the Pathophysiology of Chronic Granulomatous Disease.
Chronic granulomatous disease (CGD) is a rare genetic disease of innate immune due to the malfunction of phagocytic cells unable to destroy pathogens during infection.
- Trial name
- FIBRO CGD (NCT02926963)
- Testing
- samples collection : hair and skin biopsy
- Where
- France
- Volunteers
- 3 people
- Sponsor
- University Hospital, Grenoble
p47, Autosomal Recessive
pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)
Background: Chronic granulomatous disease (CGD) is a genetic disorder. People with CGD are missing a gene that affects their white blood cells.
- Conditions
- p47, Autosomal Recessive, Chronic Granulomatous Disease
- Trial name
- NCT06253507
- Testing
- Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Granulomatous Mastitis, Mastitis Chronic
Observation Alone for Mild Non-Lactational Mastitis
The goal of this observational study is to investigate the disease progression rate and timing in patients with mild Non-Lactational Granulomatous Lobular / Periductal Mastitis (NL-GLM/PDM) during follow-up with observation alone…
- Trial name
- NCT06665399
- Testing
- Observation
- Where
- 2 sites in China
- Volunteers
- 57 people
- Sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Cystic Fibrosis, Pseudohypoaldosteronism
Genetic Disorders of Mucociliary Clearance in Nontuberculous Mycobacterial Lung Disease
Healthy volunteers and patients with diseases that involve problems clearing mucus from the lungs will be examined and tested to better understand the reasons for recurring lung infections in these patients and to try to develop…
- Conditions
- Cystic Fibrosis, Pseudohypoaldosteronism, Nontuberculous Mycobacterial Infection, Chronic Granulomatous Disease
- Trial name
- NCT00368446
- Where
- United States
- Volunteers
- 87 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Chronic Granulomatous Disease, Granulomatous Disease, Chronic
A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with…
- Trial name
- NCT06559176
- Testing
- PM359
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 12 people
- Sponsor
- Prime Medicine, Inc.
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
OBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation for patients with primary immunodeficiencies. II.
- Conditions
- Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease
- Trial name
- NCT00006054
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center
Chronic Granulomatous Disease, Inflammatory Bowel Disease
Elemental Diet for Treatment of Inflammatory Bowel Disease in Patients With Chronic Granulomatous Disease
Background: Bacteria that live inside the stomach and intestines are important for health. Chronic granulomatous disease (CGD) and inflammatory bowel disease (IBD) can make people have unhealthy bacteria.
- Trial name
- NCT03983837
- Testing
- Neocate Splash
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
Granuloma, Granulomatous Disease, Chronic
Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes
The research goal of this study is to obtain CD34+ hematopoietic stem cells (HSC) from peripheral blood and/or bone marrow, and Mononuclear Cells (lymphocytes and monocytes), and granulocytes (grans) from peripheral blood that…
- Conditions
- Granuloma, Granulomatous Disease, Chronic, Leukocyte Disease, Genetic Disease, X-Linked
- Trial name
- NCT00001405
- Where
- United States
- Volunteers
- 850 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
X-Linked Chronic Granulomatous Disease
Gene Therapy for X-linked Chronic Granulomatous Disease
X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder, which affects boys. It is a primary immunodeficiency disorder which results from an inability of the white blood cells called phagocytic cells (or…
- Trial name
- NCT02757911
- Testing
- X vivo gene therapy
- Where
- France
- Volunteers
- 3 people
- Sponsor
- Genethon
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege