Clinical trials: Chronic Granulomatous Disease
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.
Granulomatous Disease, Chronic, X-linked, Variant
Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)
Chronic Granulomatous Disease (CGD) is a rare inherited disorder in which patients suffer from severe infection and inflammation. The first indication of disease usually appears in early childhood.
- Trial name
- NCT01381003
- Testing
- pCCLchimGp91s lentiviral vector transduced CD34+ cells infusion
- Where
- United Kingdom
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
X-Linked Chronic Granulomatous Disease
Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)
X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder, which affects boys. It is caused by an error in a gene that makes part of the immune system.
- Trial name
- CGD (NCT01855685)
- Testing
- X vivo gene therapy
- Where
- 2 sites in United Kingdom
- Volunteers
- 3 people
- Sponsor
- Genethon
Chronic Granulomatous Disease
Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
This study is a longitudinal and cross-sectional evaluation of patients with Chronic Granulomatous Disease (CGD) who received or are receiving hematopoietic cell transplantation (HCT) for their disease under a variety of…
- Trial name
- NCT02116764
- Where
- United States
- Volunteers
- 137 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease
Whole Blood Biospecimen Collection for Subjects With Chronic Granulomatous Disease (CGD)
The primary study objective is to collect biospecimen samples (e.g., blood) from participants diagnosed with Chronic Granulomatous Disease (CGD).
- Trial name
- NCT05915897
- Testing
- Specimen Donation
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Sanguine Biosciences
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)
Crohn's Disease
An Open-Label, Proof of Consent Study of Vorinostat for the Treatment of Mdoerate-to-Severe Crohn s Disease and Maintenance Therapy With Ustekinumab
Background: Crohn s disease (CD) is an inflammatory bowel disease. It causes inflammation of the gut. Symptoms may include diarrhea, abdominal pain, fatigue, weight loss and malnutrition.
- Trial name
- NCT03167437
- Testing
- Vorinostat, Ustekinumab
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
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
Chronic Granulomatous Disease (CGD)
Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease
This research study is designed to assess the prevalence of specific antibodies and inflammatory cytokines in adult and pediatric participants with CGD.
- Trial name
- NCT06605378
- Where
- 52 sites in United States, United Kingdom
- Volunteers
- 60 people
- Sponsor
- Ensoma
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
IL-1 Receptor Inhibitor for Granulomatous Complications in Patients With Chronic Granulomatous Disease
"Kineret" (INN: Anakinra) neutralizes the biological activity of interleukin-1α (IL-1α) and interleukin-1β (IL-1β) by the concurrent inhibition of binding to interleukin-1 receptor I (IL-1RI).
- Trial name
- NCT04136028
- Testing
- Kineret
- Where
- Russia
- Volunteers
- 13 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Chronic Granulomatous Disease, Communicable Disease
Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up
This protocol will follow patients who participated in NIAID's study Gene Therapy Approach for Chronic Granulomatous Diseases (95-I-0134). No further gene therapy treatments will be given under this protocol.
- Trial name
- NCT00001476
- Testing
- Gene Therapy Method for CGD, Isolex 300i Magnetic Cell Selector
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
X-linked Chronic Granulomatous Disease
Gene Therapy for X-CGD
X-linked chronic granulomatous disease (X-CGD) is a rare inherited immune defect, which is caused by the inability of phagocytic cells to produce reactive oxygen species due to a defect in the gp91phox subunit of the NADPH…
- Trial name
- NCT01906541
- Testing
- ex-vivo gene-therapy
- Where
- Germany
- Volunteers
- 5 people
- Sponsor
- Hubert Serve, Prof., MD
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Granulomatous Disease, Chronic
Gene Therapy for Chronic Granulomatous Disease
The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan)patients with chronic granulomatous…
- Trial name
- CGD (NCT00564759)
- Testing
- retroviral SF71-gp91phox transduced CD34+ cells
- Where
- Germany
- Volunteers
- 2 people
- Sponsor
- Johann Wolfgang Goethe University Hospital
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
IFN-Gamma Therapy, CGD Gene Mutation
Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease
Background: Chronic granulomatous disease (CGD) is an immunodeficiency disease in which white blood cells are unable to kill certain bacteria and fungi.
- Conditions
- IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg, CGD - Chronic Granulomatous Disease
- Trial name
- NCT01147042
- Testing
- IFN-gamma
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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 (CGD)
Open-label Gene Therapy Study in p47-CGD
Chronic granulomatous disease (CGD) caused by p47phox deficiency (p47-CGD) is a life-threatening genetic disorder causing nicotinamide adenine dinucleotide phosphate (NADPH) oxidase deficiency in phagocytes.
- Trial name
- NCT07826767
- Testing
- SGX-001
- Where
- 3 sites in Germany, Spain and 1 more
- Volunteers
- 5 people
- Sponsor
- Somagenetix AG